Gene Therapy Tested in People With Huntington's Disease
Tests a one-time gene therapy, SPK-10001, in adults with Huntington's disease.
Plain-language title generated from the registry record
Registry title: A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
Key facts
- Where
- Boston, Cincinnati, Columbus, Philadelphia, and Pittsburgh, United StatesSee the 5 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 25 and 65 years old?
Source: NCT06826612, who can take part
Where in United States
- Beth Israel Deaconess Medical CenterBoston, United StatesTaking people
- University of Cincinnati/Cincinnati Children's HospitalCincinnati, United StatesTaking people
- Ohio State UniversityColumbus, United StatesTaking people
- University of PennsylvaniaPhiladelphia, United StatesTaking people
- University of PittsburgPittsburgh, United StatesTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Hoffmann-La Roche
- Trial code
- NCT06826612
- Record updated
- 5 May 2026
- Original description
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.Read full original descriptionShow less
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
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
Inclusion: 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.
Exclusion: Have received an antisense oligonucleotide therapy during the past year; 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; History of deep brain stimulation; History of or intention to undergo gene therapy, cell transplantation, or brain surgery during the course of the study; 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.