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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
160
Westmead Hospital
Westmead, New South Wales, Australia
RECRUITINGThe University of Queensland
Herston, Queensland, Australia
NOT_YET_RECRUITINGCalvary Health Care Bethlehem
Parkdale, Victoria, Australia
NOT_YET_RECRUITINGCaudate Atrophy Rate on volumetric MRI
Blinded assessment
Time frame: Baseline through end of study (up to 3 years)
Rate of motor phenoconversion
Defined by conversion to Diagnostic Confidence Level 4, upon blinded assessment using the UHDRS motor subscale
Time frame: Baseline through end of study (up to 3 years)
UHDRS motor subscale (total score)
Measuring changes in motor function
Time frame: Baseline through end of study (up to 3 years)
Stroop Word
Change in cognition as measured by Stroop Word
Time frame: Baseline through end of study (up to 3 years)
Trail Making Test
Change in cognition as measured by Trail Making Test
Time frame: Baseline through end study (up to 3 years)
Montreal Cognitive Assessment
Change in cognition as measured by Montreal Cognitive Assessment
Time frame: Baseline through end of study (up to 3 years)
Symbol Digit Modality Test
Change in cognition as measured by Symbol Digit Modality Test
Time frame: Baseline through end of study (up to 3 years)
Changes in Mood and Behavioural symptoms
Evaluated using the PBA-s, a semi-structured interview behavioural scale
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The Royal Melbourne Hospital
Parkville, Victoria, Australia
NOT_YET_RECRUITINGPerron Institute
Nedlands, Western Australia, Australia
NOT_YET_RECRUITINGTime frame: Baseline through end of study (up to 3 years)
Changes in Daily Function
Measured using the Total Functional Capacity and Independent Scale from the broader UHDRS and the Functional Rating Scale for HD
Time frame: Baseline through end of study (up to 3 years)
Change to Quality of Life
As measured by the standardised questionnaires, HDQoL and EQ-5D
Time frame: Baseline through end of study (up to 3 years)
Study completion (Safety and Tolerability)
Measured by the proportion of participants completing NAC arm of study
Time frame: Baseline through end of study (up to 3 years)
Incidence of abnormal laboratory values and/or 12-lead ECG changes (Safety and Tolerability)
Measured by the Number of participants with abnormal laboratory values and/or 12-lead ECG changes compared to baseline
Time frame: Baseline through end of study (up to 3 years)
Incidence of adverse and/or serious adverse events (Safety and Tolerability)
Measured by the number of adverse and/or serious adverse events
Time frame: Baseline through end of study (up to 3 years)