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

Phase 2TerminatedNCT04617847

Wave Life Sciences USA, Inc.27 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Huntington Disease

Interventions

WVE-120101DRUG

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

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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.

Locations (18)

Westmead Hospital

Sydney, New South Wales, Australia

Royal Brisbane & Women's Hospital

Herston, Queensland, Australia

Outcomes

Primary Outcomes

Safety: Number of Patients With Treatment-emergent AEs (TEAEs)

Time frame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment)

Safety: Number of Patients With a Severe TEAE

Time frame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment

Safety: Number of Patients With Serious TEAEs

Time frame: 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

Time frame: First dose received (Day 1) through the Study Termination visit (maximum of 45 weeks of treatment

Data from ClinicalTrials.gov

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