Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Northwell Health
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.
Age
2–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
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
You may not be if
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
Clareo Health | Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia
