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

Huntington Disease Clinical Trial

Official title:

A Multicenter, Open-label Extension Study to Evaluate the Safety, Pharmacodynamics, and Clinical Effects of WVE-120101 in Patients With Huntington's Disease

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04617847
• Other study ID #: WVE-HDSNP1-002
• Status: Terminated
• Phase: Phase 1/Phase 2
• Start date: April 13, 2020
• Est. completion date: May 3, 2021

Study information

• Verified date: January 2022
• Source: Wave Life Sciences Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 27
• Est. completion date: May 3, 2021
• Est. primary completion date: May 3, 2021
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Key Inclusion Criteria:

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

Key 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.

Related Conditions & MeSH terms

• Huntington Disease

Intervention

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

Locations

Country	Name	City	State
Australia	Royal Melbourne Hospital	Carlton	Victoria
Australia	Monash Health	Clayton	Victoria
Australia	Royal Brisbane & Women's Hospital	Herston	Queensland
Australia	Alfred Health	Melbourne	Victoria
Australia	Calvary Health Care Bethlehem	Parkdale	Victoria
Australia	North Metropolitan Health Service	Perth	Western Australia
Australia	Westmead Hospital	Sydney	New South Wales
Canada	University of Alberta	Edmonton	Alberta
Canada	Centre Hospitalier de l-Universite de Montreal	Montreal	Quebec
Denmark	Aarhus Universitets Hospital	Aarhus
Denmark	Rigshospitalet	Copenhagen
France	Hospital Henri Mondor	Créteil
France	Institut du Cerveau et de la Moelle Epinière	Paris
Germany	George-Huntington-Institut GmbH	Muenster
Poland	Szpital Sw. Wojciecha	Gdansk
Poland	Instytut Psychiatrii i Neurologii	Warsaw
United Kingdom	Royal Devon and Exeter Hospital NHS Trust	Exeter	Devon
United Kingdom	Queen Elizabeth University Hospital - PPDS	Glasgow	Glasgow City

Sponsors (1)

Lead Sponsor	Collaborator
Wave Life Sciences Ltd.

Countries where clinical trial is conducted

Australia,  Canada,  Denmark,  France,  Germany,  Poland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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)
Primary	Safety: Number of Patients With a Severe TEAE		First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Primary	Safety: Number of Patients With Serious TEAEs		First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Primary	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