Plain-English translation of NCT07599176 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle Cell Disease research guide →Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.
This trial is testing a new approach to stem cell transplantation for patients with sickle cell disease or beta-thalassemia who have a matched family member donor. The procedure uses a combination of medications — including and — along with a controlled dose of radiation to prepare your body to accept healthy blood-forming cells from your donor. The goal is to reduce the chance of transplant failure while keeping side effects as low as possible.
Previous transplant approaches have worked well, but over time some patients' bodies slowly reject the donor cells. This trial is testing whether a slightly higher radiation dose, combined with a new supportive medication called , can help the transplanted cells take hold more permanently without causing serious complications like graft-versus-host disease.
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You will receive conditioning medications (including and radiation) over several days to prepare your body for the transplant. Your matched donor will undergo a procedure to collect their healthy stem cells, which will then be infused into you. After transplant, you will need frequent hospital visits or admissions for monitoring, blood tests, and management of any side effects. You will also complete questionnaires about your quality of life, pain, and overall wellbeing at several points during the study. Follow-up care continues for years after the transplant to ensure the graft is working well.
AI-generated summary from trial data · Jul 14, 2026 · Not medical advice
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