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Study of WVE-003 in Patients With Huntington's Disease

NCT05032196 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 47

Last updated 2025-08-12

Study results available
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Summary

This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of WVE-003 in adult patients with early-manifest HD who carry the targeted single nucleotide polymorphism (SNP) - SNP3.

Conditions

  • Huntington Disease

Interventions

DRUG

SAD: 30mg WVE-003

Single ascending dose of 30mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

DRUG

SAD: 60mg WVE-003

Single ascending dose of 60mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

DRUG

SAD: 90mg WVE-003

Single ascending dose of 90mg WVE-003, an allele-selective stereopure antisense oligonucleotide (ASO)

DRUG

SAD: Pooled Placebo

Single dose of placebo

DRUG

MD: 30mg WVE-003

Three doses of 30mg WVE-003 Q8WK an allele-selective stereopure, antisense oligonucleotide (ASO)

DRUG

MD: Placebo

Three doses of placebo Q8WK

Sponsors & Collaborators

  • Wave Life Sciences Ltd.

    lead INDUSTRY

Principal Investigators

  • Medical Director, MD · Wave Life Sciences

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Model
SEQUENTIAL

Eligibility

Min Age
25 Years
Max Age
60 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-06
Primary Completion
2024-05-24
Completion
2024-05-24
FDA Drug
Yes

Countries

  • Australia
  • Canada
  • Denmark
  • France
  • Germany
  • Italy
  • Netherlands
  • Poland
  • Spain
  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05032196 on ClinicalTrials.gov