Try our beta test site

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

This study has been completed.
Sponsor:
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
Jason Robert Gotlib, Stanford University
ClinicalTrials.gov Identifier:
NCT01034592
First received: December 15, 2009
Last updated: December 13, 2016
Last verified: December 2016
Results First Received: December 13, 2016  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Conditions: Anemia
Leukemia
Acute Myeloid Leukemia (AML)
Myelodysplastic Syndromes (MDS)
Intervention: Drug: Lenalidomide

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Lenalidomide 2.5 mg weekly as oral tablets during days 1-21 of cycle 1 (dose level 1). If no toxicity ≥3 dose will be increased to 2.5mg twice weekly on days 1-21 of cycle 2 (dose level 2). If no toxicity ≥3 dose will be increased to 5mg twice weekly on days 1-21 of cycle 3 (dose level 3). If no toxicity ≥3 dose will be increased to 5mg thrice weekly on days 1-21 of cycle 4 (dose level 4).

Participant Flow:   Overall Study
    Lenalidomide
STARTED   2 
COMPLETED   2 
NOT COMPLETED   0 



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Baseline Measures This is an open-label study. Active drug will be administered to all participants at a dosing regimen of 2.5 mg of lenalidomide weekly on days 1-21 every 28 days up to 5mg thrice weekly on days 1-21 every 28 days.

Baseline Measures
   Baseline Measures 
Overall Participants Analyzed 
[Units: Participants]
 2 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      0   0.0% 
Between 18 and 65 years      2 100.0% 
>=65 years      0   0.0% 
Gender 
[Units: Participants]
Count of Participants
 
Female      1  50.0% 
Male      1  50.0% 
Ethnicity (NIH/OMB) 
[Units: Participants]
Count of Participants
 
Hispanic or Latino      0   0.0% 
Not Hispanic or Latino      2 100.0% 
Unknown or Not Reported      0   0.0% 
Race (NIH/OMB) 
[Units: Participants]
Count of 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 
[Units: Participants]
 
United States   2 


  Outcome Measures

1.  Primary:   RBC Transfusion Independence   [ Time Frame: Assessment done every 56 days: D56, D112, D168, D224, then every month during Maintenance Phase ]

2.  Secondary:   >50% Decrease in RBC Transfusion Requirements   [ Time Frame: Assessment done every 56 days: D56, D112, D168, D224, then every month during Maintenance Phase ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

3.  Secondary:   Change of Hemoglobin Concentration From Baseline   [ Time Frame: Assessed weekly up to end of cycle 8 (Day 224) or Early Discontinuation, then every two weeks during Maintenance Phase with an additional assessment done 30 days (+/- 3 days) after last dose of study drug ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

4.  Secondary:   Neutrophil Response   [ Time Frame: Assessed weekly up to end of cycle 8 (Day 224) or Early Discontinuation, then every two weeks during Maintenance Phase with an additional assessment done 30 days (+/- 3 days) after last dose of study drug ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

5.  Secondary:   Platelet Response   [ Time Frame: Assessed weekly up to end of cycle 8 (Day 224) or Early Discontinuation, then every two weeks during Maintenance Phase with an additional assessment done 30 days (+/- 3 days) after last dose of study drug ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

6.  Secondary:   Bone Marrow Response   [ Time Frame: End of cycle 8 (Day 224) or Early Discontinuation, then every 6 months during Maintenance Phase ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

7.  Secondary:   Duration of Response   [ Time Frame: Day 56 and end of cycle 8 (Day 224) or Early Discontinuation, then every month during Maintenance Phase ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

8.  Secondary:   Safety (Type, Frequency, Severity, and Relationship of Adverse Events to Lenalidomide)   [ Time Frame: Safety is monitored on a continuous basis throughout the trial period, and for 30 days after last dose of study medication ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

9.  Secondary:   Correction of Clinical Response With Ribosomal Protein Mutation Status and ex Vivo Effects of Lenalidomide on Marrow Erythroid Colony Growth and Microarray Gene Expression Signatures   [ Time Frame: Assessment done end of cycle 8 (Day 224) ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Only 2 subjects were enrolled on this protocol. Low accrual was related to various factors. Given the accrual status, we are unable to reach substantive conclusions about the efficacy or safety of lenalidomide in adults with Diamond-Blackfan Anemia.


  More Information
  Hide More Information

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:  
Name/Title: Jason R Gotlib, MD, Professor of Medicine (Hematology)
Organization: Stanford University Medical Center
phone: 650-867-2823
e-mail: jason.gotlib@stanford.edu



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 )
Study First Received: December 15, 2009
Results First Received: December 13, 2016
Last Updated: December 13, 2016