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Trial record 24 of 848 for:    LENALIDOMIDE AND Angiogenesis

A Study of Lenalidomide in Pediatric Subjects With Relapsed or Refractory Acute Myeloid Leukemia

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ClinicalTrials.gov Identifier: NCT02538965
Recruitment Status : Completed
First Posted : September 2, 2015
Results First Posted : September 20, 2018
Last Update Posted : March 4, 2019
Sponsor:
Information provided by (Responsible Party):
Celgene

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Leukemia, Myeloid
Intervention Drug: Lenalidomide
Enrollment 17
Recruitment Details Participants were enrolled at 15 centers within the United States and Canada.
Pre-assignment Details The study population consisted of pediatric participants with relapsed or refractory Acute Myeloid Leukemia (rrAML) from 1 to ≤ 18 years of age and were required to have a fresh bone marrow aspirate and biopsy submitted for confirmation of disease.
Arm/Group Title Lenalidomide
Hide Arm/Group Description Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Period Title: Treatment Period
Started 17
Completed 0
Not Completed 17
Reason Not Completed
Other = Death due to disease progression             1
Adverse Event             2
Withdrawal by Parent/Guardian             2
Treatment Failure             9
Disease Progression             1
Physician Decision             2
Period Title: Follow-Up Period
Started 17
Completed [1] 2
Not Completed 15
Reason Not Completed
Other = Death due to disease progression             11
Withdrawal by Parent             4
[1]
Completed defined as participants continuing in follow-up period.
Arm/Group Title Lenalidomide
Hide Arm/Group Description Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Baseline Participants 17
Hide Baseline Analysis Population Description
The intention to treat (ITT) population consisted of all enrolled participants regardless of whether they received lenalidomide.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 17 participants
11.5  (4.56)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Female
7
  41.2%
Male
10
  58.8%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 17 participants
Asian
1
   5.9%
Black or African American
0
   0.0%
White
12
  70.6%
Not Collected or Reported
3
  17.6%
Other
1
   5.9%
Age Categories  
Measure Type: Count of Participants
Unit of measure:  Participants
≤ 2 years Number Analyzed 17 participants
0
   0.0%
3-6 years Number Analyzed 17 participants
4
  23.5%
7-12 years Number Analyzed 17 participants
6
  35.3%
13-16 years Number Analyzed 17 participants
4
  23.5%
≥ 17 years Number Analyzed 17 participants
3
  17.6%
Peripheral Blood Smear Blasts   [1] [2] 
Median (Full Range)
Unit of measure:  Percent of Blasts
Number Analyzed 15 participants
8.7
(0 to 91)
[1]
Measure Description: Laboratory results obtained at baseline/screening include the peripheral white blood cells and blast counts, bone marrow blast percentage, cytogenetics and molecular alterations.
[2]
Measure Analysis Population Description: Includes participants with available data.
White Blood Cell Count   [1] 
Median (Full Range)
Unit of measure:  10^9/L
Number Analyzed 17 participants
3.700
(0.13 to 158.90)
[1]
Measure Description: Laboratory results obtained at baseline/screening include the peripheral white blood cells and blast counts, bone marrow blast percentage, cytogenetics and molecular alterations.
Bone Marrow (BM) Myeloblasts Count   [1] [2] 
Median (Full Range)
Unit of measure:  Percent of Myeloblasts
Number Analyzed 16 participants
57.5
(7 to 92)
[1]
Measure Description: Laboratory results obtained at baseline/screening include the peripheral white blood cells and blast counts, bone marrow blast percentage, cytogenetics and molecular alterations.
[2]
Measure Analysis Population Description: Participants with available data.
Number of Participants With at Least One Cytogenetic Abnormality   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
t(8;21) Number Analyzed 17 participants
1
   5.9%
+8 Number Analyzed 17 participants
1
   5.9%
complex (>/= 3 abnormalities) Number Analyzed 17 participants
5
  29.4%
-7 Number Analyzed 17 participants
1
   5.9%
7q- Number Analyzed 17 participants
2
  11.8%
11q 23 abnormalities Number Analyzed 17 participants
3
  17.6%
inv (3) Number Analyzed 17 participants
1
   5.9%
Other Number Analyzed 17 participants
9
  52.9%
[1]
Measure Description: A standard cytogenetic metaphase preparation from the fresh bone marrow aspirate was prepared for the cytogenetic testing/chromosome analysis including karyotype. Results from cytogenetics, FISH studies, flow cytometry studies, and molecular studies performed locally at initial diagnosis and/or relapse was reported for t(16;16), t(8;21), MLL (11q23), t(15;17), monosomy 7, del(5q), or any other recurring abnormalities and presence or absence of mutations. More than one category can apply to a participant.
Number of Prior Systemic Anti-Cancer Regimens  
Median (Full Range)
Unit of measure:  Regimens
Number Analyzed 17 participants
5
(2 to 7)
1.Primary Outcome
Title Number of Participants Who Achieved a Morphologic Complete Response Within the First Four Cycles of Lenalidomide Treatment According to the Modified International Working Group (IWG) Criteria
Hide Description

