Clinical trial NCT04617847: Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120101 in Patients With Huntington's Disease
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Huntington Disease
Lead sponsor: Wave Life Sciences Ltd.
Sponsor class: INDUSTRY
First posted: 2020-11-05
Enrollment: 27
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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.

Primary outcomes:
measure: Safety: Number of Patients With Treatment-emergent AEs (TEAEs)
timeFrame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)
measure: Safety: Number of Patients With a Severe TEAE
timeFrame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
measure: Safety: Number of Patients With Serious TEAEs
timeFrame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
measure: Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
timeFrame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment
Secondary outcomes:
Other outcomes:
Locations:
city: Sydney
country: Australia
facility: Westmead Hospital
state: New South Wales
city: Herston
country: Australia
facility: Royal Brisbane & Women's Hospital
state: Queensland
city: Carlton
country: Australia
facility: Royal Melbourne Hospital
state: Victoria
city: Clayton
country: Australia
facility: Monash Health
state: Victoria
city: Melbourne
country: Australia
facility: Alfred Health
state: Victoria
city: Parkdale
country: Australia
facility: Calvary Health Care Bethlehem
state: Victoria
city: Perth
country: Australia
facility: North Metropolitan Health Service
state: Western Australia
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
city: Montreal
country: Canada
facility: Centre Hospitalier de l-Universite de Montreal
state: Quebec
city: Aarhus
country: Denmark
facility: Aarhus Universitets Hospital
city: Copenhagen
country: Denmark
facility: Rigshospitalet
city: Créteil
country: France
facility: Hospital Henri Mondor
city: Paris
country: France
facility: Institut du Cerveau et de la Moelle Epinière
city: Muenster
country: Germany
facility: George-Huntington-Institut GmbH
city: Gdańsk
country: Poland
facility: Szpital Sw. Wojciecha
city: Warsaw
country: Poland
facility: Instytut Psychiatrii i Neurologii
city: Exeter
country: United Kingdom
facility: Royal Devon and Exeter Hospital NHS Trust
state: Devon
city: Glasgow
country: United Kingdom
facility: Queen Elizabeth University Hospital - PPDS
state: Glasgow City
Study officials:
affiliation: Wave Life Sciences
name: Medical Director, MD
role: STUDY_DIRECTOR
References: