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

Sponsor

Wave Life Sciences Ltd. (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05683860

Collaborator

(none)

Enrollment

Location

Arm

37.6

Anticipated Duration (Months)

1.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
ALS FTD	Drug: WVE-004	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

42 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

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)

Actual Study Start Date :

Dec 14, 2022

Anticipated Primary Completion Date :

Feb 1, 2026

Anticipated Study Completion Date :

Feb 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Experimental: WVE-004 (Dose A)	Drug: WVE-004 WVE-004 is a stereopure antisense oligonucleotide. It is administered via intrathecal injection

Arm

Intervention/Treatment

Experimental: Experimental: WVE-004 (Dose A)

Drug: WVE-004

WVE-004 is a stereopure antisense oligonucleotide. It is administered via intrathecal injection

Outcome Measures

Primary Outcome Measures

Safety: Number of patients with adverse events (AEs) [Day 1 to Week 120 (end of study)]
Safety: Number of patients with a severe AE [Day 1 to Week 120 (end of study)]
Safety: Number of patients with serious AEs (SAEs) [Day 1 to Week 120 (end of study)]
Safety: Number of patients who withdraw due to AEs [Day 1 to Week 120 (end of study)]

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts 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.