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Imetelstat Sodium in Treating Participants With Primary or Secondary Myelofibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01731951
Recruitment Status : Completed
First Posted : November 22, 2012
Results First Posted : September 21, 2021
Last Update Posted : September 21, 2021
Sponsor:
Information provided by (Responsible Party):
Geron Corporation

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Primary Myelofibrosis
Secondary Myelofibrosis
Myeloid Malignancies
Intervention Drug: Imetelstat
Enrollment 80
Recruitment Details Participants were enrolled at 1 site in the United States from 29 October 2012 to 22 January 2014 (end of recruitment). Data analyses includes data through 24 May 2018. Study has 4 phases: Screening Phase, Core Phase:up to nine 21-day (Arms A,B) or 28-day (Arms D,E,F,G) cycle, Extension Phase: after Cycle 9 to continue treatment with imetelstat; Event Monitoring Phase:Follow-up for those who discontinued treatment to collect survival status, disease status, and subsequent treatment information.
Pre-assignment Details A total of 80 participants with Intermediate-2 or high-risk primary myelofibrosis (PMF)/post-polycythemia vera/essential thrombocythemia (post-PV/ET) MF (Arms A, B, C, E and F) or blast-phase MF (Arm D only) or spliceosome-mutated (or with ring sideroblasts) myelodysplastic syndromes [MDS]/ myeloproliferative neoplasm [MPN] (Arm G only) were enrolled to receive imetelstat. Arm C (Imetelstat 9.4 mg/kg [with MF]) was never initiated, participants allocated to Arm C were reassigned to Arm A or B.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 milligram per kilogram (mg/kg), intravenously (IV) as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with blast-phase myelofibrosis/acute myeloid leukemia (MF/AML) received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with either myelodysplastic syndromes/ myeloproliferative neoplasm (MDS/MPN) or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Period Title: Overall Study
Started 19 16 9 9 18 9
Completed 2 1 0 0 6 0
Not Completed 17 15 9 9 12 9
Reason Not Completed
Documented Disease Progression             1             0             0             0             0             0
Lost to Follow-up             2             0             0             1             3             1
Withdrawal of Consent             1             0             0             0             0             2
Death             13             15             9             8             9             6
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts) Total
Hide Arm/Group Description Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Total of all reporting groups
Overall Number of Baseline Participants 19 16 9 9 18 9 80
Hide Baseline Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of imetelstat.
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 9 participants 80 participants
<=18 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Between 18 and 65 years
8
  42.1%
7
  43.8%
6
  66.7%
1
  11.1%
10
  55.6%
3
  33.3%
35
  43.8%
>=65 years
11
  57.9%
9
  56.3%
3
  33.3%
8
  88.9%
8
  44.4%
6
  66.7%
45
  56.3%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 9 participants 80 participants
Female
7
  36.8%
4
  25.0%
3
  33.3%
3
  33.3%
7
  38.9%
2
  22.2%
26
  32.5%
Male
12
  63.2%
12
  75.0%
6
  66.7%
6
  66.7%
11
  61.1%
7
  77.8%
54
  67.5%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 9 participants 80 participants
Hispanic or Latino
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Not Hispanic or Latino
19
 100.0%
15
  93.8%
8
  88.9%
9
 100.0%
17
  94.4%
7
  77.8%
75
  93.8%
Unknown or Not Reported
0
   0.0%
1
   6.3%
1
  11.1%
0
   0.0%
1
   5.6%
2
  22.2%
5
   6.3%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 9 participants 80 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
2
  22.2%
0
   0.0%
0
   0.0%
0
   0.0%
2
   2.5%
Black or African American
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
White
19
 100.0%
15
  93.8%
6
  66.7%
9
 100.0%
17
  94.4%
8
  88.9%
74
  92.5%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   6.3%
1
  11.1%
0
   0.0%
1
   5.6%
1
  11.1%
4
   5.0%
Eastern Cooperative Oncology Group (ECOG) Performance Status Score   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 9 participants 80 participants
0
7
  36.8%
5
  31.3%
2
  22.2%
1
  11.1%
2
  11.1%
2
  22.2%
19
  23.8%
1
11
  57.9%
6
  37.5%
4
  44.4%
4
  44.4%
13
  72.2%
7
  77.8%
45
  56.3%
2
1
   5.3%
5
  31.3%
3
  33.3%
4
  44.4%
3
  16.7%
0
   0.0%
16
  20.0%
3
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
4
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
5
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
[1]
Measure Description: ECOG Performance Status has 5 Scores.0= Fully active, able to carry on all predisease performance without restriction;1= Restricted in physically strenuous activity but ambulatory and able to carry out work on a light/sedentary nature,2= Ambulatory and capable of all self-care but unable to carry out any work activities. Up and about more than 50% of waking hours;3= Capable of only limited self-care; confined to bed/chair more than 50% of waking hours;4= Completely disabled. Cannot carry on any self-care.Totally confined to bed/chair;5= Dead. Number of participants for each score are reported.
Myelofibrosis subtype   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 9 participants 80 participants
Myelodysplastic Syndromes/ Myeloproliferative Neoplasm (MDS/MPN) or MDS
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
9
 100.0%
9
  11.3%
Blast-phase Myelofibrosis
0
   0.0%
0
   0.0%
8
  88.9%
0
   0.0%
0
   0.0%
0
   0.0%
8
  10.0%
Primary Myelofibrosis
11
  57.9%
8
  50.0%
0
   0.0%
7
  77.8%
9
  50.0%
0
   0.0%
35
  43.8%
Post-Essential Thrombocythemia (ET) Myelofibrosis
1
   5.3%
5
  31.3%
1
  11.1%
2
  22.2%
5
  27.8%
0
   0.0%
14
  17.5%
Post-Polycythemia Vera (PV) Myelofibrosis
7
  36.8%
3
  18.8%
0
   0.0%
0
   0.0%
4
  22.2%
0
   0.0%
14
  17.5%
[1]
Measure Description: Myelofibrosis subtypes included MDS/MPN or MDS, blast-phase myelofibrosis, primary myelofibrosis, post-ET myelofibrosis, and Post-PV myelofibrosis. Number of participants for each subtype are reported.
Dynamic International Prognostic Scoring System (DIPSS)-Plus Risk Status   [1] [2] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 8 participants 79 participants
Intermediate-2 Risk (2 or 3 risk factors)
11
  57.9%
5
  31.3%
1
  11.1%
1
  11.1%
10
  55.6%
6
  75.0%
34
  43.0%
High Risk (4 or more risk factors)
8
  42.1%
11
  68.8%
8
  88.9%
8
  88.9%
8
  44.4%
2
  25.0%
45
  57.0%
[1]
Measure Description: DIPSS stratifies primary myelofibrosis (PMF) into four risk categories (low, intermediate 1, intermediate 2, and high risk), based on 5 clinical factors; Age>65, Hemoglobin <10 grams per deciliter (gm/dL), Leukocytes >10 (9)/L, circulating blasts ≥1%, and constitutional symptoms. Participants for each status are reported. Only categories with data are reported.
[2]
Measure Analysis Population Description: Number analyzed is the number of participants with data available for DIPSS-Plus risk status.
Spliceosomal Mutation Status   [1] [2] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 15 participants 7 participants 9 participants 18 participants 9 participants 77 participants
Had Spliceosomal Mutation
7
  36.8%
4
  26.7%
5
  71.4%
8
  88.9%
0
   0.0%
7
  77.8%
31
  40.3%
Had No Spliceosomal Mutation
12
  63.2%
11
  73.3%
2
  28.6%
1
  11.1%
18
 100.0%
2
  22.2%
46
  59.7%
[1]
Measure Description: Spliceosome mutations represent a group of acquired genetic alterations that affect both myeloid and lymphoid malignancies.
[2]
Measure Analysis Population Description: Number analyzed is the number of participants with data available for spliceosomal mutation status.
Spliceosomal Mutation Type   [1] [2] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants 4 participants 5 participants 8 participants 0 participants 7 participants 31 participants
Splicing Factor 3B Subunit 1 (SF3B1)
1
  14.3%
2
  50.0%
1
  20.0%
1
  12.5%
6
  85.7%
11
  35.5%
Serine And Arginine Rich Splicing Factor 2 (SRSF2)
2
  28.6%
1
  25.0%
2
  40.0%
4
  50.0%
0
   0.0%
9
  29.0%
U2 Small Nuclear RNA Auxiliary Factor 1 (U2AF1)
4
  57.1%
1
  25.0%
2
  40.0%
3
  37.5%
1
  14.3%
11
  35.5%
[1]
Measure Description: Spliceosome mutations represent a group of acquired genetic alterations that affect both myeloid and lymphoid malignancies. Spliceosomal mutation type included SF3B1, SRSF2 and U2AF1. Number of participants for each type are reported.
[2]
Measure Analysis Population Description: Number analyzed is the number of participants with data available for spliceosomal mutation type.
Ringed Sideroblasts Present   [1] [2] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 12 participants 6 participants 9 participants 17 participants 9 participants 69 participants
Yes
4
  25.0%
2
  16.7%
1
  16.7%
3
  33.3%
0
   0.0%
8
  88.9%
18
  26.1%
No
12
  75.0%
10
  83.3%
5
  83.3%
6
  66.7%
17
 100.0%
1
  11.1%
51
  73.9%
[1]
Measure Description: Ring sideroblasts are erythroblasts with iron-loaded mitochondria visualized by Prussian blue staining (Perls' reaction) as a perinuclear ring of blue granules. Refractory anemia with ring sideroblasts (RARS) is a type of MDS that is characterized by anemia and the presence of at least 15 percent ring sideroblasts in the marrow.
[2]
Measure Analysis Population Description: Number analyzed is the number of participants with data available for ringed sideroblasts present.
JAK2V617F Mutation   [1] [2] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 16 participants 14 participants 9 participants 9 participants 18 participants 9 participants 75 participants
Had JAK2V617F Mutation
15
  93.8%
8
  57.1%
5
  55.6%
6
  66.7%
14
  77.8%
3
  33.3%
51
  68.0%
Had No JAK2V617F Mutation
1
   6.3%
5
  35.7%
4
  44.4%
3
  33.3%
4
  22.2%
6
  66.7%
23
  30.7%
Unable to Determine
0
   0.0%
1
   7.1%
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
1
   1.3%
[1]
Measure Description: JAK2 V617F mutation is an acquired, somatic mutation present in the majority of participants with myeloproliferative cancer (myeloproliferative neoplasms) i.e. nearly 100% of participants with polycythemia vera and in about 50% of participants with essential thrombocytosis and primary myelofibrosis.
[2]
Measure Analysis Population Description: Number analyzed is the number of participants with data available for JAK2V617F Mutation.
Transfusion Dependent   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 19 participants 16 participants 9 participants 9 participants 18 participants 9 participants 80 participants
Had Transfusion Dependency
8
  42.1%
5
  31.3%
3
  33.3%
2
  22.2%
4
  22.2%
5
  55.6%
27
  33.8%
Had No Transfusion Dependency
11
  57.9%
11
  68.8%
6
  66.7%
7
  77.8%
14
  77.8%
4
  44.4%
53
  66.3%
[1]
Measure Description: Transfusion dependency was defined by a history of at least 2 units of red blood cell transfusions in the last month for a hemoglobin level of less than 85 grams per deciliter (g/dL) that was not associated with clinically overt bleeding.
