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 people with sickle cell disease or beta-thalassemia who have serious complications from their disease. The study uses a milder preparation regimen — involving low-dose radiation, , and a newer medicine called — to help a matched family donor's healthy blood cells take root in your body. The goal is to cure your disease while reducing the severe side effects that have been a concern with traditional transplants.
Previous transplant approaches have worked well for many patients, but over time some patients lose the donor cells and their own disease cells return. This trial is designed to find a better balance: using slightly more radiation to help donor cells stay in your body longer, while adding a new medicine to prevent the harmful immune reactions that can happen after transplant.
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You would undergo a stem cell transplant procedure where your matched family donor's healthy blood cells are infused into your body after you receive preparation medicines and low-dose radiation. Before transplant, you'll have many tests and evaluations. After transplant, you will need close monitoring with frequent clinic visits, blood tests, and imaging scans for at least the first year, and longer-term follow-up beyond that. The research team will also assess how your organs are functioning, how you're feeling emotionally, and other aspects of your health before and after the transplant.
AI-generated summary from trial data · Jun 14, 2026 · Not medical advice
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