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Study of WVE-003 in Patients With Huntington's Disease

Primary Purpose

Huntington Disease

Status

Recruiting

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

WVE-003

About this trial

This is an interventional treatment trial for Huntington Disease

Eligibility Criteria

25 Years - 60 Years (Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
Ambulatory, male or female patients aged ≥25 to ≤60 years
Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
UHDRS Total Functional Capacity Scores ≥9 and ≤13

Exclusion Criteria:

Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years
Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following:
a. Received WVE-120101 or WVE-120102 within the last 3 months
Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
Inability to undergo brain MRI (with or without sedation)
Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture
Previously received tominersen

Sites / Locations

Westmead HospitalRecruiting
Monash HealthRecruiting
Royal Melbourne HospitalRecruiting
North Metropolitan Health ServiceRecruiting
University of Alberta HospitalRecruiting
The Ottawa HospitalRecruiting
Centre Hospitalier de l-Universite de MontrealRecruiting
RigshospitaletRecruiting
Hopital Henri Mondor - HospitalRecruiting
Institut du Cerveau et de la Moelle EpiniereRecruiting
Katholisches Klinikum Bochum gGmbHRecruiting
George-Huntington-Institut GmbHRecruiting
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)Recruiting
Ospedale Casa Sollievo Della Sofferenza IRCCSRecruiting
Centro Ricerche Cliniche Di VeronaRecruiting
Szpital Sw. WojciechaRecruiting
Instytut Psychiatrii I NeurologiiRecruiting
Hospital de la Sanata Creu i Sant PauRecruiting
Hospital Universitario Ramón y CajalRecruiting
Royal Devon and Exeter Hospital NHS TrustRecruiting
Royal Hospital for Children, Pharmacy Aseptic UnitRecruiting
Cardiff University, Schools of Medicine and BiosciencesRecruiting
Royal Liverpool University HospitalRecruiting
Royal Victoria InfirmaryRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Arm Label

WVE-003 (Dose A) or placebo

WVE-003 (Dose B) or placebo

WVE-003 (Dose C) or placebo

WVE-003 (Dose D) or placebo

Arm Description

Outcomes

Primary Outcome Measures

Safety: Proportion of patients with adverse events (AEs)

Secondary Outcome Measures

Maximum concentration (Cmax) of WVE-003 in plasma

Area under the plasma concentration time curve for WVE-003 from time 0 to last quantifiable concentration (AUC0-t)

Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF)

Full Information

NCT ID

NCT05032196

First Posted

August 18, 2021

Last Updated

January 4, 2023

Sponsor

Wave Life Sciences Ltd.

1. Study Identification

Unique Protocol Identification Number

NCT05032196

Brief Title

Study of WVE-003 in Patients With Huntington's Disease

Official Title

A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease (SELECT-HD)

Study Type

Interventional

2. Study Status

Record Verification Date

January 2023

Overall Recruitment Status

Recruiting

Study Start Date

September 6, 2021 (Actual)

Primary Completion Date

June 2023 (Anticipated)

Study Completion Date

June 2023 (Anticipated)

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

Product Manufactured in and Exported from the U.S.

Yes

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) - SNP3.

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

Sequential Assignment

Masking

ParticipantInvestigator

Allocation

Randomized

Enrollment

36 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

WVE-003 (Dose A) or placebo

Arm Type

Experimental

Arm Title

WVE-003 (Dose B) or placebo

Arm Type

Experimental

Arm Title

WVE-003 (Dose C) or placebo

Arm Type

Experimental

Arm Title

WVE-003 (Dose D) or placebo

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

WVE-003

Intervention Description

WVE-003 is a stereopure antisense oligonucleotide (ASO)

Primary Outcome Measure Information:

Title

Safety: Proportion of patients with adverse events (AEs)

Time Frame

Day 1 through end of study (minimum of 36 weeks)

Secondary Outcome Measure Information:

Title

Maximum concentration (Cmax) of WVE-003 in plasma

Time Frame

Day 1 through end of study (minimum of 36 weeks)

