NCT05683860

Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)

A Multicenter, Open-label Extension (OLE) Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 8 (actual)
• Sponsor: Wave Life Sciences Ltd. · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• ALS
• FTD

Interventions

Type	Name	Description
DRUG	WVE-004	WVE-004 is a stereopure antisense oligonucleotide. It is administered via intrathecal injection

Timeline

• Start date: 2022-12-14
• Primary completion: 2023-06-30
• Completion: 2023-06-30
• First posted: 2023-01-13
• Last updated: 2023-10-23

Locations

3 sites across 2 countries: Netherlands, United Kingdom

Regulatory

• FDA-regulated drug study