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

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.

Full Title of Study: “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

  • Study Type: Interventional
  • Study Design
    • Allocation: N/A
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: May 3, 2021

Interventions

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

Arms, Groups and Cohorts

  • Experimental: WVE-120101 (Dose A)

Clinical Trial Outcome Measures

Primary Measures

  • 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

Participating in This Clinical Trial

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.

Gender Eligibility: All

Minimum Age: N/A

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Wave Life Sciences Ltd.
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Medical Director, MD, Study Director, Wave Life Sciences

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