Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
Completed · Phase 1/Phase 2
Conditions studied: Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
In brief
This study will determine the safety and possibility of giving the amino acid, leucine, in patients with Diamond Blackfan anemia(DBA)who are on dependent on red blood cell transfusions. The leucine is expected to produce a response in patients with DBA to the point where red blood cell production is increased. Red cell transfusions can then be less frequent or possibly discontinued. The investigators will study the side effects, if any, of giving leucine to DBA patients. Leucine levels of leucine will be obtained at baseline and during the study. The drug leucine will be provided in capsule form and taken 3 times a day for a total of 9 months.
Key facts
- Study ID
- NCT01362595
- Run by
- Northwell Health
- People needed
- 55
- Starts
- 2013-06-01
- Expected to finish
- 2020-11-30
- Last updated by the study team
- 2022-12-02
Who can join
Age: 2 and older. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- 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
You may not qualify if…
- 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
Where it is running
- Phoenix Children's Hospital — Phoenix, Arizona, United States
- Stanford University Medical Center — Palo Alto, California, United States
- Riley Hospital for Children — Indianapolis, Indiana, United States
- University of Louisville — Louisville, Kentucky, United States
- Boston Children's Hospital — Boston, Massachusetts, United States
- University of Michigan C.S. Mott Children's Hospital — Ann Arbor, Michigan, United States
- University of Missouri-Columbia Women's and Children's Hospital — Columbia, Missouri, United States
- Children's Specialty Center of Nevada — Las Vegas, Nevada, United States
- Cohen Children's Medical Center of New York — New Hyde Park, New York, United States
- Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States
- UT Southwestern Medical Center — Dallas, Texas, United States
Full record on ClinicalTrials.gov
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