Study drug for severe alpha thalassemia in people needing regular transfusions
Treatments studied
Part of Blood & lymphatic clinical trials.
This early-phase trial studies HGI-002 to see if it’s safe and can help people with severe alpha thalassemia who need frequent blood transfusions. It also requires planning for a special treatment approach that includes busulfan pre-treatment and stem cell transplantation.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be between 12 and 35 years old, with a parent/legal guardian able to sign consent if needed
- Have confirmed severe alpha thalassemia that requires regular transfusions (transfusion needs in the last 2 years, or already diagnosed as transfusion-dependent)
- Have had transfusions at full usual dose for at least 3 months before screening, and your hemoglobin (Hb) stays at or above 9.0 g/dL
- Have iron overload levels that are not too severe (ferritin under 3000 µg/L) and heart/liver iron that is moderate or less
- Be able to have busulfan pre-treatment and then hematopoietic stem cell transplantation, and have your organs (heart, liver, kidney, lungs, blood clotting) meet stability requirements
- Agree to frequent follow-up visits and tests for up to 2 years after reinfusion of HGI-002
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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