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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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12 December 2020 |
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Main ID: |
NCT01362595 |
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Date of registration:
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20/05/2011 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
LeucineDBA |
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Scientific title:
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The Use of Novel Therapies to Reconstitute Blood Cell Production and Promote Organ Performance Using Bone Marrow Failure as a Model: a Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia |
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Date of first enrolment:
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June 2013 |
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Target sample size:
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50 |
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Recruitment status: |
Active, not recruiting |
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URL:
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https://clinicaltrials.gov/show/NCT01362595 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Adrianna Vlachos, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Feinstein Institutes for Medical Research; Cohen Children's Medical Center |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- diagnosed with Diamond Blackfan anemia as published in British Journal of Hematology
- transfusion dependent
- age 2 years and older
- adequate renal function
- adequate liver function
- negative B-HCG if patient is a menstruating female and documentation of adequate
contraception
- signed informed consent
Exclusion Criteria:
- Known hypersensitivity to branched chain amino acids
- Diagnosis of an inborn error of amino acid metabolism disorder
- Prior hematopoietic stem cell transplantation
- Pregnancy, or plans to become pregnant during duration of trial
Age minimum:
2 Years
Age maximum:
N/A
Gender:
All
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Health Condition(s) or Problem(s) studied
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Blackfan Diamond Syndrome
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Congenital Hypoplastic Anemia
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Diamond Blackfan Anemia
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Pure Red Cell Aplasia
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DBA
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Intervention(s)
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Drug: leucine
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Primary Outcome(s)
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Response to Leucine in Transfusion dependent patients with Diamond Blackfan Anemia
[Time Frame: Patients will take leucine for 9 months. The study is expected to take 12-15 months to complete.]
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Secondary Outcome(s)
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Side effects of leucine in transfusion-dependent DBA patients
[Time Frame: Total study 12-15 months]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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