Actively Recruiting
A Randomised Controlled Trial of N-Acetyl Cysteine (NAC) for Premanifest Huntington Gene Expansion Carriers
Led by Western Sydney Local Health District · Updated on 2025-02-28
160
Participants Needed
5
Research Sites
25 weeks
Total Duration
On this page
Sponsors
W
Western Sydney Local Health District
Lead Sponsor
D
Deakin University
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are conducting a phase II randomized placebo-controlled trial to study oral N-Acetylcysteine (NAC) in individuals who carry the Huntington disease gene but do not yet show clear motor symptoms. The study aims to assess clinical and brain imaging outcomes over a three-year period, focusing on slowing disease progression in this premanifest stage. Participants will be randomly assigned to take either 1 gram of NAC capsules or matching placebo capsules twice daily by mouth. The study is quadruple-masked, meaning that participants, caregivers, investigators, and outcome assessors do not know which treatment is assigned. The treatment period lasts up to three years, with ongoing monitoring throughout. During the study, participants will undergo regular brain MRI scans to measure caudate atrophy rate and be evaluated for motor changes indicating disease progression. Various cognitive and behavioral tests, such as the UHDRS motor subscale, Stroop Word test, and Montreal Cognitive Assessment, will be performed at intervals over the three years. Safety will be monitored through laboratory tests, ECGs, and tracking of adverse events. Participants must comply with study procedures including blood draws and MRI scans for the full duration up to the study end in May 2027.
CONDITIONS
Brief Title
A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Able to provide informed consent
- Carrier of Huntington gene expansion with 39 or more CAG repeats
- No clear motor signs of Huntington Disease (Diagnostic Confidence Level less than 4)
- Expected to develop clinical Huntington Disease within 10 years based on Langbehn formula
- Availability of an informant for history confirmation
- Negative serum pregnancy test for women who could become pregnant
- If able to become pregnant, agrees to remain abstinent or use effective contraception
- Able to tolerate MRI scans and blood draws
- Able to follow all study procedures as judged by investigator
- Medically, psychiatrically, and neurologically stable at enrollment according to investigator
You will not qualify if you...
- Diagnosis of clinical Huntington Disease
- Known allergy or hypersensitivity to N-Acetylcysteine (NAC)
- Pregnancy, breastfeeding, or plans to become pregnant before study ends
- Use of any investigational drugs within 30 days before baseline visit
- Use of supplemental N-Acetylcysteine
- Abnormal lab tests, ECG, or vital signs that make participation unsafe
- Current or recent (within 1 year) substance abuse
- Unstable psychiatric or acute medical illness including cancer
- Current use of antipsychotic medications or Tetrabenazine
- History of gene therapy, cell transplantation, or experimental brain surgery
- Suicide attempt or suicidal thoughts within 12 months before screening
- Pre-existing structural brain lesion found on MRI during screening
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 3 years
Participants take either 1g of N-Acetylcysteine capsules or matching placebo capsules orally twice a day.
Regular visits throughout treatment as scheduled by the study team
Trial Site Locations
Total: 5 locations
1
Westmead Hospital
Westmead, New South Wales, Australia, 2145
Actively Recruiting
2
The University of Queensland
Herston, Queensland, Australia, 4029
Not Yet Recruiting
3
Calvary Health Care Bethlehem
Parkdale, Victoria, Australia, 3195
Not Yet Recruiting
4
The Royal Melbourne Hospital
Parkville, Victoria, Australia, 3050
Not Yet Recruiting
5
Perron Institute
Nedlands, Western Australia, Australia, 6009
Not Yet Recruiting
Research Team
C
Clement Loy
S
Sarah Samperi
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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