Plain-English translation of NCT04774536 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 is an early-stage trial testing a new treatment called for people with severe sickle cell disease. The treatment works by taking your own blood-forming stem cells, using advanced gene editing to fix the genetic error that causes sickle cell disease, and then transplanting the corrected cells back into your body. Researchers hope this will reduce or eliminate the painful and dangerous complications of sickle cell disease.
Current treatments for severe sickle cell disease can help manage pain and complications, but they don't cure the disease. This trial is testing whether fixing the genetic root cause—rather than just treating symptoms—could offer a lasting solution for people whose sickle cell disease hasn't responded well to other therapies.
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If you enroll, doctors will collect your own blood stem cells and send them to a lab where they will be corrected using gene editing technology. You will then receive chemotherapy to prepare your body, followed by a one-time transplant of your corrected cells back into your bloodstream. The trial will closely monitor your safety and health over time, with the first group of 6 adults enrolled with careful safety checks between each patient, and potentially 3 adolescents enrolled afterward if safety is confirmed.
AI-generated summary from trial data · Jun 6, 2026 · Not medical advice
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