Recruiting
Phase 1
Phase 2

SPK-10001

Sponsor:

Hoffmann-La Roche

Code:

NCT06826612

Conditions

Huntington Disease

Eligibility Criteria

Sex: All

Age: 25 - 65

Healthy Volunteers: Not accepted

Interventions

SPK-10001

Placebo Surgery Control

Study Details

Brief summary:

The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.

Conditions

Huntington Disease

Study ID

NCT06826612

Start date

Feb 21, 2025

Status verified date

Apr, 2026

Completion date

Jan 12, 2035

Anticipated

Primary completion date

Jan 12, 2035

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 25 - 65

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

53 participants

Anticipated

Allocation

Randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: SPK-10001

other: Placebo Surgery Control

Interventions

SPK-10001

Specified dose on specified days

Placebo Surgery Control

Placebo Surgery procedure for SPK-10001

Primary outcome measure

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

Central Contacts and Locations

Central contacts

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

888-662-6728global-roche-genentech-trials@gene.com

Locations

Beth Israel Deaconess Medical Center

Recruiting

Boston, Massachusetts, United States, 02215

University of Cincinnati/Cincinnati Children's Hospital

Recruiting

Cincinnati, Ohio, United States, 45221

Ohio State University

Recruiting

Columbus, Ohio, United States, 43221

University of Pennsylvania

Recruiting

Philadelphia, Pennsylvania, United States, 19107

University of Pittsburg

Recruiting

Pittsburgh, Pennsylvania, United States, 15213

More Information

Sponsor

Hoffmann-La Roche

Last update posted

May 5, 2026

Last verified

Apr, 2026

Keywords

  • Huntington's Disease
  • SPK-10001
  • Adult-onset HD
  • HD
  • Rare Disease
  • Chorea
  • Movement Disorder
  • Spark Therapeutics
  • Cognition
  • Gene Therapy
  • AAV
  • mHTT

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-05. This information was provided to ClinicalTrials.gov by Hoffmann-La Roche on 2026-05-05.