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 whether a medication called can make bone marrow transplants work better for children and young adults with sickle cell disease. The medication is added to the preparation treatment (called conditioning) before the transplant and continues afterward. The goal is to prevent a serious complication called graft failure, where the new bone marrow doesn't take hold and sickle cell symptoms come back.
Bone marrow transplants can cure sickle cell disease, but sometimes they don't work properly—especially in children—leading to graft failure and a return of symptoms. Researchers believe this medication might reduce that risk and improve transplant success rates.
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You would take hydroxyurea for at least 60 days before your transplant begins. Then you would receive conditioning chemotherapy and radiation over about 9 days, along with the new medication . After the transplant, you would continue taking along with other medications to prevent rejection and graft-versus-host disease. You would have frequent visits to the hospital for monitoring, blood tests, and evaluations over the first year, with follow-up care continuing after that. The study lasts approximately 2 years, with the main focus on your health and whether the transplant successfully cures your sickle cell disease.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
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