ALS
+1
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
Sponsored by Wave Life Sciences Ltd.
About this trial
Last updated 2 years ago
Study ID
WVE-004-002
Status
Enrolling by invitation
Type
Interventional
Phase
Phase 1/Phase 2
Placebo
No
Accepting
All
All
Not accepting
Healthy Volunteers
Trial Timing
Started 3 years ago
What is this trial about?
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.
What are the participation requirements?
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.