Tests N-acetyl cysteine in people carrying the Huntington disease gene before symptoms
Tests N-acetyl cysteine against placebo in adults with the Huntington gene, not yet symptomatic.
Plain-language title generated from the registry record
Registry title: A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
Key facts
- Where
- Herston, Nedlands, Parkdale, Parkville, and Westmead, AustraliaSee the 5 sites
- Phase
- Phase 2
Where in Australia
- The University of QueenslandHerston, AustraliaNot yet recruiting
- Perron InstituteNedlands, AustraliaNot yet recruiting
- Calvary Health Care BethlehemParkdale, AustraliaNot yet recruiting
- The Royal Melbourne HospitalParkville, AustraliaNot yet recruiting
- Westmead HospitalWestmead, AustraliaTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Western Sydney Local Health District
- Trial code
- NCT05509153
- Record updated
- 28 February 2025
- Original description
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.Read full original descriptionShow less
A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
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.
Inclusion: 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; Expected to develop clinical HD within 10 years of trial enrolment using the Langbehn formula; Availability of an informant for corroborative history; Negative serum pregnancy test for women of childbearing potential; If of childbearing potential, is able and agrees to remain abstinent or use adequate contraceptive methods; Ability to tolerate MRI scans; Ability to tolerate blood draws; Able to comply with all study protocol requirements, according to the investigators judgement; In the opinion of the investigator, medically, psychiatrically and neurologically stable at the time of enrolment.
Exclusion: 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; Current use of antipsychotic medications or Tetrabenazine; History of attempted suicide or suicidal ideation within 12 months prior to screening; Known hypersensitivity to NAC; Current or history of substance abuse within one year of Baseline visit; Unstable psychiatric or acute medical illness including cancer, as determined by investigator; History of gene therapy, cell transplantation, or any experimental brain surgery; Abnormalities in laboratory measurements, ECG or vital signs at screening, which precludes safe participation in the study; Diagnosis of clinical HD; Use of supplemental NAC; Pre-existing structural brain lesion as assessed by a centrally read MRI scan during the screening period.