Study of SPK-10001 Gene Therapy Given Once by Brain Injection for Adults Aged 25 to 65 With Huntingtons Disease

What this Trial Is About

Researchers are evaluating the safety, tolerability, and initial effects of SPK-10001 gene therapy in adults with Huntingtons Disease. This randomized Phase 12 study aims to assess these aspects through a controlled, dose-escalation approach involving a one-time infusion into specific brain regions. The study focuses on participants aged 25 to 65 years who have confirmed genetic and clinical signs of Huntingtons Disease. Participants will receive either the SPK-10001 gene therapy or a placebo surgery control in a randomized, quadruple-masked design. The gene therapy is administered as a single bilateral intraparenchymal infusion into the caudate and putamen. The study includes dose escalation and monitoring over several years to evaluate safety and early signs of efficacy. During the study, participants will be monitored for treatment-emergent adverse events for up to approximately five years. They will undergo assessments including the Unified Huntingtons Disease Rating Scale Total Functional Capacity score and motor symptom progression measured by digital motor scores and composite scales at multiple time points up to 24 months. The study involves detailed safety and efficacy evaluations, with follow-up visits scheduled for long-term observation.

A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease