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)

Wave Life Sciences Ltd.3 sites in 2 countries8 target enrollmentDecember 14, 2022

ConditionsALS FTD

InterventionsWVE-004

DrugsWVE-004

Overview

Phase: Phase 1
Intervention: WVE-004
Conditions: ALS
Sponsor: Wave Life Sciences Ltd.
Enrollment: 8
Locations: 3
Primary Endpoint: Safety: Number of patients with serious AEs (SAEs)
Status: Terminated
Last Updated: 2 years ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief 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.

Registry

clinicaltrials.gov

Start Date

December 14, 2022

End Date

June 30, 2023

Last Updated

2 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

Wave Life Sciences Ltd.

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Patient successfully completed the Phase 1b/2a study with WVE-004, WVE-004-001.

Exclusion Criteria

•Patient has a clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator or Sponsor, will make the patient unsuitable for participation in and/or completion of the trial procedures.
•Patient received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Patient received an investigational oligonucleotide within the past 6 months or 5 half-lives of the drug, whichever is longer.