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
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
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: 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
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