A Safety and Pharmacokinetics Trial of VO659 in SCA1, SCA3 and HD
Status:
Recruiting
Trial end date:
2025-09-15
Target enrollment:
Participant gender:
Summary
The goal of this first-in-human clinical trial is to assess the safety and tolerability of
four doses of a new study drug called VO659 in people with genetic disorders called
spinocerebellar ataxia type 1, type 3 or Huntington's disease. Another aim is to determine
the concentrations of the study drug in the cerebral spinal fluid and blood after single and
multiple doses. Study drug will be administered by lumbar intrathecal bolus injections.