Actively Recruiting
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
Led by Hoffmann-La Roche · Updated on 2026-05-05
53
Participants Needed
5
Research Sites
516 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official 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) cytosine-adenine-guanine (CAG) repeat length 640 on genetic testing and confirmation diagnostic test by the central laboratory at screening
- Have striatal atrophy shown by caudate/intracranial volume below age-adjusted cutoff values linked to HDISS Stage 1
- Have Unified Huntington's Disease Rating Scale (UHDRS) Total Motor Score equal or above age-adjusted cutoff for HDISS Stage 2
- Have UHDRS Total Functional Capacity score of 11 or higher
- Use of cholinesterase inhibitors, memantine, amantadine, or riluzole must be at stable doses for at least 12 weeks before screening and expected to remain stable for 12 months after treatment
- Use of antidepressants or benzodiazepines must be stable for at least 12 weeks before screening and expected to remain stable for 12 months after treatment
- Use of antipsychotics for motor symptoms or mood stabilization and/or tetrabenazine, valbenazine, or deutetrabenazine must be stable for at least 12 weeks before screening and expected to remain stable for 12 months after treatment
You will not qualify if you...
- Cannot safely place the cannula into both sides of the brain due to severe atrophy or other anatomical issues
- Received antisense oligonucleotide therapy in the past year
- History of deep brain stimulation
- History of or plans for gene therapy, cell transplantation, or brain surgery during the study
- Participated in a systemic investigational drug study within 6 weeks or 5 half-lives before screening
- Additional protocol-defined exclusion criteria apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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/
CONTACT
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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