Wave Life Sciences Ltd.
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.
A Multicenter, Randomized, Double-blind, Placebo Controlled, Phase 1b/2a Study of WVE-003 Administered Intrathecally in Patients With Huntington’s Disease.
Interventional (Clinical Trial)
Location: Centre Hospitalier de l’Universite de Montreal
Montréal, Quebec, Canada, H2X019
Principal Investigator: Dr. Sylvain Chouinard
Ages Eligible for Study: 25 Years to 60 Years (Adult)
Sexes Eligible for Study: All
Accepts Healthy Volunteers: No
- 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 (eligibility that would exclude you from this study)
- 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
For more detailed information on this study and others and for the most recent updates, please go to clinicaltrials.gov. ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.