Stopped: Despite robust, sustained reductions in poly(GP), no clinical benefit was seen at 24 weeks, and reductions in poly(GP) were not associated with stabilization in functional outcomes. Based on these data, Wave decided to stop development of WVE-004.
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult patients with ALS, FTD, or mixed ALS/FTD phenotype with a documented mutation in the C9orf72 gene. To participate in the study, patients must have successfully completed Phase 1b/2a WVE-004-001 study.
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Safety: Number of patients with adverse events (AEs)
Timeframe: Day 1 to Week 120 (end of study)
Safety: Number of patients with a severe AE
Timeframe: Day 1 to Week 120 (end of study)
Safety: Number of patients with serious AEs (SAEs)
Timeframe: Day 1 to Week 120 (end of study)
Safety: Number of patients who withdraw due to AEs
Timeframe: Day 1 to Week 120 (end of study)