Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 41 of 52 for:    LENALIDOMIDE AND Leukemia AND Acute Myeloid Leukemia

Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia

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: NCT01034592
Recruitment Status : Terminated (Poor accrual related to rarity of Diamond-Blackfan anemia (DBA))
First Posted : December 17, 2009
Results First Posted : February 7, 2017
Last Update Posted : August 2, 2017
Sponsor:
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
Jason Robert Gotlib, Stanford University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Anemia
Leukemia
Acute Myeloid Leukemia (AML)
Myelodysplastic Syndromes (MDS)
Intervention Drug: Lenalidomide
Enrollment 2
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Lenalidomide
Hide Arm/Group Description Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Period Title: Overall Study
Started 2
Completed 2
Not Completed 0
Arm/Group Title Lenalidomide
Hide Arm/Group Description Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Number of Baseline Participants 2
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants
<=18 years
0
   0.0%
Between 18 and 65 years
2
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants
Female
1
  50.0%
Male
1
  50.0%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants
Hispanic or Latino
0
   0.0%
Not Hispanic or Latino
2
 100.0%
Unknown or Not Reported
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 2 participants
American Indian or Alaska Native
0
   0.0%
Asian
0
   0.0%
Native Hawaiian or Other Pacific Islander
0
   0.0%
Black or African American
0
   0.0%
White
2
 100.0%
More than one race
0
   0.0%
Unknown or Not Reported
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 2 participants
2
1.Primary Outcome
Title Red Blood Cell (RBC) Transfusion Independence
Hide Description Red blood cell (RBC) transfusion independence is reported as the number of subjects who achieve a continuous absence of the intravenous infusion of any RBC transfusion during any consecutive "rolling" 56 days during the treatment period.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
All treated subjects were analyzed.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
0
2.Secondary Outcome
Title Red Blood Cell (RBC) Transfusions
Hide Description The effect on red blood cell (RBC) transfusions was assessed as the number of participants that achieved a greater than 50% decrease in RBC transfusion requirements.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Both participants were assessed and contributed to the analysis.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Number of Participants Analyzed 2
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
3.Secondary Outcome
Title Hemoglobin Concentration
Hide Description The effect on hemoglobin concentration was assessed as the change from baseline, measured in g/dL.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Both participants were assessed and contributed to the analysis.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Number of Participants Analyzed 2
Median (Full Range)
Unit of Measure: g/dL
0.4
(0.4 to 0.4)
4.Secondary Outcome
Title Neutrophil Response
Hide Description The effect on neutrophil levels was assessed as the change in neutrophil count from baseline.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Both participants were assessed and contributed to the analysis.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Number of Participants Analyzed 2
Median (Full Range)
Unit of Measure: 1000/uL
0.50
(0.10 to 0.91)
5.Secondary Outcome
Title Platelet Response
Hide Description The effect on platelet levels as assessed as the change in platelet count from baseline.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Both participants were assessed and contributed to the analysis.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:

Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.

Lenalidomide: 2.5 mg/wk up to 5 mg 3x/wk

Overall Number of Participants Analyzed 2
Median (Full Range)
Unit of Measure: 1000/uL
25.5
(-20.0 to 71.0)
6.Secondary Outcome
Title Duration of Response
Hide Description The response duration was measured from the last of the consecutive 56 days during which the subject was free of red blood cells (RBC) transfusions to the date of the first RBC transfusion after the 56-day RBC-transfusion-free period.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Both participants were assessed and contributed to the analysis, but never demonstrated any therapeutic response.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Number of Participants Analyzed 2
Median (Full Range)
Unit of Measure: days
0
(0 to 0)
7.Secondary Outcome
Title Toxicity
Hide Description Toxicity was assessed as the number of adverse events related to lenalidomide.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
Both participants were assessed and contributed to the analysis.
Arm/Group Title Lenalidomide
Hide Arm/Group Description:
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: Related Adverse Events
1
Time Frame Adverse event data was collected over the duration of the treatment and maintenance phase of the study, for a total of 2 years.
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Serious Adverse Events
Hide Arm/Group Description Serious Adverse Events include: adverse events that result in death, require either inpatient hospitalization or the prolongation of hospitalization, are life-threatening, result in a persistent or significant disability/incapacity or result in a congenital anomaly/birth defect. Other important medical events, based upon appropriate medical judgment, may also be considered Serious Adverse Events if a trial participant's health is at risk and intervention is required to prevent an outcome mentioned.
All-Cause Mortality
Serious Adverse Events
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Serious Adverse Events
Affected / at Risk (%) # Events
Total   0/2 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Serious Adverse Events
Affected / at Risk (%) # Events
Total   2/2 (100.00%)    
Blood and lymphatic system disorders   
Neutropenia   1/2 (50.00%)  1
General disorders   
Fatigue *  2/2 (100.00%)  2
Infections and infestations   
Ear Infection   1/2 (50.00%)  1
Investigations   
Aspartate aminotransferase, increased   1/2 (50.00%)  1
Alanine aminotransferase, increased   1/2 (50.00%)  1
Respiratory, thoracic and mediastinal disorders   
Dry cough *  1/2 (50.00%)  1
Indicates events were collected by systematic assessment
*
Indicates events were collected by non-systematic assessment
Only 2 subjects were enrolled on this protocol. Low accrual was related to various factors. Given the accrual status, no substantive conclusions about the efficacy or safety of lenalidomide in adults with Diamond-Blackfan Anemia could be reached.
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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 Point of Contact
Layout table for Results Point of Contact information
Name/Title: Jason R Gotlib, MD, Professor of Medicine (Hematology)
Organization: Stanford University Medical Center
Phone: 650-867-2823
EMail: jason.gotlib@stanford.edu
Layout table for additonal information
Responsible Party: Jason Robert Gotlib, Stanford University
ClinicalTrials.gov Identifier: NCT01034592     History of Changes
Other Study ID Numbers: IRB-16822
SU-12082009-4523 ( Other Identifier: Stanford University )
RV-0365 ( Other Identifier: Celgene Corporation )
HEMMDS0022 ( Other Identifier: OnCore )
First Submitted: December 15, 2009
First Posted: December 17, 2009
Results First Submitted: December 13, 2016
Results First Posted: February 7, 2017
Last Update Posted: August 2, 2017