Plain-English translation of NCT05477563 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
Read our Beta-Thalassemia research guide →Phase 3 — Testing in thousands of people, comparing the treatment against what doctors currently use. This is the last big step before approval.
This study is testing , a treatment that uses your own blood-forming stem cells edited with CRISPR technology (a tool that can modify genes) to treat transfusion-dependent beta-thalassemia or severe sickle cell disease. You would receive a single infusion of these edited cells through a central line, and researchers would monitor whether the treatment helps your body produce healthy blood cells on its own. The goal is to reduce or eliminate the need for regular blood transfusions or prevent the severe pain episodes that come with sickle cell disease.
People with severe thalassemia and sickle cell disease currently depend on frequent blood transfusions or face life-threatening complications, and these treatments don't address the root genetic cause. This medication is being tested to see if editing your own stem cells can correct the underlying problem and allow your body to make healthy blood cells without ongoing transfusions.
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If you enroll, you would first receive chemotherapy to prepare your bone marrow, then receive a single infusion of your own edited cells through a central venous catheter (a special IV line). After the infusion, you would be monitored closely through follow-up visits and blood tests to track how well the edited cells are working and to watch for any side effects. The study will evaluate whether the treatment reduces your need for transfusions or prevents sickle cell pain crises over time.
AI-generated summary from trial data · Jun 6, 2026 · Not medical advice
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