Gene therapy for rare blood disease shows promise in Long-Term Follow-Up
NCT ID NCT07527975
First seen Apr 19, 2026 · Last updated Apr 29, 2026 · Updated 1 time
Summary
This study checks the long-term safety and effectiveness of a gene therapy called RP-L102 for people with Fanconi Anemia, a rare genetic condition that causes bone marrow failure and raises cancer risk. About 14 patients who already received RP-L102 in earlier studies will be followed for years to see if their blood counts stay stable, if they avoid needing a bone marrow transplant, and if any side effects appear. The goal is to understand whether this one-time treatment can help control the disease over time.
Disclaimer
Read more
Show less
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.
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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for FANCONI ANEMIA are added.
By submitting, you agree to our Terms of use
Contacts and locations
Show contact details
Enter your email to view the contact information for this study.
By submitting, you agree to our Terms of use
Locations
-
Hospital Infantil Universitario Niño Jesús
Madrid, 28009, Spain
-
Lucille Packard Children's Hospital, Stanford University
Palo Alto, California, 94305, United States
-
University College London Great Ormond Street Institute of Child Health (GOSH)
London, WC1N 1EH, United Kingdom
Conditions
Explore the condition pages connected to this study.