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Clinical Trials in Australia / NCT05509153
Recruiting Phase 2

A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers

NCT05509153 · tracked via the Priya Life Science Australia tracker
Phase
Phase 2
Started
2024-06-01
Last updated
2025-02-28

Condition(s) studied

Huntington Disease

Investigational drug(s) / intervention(s)

NAC →Placebo

NAC: 1g of clinical grade N-Acetylcysteine capsules, taken orally twice a day

Placebo: Coated Placebo capsules, manufactured to match appearance and taste, taken orally twice a day

Study summary

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * 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 Criteria: * Diagnosis of clinical HD * Known hypersensitivity to NAC * 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 * Use of supplemental NAC * Abnormalities in laboratory measurements, ECG or vital signs at screening, which precludes safe participation in the study * Current or history of substance abuse within one year of Baseline visit * Unstable psychiatric or acute medical illness including cancer, as determined by investigator * Current use of antipsychotic medications or Tetrabenazine * History of gene therapy, cell transplantation, or any experimental brain surgery * History of attempted suicide or suicidal ideation within 12 months prior to screening * Pre-existing structural brain lesion as assessed by a centrally read MRI scan during the screening period

Primary outcome measure(s)

Trial sites (5)

FacilityCityRegionStatus
Westmead Hospital Westmead New South Wales Recruiting
The University of Queensland Herston Queensland Not Yet Recruiting
Calvary Health Care Bethlehem Parkdale Victoria Not Yet Recruiting
The Royal Melbourne Hospital Parkville Victoria Not Yet Recruiting
Perron Institute Nedlands Western Australia Not Yet Recruiting

More Western Sydney Local Health District trials in Australia

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05509153 on ClinicalTrials.gov ↗ ← All trials in Australia