A Fabry Disease Gene Therapy Study
brief summary
This is a multinational, open-label study to assess the safety and efficacy of FLT190 in up to 15 adult male participants with classical Fabry disease.
detailed description
Patients who provide consent to participate in this study will be screened for eligibility.
Eligible patients will attend the study site on the day prior to infusion (Day -1) for a baseline visit. On Day 0, FLT190 will be administered as a single dose, slow intravenous infusion. Following FLT190 treatment the patient will be discharged from the investigational site and will continue to be monitored at outpatient visits for a period of approximately 9 months; following which, the patient will enter a period of long-term follow-up conducted under a separate protocol.
The study will be conducted in 2 parts;
Part 1: Enrolment of previously treated patients (Dose escalation)
Part 2: Enrolment of previously untreated patients (Dose expansion).
official title
A Phase 1/2, Baseline-controlled, Non-randomized, Open-label, Single-ascending Dose Study of a Novel Adeno-associated Viral Vector (FLT190) in Patients With Fabry Disease