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

Korábbi kezelés

• 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
• Previously received tominersen

Más betegség

• Bone, spine, bleeding, or other disorder that exposes the patient to risk of injury or unsuccessful lumbar puncture

Egyéb

• Inability to undergo brain MRI (with or without sedation)

• 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