Gene therapy patients monitored for years to ensure lasting safety
NCT ID NCT05145062
First seen Nov 12, 2025 · Last updated May 16, 2026 · Updated 27 times
Summary
This study follows 8 people with severe sickle cell disease or transfusion-dependent beta-thalassemia who already received a gene therapy called BIVV003 or ST-400. Researchers will check for side effects, survival, and how well the treatment controls their disease over many years. The goal is to make sure the therapy remains safe and effective long-term.
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the original study
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
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Henry Ford Health System
Detroit, Michigan, 48202, United States
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Karmanos Cancer Institute
Detroit, Michigan, 48201, United States
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UCSF Benioff Children's Hospital
Oakland, California, 94609, United States
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University of California Davis Health System
Sacramento, California, 95817, United States
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University of Minnesota
Minneapolis, Minnesota, 55455, United States
Conditions
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