SELECT HD

Enrolment Completed

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SPONSOR

Wave Life Sciences Ltd.

PARTICIPANTS

36

The study is a phase 1/2 and will look at the safety and how participants tolerate a new antisense oligonucleotide(ASO) in early stage disease.

Estimated Study Completion Date: December 2022

Ages Eligible for Study:

25 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

No

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

COUNTRIES

Denmark

  • TRIAL SITE:Rigshospitalet
  • Address:Copenhagen, Denmark, 2100
  • Principal Investigator: Lena Hjermind, MD

France

  • TRIAL SITE: Hopital Henri Mondor – Hospital
  • Address: Créteil, France, 94010
  • Principal Investigator: Katia Youssov, MD 
  • TRIAL SITE:Institut du Cerveau et de la Moelle Epiniere
  • Address: Paris, France, 75646
  • Principal Investigator: Alexandra Durr, MD, PhD

Germany

  • 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  

Poland

  • 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 

Spain

  • TRIAL SITE: Hospital de la Sanata Creu i Sant Pau
  • Address: Barcelona, Spain, 08041
  • Principal Investigator: Jaime Kulisevsky, MD, PhD 
  • TRIAL SITE: Hospital Universitario Ramón y Cajal
  • Address: Madrid, Spain, 28034
  • Principal Investigator: Jose Luis Lopez-Sendon Moreno, MD

UK

  • TRIAL SITE: Royal Liverpool University Hospital
  • Address: Liverpool, United Kingdom, L7 8XP
  • Principal Investigator: Richard Rhys Davies, MA BMBCh PhD FRCP 
  • TRIAL SITE: Royal Hospital for Children, Pharmacy Aseptic Unit
  • Address:Glasgow, Glasgow City, United Kingdom, G51 4TF
  • Principal Investigator: Matthew Sheridan 
  • TRIAL SITE:Royal Liverpool University Hospital
  • Address: Liverpool, United Kingdom, L7 8XP
  • Principal Investigator: Richard Rhys Davies, MA BMBCh PhD FRCP     
  • TRIAL SITE: Royal Victoria Infirmary
  • Address: Newcastle, United Kingdom
  • Principal Investigator: Suresh Komati, MD   
  • TRIAL SITE: Cardiff University, Schools of Medicine and Biosciences
  • Address: Cardiff, Wales, United Kingdom, CF14 4XW
  • Principal Investigator: Anne Rosser, PhD, MBBChir, MRCP