Phase 2 Huntington's Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- You are able to give your informed consent to take part in the study
- You carry an expanded Huntingtin gene with 39 or more CAG repeats (a genetic marker linked to Huntington's disease)
- You do not yet have clear physical/movement symptoms of Huntington's disease, based on a standard assessment called the UHDRS
- Based on this same assessment, your diagnostic confidence level is below 4 at the time of joining (confirm with trial site)
- Based on a specific calculation called the Langbehn formula, you are expected to develop clinical Huntington's disease within the next 10 years
- You have a family member, friend, or carer who can provide background information about you to the research team
- If you are a woman who could become pregnant, you have a negative pregnancy blood test before joining
- If you could become pregnant, you agree to either abstain from sex or use reliable contraception throughout the study
- You are able to tolerate MRI scans
- You are able to tolerate having blood samples taken
- You are able to follow all the requirements of the study, in the opinion of the research team
- You are considered medically, mentally, and neurologically stable at the time of joining, in the opinion of the research team
Who may not be able to join:
- You have already been diagnosed with clinical Huntington's disease
- You have a known allergy or sensitivity to NAC (N-acetylcysteine)
- You are currently pregnant, breastfeeding, or plan to become pregnant or breastfeed before the study ends
- You have taken part in another clinical trial and received an experimental drug within the 30 days before your first study visit
- You are currently taking NAC as a supplement
- You have abnormal results from blood tests, a heart tracing (ECG), or vital signs at your screening appointment that could make it unsafe for you to take part
- You currently have, or have had within the past year, a problem with substance or alcohol misuse
- You have an unstable mental health condition or a serious active medical illness, including cancer, as judged by the research team
- You are currently taking antipsychotic medications or a drug called Tetrabenazine
- You have previously had gene therapy, a cell transplant, or any experimental brain surgery
- You have had thoughts of suicide or a suicide attempt within the 12 months before your screening appointment
- An MRI scan taken during screening shows a pre-existing structural problem in your brain
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Clement Loy, University of Sydney
Phone: 001164 4 8890 3560
Australian sites
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
5 site(s) in Australia. Confirm current status and contact details directly with the trial site.
Primary endpoints
Caudate Atrophy Rate on volumetric MRI; Rate of motor phenoconversion
Can't join this trial?
Data last synced from ClinicalTrials.gov: 28 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.