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

Study Timeline

• Study Start: 2020-04-13
• Study First Submitted: 2020-10-30
• Study First Submitted QC: 2020-10-30
• Study First Posted: 2020-11-05
• Primary Completion: 2021-05-03
• Study Completion: 2021-05-03
• Results First Submitted: 2021-12-17
• Status Verified: 2022-01
• Results First Submitted QC: 2022-01-19
• Last Update Submitted: 2022-01-19
• Results First Posted: 2022-02-09
• Last Update Posted: 2022-02-09

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

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.
• 2. Inability to undergo brain MRI (with or without sedation).
• 3. 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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
WVE-120101 (Dose A)	WVE-120101	-

Primary Outcome Measures

Name	Time	Method
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs	First 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 TEAEs	First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Safety: Number of Patients With a Severe TEAE	First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment

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