Trials / Recruiting
RecruitingNCT06826612
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
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
- Status
- Recruiting
- Phase
- Phase 1 / Phase 2
- Study type
- Interventional
- Enrollment
- 53 (estimated)
- Sponsor
- Hoffmann-La Roche · Industry
- Sex
- All
- Age
- 25 Years – 65 Years
- Healthy volunteers
- Not accepted
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| GENETIC | SPK-10001 | Specified dose on specified days |
| OTHER | Placebo Surgery Control | Placebo Surgery procedure for SPK-10001 |
Timeline
- Start date
- 2025-02-21
- Primary completion
- 2035-01-12
- Completion
- 2035-01-12
- First posted
- 2025-02-14
- Last updated
- 2026-04-17
Locations
5 sites across 1 country: United States
Regulatory
- FDA-regulated drug study
Source: ClinicalTrials.gov record NCT06826612. Inclusion in this directory is not an endorsement.