Plain-English translation of NCT03587272 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle Cell Disease research guide →Phase 2 — Testing in a bigger group (up to a few hundred people) to see if the treatment actually works and is still safe.
This trial is testing a bone marrow transplant approach for children with sickle cell disease that uses lower doses of radiation and chemotherapy compared to standard transplants. The study combines three medications — alemtuzumab, low-dose whole-body radiation, and sirolimus — to help the new bone marrow cells take hold while causing fewer harmful side effects. Researchers want to see if this gentler approach can cure sickle cell disease while keeping children healthier during recovery.
Standard bone marrow transplants for sickle cell disease can cause severe complications like graft-versus-host disease (when the new cells attack the child's body) and other serious toxicities. This trial exists to find a way to cure sickle cell disease with a bone marrow transplant while minimizing these dangerous side effects and protecting children's quality of life.
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If you qualify, you would be admitted to a transplant center where you receive the three medications as part of your conditioning regimen before receiving bone marrow cells from your matched sibling donor. You would stay in the hospital during the initial transplant period and then be followed closely with blood tests, imaging, and clinic visits to monitor how well the new bone marrow is working and how you're recovering. The trial tracks your health, side effects, quality of life, and how well the transplant worked for about one year after the procedure.
AI-generated summary from trial data · Jun 6, 2026 · Not medical advice
United States