1.Primary Outcome
Title MF Participants: Percentage of Participants With Overall Response (OR) - (Clinical Improvement[CI] or Partial Remission[PR] or Complete Remission[CR]) Per International Working Group - Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) Criteria
Hide Description OR:CI/PR/CR per IWG-MRT. CR:Bone marrow (BM):<5% blasts;≤Grade 1 MF, AND Peripheral blood:Hemoglobin (Hb) ≥100 g/liter (g/L) and <upper normal limit (UNL);Neutrophil count(NC) ≥1x10^9/L and <UNL; Platelet(PLT) count ≥100x10^9/L and <UNL;<2% immature myeloid cells(IMCs), AND Clinical: Resolution of disease symptoms; Spleen and liver not palpable; No evidence of extramedullary hematopoeisis(EMH). PR:All CR criteria plus peripheral blood: Hb≥85 but <100g/L and <UNL; NC ≥1x10^9/L and <UNL; PLT count ≥50 but <100x10^9/L and <UNL;<2%IMCs. CI:achievement of anemia (≥20 g/L increase in Hb level), spleen(baseline splenomegaly palpable at 5-10 cm, below left costal margin(LCM), becomes not palpable), symptoms response (50% reduction in total symptom score) without progressive disease (PD) (appearance of new splenomegaly- palpable at least [≥]5 cm below LCM)/increase in severity of anemia, thrombocytopenia/neutropenia. Data is reported separately for arms whose response was assessed per IWG-MRT.
Time Frame Up to first 9 cycles of treatment (each cycle was of 21 days for Arms A and B and 28 days for Arms E and F)
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Hide Analysis Population Description
Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Data is reported for arm groups (Arms A, B, E and F) whose overall response was assessed by IWG-MRT criteria.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])
Hide Arm/Group Description:
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 19 14 9 18
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
36.84
(15.2 to 58.5)
35.7
(10.6 to 60.8)
0 [1] 
(NA to NA)
33.3
(11.6 to 55.1)
[1]
Upper and lower limits of 95% CI were not estimable due to limited number of participants with the event.
2.Primary Outcome
Title Blastic MF/AML Participants: Percentage of Participants With Overall Response
Hide Description For this pilot study, overall response was defined as achievement of a leukemic response (i.e. a clinically meaningful reduction in Blast cells). This was quantified as <5% peripheral blood and bone marrow blasts % that lasts for at least two months. Data is reported separately for this arm where response was assessed by this specific criterion.
Time Frame Up to first 9 cycles of treatment (each cycle was of 28 days for Arm D)
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Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of study drug. Data is reported for Arm D: Imetelstat 9.4 mg/kg (Blastic MF/AML) participants whose overall response was assessed by a specific criterion.
Arm/Group Title Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia)
Hide Arm/Group Description:
Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 9
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
0 [1] 
(NA to NA)
[1]
Upper and lower limits of 95% CI were not estimable due to limited number of participants with the event.
3.Primary Outcome
Title MDS Participants: Percentage of Participants With Overall Response (Hematologic Improvement [HI] or PR or CR) Per IWG Criteria
Hide Description OR: HI/PR/CR per IWG. CR: BM:≤5% myeloblasts (all cell lines normal maturation), Peripheral blood:Hgb ≥11g/dL, PLTs ≥100x10^9/L, Neutrophils ≥1.0x10^9/L, Blasts 0%. PR: All CR criteria if abnormal before treatment except- BM blasts decreased ≥50% over pretreatment but still >5%, Cellularity, morphology not relevant. HI responses:1) Erythroid: Hgb increase ≥1.5 g/dL, Relevant reduction of red blood cell(RBC) units transfusions by absolute ≥4 RBC transfusions/8 week (wk) compared with pretreatment transfusion number in previous 8 wk. Only RBC transfusions given for Hgb of ≤9.0 g/dL.2)PLTs:Absolute increase ≥30x10^9/L starting >20x10^9/L PLTs; Increase from <20x10^9/L to >20x10^9/L and by ≥100%; 3)Neutrophil: ≥100% increase, absolute increase >0.5x10^9/L; 4)Progression/relapse after HI:≥1 of following:≥50% decrement from maximum response levels in granulocytes/PLTs, Reduction in Hgb ≥1.5 g/dL,Transfusion dependence. Data is reported separately for arm whose response was assessed per IWG.
Time Frame Up to first 9 cycles of treatment (each cycle was of 28 days for Arm G)
Hide Outcome Measure Data
Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of the study drug. Data is reported for Arm G: Imetelstat 9.4 mg/kg (MDS/MPN or MDS and spliceosome mutations or ring sideroblasts) participants whose overall response was assessed by IWG criteria.
Arm/Group Title Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description:
Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 9
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
33.3
(2.5 to 64.1)
4.Secondary Outcome
Title Percentage of Participants With Treatment Emergent Adverse Events (TEAEs), Grade >= 3 TEAEs and Treatment Related Adverse Events
Hide Description TEAEs defined as those events that 1) occur on or after the first dose of study drug, through the treatment phase, and for 30 days following the last dose of study drug or until subsequent anti-cancer therapy if earlier; 2) any event that is considered study drug-related regardless of the start date of the event; or 3) any event that is present at baseline but worsens in severity or is subsequently considered drug-related by the investigator. Grade >=3 TEAE defined as events that are severe, life-threatening or disabling, or fatal and considered related to imetelstat as per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03. An AE was considered related to the study drug if the event was assessed by the investigator as probably or possibly related.
Time Frame From first dose of study drug up to 30 days from the last dose of study drug or until subsequent anti-cancer therapy if earlier (Up to approximately 5.7 years)
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Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of imetelstat.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description:
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 19 16 9 9 18 9
Measure Type: Number
Unit of Measure: percentage of participants
TEAEs 100 100 100 100 100 100
Grade >=3 TEAEs 100 93.5 100 77.8 83.3 100
Treatment Related AEs 89.5 75 88.9 77.8 83.3 100
5.Secondary Outcome
Title Number of Participants With Spleen Response Per IWG-MRT Criteria
Hide Description Spleen response per IWG-MRT criteria defined as baseline splenomegaly that is palpable at 5-10 cm, below the LCM, becomes not palpable, OR A baseline splenomegaly that is palpable at >10 cm, below the LCM, decreases by ≥50%. Baseline splenomegaly that is palpable at <5 cm, below the LCM, is not eligible for spleen response. Confirmation by Magnetic resonance imaging (MRI) or computerized tomography (CT) showing ≥35% spleen volume reduction is recommended (but not required). Spleen response was assessed only in participants with MF.
