A Phase 1/2, Randomized, Sequential, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of a One-Time, Bilateral, Intraparenchymal Infusion of SPK-10001 Into the Caudate and Putamen in Participants With Huntington's Disease
Hoffmann-La Roche
Summary
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
Eligibility
- Age range
- 25–65 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: * Have confirmed huntingtin (HTT) cytosine-adenine-guanine (CAG) repeat length ≥40 on genetic testing and confirmation diagnostic test by the central laboratory (CL) at screening. * Have striatal atrophy demonstrated by caudate/intracranial volume less than the age-adjusted cutoff values associated with HDISS Stage 1. * Have UHDRS Total Motor Score (TMS) equal to or greater than the age-adjusted cutoff value associated with HDISS Stage 2. * Have UHDRS Total Functional Capacity (TFC) greater than or equal to 11. * Use of cholinesterase inhibitors, memantine, amantadine,…
Interventions
- GeneticSPK-10001
Specified dose on specified days
- OtherPlacebo Surgery Control
Placebo Surgery procedure for SPK-10001
Locations (5)
- Beth Israel Deaconess Medical CenterBoston, Massachusetts
- University of Cincinnati/Cincinnati Children's HospitalCincinnati, Ohio
- Ohio State UniversityColumbus, Ohio
- University of PennsylvaniaPhiladelphia, Pennsylvania
- University of PittsburgPittsburgh, Pennsylvania