Back to resultsOpen-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
Primary Purpose
Huntington Disease
Status
Terminated
Phase
Phase 1
Locations
International
Study Type
Interventional
Intervention
WVE-120102
Sponsored by
About this trial
This is an interventional treatment trial for Huntington Disease
Eligibility Criteria
Inclusion Criteria:
1. Patient successfully completed the Phase 1b/2a study with WVE-120102, WVE-HDSNP2-001.
Exclusion Criteria:
- 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.
- Inability to undergo brain MRI (with or without sedation).
- 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.
Sites / Locations
- Westmead Hospital
- Royal Brisbane & Women's Hospital
- Royal Melbourne Hospital
- Monash Health
- Alfred Health
- Calvary Health Care Bethlehem
- North Metropolitan Health Service
- University of Alberta
- Centre For Movement Disorders
- Centre Hospitalier de l-Universite de Montreal
- Aarhus Universitets Hospital
- Rigshospitalet
- Odense University Hospital and University of Southern Denmark
- Hospital Henri Mondor
- Institut du Cerveau et de la Moelle Epinière
- George-Huntington-Institut GmbH
- Szpital Sw. Wojciecha
- Instytut Psychiatrii i Neurologii
- Royal Devon and Exeter Hospital NHS Trust
- Queen Elizabeth University Hospital - PPDS
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
WVE-120102 (Dose A)
Arm Description
Outcomes
Primary Outcome Measures
Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)
Safety: Number of Patients With a Severe TEAE
Safety: Number of Patients With Serious TEAEs
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
Secondary Outcome Measures
Full Information
NCT ID
NCT04617860
First Posted
October 30, 2020
Last Updated
January 19, 2022
Sponsor
Wave Life Sciences Ltd.
1. Study Identification
Unique Protocol Identification Number
NCT04617860
Brief Title
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
Official Title
A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120102 in Patients With Huntington's Disease
Study Type
Interventional
2. Study Status
Record Verification Date
January 2022
Overall Recruitment Status
Terminated
Why Stopped
Lack of efficacy
Study Start Date
September 24, 2019 (Actual)
Primary Completion Date
April 29, 2021 (Actual)
Study Completion Date
April 29, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Wave Life Sciences Ltd.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
Yes
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington Disease
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
36 (Actual)
8. Arms, Groups, and Interventions
Arm Title
WVE-120102 (Dose A)
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
WVE-120102
Intervention Description
WVE-120102 is a stereopure antisense oligonucleotide. It is administered monthly via intrathecal injection.
Primary Outcome Measure Information:
Title
Safety: Number of Patients With Treatment-emergent Adverse Events (TEAEs)
Time Frame
Day 1 to Study Termination (maximum of 12 monthly doses)
Title
Safety: Number of Patients With a Severe TEAE
Time Frame
Day 1 to Study Termination (maximum of 12 monthly doses)
Title
Safety: Number of Patients With Serious TEAEs
Time Frame
Day 1 to Study Termination (maximum of 12 monthly doses)
Title
Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
Time Frame
Day 1 to Study Termination (maximum of 12 monthly doses)
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
1. Patient successfully completed the Phase 1b/2a study with WVE-120102, WVE-HDSNP2-001.
Exclusion Criteria:
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.
Inability to undergo brain MRI (with or without sedation).
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director, MD
Organizational Affiliation
WAVE Life Sciences
Official's Role
Study Director
Facility Information:
Facility Name
Westmead Hospital
City
Sydney
State/Province
New South Wales
ZIP/Postal Code
2145
Country
Australia
Facility Name
Royal Brisbane & Women's Hospital
City
Herston
State/Province
Queensland
ZIP/Postal Code
QLD 4006
Country
Australia
Facility Name
Royal Melbourne Hospital
City
Carlton
State/Province
Victoria
ZIP/Postal Code
3053
Country
Australia
Facility Name
Monash Health
City
Clayton
State/Province
Victoria
ZIP/Postal Code
3168
Country
Australia
Facility Name
Alfred Health
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Facility Name
Calvary Health Care Bethlehem
City
Parkdale
State/Province
Victoria
ZIP/Postal Code
3195
Country
Australia
Facility Name
North Metropolitan Health Service
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6910
Country
Australia
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G 2B7
Country
Canada
Facility Name
Centre For Movement Disorders
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M3B 2S7
Country
Canada
Facility Name
Centre Hospitalier de l-Universite de Montreal
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H2X019
Country
Canada
Facility Name
Aarhus Universitets Hospital
City
Aarhus
ZIP/Postal Code
8200
Country
Denmark
Facility Name
Rigshospitalet
City
Copenhagen
ZIP/Postal Code
2100
Country
Denmark
Facility Name
Odense University Hospital and University of Southern Denmark
City
Odense
ZIP/Postal Code
5000
Country
Denmark
Facility Name
Hospital Henri Mondor
City
Créteil
ZIP/Postal Code
94010
Country
France
Facility Name
Institut du Cerveau et de la Moelle Epinière
City
Paris
ZIP/Postal Code
75646
Country
France
Facility Name
George-Huntington-Institut GmbH
City
Muenster
ZIP/Postal Code
48149
Country
Germany
Facility Name
Szpital Sw. Wojciecha
City
Gdańsk
ZIP/Postal Code
80-462
Country
Poland
Facility Name
Instytut Psychiatrii i Neurologii
City
Warsaw
ZIP/Postal Code
02-957
Country
Poland
Facility Name
Royal Devon and Exeter Hospital NHS Trust
City
Exeter
State/Province
Devon
ZIP/Postal Code
EX2 5DW
Country
United Kingdom
Facility Name
Queen Elizabeth University Hospital - PPDS
City
Glasgow
State/Province
Glasgow City
ZIP/Postal Code
G12 0XH
Country
United Kingdom
12. IPD Sharing Statement
Learn more about this trial
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
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