Trial record 1 of 1 for:
WVE-004
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 |
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Recruitment Status :
Recruiting
First Posted : June 18, 2021
Last Update Posted : May 24, 2022
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Sponsor:
Wave Life Sciences Ltd.
Information provided by (Responsible Party):
Wave Life Sciences Ltd.
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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 |
| 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 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
CHMP2B-related frontotemporal dementia
Frontotemporal dementia with parkinsonism-17
Amyotrophic lateral sclerosis
Inclusion body myopathy with early-onset Paget disease and frontotemporal dementia
Juvenile primary lateral sclerosis
GRN-related frontotemporal lobar degeneration
| 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 :
- 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 :
- Pharmacokinetic: Concentration of WVE-004 in cerebrospinal fluid (CSF) [ Time Frame: Period 1 Day 1 to Period 2 Week 24 (end of study) ]
- 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) ]
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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:
- 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.
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
| Contact: Clinical Operations | 855-215-4687 | clinicaltrials@wavelifesci.com |
Locations
| 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
| Study Director: | Medical Director, MD | Wave Life Sciences |
| 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 |
| 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 |