Time Frame Up to approximately 5.7 years
Hide Outcome Measure Data
Hide Analysis Population Description
Arms A, B, E and F: Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Arm D: All Treated Analysis Set included all participants who received at least one dose of imetelstat.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])
Hide Arm/Group Description:
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 19 14 9 9 18
Measure Type: Count of Participants
Unit of Measure: Participants
3
  15.8%
1
   7.1%
1
  11.1%
0
   0.0%
2
  11.1%
6.Secondary Outcome
Title MF and Blastic MF/AML Participants: Number of Participants With Transfusion Independence (CI by Anemia Response) Per IWG-MRT
Hide Description Transfusion dependency was defined by a history of at least 2 units of red blood cell transfusions in the last month for a hemoglobin level of less than 85 g/L that was not associated with clinically overt bleeding. A participant who was transfusion dependent at baseline was considered transfusion independent if the participant met either of the following conditions: Received no more than 1 unit of red blood cell transfusions in a rolling time interval of 30 days or more, and had a hemoglobin level increase over baseline more than 2 g/dL if participant baseline hemoglobin level was less than 85 g/L and received no transfusion. Anemia response per IWG-MRT Criteria- Transfusion-independent participants: a ≥20 g/L increase in hemoglobin level. Transfusion-dependent participants: becoming transfusion-independent. Data is reported separately for arms assessed as per the IWG-MRT criteria.
Time Frame Up to approximately 5.7 years
Hide Outcome Measure Data
Hide Analysis Population Description
Arms A, B, E, F: Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Arm D: All Treated Analysis Set included all participants who received at least one dose of imetelstat, assessed per IWG-MRT.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])
Hide Arm/Group Description:
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 19 14 9 9 18
Measure Type: Count of Participants
Unit of Measure: Participants
1
   5.3%
0
   0.0%
0
   0.0%
0
   0.0%
3
  16.7%
7.Secondary Outcome
Title MDS Participants: Number of Participants With Transfusion Independence (HI by Erythroid Response) Per IWG Criteria
Hide Description Transfusion dependency was defined by a history of at least 2 units of red blood cell transfusions in the last month for a hemoglobin level of less than 85 g/L that was not associated with clinically overt bleeding. A participant who was transfusion dependent at baseline was considered transfusion independent if the participant met either of the following conditions: Received no more than 1 unit of red blood cell transfusions in a rolling time interval of 30 days or more, and had a hemoglobin level increase over baseline more than 2 g/dL if participant baseline hemoglobin level was less than 85 g/L and received no transfusion. HI responses included: 1) Erythroid: Hgb increase ≥1.5 g/dL, Relevant reduction of RBC units transfusions by absolute number of >=4 RBC transfusions/8 week compared with pretreatment transfusion number in previous 8 week. Only RBC transfusions given for Hgb of ≤9.0 g/dL. Data is reported separately for arm assessed as per IWG criteria.
Time Frame Up to approximately 5.7 years
Hide Outcome Measure Data
Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of study drug. Data is reported for Arm G: Imetelstat 9.4 mg/kg (MDS/MPN or MDS and spliceosome mutations or ring sideroblasts) participants whose transfusion independence (HI by erythroid response) was assessed by IWG criteria.
Arm/Group Title Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description:
Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 9
Measure Type: Count of Participants
Unit of Measure: Participants
3
  33.3%
8.Secondary Outcome
Title MF Participants: Time to Response
Hide Description Time to response was defined as the duration from study Day 1 to the earliest date that a response is first documented. The response CI/CR/PR was assessed by IWG-MRT criteria.
Time Frame From study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
Hide Outcome Measure Data
Hide Analysis Population Description
Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Data is reported for arm groups (Arms A, B, E and F) whose overall response was assessed by IWG-MRT criteria. Only responders were analyzed for this outcome measure.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])
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Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 7 5 0 6
Median (Full Range)
Unit of Measure: months
2.1
(0.7 to 3.5)
1.4
(0.7 to 5.6)
2.9
(1.9 to 12.1)
9.Secondary Outcome
Title Blastic MF/AML Participants: Time to Response
Hide Description Time to response was defined as the duration from study Day 1 to the earliest date that a response is first documented. Response was defined as achievement of a leukemic response (i.e. a clinically meaningful reduction in Blast cells). This was quantified as <5% peripheral blood and bone marrow blasts % that lasts for at least two months. Data for time to response is reported as per criteria of response assessment.
Time Frame From study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
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Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of the study drug. Only responders were analyzed for this outcome measure. Data is reported for Arm D: Imetelstat 9.4 mg/kg (Blastic MF/AML) participants whose overall response was assessed by a specific criterion.
Arm/Group Title Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia)
Hide Arm/Group Description:
Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
10.Secondary Outcome
Title MDS Participants: Time to Response
Hide Description Time to response was defined as the duration from study Day 1 to the earliest date that a response is first documented. Response was defined as HI, PR or CR per IWG criteria. Data for time to response is reported as per criteria of response assessment.
Time Frame From study Day 1 to the earliest date that a response was first documented (Up to approximately 5.7 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of the study drug. Only responders were analyzed for this outcome measure. Data is reported for Arm G: Imetelstat 9.4 mg/kg (MDS/MPN or MDS and spliceosome mutations or ring sideroblasts) participants whose overall response was assessed by IWG criteria.
