Trial of HQK-1001 in Beta Thalassemia Intermedia in Lebanon
Status:
Completed
Trial end date:
2013-01-01
Target enrollment:
Participant gender:
Summary
Beta thalassemia intermedia syndromes are genetic anemias caused by mutations which reduce
production of beta globin, a major component of adult hemoglobin A, the protein which
delivers oxygen throughout the body. Patients suffer from poor growth, fatigue, heart
failure, endocrine deficiencies, and eventually, many require chronic blood transfusions.
There is no approved therapeutic for the deficiency of beta globin chains in beta
thalassemia.
This trial will study an oral therapeutic which stimulates production of fetal globin, an
alternate type which is produced by all humans, but is normally switched off in infancy. This
type of globin can compensate for the missing protein in beta thalassemia.