The morphological complete response rate was defined as the total number of participants with morphological CR observed within the first 4 cycles of lenalidomide (regardless of whether the CR/CRi was observed at the end of Cycle 1, 2, 3 or 4) over the total number of participants evaluable for this endpoint. According to Modified IWG criteria, morphologic CR was defined as:

  1. Absolute neutrophil count (ANC) ≥ 1000/μL and platelets ≥ 100,000 without transfusions and/or exogenous growth factor support (i.e., no transfusion or exogenous growth factor within 7 days of assessment;
  2. Bone marrow < 5% blasts evidence of trilineage hematopoiesis;
  3. No evidence of extramedullary disease.

Morphologic CRi was defined as:

  1. ANC < 1000/μL and platelets < 100,000/μL or > 100,000/μL without platelet recovery (requiring transfusion within 7 days of assessment);
  2. BM with < 5% blasts and evidence of trilineage hematopoiesis;
  3. No evidence of extramedullary disease.
Time Frame From day of the first dose of IP to end of cycle 4; Response was assessed at the completion of the 21-day treatment period of cycles 1, 2, 3, and 4 and at treatment discontinuation.
Hide Outcome Measure Data
Hide Analysis Population Description
The Intent to Treat (ITT) population consisted of all enrolled participants regardless of whether they received lenalidomide. Analysis was not completed due to scarcity of relevant data. See description.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Measure Type: Count of Participants
Unit of Measure: Participants
1
   5.9%
2.Secondary Outcome
Title Number of Participants Who Achieved a Bone Marrow Confirmed CR/CRi Lasting 3 Months (Durable Response Rate)
Hide Description Durable response rate was defined as the percentage of participants who achieved a BM confirmed CR/CRi according to the Modified IWG Response Assessment Lasting 3 Months (from the time to complete response observed until treatment failure or worse) or until transplantation if earlier among all participants eligible for durable response rate analysis, provided the CR/CRi was confirmed in a bone marrow sample). Due to scarcity of relevant data, it was not practical or meaningful to analyze the durable response rate. Only 1 participant had a response and the participant was censored soon after, as the consent was withdrawn; unable to calculate duration of response.
Time Frame From date of confirmed complete response observed until treatment failure or worse; up to data cut-off date of 31 December 2017
Hide Outcome Measure Data
Hide Analysis Population Description
Due to scarcity of relevant data, it was not practical or meaningful to analyze the durable response rate. See description.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
3.Secondary Outcome
Title Duration of Response
Hide Description Duration of response was defined as the time from date of the first observed response (CR, CRi or PR) until morphologic relapse, molecular/cytogenetic relapse, or death only for participants who achieved a response. Due to scarcity of relevant data, it was not practical or meaningful to analyze the duration of response. Only 1 participant had a response and the participant was censored soon after, as the consent was withdrawn; unable to calculate duration of response.
Time Frame From date of first time of complete response observed until treatment failure or worse; up to data cut-off date of 31 December 2017
Hide Outcome Measure Data
Hide Analysis Population Description
ITT population participants that achieved at least a CR or CRi.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
4.Secondary Outcome
Title Number of Participants Who Achieved a Best Response of Morphologic Complete Remission, Morphologic Complete Remission Incomplete or Partial Remission
Hide Description

Overall response rate was defined as the number of participants with best response of CR, CRi or PR.

