Gene therapy patients monitored for years to ensure lasting safety
NCT ID NCT05145062
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows 8 people who received a one-time gene therapy (BIVV003 for sickle cell disease or ST-400 for beta-thalassemia) to see if it remains safe and effective over many years. Researchers will track serious side effects, survival, and blood markers like hemoglobin levels. Participants must have already received the therapy in an earlier study.
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This is a summary of
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
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