Title

Area under the plasma concentration time curve for WVE-003 from time 0 to last quantifiable concentration (AUC0-t)

Time Frame

Day 1 through end of study (minimum of 36 weeks)

Title

Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF)

Time Frame

Day 1 through end of study (minimum of 36 weeks)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

25 Years

Maximum Age & Unit of Time

60 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion Ambulatory, male or female patients aged ≥25 to ≤60 years Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4 UHDRS Total Functional Capacity Scores ≥9 and ≤13 Exclusion Criteria: Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following: a. Received WVE-120101 or WVE-120102 within the last 3 months Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan Inability to undergo brain MRI (with or without sedation) Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture Previously received tominersen

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Clinical Operations

Phone

855-215-4687

clinicaltrials@wavelifesci.com

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

Westmead

State/Province

New South Wales

ZIP/Postal Code

2145

Country

Australia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Shanthi Graham

shanthi.graham@health.nsw.gov.au

First Name & Middle Initial & Last Name & Degree

Clement Loy, MD, MPH

Facility Name

Monash Health

City

Clayton

State/Province

Victoria

ZIP/Postal Code

3168

Country

Australia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Yenni Lie, MD

yenni.lie@calvarycare.org.au

First Name & Middle Initial & Last Name & Degree

Yenni Lie, MD

Facility Name

Royal Melbourne Hospital

City

Melbourne

State/Province

Victoria

ZIP/Postal Code

3050

Country

Australia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Dennis Velakoulis

dennis.velakoulis@mh.org.au

First Name & Middle Initial & Last Name & Degree

Dennis Velakoulis, MBBS MMed DMedSci

Facility Name

North Metropolitan Health Service

City

Perth

State/Province

Western Australia

ZIP/Postal Code

6910

Country

Australia

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Carolyn Orr, MD

carolyn.orr2@health.wa.gov.au

First Name & Middle Initial & Last Name & Degree

Carolyn Orr, MD

Facility Name

University of Alberta Hospital

City

Edmonton

State/Province

Alberta

ZIP/Postal Code

T6G 2G3

Country

Canada

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Oksana Suchowersky

oksana.suchowersky@albertahealthservices.ca

First Name & Middle Initial & Last Name & Degree

Oksana Suchowerky, MD

Facility Name

The Ottawa Hospital

City

Ottawa

State/Province

Ontario

ZIP/Postal Code

K1H 8L6

Country

Canada

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Tanya Thompson

tanthompson@ohri.ca

First Name & Middle Initial & Last Name & Degree

Tiago Mestre, MD, MsC

Facility Name

Centre Hospitalier de l-Universite de Montreal

City

Montréal

State/Province

Quebec

ZIP/Postal Code

H2X019

Country

Canada

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Joannie Vallancourt

joanie.vallancourt.chum@ssss.gouv.qc.ca

First Name & Middle Initial & Last Name & Degree

Sylvain Chouinard, MD

Facility Name

Rigshospitalet

City

Copenhagen

ZIP/Postal Code

2100

Country

Denmark

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Lena Hjermind, MD

lena.elisabeth.hjermind.01@regionh.dk

First Name & Middle Initial & Last Name & Degree

Lena Hjermind, MD

Facility Name

Hopital Henri Mondor - Hospital

City

Créteil

ZIP/Postal Code

94010

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Katia Youssov

katia.youssov@aphp.fr

First Name & Middle Initial & Last Name & Degree

Katia Youssov, MD

Facility Name

Institut du Cerveau et de la Moelle Epiniere

City

Paris

ZIP/Postal Code

75646

Country

France

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Elodie Petit

elodie.petit@icm-institute.org

First Name & Middle Initial & Last Name & Degree

Alexandra Durr

alexandra.durr@icm-institute.org

First Name & Middle Initial & Last Name & Degree

Alexandra Durr, MD, PhD

Facility Name

Katholisches Klinikum Bochum gGmbH

City

Bochum

ZIP/Postal Code

44791

Country

Germany

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Carsten Saft, MD

carsten.saft@ruhr-uni-bochum.de

First Name & Middle Initial & Last Name & Degree

Carsten Saft, MD

Facility Name

George-Huntington-Institut GmbH

City

Muenster

ZIP/Postal Code

48149

Country

Germany

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Ralf Reilman, MD

ralf.reilmann@ghi-muenster.de

First Name & Middle Initial & Last Name & Degree

Ralf Reilman, MD

Facility Name

kbo-Isar-Amper-Klinikum Taufkirchen (Vils)

