Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
NCT ID: NCT01362595
Last Updated: 2022-12-02
Study Results
Results available
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
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Recruitment Status
COMPLETED
Clinical Phase
PHASE1/PHASE2
Total Enrollment
55 participants
Study Classification
INTERVENTIONAL
Study Start Date
2013-06-30
Study Completion Date
2020-11-30
Brief Summary
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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.
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.
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Detailed Description
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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.
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.
Conditions
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Diamond Blackfan Anemia
Blackfan Diamond Syndrome
DBA
Congenital Hypoplastic Anemia
Pure Red Cell Aplasia
Study Design
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Allocation Method
NA
Intervention Model
SINGLE_GROUP
Primary Study Purpose
TREATMENT
Blinding Strategy
NONE
Study Groups
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Leucine
No alternative treatment arm
Group Type
OTHER
leucine
Intervention Type
DRUG
Dosage of leucine will be dependent on body surface area (BSA):
leucine 700 mg/m2/dose by mouth three times a day
Interventions
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leucine
Dosage of leucine will be dependent on body surface area (BSA):
leucine 700 mg/m2/dose by mouth three times a day
Intervention Type
DRUG
Other Intervention Names
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L-leucine
Eligibility Criteria
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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
* 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
* 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
Minimum Eligible Age
2 Years
Eligible Sex
ALL
Accepts Healthy Volunteers
No
Sponsors
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Northwell Health
OTHER
Sponsor Role
lead
Responsible Party
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Adrianna Vlachos, MD
MD, Principal Investigator
Responsibility Role
PRINCIPAL_INVESTIGATOR
Principal Investigators
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Adrianna Vlachos, MD
Role: PRINCIPAL_INVESTIGATOR
Feinstein Institutes for Medical Research; Cohen Children's Medical Center
Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
Site Status
Stanford University Medical Center
Palo Alto, California, United States
Site Status
Riley Hospital for Children
Indianapolis, Indiana, United States
Site Status
University of Louisville
Louisville, Kentucky, United States
Site Status
Boston Children's Hospital
Boston, Massachusetts, United States
Site Status
University of Michigan C.S. Mott Children's Hospital
Ann Arbor, Michigan, United States
Site Status
University of Missouri-Columbia Women's and Children's Hospital
Columbia, Missouri, United States
Site Status
Children's Specialty Center of Nevada
Las Vegas, Nevada, United States
Site Status
Cohen Children's Medical Center of New York
New Hyde Park, New York, United States
Site Status
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Site Status
UT Southwestern Medical Center
Dallas, Texas, United States
Site Status
Countries
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United States
References
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Reference Type
DERIVED
Provided Documents
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Document Type: Study Protocol and Statistical Analysis Plan
Related Links
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http://www.dbar.org
Diamond Blackfan Anemia Registry - a registry for patients with DBA in North America
http://www.dmaf.org
Daniella Maria Arturi Foundation is a family sponsored group which facilitates research for DBA.
http://www.dbafoundation.org
The DBA foundation is a family sponsored organization who raise funds for research and provides emotional support to families.
Other Identifiers
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12-375B
Identifier Type: -
Identifier Source: org_study_id
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