TerminatedPhase 1 / PHASE2

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)

Lead sponsor

Wave Life Sciences USA, Inc.

Asset

WVE-004

Listed sites

Recruiting sites

Enrollment

actual

Study population

Frontotemporal dementia

Key I/E criterion

Primary endpoint

•Safety

Footprint

Where this trial recruits

Site locations as reported to ClinicalTrials.gov. Site count is not enrollment count; per-site enrollment is not available from source.

Identifiers

Registered as

NCT IDNCT05683860

Org study IDWVE-004-002

Timeline

Milestones

Study start2022-12-14actual

Study first posted2023-01-13actual

Primary completion2023-06-30actual

Study completion2023-06-30actual

Last update posted2023-10-23actual

Assets

Drug assets

WVE-004

Study populations

Who this study enrolls

Frontotemporal dementia

Eligibility

Who can enroll

SexAll

Healthy volunteersNot accepted

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.

Endpoints (4)

What's being measured

Protocol endpoints and posted registry outcome measures, grouped into outcome categories. Composite endpoints show their component event types. Standard codes (LOINC, SNOMED CT) are shown where available.

Safety / tolerability / PK

4 endpoints

Primary/protocol endpoint

Safety: Number of patients with adverse events (AEs)

Time frame:Day 1 to Week 120 (end of study)

event count, event

Primary/protocol endpoint

Safety: Number of patients with a severe AE

Time frame:Day 1 to Week 120 (end of study)

event count, event

Primary/protocol endpoint

Safety: Number of patients with serious AEs (SAEs)

Time frame:Day 1 to Week 120 (end of study)

event count, event

Primary/protocol endpoint

Safety: Number of patients who withdraw due to AEs

Time frame:Day 1 to Week 120 (end of study)

event count, event

Provenance

Sources

Trial identity, design, statusClinicalTrials.gov API v2

Snapshot dateJuly 21, 2026

Endpoint classificationDelfa ADRD endpoint taxonomy

Results tableno registry results posted yet

Trial facts come from public ClinicalTrials.gov records. Endpoint categories are Delfa's classification of those records, not a ClinicalTrials.gov field. All figures reflect the July 21, 2026 snapshot.