Arm/Group Title Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description:
Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 3
Median (Full Range)
Unit of Measure: months
3.7
(2.8 to 4.9)
11.Secondary Outcome
Title MF Participants: Duration of Response (DOR) Per IWG-MRT Criteria
Hide Description DOR measured from time of initial response (CR/PR/CI) until documented PD or death whichever occurs first. If no PD/death occurs DOR is censored at last disease evaluation date for responders. CR:BM: <5%blasts; ≤Grade 1 MF, AND Peripheral blood:Hb≥100 g/L and <UNL;Neutrophil count≥1x10^9/L and <UNL;PLT count≥100x10^9/L and <UNL;<2% IMCs, AND Clinical:Resolution of disease symptoms;Spleen and liver not palpable;No evidence of EMH.PR:All CR criteria plus peripheral blood:Hb≥85 but <100g/L and <UNL;NC≥1x10^9/L and <UNL;PLTcount ≥50 but<100x10^9/L and<UNL;<2%IMCs. CI:achievement of anemia(≥20 g/L increase Hb level),spleen(baseline splenomegaly-palpable at 5-10cm,below LCM,becomes not palpable) or symptoms response(50% reduction in total symptom score) without PD(appearance of new splenomegaly- palpable[>= 5 cm below LCM) or increase in severity of anemia, thrombocytopenia or neutropenia. Data is reported separately for arms whose DOR was assessed per IWG-MRT. Kaplan-Meier method was used.
Time Frame From time of initial response until documented disease progression or death whichever occurs first (Up to approximately 5.7 years)
Hide Outcome Measure Data
Hide Analysis Population Description
Eligible Analysis Set included subset of all participants who received at least one dose of study drug that excludes ineligible participants who did not satisfy each and every eligibility criteria for study entry. Data is reported for arm groups whose DOR was assessed by IWG-MRT criteria. Only responders were analyzed for this outcome measure.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts])
Hide Arm/Group Description:
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 7 5 0 6
Median (95% Confidence Interval)
Unit of Measure: months
24.36
(17.94 to 40.74)
NA [1] 
(16.59 to NA)
35.52 [2] 
(NA to NA)
[1]
Median and upper limit of 95% CI were not estimable due to limited number of participants with the event.
[2]
Upper and lower limits of 95% CI were not estimable due to limited number of participants with the event.
12.Secondary Outcome
Title Blastic MF/AML Participants: Duration of Response
Hide Description DOR was measured from the time of initial response until documented disease progression or death whichever occurs first. If no PD/death occurs the DOR is censored at last disease evaluation date for responders. Response is defined as achievement of <5% peripheral blood and bone marrow blast % that lasts for at least two months. PD is the appearance of new splenomegaly that is palpable at least 5 cm below the LCM. Data is reported separately for arm whose DOR was assessed by a specific criterion. Kaplan-Meier method was used.
Time Frame From time of initial response until documented disease progression or death whichever occurs first (Up to approximately 5.7 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of the study drug. Data is reported for Arm D: Imetelstat 9.4 mg/kg (Blastic MF/AML) participants whose DOR was assessed by a specific criterion. Only responders were analyzed for this outcome measure.
Arm/Group Title Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia)
Hide Arm/Group Description:
Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
13.Secondary Outcome
Title MDS Participants: Duration of Response Per IWG Criteria
Hide Description DOR: time of initial response (CR/PR/HI) until documented PD/death whichever occurs first. If no PD/death occurs DOR is censored at last disease evaluation date for responders. Data reported separately for arm assessed per IWG. Kaplan-Meier method was used.
Time Frame From the time of initial response (CR/PR/HI) until documented disease progression or death whichever occurs first (Up to approximately 5.7 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of the study drug. Data is reported for Arm G: Imetelstat 9.4 mg/kg (MDS/MPN or MDS and spliceosome mutations or ring sideroblasts) participants whose DOR was assessed by IWG criteria. Only responders were analyzed for this outcome measure.
Arm/Group Title Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description:
Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 3
Median (95% Confidence Interval)
Unit of Measure: months
NA [1] 
(NA to NA)
[1]
Median, upper and lower limits of 95% CI were not estimable as all DOR of responder participants were censored .
14.Secondary Outcome
Title Overall Survival (OS)
Hide Description OS was defined as the as the interval from Study Day 1 to the date of death from any cause. Survival time of living participants was censored on the last date a participant is known to be alive or lost to follow-up. Overall Survival was estimated by Kaplan-Meier method.
Time Frame From Study Day 1 to the date of death from any cause (Up to approximately 5.7 years)
Hide Outcome Measure Data
Hide Analysis Population Description
All Treated Analysis Set included all participants who received at least one dose of imetelstat.
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia) Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description:
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
Overall Number of Participants Analyzed 19 16 9 9 18 9
Median (95% Confidence Interval)
Unit of Measure: months
42.61
(19.12 to 56.77)
26.73
(16.46 to 36.17)
4.93
(1.05 to 15.67)
12.09
(7.06 to 27.14)
NA [1] 
(12.22 to NA)
28.42 [2] 
(6.64 to NA)
[1]
Median and upper limit of 95% CI were not estimable due to limited number of participants with the event.
[2]
Upper limit of 95% CI was not estimable due to limited number of participants with the event.
Time Frame From first dose of study drug to the last dose of study drug or until subsequent anti-cancer therapy if earlier (Up to approximately 5.7 years)
Adverse Event Reporting Description All treated analysis set included all participants who received at least 1 dose of Imetelstat. All non-serious adverse events (at 5% threshold) and serious adverse event including requirements for expedited reporting of serious adverse events occurring within 30 days of the last dose of study drug are reported.
 
Arm/Group Title Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Hide Arm/Group Description Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of each 21-day cycle in Core Phase up to 9 cycles. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF regardless of spliceosome mutation status or presence of ring sideroblasts received imetelstat 9.4 mg/kg, IV as 2-hour infusion on Days 1, 8, and 15 of 21-day cycle in Cycle 1 followed by 9.4 mg/kg on Day 1 of each 21-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with blast-phase MF/AML received imetelstat 9.4 mg/kg, IV as 2-hour infusion weekly on Days 1, 8, 15, 22 of a 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF and spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with MF without spliceosome mutations or ring sideroblasts present received 2 cycles (Cycles 1-2) of imetelstat 9.4 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or imetelstat 7.5 mg/kg twice weekly on Days 1, 3 for 2 cycles of 28-day cycle (Cycles 3-4) followed by imetelstat 7.5 mg/kg 3-times-weekly on Days 1, 3, 5 of Cycles 5 and beyond of 28-day cycle up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years). Participants with either MDS/MPN or MDS and spliceosome mutations or ring sideroblasts present received 2 cycles of 28-day cycle (Cycles 1-2) of imetelstat 7.5 mg/kg, IV as 2-hour infusion on Day 1 of a 28-day cycle followed by maintenance therapy with the same regimen or the possibility of dose escalation to imetelstat 9.4 mg/kg weekly if response to the initial dose was insufficient up to 9 cycles in the Core Phase. Participants could receive imetelstat beyond Cycle 9 in the Extension Phase if they did not meet any of the withdrawal criteria, did not have disease progression and were receiving clinical benefit from treatment as determined by the investigator. Following treatment discontinuation, participants entered the Event Monitoring Phase for collection of survival status, disease status, and subsequent treatment information (Up to approximately 5.7 years).
