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CPX-351 in Treating Patients With Relapsed or Refractory Acute Myeloid Leukemia or Myelodysplastic Syndrome

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ClinicalTrials.gov Identifier: NCT02019069
Recruitment Status : Completed
First Posted : December 24, 2013
Results First Posted : January 15, 2019
Last Update Posted : January 21, 2019
Sponsor:
Collaborator:
National Cancer Institute (NCI)
Information provided by (Responsible Party):
Rondeep Brar, Stanford University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Adult Acute Erythroid Leukemia (M6)
Adult Acute Megakaryoblastic Leukemia (M7)
Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Adult Acute Monoblastic Leukemia and Acute Monocytic Leukemia (M5)
Adult Acute Myeloblastic Leukemia With Maturation (M2)
Adult Acute Myeloblastic Leukemia Without Maturation (M1)
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
Adult Acute Myeloid Leukemia With Del(5q)
Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
Adult Acute Myeloid Leukemia With t(8;21)(q22;q22)
Adult Acute Myelomonocytic Leukemia (M4)
de Novo Myelodysplastic Syndromes
Previously Treated Myelodysplastic Syndromes
Recurrent Adult Acute Myeloid Leukemia
Secondary Myelodysplastic Syndromes
Intervention Drug: liposomal cytarabine-daunorubicin CPX-351
Enrollment 11
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Period Title: Overall Study
Started 11
Completed 11
Not Completed 0
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Baseline Participants 11
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
<=18 years
0
   0.0%
Between 18 and 65 years
0
   0.0%
>=65 years
11
 100.0%
Age, Continuous  
Median (Standard Deviation)
Unit of measure:  Years
Number Analyzed 11 participants
74  (4.9)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Female
2
  18.2%
Male
9
  81.8%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
Hispanic or Latino
2
  18.2%
Not Hispanic or Latino
9
  81.8%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 11 participants
American Indian or Alaska Native
0
   0.0%
Asian
1
   9.1%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
8
  72.7%
More than one race
0
   0.0%
Unknown or Not Reported
2
  18.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 11 participants
11
1.Primary Outcome
Title Response Rate (RR)
Hide Description

The response rate was determined as the sum of complete response calculated by adding the total complete response (CR) and complete response with incomplete count recovery (CRi). The outcome is reported as the total number without dispersion.

  • CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC > 1000/uL, with transfusion independence.
  • CRi = all the parameters for CR, but platelets < 100,000/uL and/or ANC ≤ 1000/uL.
Time Frame Day 42
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
3
  27.3%
2.Secondary Outcome
Title Complete Response With Incomplete Count Recovery (CRi)
Hide Description

Complete response (CR) with incomplete count recovery (CRi) was determined as the number of participants who achieved CRi after induction therapy. The outcome is reported as the total number or participants without dispersion.

  • CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC > 1000/uL, with transfusion independence.
  • CRi = all the parameters for CR, but platelets < 100,000/uL and/or ANC ≤ 1000/uL.
Time Frame Day 42
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
1
   9.1%
3.Secondary Outcome
Title Complete Response (CR)
Hide Description

Complete response (CR) was determined the number of participants who achieved CR by Day 42 after induction treatment. The outcome is reported as the total number of participants without dispersion.

• CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC > 1000/uL, with transfusion independence.

Time Frame Day 42
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
2
  18.2%
4.Secondary Outcome
Title Duration of Remission (DOR) Following Induction With CPX-351
Hide Description

Duration of remission (DOR) was assessed as the length of time from documented complete response (CR) or complete response with incomplete count recovery (CRi) until documented lost of response, relapse, or death. The outcome is reported as the median with full range.

  • CR = less than 5% blasts; no blasts with auer rods; and no persistence of extramedullary disease, with blood count recovery to platelets ≥ 100,000/uL and ANC > 1000/uL, with transfusion independence.
  • CRi = all the parameters for CR, but platelets < 100,000/uL and/or ANC ≤ 1000/uL.

