Stopped: Enrollment ended early due to interim data suggesting a lack of efficacy. Interim data did demonstrate that IUHSCT was safe and well tolerated, but the PI and DSMB agreed that enrollment should not continue under the current treatment protocol.
The investigators aims to evaluate the safety of in utero hematopoietic stem cell transplantation in fetuses with alpha-thalassemia major performed at the time of in utero transfusion of red blood cells.
Age range
18 Weeks – 26 Weeks
Sex
ALL
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A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Maternal participant tolerance of bone marrow harvest
Timeframe: 5 year recruitment phase to include time of bone marrow harvest through 30 days after delivery
Safety of in utero hematopoietic stem cell transplantation when performed at the same time as in utero blood transfusion for the fetal participant
Timeframe: 5 year recruitment plus 1 year data collection phase to include time of IUHCT through 1 year after delivery