Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease

Phase 1

Terminated

• Conditions: Huntington Disease
• Interventions: Drug: WVE-120102

• Registration Number: NCT04617860
• Lead Sponsor: Wave Life Sciences Ltd.

Brief Summary

WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism, rs362331 (SNP2). To participate in the study, patients must have completed the Phase 1b/2a clinical study WVE-HDSNP2-001.

Detailed Description

Not available

Study Timeline

• Study Start: 2019-09-24
• Study First Submitted: 2020-10-30
• Study First Submitted QC: 2020-10-30
• Study First Posted: 2020-11-05
• Primary Completion: 2021-04-29
• Study Completion: 2021-04-29
• 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: 36

Inclusion Criteria

1. Patient successfully completed the Phase 1b/2a study with WVE-120102, WVE-HDSNP2-001.

Exclusion Criteria

1. Received an investigational drug other than WVE-120102, 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-120102 (Dose A)	WVE-120102	-

Primary Outcome Measures

Name	Time	Method
Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)	Day 1 to Study Termination (maximum of 12 monthly doses)
Safety: Number of Patients With Serious TEAEs	Day 1 to Study Termination (maximum of 12 monthly doses)
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs	Day 1 to Study Termination (maximum of 12 monthly doses)
Safety: Number of Patients With a Severe TEAE	Day 1 to Study Termination (maximum of 12 monthly doses)

Trial Locations

Locations (20)

North Metropolitan Health Service; 🇦🇺 Perth, Western Australia, Australia

Alfred Health; 🇦🇺 Melbourne, Victoria, Australia

Instytut Psychiatrii i Neurologii; 🇵🇱 Warsaw, Poland

Centre Hospitalier de l-Universite de Montreal; 🇨🇦 Montreal, Quebec, Canada

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

Royal Devon and Exeter Hospital NHS Trust; 🇬🇧 Exeter, Devon, United Kingdom

Monash Health; 🇦🇺 Clayton, Victoria, Australia

Royal Brisbane & Women's Hospital; 🇦🇺 Herston, Queensland, Australia

Centre For Movement Disorders; 🇨🇦 Toronto, Ontario, Canada

Royal Melbourne Hospital; 🇦🇺 Carlton, Victoria, Australia

Szpital Sw. Wojciecha; 🇵🇱 Gdańsk, Poland

Odense University Hospital and University of Southern Denmark; 🇩🇰 Odense, Denmark

Hospital Henri Mondor; 🇫🇷 Créteil, France

Queen Elizabeth University Hospital - PPDS; 🇬🇧 Glasgow, Glasgow City, United Kingdom

George-Huntington-Institut GmbH; 🇩🇪 Muenster, Germany

Institut du Cerveau et de la Moelle Epinière; 🇫🇷 Paris, France

Westmead Hospital; 🇦🇺 Sydney, New South Wales, Australia

Aarhus Universitets Hospital; 🇩🇰 Aarhus, Denmark

Rigshospitalet; 🇩🇰 Copenhagen, Denmark

Calvary Health Care Bethlehem; 🇦🇺 Parkdale, Victoria, Australia