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

NCT ID: NCT04617847

Last Updated: 2022-02-09

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE1/PHASE2

Total Enrollment

27 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-04-13

Study Completion Date

2021-05-03

Brief Summary

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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

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Conditions

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Huntington Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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WVE-120101 (Dose A)

Group Type EXPERIMENTAL

WVE-120101

Intervention Type DRUG

WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection.

Interventions

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WVE-120101

WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 1\. Patient successfully completed the Phase 1b/2a study with WVE-120101, WVE-HDSNP1-001.

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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Wave Life Sciences Ltd.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director, MD

Role: STUDY_DIRECTOR

Wave Life Sciences

Locations

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Westmead Hospital

Sydney, New South Wales, Australia

Site Status

Royal Brisbane & Women's Hospital

Herston, Queensland, Australia

Site Status

Royal Melbourne Hospital

Carlton, Victoria, Australia

Site Status

Monash Health

Clayton, Victoria, Australia

Site Status

Alfred Health

Melbourne, Victoria, Australia

Site Status

Calvary Health Care Bethlehem

Parkdale, Victoria, Australia

Site Status

North Metropolitan Health Service

Perth, Western Australia, Australia

Site Status

University of Alberta

Edmonton, Alberta, Canada

Site Status

Centre Hospitalier de l-Universite de Montreal

Montreal, Quebec, Canada

Site Status

Aarhus Universitets Hospital

Aarhus, , Denmark

Site Status

Rigshospitalet

Copenhagen, , Denmark

Site Status

Hospital Henri Mondor

Créteil, , France

Site Status

Institut du Cerveau et de la Moelle Epinière

Paris, , France

Site Status

George-Huntington-Institut GmbH

Münster, , Germany

Site Status

Szpital Sw. Wojciecha

Gdansk, , Poland

Site Status

Instytut Psychiatrii i Neurologii

Warsaw, , Poland

Site Status

Royal Devon and Exeter Hospital NHS Trust

Exeter, Devon, United Kingdom

Site Status

Queen Elizabeth University Hospital - PPDS

Glasgow, Glasgow City, United Kingdom

Site Status

Countries

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Australia Canada Denmark France Germany Poland United Kingdom

Provided Documents

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Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

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WVE-HDSNP1-002

Identifier Type: -

Identifier Source: org_study_id

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