Phase 2
N=47
Study of WVE-003 in Patients With Huntington's Disease
Huntington Disease
Bottom Line
View on ClinicalTrials.gov: NCT05032196 ↗Enrolled (actual)
47
Serious AEs
2.9%
Results posted
Aug 2025
Primary outcome: Primary: Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug — 2; 1; 3; 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- SAD: 30mg WVE-003 (Drug); SAD: 60mg WVE-003 (Drug); SAD: 90mg WVE-003 (Drug); SAD: Pooled Placebo (Drug); MD: 30mg WVE-003 (Drug); MD: Placebo (Drug)
- Age
- Adult · 25+ yrs
- Sex
- All
- Sponsor
- Wave Life Sciences USA, Inc.
- Primary completion
- May 2024
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Safety: Proportion of Patients With Treatment Emergent Adverse Events (TEAEs) Related to Study Drug |
2; 1; 3; 3; 0; 8 | — |
| SECONDARY Pharmacokinetics of WVE-003 in Plasma |
240; 601; 1061; 186; 157 | — |
| SECONDARY Pharmacokinetics of WVE-003 in Plasma |
240; 601; 1061; 186; 157 | — |
| SECONDARY Concentration of WVE-003 in Cerebrospinal Fluid (CSF) |
3.6405; 5.5346; 7.0983; 4.2903 | — |
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.
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT05032196). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.