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Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease

Phase 1
Terminated
Conditions
Huntington Disease
Interventions
Drug: WVE-120101
Registration Number
NCT04617847
Lead Sponsor
Wave Life Sciences Ltd.
Brief Summary

WVE-HDSNP1-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120101 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362307 (SNP1). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP1-001.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
27
Inclusion Criteria
    1. Patient successfully completed the Phase 1b/2a study with WVE-120101, WVE-HDSNP1-001.

Key

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Exclusion Criteria
    1. Received an investigational drug other than WVE-120101, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer.
    1. Inability to undergo brain MRI (with or without sedation).
    1. Clinically significant medical finding on the physical examination other than HD that, in the judgment of the Investigator, will make the patient unsuitable for participation in and/or completion of the study procedures.
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Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
WVE-120101 (Dose A)WVE-120101-
Primary Outcome Measures
NameTimeMethod
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEsFirst dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Safety: Number of Patients With Treatment-emergent AEs (TEAEs)First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
Safety: Number of Patients With Serious TEAEsFirst dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Safety: Number of Patients With a Severe TEAEFirst dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (18)

Royal Brisbane & Women's Hospital

🇦🇺

Herston, Queensland, Australia

Calvary Health Care Bethlehem

🇦🇺

Parkdale, Victoria, Australia

Hospital Henri Mondor

🇫🇷

Créteil, France

Centre Hospitalier de l-Universite de Montreal

🇨🇦

Montreal, Quebec, Canada

Szpital Sw. Wojciecha

🇵🇱

Gdańsk, Poland

Royal Melbourne Hospital

🇦🇺

Carlton, Victoria, Australia

Alfred Health

🇦🇺

Melbourne, Victoria, Australia

Institut du Cerveau et de la Moelle Epinière

🇫🇷

Paris, France

North Metropolitan Health Service

🇦🇺

Perth, Western Australia, Australia

Aarhus Universitets Hospital

🇩🇰

Aarhus, Denmark

Rigshospitalet

🇩🇰

Copenhagen, Denmark

University of Alberta

🇨🇦

Edmonton, Alberta, Canada

Instytut Psychiatrii i Neurologii

🇵🇱

Warsaw, Poland

Westmead Hospital

🇦🇺

Sydney, New South Wales, Australia

Monash Health

🇦🇺

Clayton, Victoria, Australia

George-Huntington-Institut GmbH

🇩🇪

Muenster, Germany

Queen Elizabeth University Hospital - PPDS

🇬🇧

Glasgow, Glasgow City, United Kingdom

Royal Devon and Exeter Hospital NHS Trust

🇬🇧

Exeter, Devon, United Kingdom

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