What is SPK-10001 studying for Huntington's Disease?

SPK-10001 is being studied to evaluate its safety, tolerability, and early effects in people with Huntington's Disease. This trial looks at how the gene therapy works when given once directly into brain areas affected by the disease. The research is sponsored by Hoffmann-La Roche and aims to better understand treatment options.

Who may be eligible for this Huntington's Disease clinical trial?

Adults aged 25 to 65 with confirmed Huntington's Disease and specific genetic markers may be eligible. Participants must have certain clinical scores and stable doses of some medications. The study is open to all genders. Please review eligibility criteria for more details.

Is there any cost to participate in this Huntington's Disease clinical trial?

Study-related care, including the gene therapy or placebo surgery and all trial visits, is provided at no cost. However, personal travel and other expenses may vary and are not guaranteed to be covered.

Actively Recruiting

Phase 1

Phase 2

Age: 25Years - 65Years

All Genders

ID06826612

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

Led by Hoffmann-La Roche · Updated on 2026-05-05

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

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.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 25Years - 65Years

All Genders

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

Research Team

Reference Study ID Number: SPK-10001-101 https://forpatients.roche.com/

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