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



Status:Active, not recruiting
Conditions:Anemia
Therapuetic Areas:Hematology
Healthy:No
Age Range:2 - Any
Updated:3/15/2019
Start Date:June 2013
End Date:October 30, 2019

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Trial Summary
Trial Overview
Inclusion Criteria

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

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.

Leucine will be provided to participants in the form of a capsule and will be taken three
times daily.

Blood hemoglobin levels will be monitored every 3-4 weeks for 9 months.

The entire study will last 12-15 months in length.

Subjects must be two years of age or older and on transfusion for more than six months prior
to enrollment.

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
We found this trial at
11
sites
Boston Children's Hospital
300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Colin Sieff, MBBCh
Phone: 617-355-3748
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Children's Hospital of Philadelphia
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Helge Hartung, MD
Phone: 267-426-6765
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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University of Michigan/ CS Mott Children's Hospital
Ann Arbor, Michigan 48167
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University of Missouri-Columbia Women's and Children's Hospital
Columbia, Missouri 65201
Principal Investigator: Thomas W Loew, MD
Phone: 573-882-1960
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Columbia, MO
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U.T. Southwestern Medical Center
2201 Inwood Rd
Dallas, Texas 75235
(214) 645-8300
Principal Investigator: Zora R Rogers, MD
Phone: 214-648-3896
U.T. Southwestern Medical Center The story of UT Southwestern Medical Center is one of commitment...
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Riley Hospital for Children
705 Riley Hospital Dr
Indianapolis, Indiana 46202
(317) 944-5000
Principal Investigator: Gregorz Nalepa, MD, PhD
Phone: 317-948-0101
Riley Hospital for Children Riley Hospital for Children at IU Health is a place of...
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Children's Specialty Center of Nevada
Las Vegas, Nevada 89109
Principal Investigator: Jonathan Bernstein, MD
Phone: 702-732-1493
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Las Vegas, NV
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University of Louisville
500 S Preston St
Louisville, Kentucky
(502) 852-5555
Principal Investigator: Arun Panigrahi, MD
Phone: 502-629-7180
University of Louisville The University of Louisville is a state supported research university located in...
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Cohen Children's Medical Center of New York
New Hyde Park, New York 11040
Phone: 516-562-1504
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New Hyde Park, NY
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Stanford University Medical Center
Palo Alto, California 94304
Principal Investigator: Bertil Glader, MD, PhD
Phone: 650-724-2448
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Palo Alto, CA
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Phoenix Children's Hospital
1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Christine Knoll, MD
Phone: 602-933-0171
Phoenix Children's Hospital Phoenix Children's Hospital has provided hope, healing, and the best healthcare for...
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