For patients remaining alive, duration of remission (DOR) is reported as the length of time from documented complete response (CR) or complete response with incomplete count recovery (CRi) until the most recent assessment.

Time Frame Up to 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Median (Full Range)
Unit of Measure: days
185
(25 to 329)
5.Secondary Outcome
Title Overall Survival (OS)
Hide Description Overall survival (OS) was assessed as the number of participants remaining alive 12 months, starting from date of entry into trial. The outcome is reported as the number of participants (without dispersion).
Time Frame At 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
1
   9.1%
6.Secondary Outcome
Title Early Induction Mortality (Day 30 After 1st Induction)
Hide Description Early induction mortality was assessed as the number of participants who died within 30 days of completing the 1st cycle of CPX-351 (1st induction). The outcome is reported as the number of participants without dispersion.
Time Frame 30 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
2
  18.2%
7.Secondary Outcome
Title Mortality at Day 60 After 1st Induction
Hide Description Mortality at Day 60 after 1st induction was assessed as the number of participants who died within 60 days of completing the 1st cycle of CPX-351 (1st induction). The outcome is reported as the number of participants without dispersion.
Time Frame 60 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
3
  27.3%
8.Secondary Outcome
Title Participants Experiencing of Serious Adverse Events
Hide Description Serious adverse events per participant were assessed as serious adverse events per 21CFR§312.32 that were Grade 3 or greater, and independent of relationship to CPX-351. The outcome is reported as the number of participants that experienced any defined SAE, a number without dispersion.
Time Frame Up to 4 weeks after completion of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Count of Participants
Unit of Measure: Participants
5
  45.5%
9.Secondary Outcome
Title Serious Adverse Events
Hide Description Serious adverse events were assessed as serious adverse events per 21CFR§312.32 that were Grade 3 or greater, and independent of relationship to CPX-351. The outcome is reported as the total number of the defined SAEs, a number without dispersion.
Time Frame Up to 4 weeks after completion of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Per protocol, the outcome only includes SAEs that are Grade 3 or greater. Adverse events that were Grade 2 or less, (eg, Grade 2 event with hospitalization) are not included.
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description:
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

Overall Number of Participants Analyzed 11
Measure Type: Number
Unit of Measure: Adverse events
8
Time Frame Up to 4 weeks after completion of treatment]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Liposomal Cytarabine-daunorubicin CPX-351
Hide Arm/Group Description
  • 1st INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV at a dose of 65 units/m2/day over 90 minutes on days 1, 3, and 5.
  • 2nd INDUCTION: Patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.
  • CONSOLIDATION: Beginning on day 28, patients receive liposomal cytarabine-daunorubicin CPX-351 IV a dose of 65 units/m2/day over 90 minutes on days 1 and 3.

