Actively Recruiting
A Phase 1/2 Randomized Study Evaluating Safety, Tolerability, and Efficacy of One-Time SPK-10001 Gene Therapy Infusion in Adults with Huntington's Disease
Led by Hoffmann-La Roche · Updated on 2026-05-05
53
Participants Needed
5
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and initial effects of SPK-10001 gene therapy in adults with Huntington's Disease. This randomized Phase 1/2 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 Huntington's 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 Huntington's 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.
CONDITIONS
Brief Title
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Have confirmed huntingtin (HTT) CAG repeat length of 40 or more on genetic testing
- Show striatal atrophy with caudate/intracranial volume below age-adjusted cutoffs for HDISS Stage 1
- Have UHDRS Total Motor Score at or above age-adjusted cutoff for HDISS Stage 2
- Have UHDRS Total Functional Capacity score of 11 or higher
- Use cholinesterase inhibitors, memantine, amantadine, or riluzole at stable doses for at least 12 weeks before screening and baseline, expected to remain stable for 12 months after therapy
- Use antidepressants or benzodiazepines at stable doses for at least 12 weeks before screening and baseline, expected to remain stable for 12 months after therapy
- Use antipsychotics for motor or mood symptoms and/or tetrabenazine, valbenazine, or deutetrabenazine at stable doses for at least 12 weeks before screening and baseline, expected to remain stable for 12 months after therapy
You will not qualify if you...
- Unable to identify a safe path for cannula placement into both sides of the brain due to atrophy or anatomy
- Received antisense oligonucleotide therapy within the past year
- History of deep brain stimulation
- History of or plans for gene therapy, cell transplantation, or brain surgery during the study
- Participation in another investigational drug study with systemic administration within 6 weeks or 5 half-lives before screening
- Additional protocol-defined exclusion criteria apply
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - One-time intervention with follow-up lasting up to approximately 5 years
Participants receive a one-time infusion of SPK-10001 gene therapy or placebo surgery.
1 baseline visit and periodic follow-up visits over 5 years
Trial Site Locations
Total: 5 locations
1
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States, 02215
Actively Recruiting
2
University of Cincinnati/Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45221
Actively Recruiting
3
Ohio State University
Columbus, Ohio, United States, 43221
Actively Recruiting
4
University of Pennsylvania
Philadelphia, Pennsylvania, United States, 19107
Actively Recruiting
5
University of Pittsburg
Pittsburgh, Pennsylvania, United States, 15213
Actively Recruiting
Research Team
R
Reference Study ID Number: SPK-10001-101 https://forpatients.roche.com/
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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