A Phase 3 Safety and Efficacy Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1

Condition studied

Gaucher Disease Type 1

Investigational medicinal product(s)

Eligibility

Primary endpoint

1. Proportion of participants with stable hemoglobin concentration (decrease from baseline of no more than 1.5 g/dL) at Week 52.

Endpoint detail

1. • Proportion of participants with stable platelet count (decrease from baseline of no more than 25%) at Week 52 • Proportion of participants with stable spleen volume (increase from baseline of no more than 25%) at Week 52 • Proportion of participants with stable liver volume (increase from baseline of no more than 20%) at Week 52, 2. • Incidence of treatment-emergent adverse events (AEs) • Incidence of treatment-emergent serious adverse events (SAEs) • Changes from baseline in vital signs and routine laboratory assessments • Incidence of physical examination and 12-lead ECG clinically significant abnormalities, 3. • Change from baseline to each assessment point in plasma β-glucocerebrosidase (GCase) activity level • Change from baseline to each assessment point in leukocyte GCase activity level, 4. • Change from baseline in glucosylsphingosine (lyso-GL1) concentration • Change from baseline to each assessment point in bone marrow burden • Change from baseline to each assessment point in bone mineral density • Change from baseline to each assessment point in GD-DS3 score, 5. • Change from baseline in 36-Item Short Form Survey, version 2 (SF-36v2®) score • Change from baseline in Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) score • Change from baseline in Gaucher Disease Type 1 Patient Reported Outcome Measure (GD1-PROM), 6. Change from baseline to each assessment point in anti-Gcase antibody titer (total and neutralizing), 7. Clearance of vector genome in plasma and semen