Clinical Trial

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Active, Not Recruiting
View on ClinicalTrials.gov →
Summary
This is a long-term safety and efficacy follow-up study for subjects with Fanconi Anaemia Subtype A who have been treated with ex vivo gene therapy on the FANCOLEN-I trial. After completion of the FANCOLEN-I study, eligible subjects will be followed for a total of 15 years post gene therapy treatment. No investigational drug product will be administered during this study.
Protocol Amendment History 2 amendments
This ClinicalTrials.gov record has been amended 2 times since 2020-06-16; most recent amendment 2026-04-07.
Status change: Enrolling by Invitation → Active, Not Recruiting 2026-04-07
Trial Details
NCT Number NCT04437771
Lead Sponsor Rocket Pharmaceuticals Inc.
Conditions Fanconi Anemia Complementation Group A, Fanconi Anemia
Enrollment 9 participants
Start Date 2020-06-01
Primary Completion 2034-01-17 (estimated)
Study Completion 2034-01-17 (estimated)
Updated on ClinicalTrials.gov 2026-04-13