A CR =

  1. ANC ≥ 1000/μL and platelets ≥ 100,000 without transfusions and/or exogenous growth factor (GF) support (no transfusion or exogenous growth factor within 7 days of assessment);
  2. BM < 5% blasts evidence of trilineage hematopoiesis;
  3. No evidence of extramedullary disease

A CRi was defined as:

  1. ANC < 1000/μL and platelets < 100,000/μL or > 100,000/μL without platelet recovery (requiring transfusion within 7 days of assessment);
  2. BM with < 5% blasts and evidence of trilineage hematopoiesis;
  3. No evidence of extramedullary disease. A PR =

1. ANC of ≥ 1000/μL and platelets ≥ 100,000 without transfusions and/or exogenous growth factor support (no transfusion or exogenous growth factor within 7 days of assessment); 2. BM with 5% to 25% blasts and at least a 50% decrease in BM blast percent from baseline; 3. No evidence of extramedullary disease

Time Frame Response was assessed at the completion of the 21-day treatment period of cycles 1, 2, and 3
Hide Outcome Measure Data
Hide Analysis Population Description
Intent to Treat Population consisted of all enrolled participants regardless of whether they received lenalidomide.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Measure Type: Count of Participants
Unit of Measure: Participants
1
   5.9%
5.Secondary Outcome
Title Number of Participants With a Morphologic CR, CRi, PR or Treatment Failure at Cycles 1, 2 and 3
Hide Description

Disease assessment outcome at the end of Cycles 1-3 based on Cheson criteria:

Morphologic CR =

  1. ANC ≥ 1000/μL and platelet ≥ 100,000 without transfusions and/or exogenous growth factor support (no transfusion or exogenous GF within 7 days of assessment)
  2. BM < 5% blasts evidence of trilineage hematopoiesis
  3. No evidence of extramedullary disease Morphologic CRi =

1. ANC< 1000/μL and Platelets < 100,000/μL or > 100,000/μL without platelet recovery (requiring transfusion within 7 days of assessment) 2. BM with < 5% blasts and evidence of trilineage hematopoiesis 3. No evidence of extramedullary disease PR =

  1. ANC ≥ 1000/μL and platelets ≥ 100,000 without transfusions and/or exogenous growth factor support
  2. BM with < 5%-25% blasts and at least a 50% decrease in BM blast percent from baseline
  3. No evidence of extramedullary disease Treatment Failure = resistant disease; survival ≥ 7 days post-therapy; failed to achieve CR, CRi, or PR but stable with pers
Time Frame Response was assessed at the completion of the 21-day treatment period of cycles 1, 2, and 3
Hide Outcome Measure Data
Hide Analysis Population Description
The intent to treat population consisted of all enrolled participants regardless of whether they received lenalidomide. Data are reported for participants who began each treatment cycle.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: participants
Cycle 1 Morphologic CR Number Analyzed 17 participants
0
Cycle 1 Morphologic CRi Number Analyzed 17 participants
0
Cycle 1 PR Number Analyzed 17 participants
1
Cycle 1 Treatment Failure Number Analyzed 17 participants
13
Cycle 2 Morphologic CR Number Analyzed 8 participants
0
Cycle 2 Morphologic CRi Number Analyzed 8 participants
1
Cycle 2 PR Number Analyzed 8 participants
0
Cycle 2 Treatment Failure Number Analyzed 8 participants
5
Cycle 3 Morphologic CR Number Analyzed 1 participants
0
Cycle 3 Morphologic CRi Number Analyzed 1 participants
1
Cycle 3 PR Number Analyzed 1 participants
0
Cycle 3 Treatment Failure Number Analyzed 1 participants
0
6.Secondary Outcome
Title Number of Participants Who Received a Haematopoietic Stem Cell Transplant (HSCT)
Hide Description The number of participants who had undergone a haematopoietic stem cell transplant was calculated over the total number of participants in the ITT population. Percentages were also calculated based on whether the transplantation was the first, second, or subsequent transplant post IP administration.
Time Frame From first dose of study drug up to 5 years post HSCT
Hide Outcome Measure Data
Hide Analysis Population Description
The intent to treat population consisted of all enrolled participants regardless of whether they received lenalidomide.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: Participants
Any Transplant After Treatment Start 2
First Transplant After Treatment Start 2
Second Transplant After Treatment Start 0
Any Subsequent Transplant After Treatment Start 0
7.Secondary Outcome
Title Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAE)
Hide Description A TEAE was defined as any adverse event (AE) occurring or worsening on or after the first treatment of lenalidomide and within 28 days after the last dose. A serious AE = any AE which results in death; is life-threatening; requires inpatient hospitalization or prolongation of existing hospitalization; results in persistent or significant disability/incapacity; is a congenital anomaly/birth defect; constitutes an important medical event. The severity of AEs was graded based on National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), Version 4.3 and based on the following scale: Grade 1 = Mild Grade 2 = Moderate Grade 3 = Severe Grade 4 = Life threatening Grade 5 = Death.
Time Frame From the first dose of study drug until 28 days after the last dose of study drug; up to data cut off date of 31 December 2017; maximum duration of treatment was 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population consisted of all participants who received at least one dose of lenalidomide.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Measure Type: Count of Participants
Unit of Measure: Participants
Any TEAE
17
 100.0%
Any TEAE Related to Lenalidomide
15
  88.2%
Any Grade 3/4 TEAE
17
 100.0%
Any Grade 3/4 TEAE Related to Lenalidomide
11
  64.7%
Any Grade 5 TEAE
1
   5.9%
Any Serious TEAE
13
  76.5%
Any Serious TEAE Related to Lenalidomide
5
  29.4%
Any Serious TEAE Leading to Dose Discontinuation
3
  17.6%
Any TEAE Leading to Dose Discontinuation
3
  17.6%
Any TEAE Leading to Dose Reduction
4
  23.5%
Any TEAE Leading to Dose Interruption
7
  41.2%
Any TEAE Leading to Death
1
