Plain-English translation of NCT07252050 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 medication that may help prevent transplant failure in children and young adults with sickle cell disease who are receiving a bone marrow transplant from a half-matched family member. The study hopes that adding the medication to the standard transplant process will reduce the chance that the new bone marrow fails to take hold in the body—a complication that can cause sickle cell symptoms to return.
Bone marrow transplants can cure sickle cell disease, but sometimes the new marrow fails to engraft properly, especially in young patients, allowing the disease to come back. This trial aims to find out whether this medication can reduce that risk and improve long-term survival without causing extra side effects.
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You would receive a hydroxyurea medication for at least 60 days before your transplant to prepare your body. Then you would start the conditioning regimen (strong chemotherapy and low-dose radiation) along with , which you would continue taking for several months after receiving the bone marrow transplant. You would also receive additional medications to prevent graft-versus-host disease (a complication where the new marrow attacks your body). Throughout the study, you would have regular clinic visits, blood tests, and scans to monitor how well the transplant is working and how you are tolerating the treatment.
AI-generated summary from trial data · Jun 5, 2026 · Not medical advice
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