All-Cause Mortality
Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   13/19 (68.42%)   15/16 (93.75%)   9/9 (100.00%)   8/9 (88.89%)   9/18 (50.00%)   6/9 (66.67%) 
Hide Serious Adverse Events
Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   17/19 (89.47%)   12/16 (75.00%)   9/9 (100.00%)   7/9 (77.78%)   14/18 (77.78%)   9/9 (100.00%) 
Blood and lymphatic system disorders             
Anaemia  1  9/19 (47.37%)  10/16 (62.50%)  8/9 (88.89%)  5/9 (55.56%)  7/18 (38.89%)  8/9 (88.89%) 
Cardiac disorders             
Atrial fibrillation  1  2/19 (10.53%)  1/16 (6.25%)  2/9 (22.22%)  0/9 (0.00%)  0/18 (0.00%)  0/9 (0.00%) 
Cardiac failure  1  1/19 (5.26%)  0/16 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  2/18 (11.11%)  1/9 (11.11%) 
General disorders             
Fatigue  1  1/19 (5.26%)  1/16 (6.25%)  2/9 (22.22%)  1/9 (11.11%)  0/18 (0.00%)  1/9 (11.11%) 
Infections and infestations             
Lung infection  1  0/19 (0.00%)  1/16 (6.25%)  3/9 (33.33%)  1/9 (11.11%)  2/18 (11.11%)  1/9 (11.11%) 
Sepsis  1  0/19 (0.00%)  0/16 (0.00%)  3/9 (33.33%)  0/9 (0.00%)  0/18 (0.00%)  1/9 (11.11%) 
Investigations             
Platelet count decreased  1  10/19 (52.63%)  5/16 (31.25%)  8/9 (88.89%)  5/9 (55.56%)  3/18 (16.67%)  1/9 (11.11%) 
Neutrophil count decreased  1  4/19 (21.05%)  6/16 (37.50%)  5/9 (55.56%)  1/9 (11.11%)  3/18 (16.67%)  5/9 (55.56%) 
White blood cell count decreased  1  3/19 (15.79%)  6/16 (37.50%)  1/9 (11.11%)  0/9 (0.00%)  4/18 (22.22%)  2/9 (22.22%) 
Respiratory, thoracic and mediastinal disorders             
Epistaxis  1  2/19 (10.53%)  0/16 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (12.0)
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Arm A: Imetelstat 9.4 mg/kg (MF) Arm B: Imetelstat 9.4 mg/kg as Induction + Maintenance (MF) Arm D: Imetelstat 9.4 mg/kg (Blast-phase MF/Acute Myeloid Leukemia Arm E: Imetelstat 7.5 - 9.4 mg/kg (MF [With Spliceosome Mutation or Ring Sideroblasts]) Arm F: Imetelstat 7.5 - 9.4 mg/kg (MF [Without Spliceosome Mutation and Ring Sideroblasts]) Arm G: Imetelstat 7.5 - 9.4 mg/kg (MDS/MPN or MDS With Spliceosome Mutations or Ring Sideroblasts)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   19/19 (100.00%)   16/16 (100.00%)   9/9 (100.00%)   9/9 (100.00%)   18/18 (100.00%)   9/9 (100.00%) 
Blood and lymphatic system disorders             
Anaemia  1  16/19 (84.21%)  9/16 (56.25%)  4/9 (44.44%)  6/9 (66.67%)  11/18 (61.11%)  7/9 (77.78%) 
Cardiac disorders             
Atrial fibrillation  1  2/19 (10.53%)  0/16 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  1/9 (11.11%) 
Cardiac failure  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  2/18 (11.11%)  2/9 (22.22%) 
Sinus tachycardia  1  1/19 (5.26%)  0/16 (0.00%)  2/9 (22.22%)  0/9 (0.00%)  3/18 (16.67%)  0/9 (0.00%) 
Eye disorders             
Eye disorders  1  1/19 (5.26%)  1/16 (6.25%)  1/9 (11.11%)  0/9 (0.00%)  4/18 (22.22%)  0/9 (0.00%) 
Gastrointestinal disorders             
Abdominal distension  1  1/19 (5.26%)  0/16 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Abdominal pain  1  7/19 (36.84%)  1/16 (6.25%)  6/9 (66.67%)  2/9 (22.22%)  6/18 (33.33%)  0/9 (0.00%) 
Abdominal pain upper  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Constipation  1  4/19 (21.05%)  1/16 (6.25%)  1/9 (11.11%)  1/9 (11.11%)  1/18 (5.56%)  2/9 (22.22%) 
Diarrhoea  1  19/19 (100.00%)  15/16 (93.75%)  8/9 (88.89%)  9/9 (100.00%)  18/18 (100.00%)  9/9 (100.00%) 
Nausea  1  8/19 (42.11%)  7/16 (43.75%)  6/9 (66.67%)  1/9 (11.11%)  12/18 (66.67%)  4/9 (44.44%) 
Vomiting  1  7/19 (36.84%)  3/16 (18.75%)  1/9 (11.11%)  1/9 (11.11%)  5/18 (27.78%)  1/9 (11.11%) 
General disorders             
Chills  1  3/19 (15.79%)  2/16 (12.50%)  2/9 (22.22%)  1/9 (11.11%)  2/18 (11.11%)  0/9 (0.00%) 
Early satiety  1  4/19 (21.05%)  3/16 (18.75%)  3/9 (33.33%)  3/9 (33.33%)  1/18 (5.56%)  1/9 (11.11%) 
Fatigue  1  18/19 (94.74%)  15/16 (93.75%)  8/9 (88.89%)  8/9 (88.89%)  18/18 (100.00%)  9/9 (100.00%) 