   5.9%
8.Secondary Outcome
Title Percentage of Participants With of Graft Versus Host Disease (GVHD)
Hide Description Acute graft versus host disease generally occurs after allogeneic hematopoietic stem cell transplantation. It is a reaction of donor immune cells against host tissues. The 3 main tissues that acute GVHD affects are the skin, liver, and gastrointestinal tract. Chronic GVHD is scored per the National Institute of Health consensus conference grading system. Clinical manifestations of chronic GVHD include skin involvement resembling lichen planus or the cutaneous manifestations of scleroderma; dry oral mucosa with ulcerations and sclerosis of the gastrointestinal tract; and a rising serum bilirubin concentration.
Time Frame From the first dose of study drug to 28 days after the last dose of study drug; up to data cut off date of 31 December 2017; maximum treatment was 12 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety population consisted of all participants who received at least 1 dose of lenalidomide.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Measure Type: Number
Unit of Measure: Percentage of Participants
0
9.Secondary Outcome
Title Area Under the Plasma Concentration-time Curve From Time 0 to the Time of the Last Quantifiable Concentration of Lenalidomide (AUC-t)
Hide Description Area under the plasma concentration-time curve from time 0 to the time of the last quantifiable concentration, calculated by linear trapezoidal method when concentrations were increasing and the logarithmic trapezoidal method when concentrations were decreasing.
Time Frame Pharmacokinetic (PK) sampling was conducted after lenalidomide administration at Cycle 1 and was weight dependent at these timepoints: 0.5, 1, 2, 4, 6, 8 and 24 hours. The 24 hour PK sample was taken prior to the lenalidomide dose on Day 2.
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic population included participants who received at least one dose of lenalidomide and had at least one measurable lenalidomide concentration.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng*h/mL
5378.83
(57.3%)
10.Secondary Outcome
Title Area Under the Plasma Concentration Time Curve From 0 Extrapolated to Infinity (AUC-inf, AUC0∞) Of Lenalidomide
Hide Description Area under the plasma concentration-time curve from time 0 extrapolated to infinity, calculated as [AUCt + Ct/ λz]. Ct is the last quantifiable concentration. No AUC extrapolation was performed with unreliable λz.
Time Frame Pharmacokinetic sampling was conducted after lenalidomide administration at Cycle 1 and was weight dependent at these timepoints: 0.5, 1, 2, 4, 6, 8 and 24 hours. The 24 hour PK sample was taken prior to the lenalidomide dose on Day 2.
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic population included participants who received at least one dose of lenalidomide and had at least one measurable lenalidomide concentration.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng*h/mL
5656.67
(52.8%)
11.Secondary Outcome
Title Maximum Observed Concentration (Cmax) of Lenalidomide
Hide Description Maximum observed plasma concentration, obtained directly from the observed concentration versus time data.
Time Frame PK sampling was conducted after lenalidomide administration at Cycle 1 and was weight dependent at these timepoints: 0.5, 1, 2, 4, 6, 8 and 24 hours. The 24 hour PK sample was taken prior to the lenalidomide dose on Day 2.
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic population included participants who received at least one dose of lenalidomide and had at least one measurable lenalidomide concentration.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ng/mL
1252.08
(43.8%)
12.Secondary Outcome
Title Time to Reach Maximum Concentration (Tmax) of Lenalidomide
Hide Description Time to cmax was obtained directly from the observed concentration versus time data.
Time Frame Pk sampling was conducted after lenalidomide administration at Cycle 1 and was weight dependent at these timepoints: 0.5, 1, 2, 4, 6, 8 and 24 hours. The 24 hour PK sample was taken prior to the lenalidomide dose on Day 2.
