Study Purpose: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.
ALS , FTD
Type of Intervention:
Phase 1/Phase 2
Study Chair(s)/Principal Investigator(s):
Medical Director, MD, Wave Life Sciences
Coordinating Center Contact Information
Wave Life Sciences Ltd.
Estimated Study Start Date:
12 / 14 / 2022
Estimated Study Completion Date:
02 / 01 / 2026
Posting Last Modified Date:
03 / 22 / 2023
Date Study Added to neals.org:
01 / 13 / 2023
- Patient successfully completed the Phase 1b/2a study with WVE-004, WVE-004-001.
- 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.
Universitair Medisch Centrum Utrecht
University of Oxford - Nuffield Department of Clinical Neurosciences
Oxford OX3 7LF