Last reviewed · How we verify

NCT06826612

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

Recruiting now Phase 1, PHASE2 Last updated 17 April 2026
What this trial tests

Phase 1, PHASE2 trial testing SPK-10001 in Huntington Disease in 53 participants. Currently enrolling.

Timeline
21 February 2025
Primary endpoint
12 January 2035
12 January 2035

Quick facts

Lead sponsorHoffmann-La Roche
PhasePhase 1, PHASE2
StatusRecruiting now
Study typeINTERVENTIONAL
Allocationrandomized
Designsequential
Maskingquadruple
Primary purposetreatment
Enrollment53
Start date21 February 2025
Primary completion12 January 2035
Estimated completion12 January 2035
Sites5 locations across United States

Drugs / interventions tested

Conditions studied

Sponsor

Hoffmann-La Roche — full company profile →

Who can join

Adults 25 to 65, any sex, with Huntington Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own 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.

Publications & conference data

3 peer-reviewed publications reference this trial (live from Europe PMC):

  1. Current trends in gene therapy to treat inherited disorders of the brain.
    Matuszek Z, Brown BL, Yrigollen CM, Keiser MS, et al · · 2025 · cited 6× · PMID 40181540 · DOI 10.1016/j.ymthe.2025.03.057
  2. Huntington's disease clinical trials update: October 2025.
    Farag M, Tabrizi SJ, Wild EJ. · · 2026 · PMID 41295902 · DOI 10.1177/18796397251399751
  3. Brain organoid models of Huntington's disease shift the focus towards neurodevelopment.
    Xu W, Prigione A. · · 2025 · PMID 41147161 · DOI 10.1242/dmm.052510

Verify or expand the search:

Other recruiting trials for Huntington Disease

Currently open trials in the same condition.

Other Hoffmann-La Roche trials

Trials by the same sponsor.

Verify against primary sources

Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT06826612.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing