Study of WVE-003 in Patients With Huntington's Disease
- Registration Number
- NCT05032196
- Lead Sponsor
- Wave Life Sciences Ltd.
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 47
- Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
- Ambulatory, male or female patients aged โฅ25 to โค60 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 โฅ9 and โค13
-
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description WVE-003 (Dose B) or placebo WVE-003 - WVE-003 (Dose A) or placebo WVE-003 - WVE-003 (Dose C) or placebo WVE-003 -
- Primary Outcome Measures
Name Time Method Safety: Proportion of patients with adverse events (AEs) Day 1 through end of study (minimum of 36 weeks)
- Secondary Outcome Measures
Name Time Method Area under the plasma concentration time curve for WVE-003 from time 0 to last quantifiable concentration (AUC0-t) Day 1 through end of study (minimum of 36 weeks) Terminal half-life (t1/2) of WVE-003 in cerebrospinal fluid (CSF) Day 1 through end of study (minimum of 36 weeks) Maximum concentration (Cmax) of WVE-003 in plasma Day 1 through end of study (minimum of 36 weeks)
Trial Locations
- Locations (23)
Leiden University Medical Center
๐ณ๐ฑLeiden, Netherlands
Maastricht University Medical Center
๐ณ๐ฑMaastricht, Netherlands
Cardiff University, Schools of Medicine and Biosciences
๐ฌ๐งCardiff, Wales, United Kingdom
Centre Hospitalier de l-Universite de Montreal
๐จ๐ฆMontrรฉal, Quebec, Canada
Szpital Sw. Wojciecha
๐ต๐ฑGdaลsk, Poland
Royal Devon and Exeter Hospital NHS Trust
๐ฌ๐งExeter, Devon, United Kingdom
Monash Health
๐ฆ๐บClayton, Victoria, Australia
Royal Melbourne Hospital
๐ฆ๐บMelbourne, Victoria, Australia
University of Alberta Hospital
๐จ๐ฆEdmonton, Alberta, Canada
Instytut Psychiatrii I Neurologii
๐ต๐ฑWarsaw, Poland
Westmead Hospital
๐ฆ๐บWestmead, New South Wales, Australia
The Ottawa Hospital
๐จ๐ฆOttawa, Ontario, Canada
Hopital Henri Mondor - Hospital
๐ซ๐ทCrรฉteil, France
George-Huntington-Institut GmbH
๐ฉ๐ชMuenster, Germany
Katholisches Klinikum Bochum gGmbH
๐ฉ๐ชBochum, Germany
Rigshospitalet
๐ฉ๐ฐCopenhagen, Denmark
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)
๐ฉ๐ชTaufkirchen, Germany
Hospital de la Sanata Creu i Sant Pau
๐ช๐ธBarcelona, Spain
Hospital Universitario Ramรณn y Cajal
๐ช๐ธMadrid, Spain
Institut du Cerveau et de la Moelle Epiniere
๐ซ๐ทParis, France
Centro Ricerche Cliniche Di Verona
๐ฎ๐นVerona, Italy
Royal Hospital for Children, Pharmacy Aseptic Unit
๐ฌ๐งGlasgow, Glasgow City, United Kingdom
Royal Liverpool University Hospital
๐ฌ๐งLiverpool, United Kingdom