Recent Updates
Recently added Catalysts

SPK-10001

Phase 1

Huntington Disease | Gene therapy | Neurology |Roche Holding AG|Last Updated: May 5, 2026

Success Probability

Subscribe to view

Market & Valuation

Subscribe to view

Trial Design

RandomizedDouble-BlindCONTROLLEDDMC
Total Trials1
Total Enrollment53

FDA Designations

No designations recorded

Clinical trial landscape

SPK-10001 · 1 trial · 1 indication

Phase 1 1
NCT06826612A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's DiseaseHuntington Disease
RECRUITING53 Analytics
PHASE1RECRUITING
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
Huntington Disease
NCT0682661253
Unlock trial analytics

Study Endpoints

Primary Endpoints

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

Secondary Endpoints

Change from Baseline in Motor Symptom Progression Based on Huntington's Disease Digital Motor Score (HDDMS)
Baseline, Months 12, 18, and 24
Change from Baseline in Composite UHDRS (cUHDRS) Score
Baseline, Months 12, 18, and 24
Change from Baseline in UHDRS TFC Score
Baseline, Months 12 and 18
Unlock Study Endpoints

Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
SPK-10001EXPERIMENTAL -
Placebo Surgery ControlOTHER -

Interventions

NameTypeDescription
SPK-10001GENETICSpecified dose on specified days
Placebo Surgery ControlOTHERPlacebo Surgery procedure for SPK-10001
Unlock Study Design Details

Eligibility Criteria

Age Range25 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites5

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 ...

Countries:United States
Unlock Eligibility Criteria

Competitive Landscape -Huntington's Disease 8 trials (matched to "Huntington Disease")

CompanyTickerTrialsLead PhaseDrugs
Novartis AG Sponsored ADRNVS2PHASE3Votoplam
Neurocrine Biosciences, Inc.NBIX1PHASE3Valbenazine
Alnylam Pharmaceuticals, IncALNY1PHASE1ALN-HTT02
uniQure N.V.QURE2PHASE1intra-striatal rAAV5-miHTT
Sarepta Therapeutics, Inc.SRPT1PHASE1SRP-1005
Unlock Competitive Intelligence

Frequently asked questions about SPK-10001

What is SPK-10001 used for?

SPK-10001 is an investigational gene therapy being developed for the treatment of Huntington's disease, a progressive neurological disorder. It is currently in Phase 1 clinical development and has not been approved by regulatory authorities. The therapy is being studied in a randomized, double-blind, controlled trial to evaluate its safety and efficacy.

Who makes SPK-10001?

SPK-10001 is being developed by Roche Holding AG, a multinational healthcare company. Roche's stock is traded on the OTC market under the ticker symbol RHHBY. The company is conducting a Phase 1 clinical trial to evaluate this gene therapy candidate for Huntington's disease.

What phase is SPK-10001 in?

SPK-10001 is currently in Phase 1 clinical development. It is an investigational gene therapy for Huntington's disease and has not yet received regulatory approval. The ongoing Phase 1 trial is actively recruiting participants in the United States and is designed as a randomized, double-blind, controlled study.

What clinical trials is SPK-10001 in?

SPK-10001 is being evaluated in a Phase 1 clinical trial registered as NCT06826612. This study is a randomized, double-blind, controlled trial enrolling approximately 53 participants with Huntington's disease. The trial is currently recruiting in the United States and includes participants aged 25 years and older.

How does SPK-10001 work?

SPK-10001 is a gene therapy designed to treat Huntington's disease by delivering genetic material to target cells. As a gene therapy, it aims to address the underlying genetic cause of the disease. The specific molecular target of SPK-10001 has not been disclosed in available information.