liposomal cytarabine-daunorubicin CPX-351: Given IV

All-Cause Mortality
Liposomal Cytarabine-daunorubicin CPX-351
Affected / at Risk (%)
Total   10/11 (90.91%)    
Show Serious Adverse Events Hide Serious Adverse Events
Liposomal Cytarabine-daunorubicin CPX-351
Affected / at Risk (%) # Events
Total   5/11 (45.45%)    
Blood and lymphatic system disorders   
Febrile neutropenia  1  4/11 (36.36%)  5
Anemia  1  1/11 (9.09%)  1
Cardiac disorders   
Cardio-pulmonary arrest  1  1/11 (9.09%)  1
General disorders   
Chills  1  1/11 (9.09%)  1
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Neoplasms - Other, Acute myelogenous leukemia  1  1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders   
Pneumonia  1  2/11 (18.18%)  2
Adult repiratory distress syndrome (ARDS)  1  1/11 (9.09%)  1
Respiratory failure  1  1/11 (9.09%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Liposomal Cytarabine-daunorubicin CPX-351
Affected / at Risk (%) # Events
Total   11/11 (100.00%)    
Blood and lymphatic system disorders   
Blood and lymphatic system disorders, other - clot in cheek  1  1/11 (9.09%)  1
Febrile neutropenia  1  8/11 (72.73%)  8
Thrombotic thrombocytopenic purpura  1  2/11 (18.18%)  2
Cardiac disorders   
Atrial fibrillation  1  1/11 (9.09%)  1
Cardiac disorders, other - tachycardia  1  1/11 (9.09%)  1
Sinus tachycardia  1  1/11 (9.09%)  1
Eye disorders   
Eye disorders, other - eye itching  1  1/11 (9.09%)  1
Papilledema  1  1/11 (9.09%)  1
Gastrointestinal disorders   
Abdominal pain  1  3/11 (27.27%)  3
Colitis  1  3/11 (27.27%)  3
Constipation  1  4/11 (36.36%)  4
Diarrhea  1  7/11 (63.64%)  7
Mucositis oral  1  6/11 (54.55%)  6
Nausea  1  1/11 (9.09%)  1
Rectal hemorrhage  1  1/11 (9.09%)  1
General disorders   
Pain, hip  1  1/11 (9.09%)  1
Pain, leg  1  1/11 (9.09%)  1
Fatigue  1  1/11 (9.09%)  1
Fever  1  4/11 (36.36%)  4
Infections and infestations   
Lung infection  1  2/11 (18.18%)  2
Investigations   
Alanine aminotransferase increased  1  2/11 (18.18%)  2
Investigations, other - fluid overload  1  1/11 (9.09%)  1
Metabolism and nutrition disorders   
Hepatobiliary disorders, other - hyperbilirubinemia  1  1/11 (9.09%)  1
Anorexia (decreased food consumption)  1  1/11 (9.09%)  1
Hypertriglyceridemia  1  1/11 (9.09%)  1
Musculoskeletal and connective tissue disorders   
Bone pain  1  1/11 (9.09%)  1
Musculoskeletal and connective tissue disorder, other - broken leg  1  1/11 (9.09%)  1
Nervous system disorders   
Brachial plexopathy  1  1/11 (9.09%)  1
Dizziness  1  2/11 (18.18%)  2
Headache  1  1/11 (9.09%)  1
Peripheral motor neuropathy  1  1/11 (9.09%)  1
Psychiatric disorders   
Hallucinations  1  4/11 (36.36%)  4
Renal and urinary disorders   
Hematuria  1  3/11 (27.27%)  3
Renal and urinary disorders, other - urinary discomfort  1  1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders   
Aspiration  1  1/11 (9.09%)  1
Cough  1  3/11 (27.27%)  3
Epistaxis (nose bleed)  1  1/11 (9.09%)  1
Hypoxia  1  1/11 (9.09%)  1
Respiratory, thoracic and mediastinal disorders, other - chest tightness  1  1/11 (9.09%)  1
Pneumothorax  1  1/11 (9.09%)  1
Skin and subcutaneous tissue disorders   
Rash  1  3/11 (27.27%)  3
Skin and subcutaneous tissue disorders, other - multiple subcutaneous nodules  1  1/11 (9.09%)  1
Vascular disorders   
Thromboembolic event  1  1/11 (9.09%)  1
1
Term from vocabulary, CTCAE (4.0)
Indicates events were collected by systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Rondeep Singh Brar
Organization: Stanford University
Phone: (650) 498-6000
EMail: rbrar@stanford.edu
Layout table for additonal information
Responsible Party: Rondeep Brar, Stanford University
ClinicalTrials.gov Identifier: NCT02019069     History of Changes
Other Study ID Numbers: IRB-28524
NCI-2013-01982 ( Registry Identifier: CTRP (Clinical Trial Reporting Program) )
HEM0036 ( Other Identifier: OnCore )
P30CA124435 ( U.S. NIH Grant/Contract )
First Submitted: November 25, 2013
First Posted: December 24, 2013
Results First Submitted: October 3, 2018
Results First Posted: January 15, 2019
Last Update Posted: January 21, 2019