Infusion related reaction  1  2/19 (10.53%)  3/16 (18.75%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Non-cardiac chest pain  1  2/19 (10.53%)  0/16 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Oedema peripheral  1  7/19 (36.84%)  3/16 (18.75%)  6/9 (66.67%)  3/9 (33.33%)  5/18 (27.78%)  2/9 (22.22%) 
Pain  1  6/19 (31.58%)  5/16 (31.25%)  3/9 (33.33%)  2/9 (22.22%)  8/18 (44.44%)  5/9 (55.56%) 
Pyrexia  1  5/19 (26.32%)  2/16 (12.50%)  4/9 (44.44%)  1/9 (11.11%)  2/18 (11.11%)  1/9 (11.11%) 
Infections and infestations             
Lung infection  1  1/19 (5.26%)  1/16 (6.25%)  3/9 (33.33%)  1/9 (11.11%)  2/18 (11.11%)  0/9 (0.00%) 
Sinusitis  1  2/19 (10.53%)  0/16 (0.00%)  2/9 (22.22%)  0/9 (0.00%)  0/18 (0.00%)  1/9 (11.11%) 
Upper respiratory tract infection  1  1/19 (5.26%)  0/16 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  4/18 (22.22%)  2/9 (22.22%) 
Injury, poisoning and procedural complications             
Contusion  1  3/19 (15.79%)  3/16 (18.75%)  1/9 (11.11%)  1/9 (11.11%)  3/18 (16.67%)  3/9 (33.33%) 
Fall  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Infusion related reaction  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  3/18 (16.67%)  1/9 (11.11%) 
Investigations             
Activated partial thromboplastin time prolonged  1  10/19 (52.63%)  3/16 (18.75%)  5/9 (55.56%)  2/9 (22.22%)  7/18 (38.89%)  0/9 (0.00%) 
Alanine aminotransferase increased  1  8/19 (42.11%)  3/16 (18.75%)  4/9 (44.44%)  2/9 (22.22%)  6/18 (33.33%)  5/9 (55.56%) 
Aspartate aminotransferase increased  1  10/19 (52.63%)  8/16 (50.00%)  4/9 (44.44%)  3/9 (33.33%)  6/18 (33.33%)  5/9 (55.56%) 
Blood alkaline phosphatase increased  1  10/19 (52.63%)  10/16 (62.50%)  3/9 (33.33%)  4/9 (44.44%)  5/18 (27.78%)  1/9 (11.11%) 
Blood amylase increased  1  3/19 (15.79%)  0/16 (0.00%)  2/9 (22.22%)  1/9 (11.11%)  2/18 (11.11%)  2/9 (22.22%) 
Blood bilirubin increased  1  7/19 (36.84%)  5/16 (31.25%)  1/9 (11.11%)  1/9 (11.11%)  5/18 (27.78%)  3/9 (33.33%) 
Blood creatinine increased  1  4/19 (21.05%)  2/16 (12.50%)  4/9 (44.44%)  0/9 (0.00%)  4/18 (22.22%)  4/9 (44.44%) 
Gamma-glutamyltransferase increased  1  2/19 (10.53%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  4/18 (22.22%)  1/9 (11.11%) 
Lipase increased  1  3/19 (15.79%)  1/16 (6.25%)  3/9 (33.33%)  2/9 (22.22%)  3/18 (16.67%)  3/9 (33.33%) 
Lymphocyte count decreased  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  2/18 (11.11%)  1/9 (11.11%) 
Neutrophil count decreased  1  19/19 (100.00%)  16/16 (100.00%)  9/9 (100.00%)  9/9 (100.00%)  18/18 (100.00%)  9/9 (100.00%) 
Platelet count decreased  1  19/19 (100.00%)  16/16 (100.00%)  9/9 (100.00%)  9/9 (100.00%)  18/18 (100.00%)  9/9 (100.00%) 
Weight decreased  1  1/19 (5.26%)  4/16 (25.00%)  0/9 (0.00%)  0/9 (0.00%)  6/18 (33.33%)  2/9 (22.22%) 
Weight increased  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  2/18 (11.11%)  2/9 (22.22%) 
White blood cell count decreased  1  19/19 (100.00%)  16/16 (100.00%)  9/9 (100.00%)  9/9 (100.00%)  18/18 (100.00%)  9/9 (100.00%) 
Metabolism and nutrition disorders             
Anorexia  1  4/19 (21.05%)  5/16 (31.25%)  4/9 (44.44%)  1/9 (11.11%)  4/18 (22.22%)  2/9 (22.22%) 
Decreased appetite  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  3/18 (16.67%)  2/9 (22.22%) 
Hyperglycaemia  1  9/19 (47.37%)  10/16 (62.50%)  7/9 (77.78%)  4/9 (44.44%)  13/18 (72.22%)  7/9 (77.78%) 
Hyperkalaemia  1  3/19 (15.79%)  4/16 (25.00%)  2/9 (22.22%)  2/9 (22.22%)  3/18 (16.67%)  1/9 (11.11%) 
Hypernatraemia  1  0/19 (0.00%)  0/16 (0.00%)  2/9 (22.22%)  0/9 (0.00%)  2/18 (11.11%)  2/9 (22.22%) 
Hyperuricaemia  1  8/19 (42.11%)  5/16 (31.25%)  2/9 (22.22%)  2/9 (22.22%)  8/18 (44.44%)  1/9 (11.11%) 
Hypocalcaemia  1  4/19 (21.05%)  2/16 (12.50%)  4/9 (44.44%)  0/9 (0.00%)  3/18 (16.67%)  2/9 (22.22%) 
Hypoglycaemia  1  3/19 (15.79%)  1/16 (6.25%)  3/9 (33.33%)  0/9 (0.00%)  1/18 (5.56%)  0/9 (0.00%) 
