Study of SDMB (2,2 Dimethylbutyrate, Sodium Salt) in Beta Thalassemia Intermedia in Thailand
Status:
Completed
Trial end date:
2012-12-01
Target enrollment:
Participant gender:
Summary
Beta thalassemia intermedia is an inherited blood disease caused by molecular mutations which
reduce the beta globin protein chain of adult hemoglobin A, the protein in red blood cells
which carries oxygen throughout the body. Beta thalassemias cause progressively severe
anemia, widespread organ damage, and often require blood transfusions. There is no FDA
approved therapeutic to treat the underlying cause of beta thalassemia. Fetal hemoglobin is
another type of endogenous hemoglobin which can replace the reduced beta globin protein,
reduce the anemia, and even abolish transfusion requirements. This type of hemoglobin is
normally suppressed in infancy.
Sodium 2,2 dimethylbutyrate (ST20, or HQK-1001) is a small molecule which stimulates
production of fetal hemoglobin in nonhuman primates and in human patients in Phase I/II
trials.
This is a Phase 2 open-label trial to evaluate the ability of this oral therapeutic to reduce
anemia in patients with beta thalassemia intermedia, when administered once daily for 26
weeks. All participants will receive the study drug.