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.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
Monitor long term safety of patients through blood laboratory evaluations and general health status
Timeframe: 15 years post-drug product infusion
Long term genetic correction assessed in bone marrow and blood
Timeframe: 15 years post-drug product infusion
Replication competent lentivirus (RCL)
Timeframe: 15 years post-drug product infusion
Insertion site analysis in blood
Timeframe: 15 years post-drug product infusion
Phenotypic correction
Timeframe: 15 years post-drug product infusion
Assessment for Malignancies
Timeframe: 15 years post-drug product infusion
Hematologic stabilization
Timeframe: 15 years post-drug product infusion