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 ≥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
-
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
🇳🇱Leiden, Netherlands
Maastricht University Medical Center
🇳🇱Maastricht, Netherlands
Cardiff University, Schools of Medicine and Biosciences
🇬🇧Cardiff, Wales, United Kingdom
Centre Hospitalier de l-Universite de Montreal
🇨🇦Montréal, Quebec, Canada
Szpital Sw. Wojciecha
🇵🇱Gdańsk, Poland
Royal Devon and Exeter Hospital NHS Trust
🇬🇧Exeter, Devon, United Kingdom
Monash Health
🇦🇺Clayton, Victoria, Australia
Royal Melbourne Hospital
🇦🇺Melbourne, Victoria, Australia
University of Alberta Hospital
🇨🇦Edmonton, Alberta, Canada
Instytut Psychiatrii I Neurologii
🇵🇱Warsaw, Poland
Westmead Hospital
🇦🇺Westmead, New South Wales, Australia
The Ottawa Hospital
🇨🇦Ottawa, Ontario, Canada
Hopital Henri Mondor - Hospital
🇫🇷Créteil, France
George-Huntington-Institut GmbH
🇩🇪Muenster, Germany
Katholisches Klinikum Bochum gGmbH
🇩🇪Bochum, Germany
Rigshospitalet
🇩🇰Copenhagen, Denmark
kbo-Isar-Amper-Klinikum Taufkirchen (Vils)
🇩🇪Taufkirchen, Germany
Hospital de la Sanata Creu i Sant Pau
🇪🇸Barcelona, Spain
Hospital Universitario Ramón y Cajal
🇪🇸Madrid, Spain
Institut du Cerveau et de la Moelle Epiniere
🇫🇷Paris, France
Centro Ricerche Cliniche Di Verona
🇮🇹Verona, Italy
Royal Hospital for Children, Pharmacy Aseptic Unit
🇬🇧Glasgow, Glasgow City, United Kingdom
Royal Liverpool University Hospital
🇬🇧Liverpool, United Kingdom