Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
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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
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Tracking Information | |||||
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First Submitted Date ICMJE | December 15, 2009 | ||||
First Posted Date ICMJE | December 17, 2009 | ||||
Results First Submitted Date ICMJE | December 13, 2016 | ||||
Results First Posted Date ICMJE | February 7, 2017 | ||||
Last Update Posted Date | August 2, 2017 | ||||
Study Start Date ICMJE | November 2009 | ||||
Actual Primary Completion Date | October 2012 (Final data collection date for primary outcome measure) | ||||
Current Primary Outcome Measures ICMJE |
Red Blood Cell (RBC) Transfusion Independence [ Time Frame: 6 months ] 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.
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Original Primary Outcome Measures ICMJE |
RBC Transfusion Independence [ Time Frame: Assessment done every 56 days: D56, D112, D168, D224, then every month during Maintenance Phase ] | ||||
Change History | Complete list of historical versions of study NCT01034592 on ClinicalTrials.gov Archive Site | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | ||||
Original Other Pre-specified Outcome Measures | Not Provided | ||||
Descriptive Information | |||||
Brief Title ICMJE | Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia | ||||
Official Title ICMJE | A Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent Anemia | ||||
Brief Summary | This is a single-center, single arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion dependent adult subjects with Diamond-Blackfan Anemia (DBA). Primary Objective: To evaluate the erythroid response rate as measured by rate of red blood cell transfusion independence [MDS International Working Group (IWG) 2000 Criteria will be applied]. Secondary Objective: 1)To evaluate the tolerability and safety profile of lenalidomide in patients with DBA and other inherited marrow failure syndromes 2) To correlate response to lenalidomide with biologic surrogates of DBA including ribosomal protein mutation status, ex vivo erythroid colony growth, and microarray gene expression |
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Detailed Description | This pilot study will utilize an intra-patient dose escalation design. Cycles are 28 days in length. Subjects will receive lenalidomide 2.5 mg weekly during days 1 to 21 of cycle 1 (dose level 1). If patients do not experience any grade > 3 hematologic or non-hematologic toxicity, the dose will be increased to 2.5 mg twice weekly on days 1 to 21 of cycle 2 (dose level 2). If patients do not experience any grade > 3 hematologic or non-hematologic toxicity, the dose will be increased to 5 mg twice weekly on days 1 to 21 of cycle 3 (dose level 3). If patients do not experience any grade >3 hematologic or non-hematologic toxicity, the dose will be increased to 5 mg thrice weekly on days 1 to 21 of cycle 4 (dose level 4). Patients who experience grade >3 hematologic or non-hematologic toxicity at dose level 1 will be discontinued from study. Patients who experience grade > 3 hematologic or non-hematologic toxicity at dose level 2, 3, or 4 will have the lenalidomide held and dose reduced according to protocol dose interruption/modification algorithms (section 5.5.3). If at least a minor erythroid response is not achieved at the end of 8 cycles of treatment, patients will be discontinued from study. If a minor or major erythroid response is achieved after completion of 8 cycles of treatment, patients can continue study drug on a maintenance phase until loss of erythroid response (return to baseline hemoglobin or transfusion requirement) or unacceptable toxicity. | ||||
Study Type ICMJE | Interventional | ||||
Study Phase ICMJE | Phase 1 | ||||
Study Design ICMJE | Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE |
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Intervention ICMJE | Drug: Lenalidomide
2.5 mg/wk up to 5 mg 3x/wk
Other Names:
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Study Arms ICMJE | Experimental: 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.
Intervention: Drug: Lenalidomide
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Publications * | Not Provided | ||||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | |||||
Recruitment Status ICMJE | Terminated | ||||
Actual Enrollment ICMJE |
2 | ||||
Original Estimated Enrollment ICMJE |
11 | ||||
Actual Study Completion Date ICMJE | December 2012 | ||||
Actual Primary Completion Date | October 2012 (Final data collection date for primary outcome measure) | ||||
Eligibility Criteria ICMJE | INCLUSION CRITERIA
EXCLUSION CRITERIA
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Sex/Gender ICMJE |
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Ages ICMJE | 18 Years and older (Adult, Older Adult) | ||||
Accepts Healthy Volunteers ICMJE | No | ||||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | ||||
Listed Location Countries ICMJE | United States | ||||
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Administrative Information | |||||
NCT Number ICMJE | NCT01034592 | ||||
Other Study ID Numbers ICMJE | IRB-16822 SU-12082009-4523 ( Other Identifier: Stanford University ) RV-0365 ( Other Identifier: Celgene Corporation ) HEMMDS0022 ( Other Identifier: OnCore ) |
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Has Data Monitoring Committee | Yes | ||||
U.S. FDA-regulated Product |
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IPD Sharing Statement ICMJE |
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Responsible Party | Jason Robert Gotlib, Stanford University | ||||
Study Sponsor ICMJE | Jason Robert Gotlib | ||||
Collaborators ICMJE | Celgene Corporation | ||||
Investigators ICMJE |
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PRS Account | Stanford University | ||||
Verification Date | June 2017 | ||||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |