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Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) (FOCUS-C9)

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ClinicalTrials.gov Identifier: NCT04931862
Recruitment Status : Recruiting
First Posted : June 18, 2021
Last Update Posted : July 22, 2021
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease Intervention/treatment Phase
ALS FTD Drug: WVE-004 Drug: Placebo Phase 1 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 42 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Multicenter, Randomized, Double-blind, Placebo-controlled, Phase 1b/2a Study of WVE-004 Administered Intrathecally to Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD)
Actual Study Start Date : June 28, 2021
Estimated Primary Completion Date : February 2023
Estimated Study Completion Date : February 2023


Arm Intervention/treatment
Experimental: WVE-004 (Dose A) or placebo Drug: WVE-004
WVE-004 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Artificial cerebrospinal fluid (aCSF)

Experimental: WVE-004 (Dose B) or placebo Drug: WVE-004
WVE-004 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Artificial cerebrospinal fluid (aCSF)

Experimental: WVE-004 (Dose C) or placebo Drug: WVE-004
WVE-004 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Artificial cerebrospinal fluid (aCSF)

Experimental: WVE-004 (Dose D) or placebo Drug: WVE-004
WVE-004 is a stereopure antisense oligonucleotide (ASO)

Drug: Placebo
Artificial cerebrospinal fluid (aCSF)




Primary Outcome Measures :
  1. Safety: Proportion of patients with adverse events (AEs) [ Time Frame: Period 1 Day 1 to Period 2 Week 24 (end of study) ]

Secondary Outcome Measures :
  1. Pharmacokinetic: Concentration of WVE-004 in cerebrospinal fluid (CSF) [ Time Frame: Period 1 Day 1 to Period 2 Week 24 (end of study) ]
  2. Pharmacodynamic: Change from baseline in concentration of poly-GP levels in the CSF [ Time Frame: Period 1 Day 1 to Period 2 Week 24 (end of study) ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion 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.

Exclusion 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04931862


Contacts
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Contact: Clinical Operations 855-215-4687 clinicaltrials@wavelifesci.com

Locations
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Canada, Quebec
McGill University Health Center - Research Institute Not yet recruiting
Montréal, Quebec, Canada
Principal Investigator: Simon Ducharme, MD, MSc, FRCPC         
Netherlands
Universitair Medisch Centrum Utrecht Recruiting
Utrecht, Netherlands
Principal Investigator: Leonard van den Berg, MD, PhD         
Sponsors and Collaborators
Wave Life Sciences Ltd.
Investigators
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Study Director: Medical Director, MD Wave Life Sciences
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Responsible Party: Wave Life Sciences Ltd.
ClinicalTrials.gov Identifier: NCT04931862    
Other Study ID Numbers: WVE-004-001
First Posted: June 18, 2021    Key Record Dates
Last Update Posted: July 22, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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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
Additional relevant MeSH terms:
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Dementia
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Frontotemporal Dementia
Aphasia, Primary Progressive
Pick Disease of the Brain
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurocognitive Disorders
Mental Disorders
Neurodegenerative Diseases
Neuromuscular Diseases
Spinal Cord Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Frontotemporal Lobar Degeneration
Aphasia
Speech Disorders
Language Disorders
Communication Disorders
Neurobehavioral Manifestations
Neurologic Manifestations