An Operationally Seamless Phase 1/2/3 Study Consisting of a Safety and Dose-finding Phase 1/2 and Randomized, Open-label, Active-controlled Phase 3 to Evaluate UX701 AAV Gene Therapy in Adults with Wilson Disease

Wilson disease

Stage 1 (Phase 1/2) Primary Endpoints include the following assessments for UX701: Incidence of TEAEs, TESAEs, AESIs, treatment-related TEAEs, and treatment-related TESAEs, Stage 1 (Phase 1/2) Primary Endpoints include the following assessments for UX701: Change in 24-hour urinary copper concentration from Baseline at Week 52, Stage 1 (Phase 1/2) Primary Endpoints include the following assessments for UX701: Change in total copper, ceruloplasmin, NCC, free copper, and ceruloplasmin activity levels from Baseline at Week 52, Stage 1 (Phase 1/2) Primary Endpoints include the following assessments for UX701: Percent reduction in SOC medication by Week 52, Stage 1 (Phase 1/2) Primary Endpoints include the following assessments for UX701: Number of subjects who discontinue SOC medication by Week 52 (measured as complete response, response, or no response), Stage 1 (Phase 1/2) Primary Endpoints include the following assessments for UX701: Number of consecutive weeks off SOC medication at Week 52, Stage 2 (Phase 3):Change in 24-hour urinary copper from Baseline at Week 52, evaluated for superiority, Stage 2 (Phase 3) Primary Endpoints;Percent reduction in SOC medication by Week 52, evaluated for superiority

Stage 2 (Phase 3) Secondary Endpoints include the following comparisons between UX701 and placebo: Change in ceruloplasmin activity levels from Baseline at Week 52, evaluated for superiority, Stage 2 (Phase 3) Secondary Endpoints Number of subjects who discontinue SOC medication by Week 52, Stage 2 (Phase 3) Secondary Endpoints Change in FACIT-Fatigue scale score from Baseline at Week 52;Change in liver copper concentration assessed by liver biopsy from Baseline at Week 52

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