NCT04617847

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.

Trial Health

63
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
27

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Apr 2020

Geographic Reach
7 countries

18 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 13, 2020

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

October 30, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

November 5, 2020

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 3, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

May 3, 2021

Completed
9 months until next milestone

Results Posted

Study results publicly available

February 9, 2022

Completed
Last Updated

February 9, 2022

Status Verified

January 1, 2022

Enrollment Period

1.1 years

First QC Date

October 30, 2020

Results QC Date

December 17, 2021

Last Update Submit

January 19, 2022

Conditions

Huntington Disease

Outcome Measures

Primary Outcomes (4)

  • 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 a Severe TEAE

    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 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

Study Arms (1)

WVE-120101 (Dose A)

EXPERIMENTAL
Drug: WVE-120101

Interventions

WVE-120101DRUG

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

WVE-120101 (Dose A)

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

  • \. 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.
  • \. 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

Westmead Hospital

Sydney, New South Wales, 2145, Australia

Location

Royal Brisbane & Women's Hospital

Herston, Queensland, QLD 4006, Australia

Location

Royal Melbourne Hospital

Carlton, Victoria, 3053, Australia

Location

Monash Health

Clayton, Victoria, 3168, Australia

Location

Alfred Health

Melbourne, Victoria, 3004, Australia

Location

Calvary Health Care Bethlehem

Parkdale, Victoria, 3195, Australia

Location

North Metropolitan Health Service

Perth, Western Australia, 6910, Australia

Location

University of Alberta

Edmonton, Alberta, T6G 2B7, Canada

Location

Centre Hospitalier de l-Universite de Montreal

Montreal, Quebec, H2X019, Canada

Location

Aarhus Universitets Hospital

Aarhus, 8200, Denmark

Location

Rigshospitalet

Copenhagen, 2100, Denmark

Location

Hospital Henri Mondor

Créteil, 94010, France

Location

Institut du Cerveau et de la Moelle Epinière

Paris, 75646, France

Location

George-Huntington-Institut GmbH

Münster, 48149, Germany

Location

Szpital Sw. Wojciecha

Gdansk, 80-462, Poland

Location

Instytut Psychiatrii i Neurologii

Warsaw, 02-957, Poland

Location

Royal Devon and Exeter Hospital NHS Trust

Exeter, Devon, EX2 5DW, United Kingdom

Location

Queen Elizabeth University Hospital - PPDS

Glasgow, Glasgow City, G12 0XH, United Kingdom

Location

MeSH Terms

Conditions

Huntington Disease

Condition Hierarchy (Ancestors)

Basal Ganglia DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesDementiaChoreaDyskinesiasMovement DisordersHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCognition DisordersNeurocognitive DisordersMental Disorders

Results Point of Contact

Title
Medical Director
Organization
Wave Life Sciences
clinicaltrials@wavelifesci.com
855-215-4687

Study Officials

  • Medical Director, MD

    Wave Life Sciences

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 30, 2020

First Posted

November 5, 2020

Study Start

April 13, 2020

Primary Completion

May 3, 2021

Study Completion

May 3, 2021

Last Updated

February 9, 2022

Results First Posted

February 9, 2022

Record last verified: 2022-01

Data Sharing

IPD Sharing
Will not share

Locations

PL(2)CA(2)GB(2)DE(1)AU(7)DK(2)FR(2)