Clinical trial NCT04617860: 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
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Huntington Disease
Lead sponsor: Wave Life Sciences Ltd.
Sponsor class: INDUSTRY
First posted: 2020-11-05
Enrollment: 36
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: Inclusion Criteria:

1. Patient successfully completed the Phase 1b/2a study with WVE-120102, WVE-HDSNP2-001.

Exclusion Criteria:

1. 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.
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 Adverse Events (TEAEs)
timeFrame: Day 1 to Study Termination (maximum of 12 monthly doses)
measure: Safety: Number of Patients With a Severe TEAE
timeFrame: Day 1 to Study Termination (maximum of 12 monthly doses)
measure: Safety: Number of Patients With Serious TEAEs
timeFrame: Day 1 to Study Termination (maximum of 12 monthly doses)
measure: Safety and Tolerability: Number of Patients Who Withdraw Due to TEAEs
timeFrame: Day 1 to Study Termination (maximum of 12 monthly doses)
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: Toronto
country: Canada
facility: Centre For Movement Disorders
state: Ontario
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: Odense
country: Denmark
facility: Odense University Hospital and University of Southern Denmark
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: