Plain-English translation of NCT06506461 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 is an early-stage trial testing a new approach to sickle cell disease using your own stem cells that have been genetically modified with CRISPR technology. Researchers will collect your stem cells, edit them in a laboratory to fix the genetic problem that causes sickle cell disease, and then put the edited cells back into your body. The goal is to reduce or stop the painful crises and other complications of sickle cell disease.
Many people with sickle cell disease don't respond well to current treatments like hydroxyurea, and some need frequent blood transfusions that come with their own risks. This trial is exploring whether gene editing—a newer technology—can offer a lasting solution by correcting the root genetic cause of the disease.
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You will receive injections under the skin for 3 to 5 days to help your stem cells move into your bloodstream, then undergo a procedure to collect those stem cells. Your cells will be sent to a lab for genetic editing and freezing while you receive chemotherapy preparation at the hospital. Once ready, your edited cells will be infused back into you through an IV. You will be followed closely for 3 years after treatment, then enrolled in a long-term follow-up study for 12 additional years to monitor your health and response to the treatment.
AI-generated summary from trial data · Jun 12, 2026 · Not medical advice
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