A Fabry Disease Gene Therapy Study (MARVEL1)
Fabry Disease / Posted 6 months ago
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 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).