Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
Status:
Recruiting
Trial end date:
2023-02-01
Target enrollment:
Participant gender:
Summary
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to
evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients
with C9orf72-associated ALS or FTD. To participate in the study, patients must have a
documented mutation (GGGGCC [G4C2] repeat expansion) in the first intronic region of the
C9orf72 gene and be diagnosed with ALS or FTD.