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic population included participants who received at least one dose of lenalidomide and had at least one measurable lenalidomide concentration.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Median (Full Range)
Unit of Measure: Hours
2.000
(0.5500 to 4.000)
13.Secondary Outcome
Title Terminal Half-Life (t1/2) of Lenalidomide
Hide Description Terminal phase half-life in plasma, calculated as [(ln 2)/λz]. Terminal half-life was only calculated when a reliable estimate for λz could be obtained.
Time Frame Pharmacokinetic sampling was conducted after lenalidomide administration at Cycle 1 and was weight dependent at these timepoints: 0.5, 1, 2, 4, 6, 8 and 24 hours. The 24 hour PK sample was taken prior to the lenalidomide dose on Day 2.
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic population included participants who received at least one dose of lenalidomide and had at least one measurable lenalidomide concentration.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: hours
2.311
(43.3%)
14.Secondary Outcome
Title Apparent Total Clearance (CL/F) of Lenalidomide
Hide Description Apparent volume of distribution, calculated as [(CL/F)/λz].
Time Frame Pharmacokinetic sampling was conducted after lenalidomide administration at Cycle 1 and was weight dependent at these timepoints: 0.5, 1, 2, 4, 6, 8 and 24 hours. The 24 hour PK sample was taken prior to the lenalidomide dose on Day 2.
Hide Outcome Measure Data
Hide Analysis Population Description
The pharmacokinetic population included participants who received at least one dose of lenalidomide and had at least one measurable lenalidomide concentration.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: ml/min
172.09
(52.5%)
15.Secondary Outcome
Title Apparent Volume of Distribution (Vz/F) of Lenalidomide
Hide Description Apparent volume of distribution, calculated as [(CL/F)/λz].
Time Frame Pharmacokinetic sampling was conducted after lenalidomide administration at Cycle 1 and was weight dependent at these timepoints: 0.5, 1, 2, 4, 6, 8 and 24 hours. The 24 hour PK sample was taken prior to the lenalidomide dose on Day 2.
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Hide Analysis Population Description
The pharmacokinetic population included participants who received at least one dose of lenalidomide and had at least one measurable lenalidomide concentration.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 17
Geometric Mean (Geometric Coefficient of Variation)
Unit of Measure: Liters
34.42
(57.0%)
16.Secondary Outcome
Title Correlation of Peripheral White Blood Cell Count, Absolute Blast Count and Cytogenetics With Response to Lenalidomide
Hide Description Correlation of peripheral white blood cell count, absolute blast count and cytogenetics with response to lenalidomide was not performed since only 1 participant met the primary efficacy endpoint of morphological CR/CRi, and due to scarcity of relevant data, it was not practical or meaningful to analyze to perform the analysis on blood counts and response to lenalidomide.
Time Frame Not Performed
Hide Outcome Measure Data
Hide Analysis Population Description
Due to scarcity of relevant data, it was not practical or meaningful to analyze or perform the analysis on blood counts and response to lenalidomide.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
Time Frame From the date of the first dose of lenalidomide until 28 days after the last dose of lenalidomide; up to final cut- off date of 31 December 2017; maximum treatment duration was 12 weeks.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Lenalidomide
