Study Purpose: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.
ALS , FTD
Type of Intervention:
Phase 1/Phase 2
Study Chair(s)/Principal Investigator(s):
Medical Director, MD, Wave Life Sciences
Coordinating Center Contact Information
Wave Life Sciences Ltd.
Estimated Study Start Date:
06 / 28 / 2021
Estimated Study Completion Date:
12 / 01 / 2023
Posting Last Modified Date:
02 / 03 / 2023
Date Study Added to neals.org:
06 / 18 / 2021
80 YearsInclusion Criteria:
1. ALS-specific: Diagnosis of ALS based on clinical manifestations.
2. 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.
3. ALS-specific: Patients receiving riluzole have been on a stable dose for a minimum of 30 days.
4. ALS-specific: Patients on edaravone have received a minimum of 1 cycle (28 days).
5. ALS-specific: Patients discontinuing riluzole or edaravone had the last dose administered ≥1 month prior to Screening.
6. FTD-specific: Must have Global Clinical Dementia Rating - Frontotemporal Lobar Degeneration (CDR® plus NACC FTLD) score of 0.5 or 1.
7. 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.
8. Mixed-phenotype: Patients who are mixed phenotype (ALS and FTD) must meet both the ALS-specific and FTD-specific criteria.
1. 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
2. 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.
North Ryde, New South Wales
The Wesley Hospital
Nedlands, Western Australia
Sunnybrook Health Sciences Centre
McGill University Health Center - Research Institute
St James Hospital - Ireland
Dublin D08 NHY1
Universitair Medisch Centrum Utrecht
Erasmus University MC
Auckland City Hospital
New Zealand Brain Research Institute
Karolinska University Hospital
University of Cambridge
University College London Hospital
London WC1N 3BG
King's College Hospital
London WC2R 2LS
University of Oxford - Nuffield Department of Clinical Neurosciences
Oxford OX3 7LF
University of Sheffield
Sheffield S10 2TN