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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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