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Trial record 1 of 1 for:    WVE-004 | ALS (Amyotrophic Lateral Sclerosis)
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Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) (FOCUS-C9)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04931862
Recruitment Status : Recruiting
First Posted : June 18, 2021
Last Update Posted : May 24, 2022
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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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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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Australia, New South Wales
Macquarie University Recruiting
North Ryde, New South Wales, Australia, 2109
Principal Investigator: Dominic Rowe, AM BSc, MBBS, PhD         
Australia, Queensland
The Wesley Hospital Recruiting
Brisbane, Queensland, Australia, QLD 4066
Principal Investigator: Robert Henderson, MD, PhD         
Australia, Western Australia
Perron Institute Recruiting
Nedlands, Western Australia, Australia, 6009
Principal Investigator: Merrilee Needham, MBBS, PhD, FRACP         
Belgium
UZ Leuven Recruiting
Leuven, Belgium, 3000
Contact: Rick Vandenberghe, MD, PhD    016344280    rik.vandenberghe@uz.kuleuven.ac.be   
Principal Investigator: Rick Vandenberghe, MD, PhD         
Canada, Ontario
Sunnybrook Health Sciences Centre Recruiting
Toronto, Ontario, Canada, M4N 3M5
Principal Investigator: Mario Masellis, MD, PhD, M.Sc., Hon B.Sc,         
Canada, Quebec
McGill University Health Center - Research Institute Recruiting
Montréal, Quebec, Canada
Contact       Neurocog-cru.neuro@mcgill.ca   
Contact       als-cru.neuro@mcgill.ca   
Principal Investigator: Simon Ducharme, MD, MSc, FRCPC         
Ireland
St James Hospital - Ireland Recruiting
Dublin, Ireland, D08 NHY1
Principal Investigator: Orla Hardiman, BSc MB BCh BAO MD FRCPI FAAN         
Netherlands
Universitair Medisch Centrum Utrecht Recruiting
Utrecht, Netherlands
Principal Investigator: Leonard van den Berg, MD, PhD         
New Zealand
New Zealand Brain Research Institute Recruiting
Christchurch, New Zealand, 8011
Principal Investigator: John Anderson, BSc(Hons), MBChB, MD, FRACP         
United Kingdom
University College London Hospital Recruiting
London, United Kingdom, WC1N 3BG
Principal Investigator: Jonathan Rohrer, MD, PhD         
King's College Hospital Recruiting
London, United Kingdom, WC2R 2LS
Principal Investigator: Christopher Shaw, MD, FRACP         
University of Oxford - Nuffield Department of Clinical Neurosciences Recruiting
Oxford, United Kingdom, OX3 7LF
Principal Investigator: Kevin Talbot, MB BS, DPhil, FRCP         
University of Sheffield Recruiting
Sheffield, United Kingdom, S10 2TN
Contact: Jonathon Cooper-Knock, MD, PhD       j.cooper-knock@sheffield.ac.uk   
Principal Investigator: Jonathon Cooper-Knock, 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: May 24, 2022
Last Verified: May 2022
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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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Dementia
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