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Completed

NCT Number: NCT05032196

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

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.

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Key information

Conditions

Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases

Age range

25 year–60 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Westmead Hospital, Westmead, New South Wales, Australia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
  • Ambulatory, male or female patients aged ≥25 to ≤60 years
  • Clinical diagnostic motor features of HD, defined as Unified Huntington's Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
  • UHDRS Total Functional Capacity Scores ≥9 and ≤13

Exclusion criteria

  • Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years
  • Received any other study drug, including an investigational oligonucleotide, within the past 1 year or 5 half-lives of the drug, whichever is longer, with the exception of the following:

a. Received WVE-120101 or WVE-120102 within the last 3 months

  • Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
  • Inability to undergo brain MRI (with or without sedation)
  • Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture
  • Previously received tominersen

Treatment and study plan

SAD: 30mg WVE-003

Drug

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

SAD: 60mg WVE-003

Drug

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

SAD: 90mg WVE-003

Drug

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

SAD: Pooled Placebo

Drug

Single dose of placebo

MD: 30mg WVE-003

Drug

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

MD: Placebo

Drug

Three doses of placebo Q8WK

Primary outcomes

  1. Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug

    Time frame: Day 1 through Week 24 (single ascending dose Period 1); Day 1 through Week 28 (multi dose Period 2)

    The primary outcome for this study was safety and is reported as the proportion of patients with TEAEs related to study drug.

Secondary outcomes

  1. Pharmacokinetics of WVE-003 in Plasma

    Time frame: Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)

    Parameter analyzed: AUC0-6 = area under the concentration-time curve from time 0 to 6 hrs

  2. Pharmacokinetics of WVE-003 in Plasma

    Time frame: Day 1 (single ascending dose Period 1); Day 1 and Day 113 (multi dose Period 2)

    Parameter analyzed: Cmax = maximum observed concentration.

  3. Concentration of WVE-003 in Cerebrospinal Fluid (CSF)

    Time frame: 28 days post-dose during Period 1 (P1:Day29); 28 days post last dose during Period 2 (P2: Day141)

    WVE-003 concentration in cerebrospinal fluid (CSF) is reported in ng/mL.

Sponsors and collaborators

Lead sponsor

Wave Life Sciences USA, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2021
Primary completion
2024
Study completion
2024
First posted
Sep 2, 2021
Registry last updated
Aug 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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