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

Study 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.

Conditions

Fanconi Anemia Complementation Group A Fanconi Anemia

Interventions / Drugs

Safety and efficacy assessments

View on ClinicalTrials.gov ↗

Sponsor

RCKT

Rocket Pharmaceuticals

Gene Therapy

C51/100

Study Details

Enrollment9 participants

Primary Completion—

Lead SponsorRocket Pharmaceuticals Inc.

Data Retrieved2026-04-11 04:25:37.504858+00:00

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