Safety and Tolerability of Monthly IV Administration of Study Drug or Placebo in those who carry the HD gene, and those with early symptoms of Huntington's Disease

What we are studying

Eligible subjects will undergo a monthly IV infusion of the study drug (VX15/2503) or placebo over an 18 month period. Subjects will also undergo repeated safety specimen collections (both blood and urine), MRI scans, PET scans (optional), neurological evaluations, motor evaluations and cognitive testing throughout the duration of the study.

Who is Eligible

  • All Genders
  • Races:
    • White
    • African American
    • Asian
    • American Indian or Alaska Native
    • Native Hawaiian or Pacific Islander
    • Other
  • All Ethnicities
  • Ages 21 - 75

Eligibility Criteria

  • Subjects must be at least 21 years of age and carry the HD gene. Subjects must meet the eligibility "score" and be able to provide documentation of genetic testing. Subjects must not be pregnant or plan to become pregnant during the course of the study. If male
  • Must agree to use a reliable method of birth control (condoms with contraceptive foams or sexual abstinence) during the study and for 3 months following the last infusion.

What is involved

  • Enrolled subjects who complete the study
  • Will have 21 in person visits and 1 telephone visit. Subjects will undergo monthly infusions of study drug/placebo
  • Monthly specimen collections
  • Motor evaluations
  • Cognitive and mood/behavior questionnaires. There are 4 or 5 MRIs and 4 or 5 PET scans (optional) throughout the study.


Subjects will be compensated $450 for each infusion visit

Contact Information

Study Coordinator
Christine O'Neill
Principal Investigator
Francis Walker

Disclaimer: The information on this website is for general informational purposes only and SHOULD NOT be relied upon as a substitute for sound professional medical advice, evaluation or care from your physician or other qualified health care provider.