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
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
Treatments tested in this trial
- SPK-10001
- Placebo Surgery Control
Treatment groups
53 Participants
are divided into 2 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov