Gene therapy patients tracked for 15 years in groundbreaking Follow-Up
NCT ID NCT07527975
First seen Apr 19, 2026 · Last updated Apr 19, 2026
Summary
This study follows patients who previously received an experimental gene therapy (RP-L102) for Fanconi anemia, a rare genetic disorder that affects bone marrow. Researchers will monitor 14 participants for up to 15 years to check long-term safety, see if the treatment effect lasts, and track blood counts and cancer risks. The goal is to understand if this one-time gene therapy can provide lasting control of the disease without needing a bone marrow transplant.
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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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Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
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Lucille Packard Children's Hospital, Stanford University
Palo Alto, California, 94305, United States
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University College London Great Ormond Street Institute of Child Health (GOSH)
London, WC1N 1EH, United Kingdom
Conditions
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