Gene therapy for Huntington's Disease using SPK-10001
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
This study is testing a new gene therapy called SPK-10001 to see if it can safely help people with Huntington's Disease feel better.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 53 (estimated) |
| Ages | 25 Years to 65 Years |
| Sex | All |
| Sponsor | Hoffmann-La Roche Industry-sponsored |
| Locations | 5 sites (Boston, Massachusetts and 4 other locations) |
| Trial ID | NCT06826612 on ClinicalTrials.gov |
What this trial studies
This study evaluates the safety, tolerability, and preliminary efficacy of SPK-10001 gene therapy in individuals diagnosed with Huntington's Disease. Participants will be randomly assigned to receive either the gene therapy or a placebo control. The study focuses on patients with specific genetic markers and clinical characteristics associated with Huntington's Disease. The goal is to gather data that could inform future treatment options for this rare and progressive condition.
Who should consider this trial
Good fit: Ideal candidates are adults with confirmed Huntington's Disease who meet specific genetic and clinical criteria.
Not a fit: Patients with Huntington's Disease who do not meet the genetic and clinical inclusion criteria may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a new treatment option that may slow the progression of Huntington's Disease.
How similar studies have performed: Other studies exploring gene therapy for neurodegenerative diseases have shown promise, but this specific approach is novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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, or riluzole must have been at stable dosing for at least 12 weeks before screening and baseline and anticipated to remain stable during the first 12 months after SPK-10001 administration. * Antidepressant or benzodiazepine use must have been at stable dosing for at least 12 weeks before screening and baseline and anticipated to remain stable during the first 12 months after SPK-10001 administration. * Antipsychotics for motor symptoms or mood stabilization (i.e., irritability or aggressive behavior) and/or tetrabenazine, valbenazine, or deutetrabenazine must have been at a stable dose for at least 12 weeks before screening and baseline and are anticipated to remain stable during the first 12 months after SPK-10001 administration. Key Exclusion Criteria: * A safe trajectory is not able to be identified for targeting placement of the cannula into the caudate or putamen on both sides of the brain due to extent of atrophy or other anatomical features. * Have received an antisense oligonucleotide therapy during the past year. * History of deep brain stimulation. * History of or intention to undergo gene therapy, cell transplantation, or brain surgery during the course of the study. * Have participated in an investigational drug study with a systemic administration within 6 weeks or 5 half-lives of screening, whichever is longer. Additional protocol-defined inclusion/exclusion criteria apply.
Where this trial is running
Boston, Massachusetts and 4 other locations
- Beth Israel Deaconess Medical Center — Boston, Massachusetts, United States (Recruiting)
- University of Cincinnati/Cincinnati Children's Hospital — Cincinnati, Ohio, United States (Recruiting)
- Ohio State University — Columbus, Ohio, United States (Recruiting)
- University of Pennsylvania — Philadelphia, Pennsylvania, United States (Recruiting)
- University of Pittsburg — Pittsburgh, Pennsylvania, United States (Recruiting)
Study contacts
- Study coordinator: Reference Study ID Number: SPK-10001-101 https://forpatients.roche.com/
- Email: global-roche-genentech-trials@gene.com
- Phone: 888-662-6728
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.