A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
NCT ID: NCT06826612
Last Updated: 2025-12-17
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1/PHASE2
53 participants
INTERVENTIONAL
2025-02-21
2035-01-12
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
RANDOMIZED
SEQUENTIAL
* Part A is an open-label cohort in which all participants will receive SPK-10001.
* Part B is randomized, double-blind and placebo-surgery-controlled.
* Part C is a crossover open-label portion where participants who received placebo-surgery control in Part B will receive SPK-10001.
* Part D is long term follow-up after completion of active treatment in any of Parts A, B, or C.
TREATMENT
QUADRUPLE
* Part B is blinded and randomized.
* Part C is open-label and non-randomized.
* Part D is open-label and non-randomized.
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
SPK-10001
SPK-10001
Specified dose on specified days
Placebo Surgery Control
Placebo Surgery Control
Placebo Surgery procedure for SPK-10001
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
SPK-10001
Specified dose on specified days
Placebo Surgery Control
Placebo Surgery procedure for SPK-10001
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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 Criteria
* 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.
25 Years
65 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Hoffmann-La Roche
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Beth Israel Deaconess Medical Center
Boston, Massachusetts, United States
University of Cincinnati/Cincinnati Children's Hospital
Cincinnati, Ohio, United States
Ohio State University
Columbus, Ohio, United States
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
Clinical Trial Director
Role: CONTACT
Facility Contacts
Find local site contact details for specific facilities participating in the trial.
Andrew Duker, MD
Role: primary
Sandra Kostyk, MD, PhD
Role: primary
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
SPK-10001-101
Identifier Type: -
Identifier Source: org_study_id