Phoenix Children’s Hospital Research
Institute (PCRI) Website Clinical Trial Listing
** Please complete and return to Shy Walker at
swalker@phoenixchildrens.com
Study Title: A Pilot, Phase I/II Study of the Amino Acid Leucine in the Treatment of Patients With
Transfusion-Dependent Diamond Blackfan Anemia (LeucineDBA)
Study Purpose:
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.
Study Summary:
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.
Basic Eligibility Criteria:
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
Study Location(s):
Phoenix Children’s Hospital
Study Contact(s):
Erica Olson, RN
Eolson1@phoenixchildrens.com