Tested injection for severe transfusion-dependent thalassemia
Treatments studied
Part of Blood & lymphatic clinical trials.
This early-stage study tests the safety and possible benefits of an injection called HGI-001 for people with severe transfusion-dependent beta thalassemia. It may help by preparing you for stem cell transplant, but you must meet several health and lab criteria and be able to return often for follow-up tests.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Be age 18 to 35 and able to give consent (or have a legal guardian consent).
- Have a confirmed diagnosis of severe transfusion-dependent beta thalassemia (TDT) and provide proof from tests.
- Have needed blood transfusions frequently or in large amounts in the last 2 years, and have been on regular full-volume transfusions for at least 3 months.
- Have hemoglobin (Hb) at or above 9.0 g/dL, and iron overload that is not too severe (ferritin < 3000 μg/L; heart and liver iron should be “moderate or less”).
- Be able to safely receive busulfan pre-treatment and then hematopoietic stem cell (HSC) transplant, with acceptable heart, liver, kidney, lung, and blood-clotting function as judged by the study doctor.
- Agree to frequent hospital visits and complete all follow-up testing for up to 2 years after the stem cells are returned. Have no major excluded infections, cancers, certain blood disorders, or recent prohibited medicines. (This includes avoiding several specific drugs in the prior 3 months and avoiding other gene therapies).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
Similar studies
Other trials that look related to this one.
This early-phase trial tests a new way to restore working blood-making cells using your own stem cells for people with transfusion-dependent beta thalassemia major. The goal is to improve safety and blood results, and possibly reduce problems from the disease.
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This trial tests a new gene therapy that uses a modified virus to add a working copy of the gene for beta globin, which may help your body make healthy red blood cells. It is designed for people with beta thalassemia who have not had a bone marrow transplant and do not have a matching sibling donor.
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