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



Wave Life Sciences Ltd.



This study 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.

Estimated Study Completion Date: December 2022

Ages Eligible for Study:

25 Years to 60 Years 

Sexes Eligible for Study:


Accepts Healthy Volunteers:


Inclusion criteria:

  1. Presence of the A variant of SNP3 on the same allele as the pathogenic CAG triplet expansion
  2. Ambulatory, male or female patients aged ≥25 to ≤60 years
  3. Clinical diagnostic motor features of HD, defined as Unified Huntington’s Disease Rating Scale (UHDRS) Diagnostic Confidence Score = 4
  4. UHDRS Total Functional Capacity Scores ≥9 and ≤13

Exclusion criteria:

  1. Malignancy or received treatment for malignancy, other than treated basal cell or squamous cell carcinoma of the skin, within the previous 5 years
  2. 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.
  3. Implantable CNS device that may interfere with ability to administer study drug via lumbar puncture or undergo MRI scan
  4. Inability to undergo brain MRI (with or without sedation)
  5. Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture



  • TRIAL SITE: Monash Health
  • Address: Clayton, Victoria, Australia, 3168
  • Principal Investigator: Yenni Lie, MD
  • TRIAL SITE: Royal Melbourne Hospital
  • Address: Melbourne, Victoria, Australia, 3050
  • Principal Investigator: Dennis Velakoulis, MBBS MMed DMedSci 

Western Australia

  • TRIAL SITE: North Metropolitan Health Service
  • Address: Perth, Western Australia, Australia, 6910
  • Principal Investigator: Carolyn Orr, MD


  • TRIAL SITE: Katholisches Klinikum Bochum gGmbH
  • Address: Bochum, Germany, 44791
  • Principal Investigator: Carsten Saft, MD     
  • TRIAL SITE: George-Huntington-Institut GmbH
  • Address: Muenster, Germany, 48149
  • Principal Investigator: Ralf Reilman, MD  
  • TRIAL SITE: kbo-Isar-Amper-Klinikum Taufkirchen (Vils)
  • Address: Taufkirchen, Germany, 84416
  • Principal Investigator:  Alzbeta Mühlbäck, MD  


  • TRIAL SITE: Szpital Sw. Wojciecha
  • Address:Gdańsk, Poland, 80-462
  • Principal Investigator: Jaroslaw Slawek, MD     
  • TRIAL SITE: Instytut Psychiatrii I Neurologii
  • Address:Warsaw, Poland, 02-957
  • Principal Investigator: Grzegorz Witkowski, MD, PhD 

United Kingdom

  • TRIAL SITE:Royal Liverpool University Hospital
  • Address: Liverpool, United Kingdom, L7 8XP
  • Principal Investigator: Richard Rhys Davies, MA BMBCh PhD FRCP