City

Taufkirchen

ZIP/Postal Code

84416

Country

Germany

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Alzbeta Mühlbäck

alzbeta.muehlbaeck@kbo.de

First Name & Middle Initial & Last Name & Degree

Alzbeta Mühlbäck, MD

Facility Name

Ospedale Casa Sollievo Della Sofferenza IRCCS

City

Foggia

ZIP/Postal Code

71013

Country

Italy

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Fernando Squitieri, MD

ferdinandosquitieri@yahoo.it

First Name & Middle Initial & Last Name & Degree

Fernando Squitieri, MD

Facility Name

Centro Ricerche Cliniche Di Verona

City

Verona

Country

Italy

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Tomasso Bovi, MD

Facility Name

Szpital Sw. Wojciecha

City

Gdańsk

ZIP/Postal Code

80-462

Country

Poland

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jaroslaw Slawek

jaroslawek@gumed.edu.pl

First Name & Middle Initial & Last Name & Degree

Jaroslaw Slawek, MD

Facility Name

Instytut Psychiatrii I Neurologii

City

Warsaw

ZIP/Postal Code

02-957

Country

Poland

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Grzegorz Witkowski

9greg@wp.pl

First Name & Middle Initial & Last Name & Degree

Grzegorz Witkowski, MD, PhD

Facility Name

Hospital de la Sanata Creu i Sant Pau

City

Barcelona

ZIP/Postal Code

08041

Country

Spain

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jaime Kulisevsky Bojarski, MD, PhD

jkulisevsky@santpau.cat

First Name & Middle Initial & Last Name & Degree

Jaime Kulisevsky Bojarski, MD, PhD

Facility Name

Hospital Universitario Ramón y Cajal

City

Madrid

ZIP/Postal Code

28034

Country

Spain

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Jose Luis Lopez-Sendon Moreno, MD

joselopezsendon@hotmail.com

First Name & Middle Initial & Last Name & Degree

Jose Luis Lopez-Sendon Moreno, MD

Facility Name

Royal Devon and Exeter Hospital NHS Trust

City

Exeter

State/Province

Devon

ZIP/Postal Code

EX2 5DW

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Timothy P Harrower, PhD

timothy.harrower@nhs.net

First Name & Middle Initial & Last Name & Degree

Timothy P Harrower, PhD

Facility Name

Royal Hospital for Children, Pharmacy Aseptic Unit

City

Glasgow

State/Province

Glasgow City

ZIP/Postal Code

G51 4TF

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Matthew Sheridan

matthew.sheridan@ggc.scot.nhs.uk

First Name & Middle Initial & Last Name & Degree

Matthew Sheridan

Facility Name

Cardiff University, Schools of Medicine and Biosciences

City

Cardiff

State/Province

Wales

ZIP/Postal Code

CF14 4XW

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Anne Rosser, PhD, MBBChir, MRCP

rosserae@cardiff.ac.uk

First Name & Middle Initial & Last Name & Degree

Anne Rosser, PhD, MBBChir, MRCP

Facility Name

Royal Liverpool University Hospital

City

Liverpool

ZIP/Postal Code

L7 8XP

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Richard Rhys Davies, MA BMBCh PhD FRCP

rhys.davies@thewaltoncentre.nhs.uk

First Name & Middle Initial & Last Name & Degree

Richard Rhys Davies, MA BMBCh PhD FRCP

Facility Name

Royal Victoria Infirmary

City

Newcastle

Country

United Kingdom

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Suresh Komati, MD

suresh.komati@nhs.net

First Name & Middle Initial & Last Name & Degree

Suresh Komati, MD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

Study of WVE-003 in Patients With Huntington's Disease

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