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

ConditionHuntington Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age25-65
SponsorHoffmann-La Roche

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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.

Trial design

Design model

Sequential

Treatments tested in this trial

  • SPK-10001

    Genetic

    Specified dose on specified days

  • Placebo Surgery Control

    Other intervention

    Placebo Surgery procedure for SPK-10001

Treatment groups

53 Participants
are divided into 2 treatment groups
Group A: SPK-10001Experimental treatment 1 intervention
Group B: Placebo Surgery ControlOther 1 intervention

Trial outcomes

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Hoffmann-La Roche

Lead sponsor

Source ClinicalTrials.gov
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