NCT05032196

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

A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington's Disease (SELECT-HD)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 47 (actual)
• Sponsor: Wave Life Sciences Ltd. · Industry
• Sex: All
• Age: 25 Years – 60 Years
• Healthy volunteers: Not accepted

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

Type	Name	Description
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

Timeline

• Start date: 2021-09-06
• Primary completion: 2024-05-24
• Completion: 2024-05-24
• First posted: 2021-09-02
• Last updated: 2025-08-12
• Results posted: 2025-08-12

Locations

23 sites across 10 countries: Australia, Canada, Denmark, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom

Regulatory

• FDA-regulated drug study