NCT05509153 A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
| NCT ID | NCT05509153 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Western Sydney Local Health District |
| Condition | Huntington Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 160 participants |
| Start Date | 2024-06-01 |
| Primary Completion | 2026-11-01 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 160 participants in total. It began in 2024-06-01 with a primary completion date of 2026-11-01.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
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
Contact & Investigator
Clement Loy
PRINCIPAL INVESTIGATOR
University of Sydney
Frequently Asked Questions
Who can join the NCT05509153 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Huntington Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05509153 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT05509153 currently recruiting?
Yes, NCT05509153 is actively recruiting participants. Contact the research team at clement.loy@sydney.edu.au for enrollment information.
Where is the NCT05509153 trial being conducted?
This trial is being conducted at Westmead, Australia, Herston, Australia, Parkdale, Australia, Parkville, Australia and 1 additional location.
Who is sponsoring the NCT05509153 clinical trial?
NCT05509153 is sponsored by Western Sydney Local Health District. The principal investigator is Clement Loy at University of Sydney. The trial plans to enroll 160 participants.