Plain-English translation of NCT03279094 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Sickle Cell Disease research guide →Phase 1 — Testing in a small group (usually 20–80 people) to find a safe dose and watch for side effects.
This trial is testing a stem cell transplant approach for people with severe sickle cell disease. You would receive special medications before the transplant to prepare your body, then receive stem cells from a family member who is a half-match for your immune system genes. The goal is to replace your damaged blood-forming cells with healthy ones and potentially cure your sickle cell disease.
Many people with sickle cell disease have severe complications like strokes, repeated pain crises, and organ damage that aren't fully controlled by current medications. This treatment aims to offer a potential cure by using a stem cell transplant from family members who don't need to be a perfect genetic match, which could help more patients access this life-changing therapy.
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You would receive two cycles of preparatory medications before your transplant. Then you would be admitted to the hospital to receive high-dose chemotherapy and other conditioning medications over several days, followed by the stem cell transplant from your family member. After the transplant, you would receive additional medications to prevent your immune system from rejecting the new cells, and you would need close medical monitoring for several months as your new immune system develops.
AI-generated summary from trial data · Jun 3, 2026 · Not medical advice
United States