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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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
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: Colin Sieff, MBBCh
Phone: 617-355-3748
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Helge Hartung, MD
Phone: 267-426-6765
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Columbia, Missouri 65201
Principal Investigator: Thomas W Loew, MD
Phone: 573-882-1960
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2201 Inwood Rd
Dallas, Texas 75235
(214) 645-8300
Principal Investigator: Zora R Rogers, MD
Phone: 214-648-3896
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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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Las Vegas, Nevada 89109
Principal Investigator: Jonathan Bernstein, MD
Phone: 702-732-1493
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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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New Hyde Park, New York 11040
Phone: 516-562-1504
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Palo Alto, California 94304
Principal Investigator: Bertil Glader, MD, PhD
Phone: 650-724-2448
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1919 E Thomas Rd
Phoenix, Arizona 85006
(602) 933-1000
Principal Investigator: Christine Knoll, MD
Phone: 602-933-0171
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