CompletedPhase 1/2

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

United States55 participantsStarted 2013-06

Plain-language summary

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.

Who can participate

Age range2 Years

SexALL

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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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

What they're measuring

Response to Leucine in Transfusion Dependent Patients With Diamond Blackfan Anemia

Timeframe: 9 Months

Trial details

NCT IDNCT01362595

SponsorNorthwell Health

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2018-06-30

Results submitted2021-03-02

View on ClinicalTrials.gov More Diamond Blackfan Anemia trials