Hypokalaemia  1  4/19 (21.05%)  0/16 (0.00%)  2/9 (22.22%)  0/9 (0.00%)  3/18 (16.67%)  2/9 (22.22%) 
Hyponatraemia  1  4/19 (21.05%)  2/16 (12.50%)  3/9 (33.33%)  0/9 (0.00%)  2/18 (11.11%)  2/9 (22.22%) 
Musculoskeletal and connective tissue disorders             
Arthralgia  1  2/19 (10.53%)  4/16 (25.00%)  2/9 (22.22%)  1/9 (11.11%)  2/18 (11.11%)  2/9 (22.22%) 
Back pain  1  5/19 (26.32%)  4/16 (25.00%)  1/9 (11.11%)  1/9 (11.11%)  3/18 (16.67%)  4/9 (44.44%) 
Bone pain  1  5/19 (26.32%)  1/16 (6.25%)  0/9 (0.00%)  1/9 (11.11%)  2/18 (11.11%)  0/9 (0.00%) 
Muscular weakness  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  3/18 (16.67%)  2/9 (22.22%) 
Musculoskeletal pain  1  2/19 (10.53%)  1/16 (6.25%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Myalgia  1  3/19 (15.79%)  2/16 (12.50%)  2/9 (22.22%)  1/9 (11.11%)  1/18 (5.56%)  0/9 (0.00%) 
Neck pain  1  0/19 (0.00%)  0/16 (0.00%)  1/9 (11.11%)  0/9 (0.00%)  1/18 (5.56%)  2/9 (22.22%) 
Pain in extremity  1  3/19 (15.79%)  3/16 (18.75%)  3/9 (33.33%)  1/9 (11.11%)  2/18 (11.11%)  2/9 (22.22%) 
Nervous system disorders             
Dizziness  1  3/19 (15.79%)  0/16 (0.00%)  3/9 (33.33%)  1/9 (11.11%)  0/18 (0.00%)  1/9 (11.11%) 
Headache  1  4/19 (21.05%)  2/16 (12.50%)  0/9 (0.00%)  0/9 (0.00%)  3/18 (16.67%)  2/9 (22.22%) 
Peripheral sensory neuropathy  1  2/19 (10.53%)  0/16 (0.00%)  1/9 (11.11%)  1/9 (11.11%)  0/18 (0.00%)  0/9 (0.00%) 
Psychiatric disorders             
Anxiety  1  0/19 (0.00%)  2/16 (12.50%)  1/9 (11.11%)  0/9 (0.00%)  2/18 (11.11%)  1/9 (11.11%) 
Insomnia  1  3/19 (15.79%)  2/16 (12.50%)  2/9 (22.22%)  0/9 (0.00%)  1/18 (5.56%)  2/9 (22.22%) 
Respiratory, thoracic and mediastinal disorders             
Cough  1  5/19 (26.32%)  1/16 (6.25%)  4/9 (44.44%)  3/9 (33.33%)  5/18 (27.78%)  2/9 (22.22%) 
Dyspnoea  1  5/19 (26.32%)  2/16 (12.50%)  7/9 (77.78%)  2/9 (22.22%)  5/18 (27.78%)  2/9 (22.22%) 
Epistaxis  1  5/19 (26.32%)  3/16 (18.75%)  1/9 (11.11%)  1/9 (11.11%)  4/18 (22.22%)  1/9 (11.11%) 
Pulmonary hypertension  1  3/19 (15.79%)  0/16 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Skin and subcutaneous tissue disorders             
Alopecia  1  1/19 (5.26%)  3/16 (18.75%)  2/9 (22.22%)  0/9 (0.00%)  1/18 (5.56%)  2/9 (22.22%) 
Hyperhidrosis  1  1/19 (5.26%)  1/16 (6.25%)  0/9 (0.00%)  1/9 (11.11%)  2/18 (11.11%)  2/9 (22.22%) 
Night sweats  1  0/19 (0.00%)  2/16 (12.50%)  1/9 (11.11%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
Pruritus  1  5/19 (26.32%)  1/16 (6.25%)  0/9 (0.00%)  1/9 (11.11%)  4/18 (22.22%)  0/9 (0.00%) 
Vascular disorders             
Haematoma  1  1/19 (5.26%)  2/16 (12.50%)  1/9 (11.11%)  0/9 (0.00%)  0/18 (0.00%)  0/9 (0.00%) 
Hypertension  1  1/19 (5.26%)  0/16 (0.00%)  0/9 (0.00%)  0/9 (0.00%)  5/18 (27.78%)  2/9 (22.22%) 
Hypotension  1  1/19 (5.26%)  0/16 (0.00%)  3/9 (33.33%)  0/9 (0.00%)  1/18 (5.56%)  1/9 (11.11%) 
1
Term from vocabulary, MedDRA (12.0)
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Consistent with Good Publication Practices and ICMJE guidelines, the sponsor shall have right to publish such primary (multicenter) data and information without approval from investigator. Investigator has right to publish study site-specific data after primary data published. If an investigator wishes to publish information from study, a copy of manuscript must be provided to sponsor for review at least 60 days before submission for publication or presentation
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Geron Corp.
Phone: 650-473-7700
EMail: myf2001-info@geron.com
Layout table for additonal information
Responsible Party: Geron Corporation
ClinicalTrials.gov Identifier: NCT01731951    
Other Study ID Numbers: CR107110
CP14B019 ( Other Identifier: Janssen Research & Development, LLC )
First Submitted: November 18, 2012
First Posted: November 22, 2012
Results First Submitted: July 8, 2021
Results First Posted: September 21, 2021
Last Update Posted: September 21, 2021