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Clinical Trial Details — Status: Terminated

Administrative data

NCT number NCT04931862
Other study ID # WVE-004-001
Secondary ID
Status Terminated
Phase Phase 1/Phase 2
First received
Last updated
Start date June 28, 2021
Est. completion date June 27, 2023

Study information

Verified date August 2023
Source Wave Life Sciences Ltd.
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Recruitment information / eligibility

Status Terminated
Enrollment 35
Est. completion date June 27, 2023
Est. primary completion date June 27, 2023
Accepts healthy volunteers No
Gender All
Age group 18 Years to 80 Years
Eligibility 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.

Study Design


Intervention

Drug:
WVE-004
WVE-004 is a stereopure antisense oligonucleotide (ASO)
Placebo
Artificial cerebrospinal fluid (aCSF)

Locations

Country Name City State
Australia The Wesley Hospital Brisbane Queensland
Australia Perron Institute Nedlands Western Australia
Australia Macquarie University North Ryde New South Wales
Belgium UZ Leuven Leuven
Canada McGill University Health Center - Research Institute Montréal Quebec
Canada Sunnybrook Health Sciences Centre Toronto Ontario
Ireland St James Hospital - Ireland Dublin
Netherlands Erasmus University MC Rotterdam
Netherlands Universitair Medisch Centrum Utrecht Utrecht
New Zealand Auckland City Hospital Auckland
New Zealand New Zealand Brain Research Institute Christchurch
Sweden Karolinska University Hospital Solna
United Kingdom University of Cambridge Cambridge
United Kingdom King's College Hospital London
United Kingdom University College London Hospital London
United Kingdom University of Oxford - Nuffield Department of Clinical Neurosciences Oxford
United Kingdom University of Sheffield Sheffield

Sponsors (1)

Lead Sponsor Collaborator
Wave Life Sciences Ltd.

Countries where clinical trial is conducted

Australia,  Belgium,  Canada,  Ireland,  Netherlands,  New Zealand,  Sweden,  United Kingdom, 

Outcome

Type Measure Description Time frame Safety issue
Primary Safety: Proportion of patients with adverse events (AEs) Period 1 Day 1 to Period 2 Week 24 (end of study)
Secondary Pharmacokinetic: Concentration of WVE-004 in cerebrospinal fluid (CSF) Period 1 Day 1 to Period 2 Week 24 (end of study)
Secondary Pharmacodynamic: Change from baseline in concentration of poly-GP levels in the CSF Period 1 Day 1 to Period 2 Week 24 (end of study)
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