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

Phase 1

Terminated

• Conditions: FTD; ALS
• Interventions: Drug: WVE-004

• Registration Number: NCT05683860
• Lead Sponsor: Wave Life Sciences Ltd.

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-12-14
• Status Verified: 2023-01
• Study First Submitted: 2023-01-04
• Study First Submitted QC: 2023-01-04
• Study First Posted: 2023-01-13
• Primary Completion: 2023-06-30
• Study Completion: 2023-06-30
• Last Update Submitted: 2023-10-18
• Last Update Posted: 2023-10-23

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 8

Inclusion Criteria

• Patient successfully completed the Phase 1b/2a study with WVE-004, WVE-004-001.

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Experimental: WVE-004 (Dose A)	WVE-004	-

Primary Outcome Measures

Name	Time	Method
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)
Safety: Number of patients with a severe AE	Day 1 to Week 120 (end of study)
Safety: Number of patients with adverse events (AEs)	Day 1 to Week 120 (end of study)

Trial Locations

Locations (3)

Erasmus MC; 🇳🇱 Rotterdam, Netherlands

Universitair Medisch Centrum Utrecht; 🇳🇱 Utrecht, Netherlands

University of Oxford - Nuffield Department of Clinical Neurosciences; 🇬🇧 Oxford, United Kingdom