Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
- Registration Number
- NCT04931862
- Lead Sponsor
- Wave Life Sciences Ltd.
- Brief Summary
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC \[G4C2\] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 35
- ALS-specific: Diagnosis of ALS based on clinical manifestations.
- ALS-specific: Clinically diagnosed possible, laboratory supported probable, probable, or definite criteria for diagnosing ALS according to the World Federation of Neurology revised El Escorial criteria.
- ALS-specific: Patients receiving riluzole have been on a stable dose for a minimum of 30 days.
- ALS-specific: Patients on edaravone have received a minimum of 1 cycle (28 days).
- ALS-specific: Patients discontinuing riluzole or edaravone had the last dose administered ≥1 month prior to Screening.
- FTD-specific: Must have Global Clinical Dementia Rating - Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD) score of 0.5 or 1.
- FTD-specific: Able to undergo periodic magnetic resonance imaging (MRI) of the brain. Participants with mixed phenotype (ALS and FTD) need not undergo MRI if their ALS symptoms prevent it.
- Mixed-phenotype: Patients who are mixed phenotype (ALS and FTD) must meet both the ALS-specific and FTD-specific criteria.
- Clinically significant medical finding on the physical examination other than C9orf72-associated ALS or FTD that, in the judgment of the Investigator, will make the patient unsuitable for participation in, and/or completion of the trial procedures
- Received any other investigational drug, biological agent, or device within 1 month or 5 half-lives of study agent, whichever is longer. Received an investigational oligonucleotide, within the past 6 months or 5 half-lives of the drug, whichever is longer.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description WVE-004 (Dose D) or placebo WVE-004 - WVE-004 (Dose A) or placebo Placebo - WVE-004 (Dose B) or placebo WVE-004 - WVE-004 (Dose B) or placebo Placebo - WVE-004 (Dose C) or placebo Placebo - WVE-004 (Dose C) or placebo WVE-004 - WVE-004 (Dose D) or placebo Placebo - WVE-004 (Dose A) or placebo WVE-004 -
- Primary Outcome Measures
Name Time Method Safety: Proportion of patients with adverse events (AEs) Period 1 Day 1 to Period 2 Week 24 (end of study)
- Secondary Outcome Measures
Name Time Method Pharmacodynamic: Change from baseline in concentration of poly-GP levels in the CSF Period 1 Day 1 to Period 2 Week 24 (end of study) Pharmacokinetic: Concentration of WVE-004 in cerebrospinal fluid (CSF) Period 1 Day 1 to Period 2 Week 24 (end of study)
Trial Locations
- Locations (17)
Macquarie University
🇦🇺North Ryde, New South Wales, Australia
The Wesley Hospital
🇦🇺Brisbane, Queensland, Australia
Perron Institute
🇦🇺Nedlands, Western Australia, Australia
UZ Leuven
🇧🇪Leuven, Belgium
Sunnybrook Health Sciences Centre
🇨🇦Toronto, Ontario, Canada
McGill University Health Center - Research Institute
🇨🇦Montréal, Quebec, Canada
St James Hospital - Ireland
🇮🇪Dublin, Ireland
Erasmus University MC
🇳🇱Rotterdam, Netherlands
Universitair Medisch Centrum Utrecht
🇳🇱Utrecht, Netherlands
Auckland City Hospital
🇳🇿Auckland, New Zealand
Scroll for more (7 remaining)Macquarie University🇦🇺North Ryde, New South Wales, Australia