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NCT Number: NCT06826612

A Randomized Study of SPK-10001 Gene Therapy 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.

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Key information

Conditions

Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Chorea Cognition Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dementia Disease Attributes Dyskinesias Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Mental Disorders Movement Disorders Nervous System Diseases Neurocognitive Disorders Neurodegenerative Diseases Neurologic Manifestations Pathologic Processes Pathological Conditions, Signs and Symptoms Rare Diseases Signs and Symptoms

Age range

25 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

SPK-10001

Genetic

Specified dose on specified days

Placebo Surgery Control

Other

Placebo Surgery procedure for SPK-10001

Primary outcomes

  1. Number of Participants with Treatment-emergent Adverse Events (TEAEs)

    Time frame: Day 1 up to approximately 5 years

  2. Severity of TEAEs

    Time frame: Day 1 up to approximately 5 years

  3. Change from Baseline in Unified Huntington's Disease Rating Scale (UHDRS®) Total Functional Capacity (TFC) Score

    Time frame: Baseline, Month 24

Secondary outcomes

  1. Change from Baseline in Motor Symptom Progression Based on Huntington's Disease Digital Motor Score (HDDMS)

    Time frame: Baseline, Months 12, 18, and 24

  2. Change from Baseline in Composite UHDRS (cUHDRS) Score

    Time frame: Baseline, Months 12, 18, and 24

  3. Change from Baseline in UHDRS TFC Score

    Time frame: Baseline, Months 12 and 18

Study contacts

Contact information is provided by the study sponsor or research team.

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

CONTACT

[email protected]

888-662-6728

Sponsors and collaborators

Lead sponsor

Hoffmann-La Roche

Industry

Registry information

Official study title

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

Important dates

Study start
2025
Primary completion
2035
Study completion
2035
First posted
Feb 14, 2025
Registry last updated
May 5, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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