CompletedPhase 1/2

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

Australia, Canada47 participantsStarted 2021-09-06

Plain-language 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.

Who can participate

Age range25 Years – 60 Years

SexALL

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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:
✕. 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

What they're measuring

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

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

Trial details

NCT IDNCT05032196

SponsorWave Life Sciences Ltd.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-05-24

Results submitted2025-06-06

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