Plain-English translation of NCT03121001 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 stem cell transplant procedure for people with severe sickle cell disease who have complications like strokes, frequent pain crises, or organ damage. You would receive stem cells from a half-matched family member (a parent, sibling, or other relative who shares about half of your immune system markers). Before the transplant, you'll receive low-dose radiation and chemotherapy medications to prepare your body to accept the new cells, along with immunosuppressive drugs afterward to help prevent rejection.
Most sickle cell patients who need a transplant don't have a perfectly matched sibling donor available. This trial is testing whether using a half-matched family member as a donor—combined with special conditioning and rejection-prevention medications—can make transplants possible and successful for more patients.
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You will come to the hospital for intensive conditioning treatment with low-dose radiation and chemotherapy medications over several days, followed by infusion of stem cells from your half-matched family member. After the transplant, you'll receive immunosuppressive medications and stay in close contact with the study team for monitoring and medical care. You'll have study visits and blood tests at days 30, 60, 100, 180, 365, and yearly afterward to track how well the transplant is working and watch for any side effects.
AI-generated summary from trial data · Jun 9, 2026 · Not medical advice
United States