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

ALS Clinical Trial

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)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05683860
• Other study ID #: WVE-004-002
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: December 14, 2022
• Est. completion date: June 30, 2023

Study information

• Verified date: January 2023
• Source: Wave Life Sciences Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 8
• Est. completion date: June 30, 2023
• Est. primary completion date: June 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• ALS
• Amyotrophic Lateral Sclerosis
• Aphasia, Primary Progressive
• Frontotemporal Dementia
• FTD
• Motor Neuron Disease
• Pick Disease of the Brain

Intervention

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

Locations

Country	Name	City	State
Netherlands	Erasmus MC	Rotterdam
Netherlands	Universitair Medisch Centrum Utrecht	Utrecht
United Kingdom	University of Oxford - Nuffield Department of Clinical Neurosciences	Oxford

Sponsors (1)

Lead Sponsor	Collaborator
Wave Life Sciences Ltd.

Countries where clinical trial is conducted

Netherlands,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety: Number of patients with adverse events (AEs)		Day 1 to Week 120 (end of study)
Primary	Safety: Number of patients with a severe AE		Day 1 to Week 120 (end of study)
Primary	Safety: Number of patients with serious AEs (SAEs)		Day 1 to Week 120 (end of study)
Primary	Safety: Number of patients who withdraw due to AEs		Day 1 to Week 120 (end of study)