Plain-English translation of NCT03214354 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 whether a gentler form of stem cell transplant can safely cure sickle cell disease in children who have a brother or sister whose blood cells match theirs — even if they have different blood types. Normally, stem cell transplants use very strong chemotherapy to prepare the body, but this study uses milder conditioning to see if it works just as well while causing fewer side effects.
Sickle cell disease causes serious pain, organ damage, and shortened lifespans, and stem cell transplant is the only cure. However, most children don't have access to this treatment because it's only safe with a perfectly matched donor — usually a sibling. This trial explores whether a gentler approach can work for more children, even when the donor has a different blood type.
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You would receive chemotherapy and other medicines to prepare your body for the transplant — using gentler doses than standard transplants. Then you would receive stem cells from your matched sibling donor through an infusion. Afterward, you would have frequent visits to monitor how well the new blood cells take hold and to watch for any side effects. The trial will pay special attention to tracking your red blood cell recovery to catch and treat any complications early.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
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