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

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

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.

Study Overview

• Status: Terminated
• Conditions: Huntington Disease
• Intervention / Treatment: Drug: WVE-120101

• Study Type: Interventional
• Enrollment (Actual): 27
• Phase: Phase 2; Phase 1

Contacts and Locations

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2145
      • Westmead Hospital
  • Queensland
    • Herston, Queensland, Australia, QLD 4006
      • Royal Brisbane & Women's Hospital
  • Victoria
    • Carlton, Victoria, Australia, 3053
      • Royal Melbourne Hospital
    • Clayton, Victoria, Australia, 3168
      • Monash Health
    • Melbourne, Victoria, Australia, 3004
      • Alfred Health
    • Parkdale, Victoria, Australia, 3195
      • Calvary Health Care Bethlehem
  • Western Australia
    • Perth, Western Australia, Australia, 6910
      • North Metropolitan Health Service
• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G 2B7
      • University of Alberta
  • Quebec
    • Montreal, Quebec, Canada, H2X019
      • Centre Hospitalier de l-Universite de Montreal
• Denmark
  • Aarhus, Denmark, 8200
    • Aarhus Universitets Hospital
  • Copenhagen, Denmark, 2100
    • Rigshospitalet
• France
  • Créteil, France, 94010
    • Hospital Henri Mondor
  • Paris, France, 75646
    • Institut du Cerveau et de la Moelle Epinière
• Germany
  • Muenster, Germany, 48149
    • George-Huntington-Institut GmbH
• Poland
  • Gdańsk, Poland, 80-462
    • Szpital Sw. Wojciecha
  • Warsaw, Poland, 02-957
    • Instytut Psychiatrii i Neurologii
• United Kingdom
  • Devon
    • Exeter, Devon, United Kingdom, EX2 5DW
      • Royal Devon and Exeter Hospital NHS Trust
  • Glasgow City
    • Glasgow, Glasgow City, United Kingdom, G12 0XH
      • Queen Elizabeth University Hospital - PPDS

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: ADULT; OLDER_ADULT; CHILD
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: WVE-120101 (Dose A)	Drug: WVE-120101; WVE-120101 is a stereopure antisense oligonucleotide (ASO). It is administered monthly via intrathecal injection.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Collaborators and Investigators

• Sponsor: Wave Life Sciences Ltd.

Study record dates

Study Major Dates

• Study Start (ACTUAL): April 13, 2020
• Primary Completion (ACTUAL): May 3, 2021
• Study Completion (ACTUAL): May 3, 2021

Study Registration Dates

• First Submitted: October 30, 2020
• First Submitted That Met QC Criteria: October 30, 2020
• First Posted (ACTUAL): November 5, 2020

Study Record Updates

• Last Update Posted (ACTUAL): February 9, 2022
• Last Update Submitted That Met QC Criteria: January 19, 2022
• Last Verified: January 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Chorea; Huntington Disease
• Other Study ID Numbers: WVE-HDSNP1-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes