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

Wave Life Sciences

Status and phase

Terminated

Phase 2

Phase 1

Conditions

ALS

FTD

Treatments

Drug: WVE-004

Study type

Interventional

Funder types

Industry

Identifiers

NCT05683860

WVE-004-002

Details and patient eligibility

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.

Enrollment

8 patients

Sex

All

Volunteers

No Healthy Volunteers

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.