Hide Arm/Group Description Participants received oral lenalidomide (capsules or suspension) at 2 mg/kg/day with a maximum dose of 70 mg/day for the first 21 days of each 28-day treatment cycle up to a maximum of 12 cycles of study treatment unless there was the development of a toxicity or adverse event (AE), death, withdrawal by parent/guardian of participant, lost to follow-up, pregnancy, non-compliance with investigational product (IP), physician decision, protocol violation or other reason. Upon completion or discontinuation of study treatment, participants entered the follow-up period for up to 5 years.
All-Cause Mortality
Lenalidomide
Affected / at Risk (%)
Total   11/17 (64.71%) 
Show Serious Adverse Events Hide Serious Adverse Events
Lenalidomide
Affected / at Risk (%)
Total   13/17 (76.47%) 
Blood and lymphatic system disorders   
Febrile neutropenia  1  6/17 (35.29%) 
Haemolytic anaemia  1  1/17 (5.88%) 
Leukocytosis  1  1/17 (5.88%) 
Eye disorders   
Vitreous haemorrhage  1  1/17 (5.88%) 
Gastrointestinal disorders   
Diarrhoea  1  1/17 (5.88%) 
Stomatitis  1  1/17 (5.88%) 
Vomiting  1  1/17 (5.88%) 
General disorders   
General physical health deterioration  1  1/17 (5.88%) 
Pyrexia  1  1/17 (5.88%) 
Infections and infestations   
Cellulitis  1  2/17 (11.76%) 
Lung infection  1  1/17 (5.88%) 
Pneumonia  1  2/17 (11.76%) 
Sinusitis  1  1/17 (5.88%) 
Injury, poisoning and procedural complications   
Subdural haematoma  1  1/17 (5.88%) 
Investigations   
Activated partial thromboplastin time prolonged  1  1/17 (5.88%) 
International normalised ratio increased  1  1/17 (5.88%) 
Metabolism and nutrition disorders   
Dehydration  1  1/17 (5.88%) 
Hypokalaemia  1  1/17 (5.88%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  1/17 (5.88%) 
Pain in extremity  1  1/17 (5.88%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Paraneoplastic syndrome  1  1/17 (5.88%) 
Tumour flare  1  1/17 (5.88%) 
Nervous system disorders   
Cerebrovascular accident  1  1/17 (5.88%) 
Somnolence  1  1/17 (5.88%) 
Renal and urinary disorders   
Renal failure  1  1/17 (5.88%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnoea  1  1/17 (5.88%) 
Hypoxia  1  2/17 (11.76%) 
Pulmonary oedema  1  1/17 (5.88%) 
Skin and subcutaneous tissue disorders   
Rash pruritic  1  1/17 (5.88%) 
Urticaria  1  1/17 (5.88%) 
1
Term from vocabulary, MedDRA 20.0
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 5%
Lenalidomide
Affected / at Risk (%)
Total   17/17 (100.00%) 
Blood and lymphatic system disorders   
Anaemia  1  10/17 (58.82%) 
Disseminated intravascular coagulation  1  1/17 (5.88%) 
Febrile neutropenia  1  2/17 (11.76%) 
Leukopenia  1  2/17 (11.76%) 
Lymphopenia  1  1/17 (5.88%) 
Neutropenia  1  3/17 (17.65%) 
Splenic infarction  1  1/17 (5.88%) 
Thrombocytopenia  1  10/17 (58.82%) 
Cardiac disorders   
Sinus bradycardia  1  1/17 (5.88%) 
Supraventricular tachycardia  1  1/17 (5.88%) 
Tachycardia  1  4/17 (23.53%) 
Ear and labyrinth disorders   
Hypoacusis  1  1/17 (5.88%) 
Eye disorders   
Diplopia  1  1/17 (5.88%) 
Dry eye  1  1/17 (5.88%) 
Photophobia  1  1/17 (5.88%) 
Vision blurred  1  1/17 (5.88%) 
Gastrointestinal disorders   
Abdominal pain  1  6/17 (35.29%) 
Abdominal pain upper  1  1/17 (5.88%) 
Constipation  1  7/17 (41.18%) 
Diarrhoea  1  3/17 (17.65%) 
Nausea  1  9/17 (52.94%) 
Neutropenic colitis  1  1/17 (5.88%) 
Oral mucosal erythema  1  1/17 (5.88%) 
Oral pain  1  3/17 (17.65%) 
Peritoneal haemorrhage  1  1/17 (5.88%) 
Proctalgia  1  1/17 (5.88%) 
Stomatitis  1  4/17 (23.53%) 
Vomiting  1  4/17 (23.53%) 
General disorders   
Catheter site haematoma  1  1/17 (5.88%) 
Face oedema  1  1/17 (5.88%) 
Fatigue  1  4/17 (23.53%) 
Non-cardiac chest pain  1  2/17 (11.76%) 
Oedema peripheral  1  4/17 (23.53%) 
Pain  1  2/17 (11.76%) 
Peripheral swelling  1  1/17 (5.88%) 
Pyrexia  1  7/17 (41.18%) 
Hepatobiliary disorders   
Hepatosplenomegaly  1  1/17 (5.88%) 
Hyperbilirubinaemia  1  3/17 (17.65%) 
Infections and infestations   
Sinusitis  1  2/17 (11.76%) 
Staphylococcal bacteraemia  1  1/17 (5.88%) 
Staphylococcal infection  1  2/17 (11.76%) 
Upper respiratory tract infection  1  1/17 (5.88%) 
Urinary tract infection  1  1/17 (5.88%) 
Injury, poisoning and procedural complications   
Contusion  1  1/17 (5.88%) 
Laceration  1  1/17 (5.88%) 
Skin wound  1  1/17 (5.88%) 
Investigations   
