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:
02 / 01 / 2023
Posting Last Modified Date:
09 / 01 / 2022
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.
Macquarie University | Recruiting
Principal Investigator : Dominic Rowe, AM BSc, MBBS, PhD
North Ryde, New South Wales
The Wesley Hospital | Recruiting
Principal Investigator : Robert Henderson, MD, PhD
Perron Institute | Recruiting
Principal Investigator : Merrilee Needham, MBBS, PhD, FRACP
Nedlands, Western Australia
UZ Leuven | Recruiting
Principal Investigator : Rick Vandenberghe, MD, PhD
Sunnybrook Health Sciences Centre | Recruiting
Principal Investigator : Mario Masellis, MD, PhD, M.Sc., Hon B.Sc,
McGill University Health Center - Research Institute | Recruiting
Principal Investigator : Simon Ducharme, MD, MSc, FRCPC
St James Hospital - Ireland | Recruiting
Principal Investigator : Orla Hardiman, BSc MB BCh BAO MD FRCPI FAAN
Dublin D08 NHY1
Universitair Medisch Centrum Utrecht | Recruiting
Principal Investigator : Leonard van den Berg, MD, PhD
Erasmus University MC | Recruiting
Principal Investigator : Harro Seelar, MD, PhD, MSc
Auckland City Hospital | Recruiting
Principal Investigator : Julian Bauer, MD
New Zealand Brain Research Institute | Recruiting
Principal Investigator : John Anderson, BSc(Hons), MBChB, MD, FRACP
Karolinska University Hospital | Recruiting
Principal Investigator : Anne B Hanson, MD, PhD
University of Cambridge | Recruiting
Principal Investigator : James Rowe, MD, PhD
University College London Hospital | Recruiting
Principal Investigator : Jonathan Rohrer, MD, PhD
London WC1N 3BG
King's College Hospital | Recruiting
Principal Investigator : Christopher Shaw, MD, FRACP
London WC2R 2LS
University of Oxford - Nuffield Department of Clinical Neurosciences | Recruiting
Principal Investigator : Kevin Talbot, MB BS, DPhil, FRCP
Oxford OX3 7LF
University of Sheffield | Recruiting
Principal Investigator : Jonathan Cooper-Knock, MD, PhD
Sheffield S10 2TN