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

This study has been terminated.
(Poor accrual related to rarity of Diamond-Blackfan anemia (DBA))
Sponsor:
ClinicalTrials.gov Identifier:
NCT01034592
First Posted: December 17, 2009
Last Update Posted: August 2, 2017
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.
Collaborator:
Celgene Corporation
Information provided by (Responsible Party):
Jason Robert Gotlib, Stanford University
Results First Submitted: December 13, 2016  
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

  Participant Flow
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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 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.

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



  Baseline Characteristics
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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
Lenalidomide 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.

Baseline Measures
   Lenalidomide 
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% 
Sex: Female, Male 
[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
  Show All Outcome Measures

1.  Primary:   Red Blood Cell (RBC) Transfusion Independence   [ Time Frame: 6 months ]

2.  Secondary:   Red Blood Cell (RBC) Transfusions   [ Time Frame: 6 months ]

3.  Secondary:   Hemoglobin Concentration   [ Time Frame: 6 months ]

4.  Secondary:   Neutrophil Response   [ Time Frame: 6 months ]

5.  Secondary:   Platelet Response   [ Time Frame: 6 months ]

6.  Secondary:   Duration of Response   [ Time Frame: 6 months ]

7.  Secondary:   Toxicity   [ Time Frame: 6 months ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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, no substantive conclusions about the efficacy or safety of lenalidomide in adults with Diamond-Blackfan Anemia could be reached.


  More Information
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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 )
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