Activated partial thromboplastin time prolonged  1  2/17 (11.76%) 
Alanine aminotransferase increased  1  4/17 (23.53%) 
Anti-platelet antibody positive  1  1/17 (5.88%) 
Aspartate aminotransferase increased  1  4/17 (23.53%) 
Blood fibrinogen decreased  1  1/17 (5.88%) 
Electrocardiogram QT prolonged  1  2/17 (11.76%) 
Gamma-glutamyltransferase increased  1  1/17 (5.88%) 
International normalised ratio increased  1  3/17 (17.65%) 
Weight decreased  1  1/17 (5.88%) 
Metabolism and nutrition disorders   
Decreased appetite  1  4/17 (23.53%) 
Dehydration  1  3/17 (17.65%) 
Fluid overload  1  1/17 (5.88%) 
Hypermagnesaemia  1  1/17 (5.88%) 
Hyperuricaemia  1  1/17 (5.88%) 
Hypoalbuminaemia  1  3/17 (17.65%) 
Hypocalcaemia  1  4/17 (23.53%) 
Hypokalaemia  1  9/17 (52.94%) 
Hypomagnesaemia  1  4/17 (23.53%) 
Hyponatraemia  1  3/17 (17.65%) 
Hypophosphataemia  1  2/17 (11.76%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  2/17 (11.76%) 
Back pain  1  5/17 (29.41%) 
Bone pain  1  3/17 (17.65%) 
Muscle spasms  1  1/17 (5.88%) 
Muscular weakness  1  2/17 (11.76%) 
Musculoskeletal pain  1  2/17 (11.76%) 
Myalgia  1  3/17 (17.65%) 
Pain in extremity  1  3/17 (17.65%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Chloroma  1  1/17 (5.88%) 
Nervous system disorders   
Cerebrovascular accident  1  1/17 (5.88%) 
Dizziness  1  1/17 (5.88%) 
Headache  1  6/17 (35.29%) 
Lethargy  1  1/17 (5.88%) 
Somnolence  1  1/17 (5.88%) 
Product Issues   
Device issue  1  1/17 (5.88%) 
Device occlusion  1  1/17 (5.88%) 
Thrombosis in device  1  1/17 (5.88%) 
Psychiatric disorders   
Anxiety  1  1/17 (5.88%) 
Insomnia  1  1/17 (5.88%) 
Phonophobia  1  1/17 (5.88%) 
Renal and urinary disorders   
Chromaturia  1  1/17 (5.88%) 
Haematuria  1  1/17 (5.88%) 
Urinary hesitation  1  1/17 (5.88%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  4/17 (23.53%) 
Dry throat  1  1/17 (5.88%) 
Dyspnoea  1  2/17 (11.76%) 
Epistaxis  1  3/17 (17.65%) 
Haemoptysis  1  1/17 (5.88%) 
Hypoxia  1  2/17 (11.76%) 
Nasal congestion  1  1/17 (5.88%) 
Oropharyngeal pain  1  5/17 (29.41%) 
Painful respiration  1  1/17 (5.88%) 
Pleural effusion  1  2/17 (11.76%) 
Pulmonary oedema  1  2/17 (11.76%) 
Wheezing  1  1/17 (5.88%) 
Skin and subcutaneous tissue disorders   
Drug eruption  1  1/17 (5.88%) 
Hyperhidrosis  1  1/17 (5.88%) 
Pruritus  1  1/17 (5.88%) 
Purpura  1  1/17 (5.88%) 
Rash macular  1  1/17 (5.88%) 
Rash maculo-papular  1  5/17 (29.41%) 
Rash papular  1  1/17 (5.88%) 
Skin hyperpigmentation  1  1/17 (5.88%) 
Vascular disorders   
Flushing  1  1/17 (5.88%) 
Hypertension  1  1/17 (5.88%) 
1
Term from vocabulary, MedDRA 20.0
Indicates events were collected by systematic assessment
The results of the efficacy analysis were reviewed by an independent Data Monitoring Committee, which concluded that the study would not proceed to Stage 2, given that the efficacy criteria for continuation of the study to Stage 2 had not been met.
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.
Results from a center cannot be submitted for publication before results of multicenter study are published unless it is more than 1 year since study completion. Then, Investigator can publish if manuscript is submitted to Celgene 30 days prior to submission. If Celgene decides publication would hinder drug development, Investigator must delay submission for up to 60 additional days. Investigator must delete confidential data before submission or defer publication to permit patent applications.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Anne McClain, Senior Manager, Clinical Trial Disclosur
Organization: Celgene Corporation
Phone: 888-260-1599
EMail: ClinicalTrialDisclosure@Celgene.com
Layout table for additonal information
Responsible Party: Celgene
ClinicalTrials.gov Identifier: NCT02538965     History of Changes
Other Study ID Numbers: CC-5013-AML-002
First Submitted: August 25, 2015
First Posted: September 2, 2015
Results First Submitted: July 13, 2018
Results First Posted: September 20, 2018
Last Update Posted: March 4, 2019