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)

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.

Study Overview

• Status: Terminated
• Conditions: ALS; FTD
• Intervention / Treatment: Drug: WVE-004

• Study Type: Interventional
• Enrollment (Actual): 8
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Netherlands
  • Rotterdam, Netherlands, 3015
    • Erasmus MC
  • Utrecht, Netherlands
    • Universitair Medisch Centrum Utrecht
• United Kingdom
  • Oxford, United Kingdom, OX3 7LF
    • University of Oxford - Nuffield Department of Clinical Neurosciences

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / 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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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)

Collaborators and Investigators

• Sponsor: Wave Life Sciences Ltd.
• Investigators: Study Director: Medical Director, MD, Wave Life Sciences

Study record dates

Study Major Dates

• Study Start (Actual): December 14, 2022
• Primary Completion (Actual): June 30, 2023
• Study Completion (Actual): June 30, 2023

Study Registration Dates

• First Submitted: January 4, 2023
• First Submitted That Met QC Criteria: January 4, 2023
• First Posted (Actual): January 13, 2023

Study Record Updates

• Last Update Posted (Actual): October 23, 2023
• Last Update Submitted That Met QC Criteria: October 18, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Neurocognitive Disorders; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Dementia; Language Disorders; Communication Disorders; Speech Disorders; Frontotemporal Lobar Degeneration; Aphasia; Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Frontotemporal Dementia; Aphasia, Primary Progressive; Pick Disease of the Brain
• Other Study ID Numbers: WVE-004-002

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes