Gene therapy for rare blood disease shows promise in Long-Term Follow-Up
NCT ID NCT07527975
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study follows 14 people with Fanconi Anemia who previously received RP-L102 gene therapy. Researchers will monitor their health for years to see if the treatment safely improves blood counts and reduces the need for a bone marrow transplant. The goal is to understand long-term benefits and risks.
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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
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
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Other studies related to the condition(s) this trial covers.
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- New MRI method could spot oral cancer early in rare disease patients
- New hope for rare cancer: targeted drug afatinib tested in fanconi anemia patients
- New drug combo aims to make bone marrow transplants safer for kids