Back to resultsOpen-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
Primary Purpose
ALS, FTD
Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
WVE-004
Sponsored by
About this trial
This is an interventional treatment trial for ALS
Eligibility Criteria
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.
Sites / Locations
- Erasmus MC
- Universitair Medisch Centrum Utrecht
- University of Oxford - Nuffield Department of Clinical Neurosciences
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Experimental: WVE-004 (Dose A)
Arm Description
Outcomes
Primary Outcome Measures
Safety: Number of patients with adverse events (AEs)
Safety: Number of patients with a severe AE
Safety: Number of patients with serious AEs (SAEs)
Safety: Number of patients who withdraw due to AEs
Secondary Outcome Measures
Full Information
NCT ID
NCT05683860
First Posted
January 4, 2023
Last Updated
October 18, 2023
Sponsor
Wave Life Sciences Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT05683860
Brief Title
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
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)
Study Type
Interventional
2. Study Status
Record Verification Date
January 2023
Overall Recruitment Status
Terminated
Why Stopped
Despite robust, sustained reductions in poly(GP), no clinical benefit was seen at 24 weeks, and reductions in poly(GP) were not associated with stabilization in functional outcomes. Based on these data, Wave decided to stop development of WVE-004.
Study Start Date
December 14, 2022 (Actual)
Primary Completion Date
June 30, 2023 (Actual)
Study Completion Date
June 30, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Wave Life Sciences Ltd.
4. Oversight
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
Data Monitoring Committee
Yes
5. 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
ALS, FTD
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
8 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Experimental: WVE-004 (Dose A)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
WVE-004
Intervention Description
WVE-004 is a stereopure antisense oligonucleotide. It is administered via intrathecal injection
Primary Outcome Measure Information:
Title
Safety: Number of patients with adverse events (AEs)
Time Frame
Day 1 to Week 120 (end of study)
Title
Safety: Number of patients with a severe AE
Time Frame
Day 1 to Week 120 (end of study)
Title
Safety: Number of patients with serious AEs (SAEs)
Time Frame
Day 1 to Week 120 (end of study)
Title
Safety: Number of patients who withdraw due to AEs
Time Frame
Day 1 to Week 120 (end of study)
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director, MD
Organizational Affiliation
Wave Life Sciences
Official's Role
Study Director
Facility Information:
Facility Name
Erasmus MC
City
Rotterdam
ZIP/Postal Code
3015
Country
Netherlands
Facility Name
Universitair Medisch Centrum Utrecht
City
Utrecht
Country
Netherlands
Facility Name
University of Oxford - Nuffield Department of Clinical Neurosciences
City
Oxford
ZIP/Postal Code
OX3 7LF
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
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