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