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SPK-10001 Gene Therapy for Huntington's Disease

This study is testing a gene therapy called SPK-10001 for people with Huntington's Disease. Researchers want to see if SPK-10001 is safe, how well people tolerate it, and if it helps with the disease. You might be able to join if you are between 25 and 65 years old, have a confirmed genetic diagnosis of Huntington's Disease with a specific CAG repeat length (40 or more), and show signs of striatal atrophy (shrinkage of a part of the brain). The study will measure side effects and their severity, and also look at changes in your ability to perform daily activities over 24 months using a scale called the Unified Huntington's Disease Rating Scale (UHDRS®) Total Functional Capacity (TFC) Score. The study plans to enroll 53 participants, but its current status is unclear.

Study design
This is a randomized study, meaning participants will be assigned by chance to receive either SPK-10001 or a placebo surgery. It plans to enroll 53 participants.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for treatment-emergent adverse events (side effects) from Day 1 up to approximately 5 years. Changes in functional capacity will be measured at Baseline and Month 24.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT06826612

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

Recruiting
PHASE1Ages 25–65InterventionalTreatment
Hoffmann-La Roche
~53 participants
Updated 2026-05-05 on ClinicalTrials.gov
What's tested:SPK-10001Placebo Surgery Control

At a glance

Recruiting sites
5 of 5 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Participants with Treatment-emergent Adverse Events (TEAEs)
Measured over Day 1 up to approximately 5 years
+2 more outcomes measured

Conditions

Huntington Disease

Explore related trials

Huntington's DiseaseHTT Gene

NCT06826612

Where you'd take part

This study runs at 5 sites. They're the same protocol — you choose where, and that choice sets who your contact draft is addressed to.

  • Beth Israel Deaconess Medical Center

    Boston, Massachusettsno site contact published

    Recruiting

  • Ohio State University

    Columbus, Ohiono site contact published

    Recruiting

  • University of Cincinnati/Cincinnati Children's Hospital

    Cincinnati, Ohiono site contact published

    Recruiting

  • University of Pennsylvania

    Philadelphia, Pennsylvaniano site contact published

    Recruiting

  • University of Pittsburg

    Pittsburgh, Pennsylvaniano site contact published

    Recruiting

Sites open and close at different times, so the status above is per site — it can differ from the study's overall status.

Study leadership

  • Clinical Trials · STUDY_DIRECTOR · Hoffmann-LaRoche

Who to contact

Reference Study ID Number: SPK-10001-101 https://forpatients.roche.com/
[email protected]
888-662-6728
Email the study team

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Eligibility criteria

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

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.

What this trial measures

  • Number of Participants with Treatment-emergent Adverse Events (TEAEs)Day 1 up to approximately 5 years
  • Severity of TEAEsDay 1 up to approximately 5 years
  • Change from Baseline in Unified Huntington's Disease Rating Scale (UHDRS®) Total Functional Capacity (TFC) ScoreBaseline, Month 24