NAC-preHD is a phase II randomized placebo controlled study of oral NAC among premanifest HD gene expansion carriers, with clinical and radiological outcome at three years.
Able to provide informed consent
Huntingtin gene expansion carrier with >= 39 CAG repeats
Absence of unequivocal motor signs of HD - that is, UHDRS
Diagnostic Confidence Level needs to be <4 upon enrolment
Diagnosis of clinical HD
Known hypersensitivity to NAC
Pregnancy, breastfeeding or intention to do so prior to the end of the study
Exposure to any investigational drugs within 30 days of Baseline Visit
Parallel
NAC
Drug1g of clinical grade N-Acetylcysteine capsules, taken orally twice a day
Placebo
DrugCoated Placebo capsules, manufactured to match appearance and taste, taken orally twice a day
Caudate Atrophy Rate on volumetric MRI
Blinded assessment
Rate of motor phenoconversion
Defined by conversion to Diagnostic Confidence Level 4, upon blinded assessment using the UHDRS motor subscale
UHDRS motor subscale (total score)
Measuring changes in motor function
Stroop Word
Change in cognition as measured by Stroop Word
Trail Making Test
Change in cognition as measured by Trail Making Test
Montreal Cognitive Assessment
Change in cognition as measured by Montreal Cognitive Assessment
Click on the lead sponsor to view all of their trials.
Western Sydney Local Health District
Lead sponsor
Deakin University
Collaborator
Monash University
Collaborator
Royal Perth Hospital
Collaborator
The University of Queensland
Collaborator
University of Melbourne
Collaborator
