Actively Recruiting
A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
Led by Western Sydney Local Health District · Updated on 2025-02-28
160
Participants Needed
5
Research Sites
152 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
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.
CONDITIONS
Official 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
- Huntingtin gene expansion carrier with �39 CAG repeats
- No clear motor signs of Huntington disease (Diagnostic Confidence Level <4)
- Expected to develop clinical Huntington disease within 10 years using Langbehn formula
- Availability of an informant for corroborative history
- Negative pregnancy test for women of childbearing potential
- If of childbearing potential, agrees to abstain or use contraception
- Ability to tolerate MRI scans
- Ability to tolerate blood draws
- Able to comply with all study protocol requirements
- Medically, psychiatrically, and neurologically stable at enrolment as judged by investigator
You will not qualify if you...
- Diagnosis of clinical Huntington disease
- Known allergy to N-Acetylcysteine (NAC)
- Pregnancy, breastfeeding, or plans to become pregnant before study end
- Use of investigational drugs within 30 days before baseline
- Use of supplemental NAC
- Abnormal lab results, ECG, or vital signs that prevent safe participation
- Substance abuse within one year before baseline
- 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 screening MRI
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
CONTACT
S
Sarah Samperi
CONTACT
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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