Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)

  • End date
    Feb 1, 2023
  • participants needed
  • sponsor
    Wave Life Sciences Ltd.
Updated on 1 October 2021
frontotemporal lobar degeneration
primary lateral sclerosis
frontotemporal dementia
amyotrophic lateral sclerosis


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.

Condition Frontotemporal Dementia, Amyotrophic Lateral Sclerosis, Antilymphocyte Serum, dementia, frontotemporal, lou gehrig's disease, C9orf72-associated ALS or FTD
Treatment Placebo, WVE-004
Clinical Study IdentifierNCT04931862
SponsorWave Life Sciences Ltd.
Last Modified on1 October 2021


Yes No Not Sure

Inclusion Criteria

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

Exclusion 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
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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