NCT01362595 - Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

June 30, 2013

Primary Completion Date

June 30, 2018

Study Completion Date

November 30, 2020

Conditions

Diamond Blackfan AnemiaBlackfan Diamond SyndromeDBACongenital Hypoplastic AnemiaPure Red Cell Aplasia

Interventions

DRUG

leucine

"Dosage of leucine will be dependent on body surface area (BSA):~leucine 700 mg/m2/dose by mouth three times a day"

Trial Locations (11)

Cohen Children's Medical Center of New York, New Hyde Park

Children's Hospital of Philadelphia, Philadelphia

University of Louisville, Louisville

Riley Hospital for Children, Indianapolis

University of Michigan C.S. Mott Children's Hospital, Ann Arbor

University of Missouri-Columbia Women's and Children's Hospital, Columbia

UT Southwestern Medical Center, Dallas

Phoenix Children's Hospital, Phoenix

Children's Specialty Center of Nevada, Las Vegas

Stanford University Medical Center, Palo Alto

Boston Children's Hospital, Boston