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

Recruiting now · Phase 2 · Has a placebo group

Conditions studied: Huntington Disease

In brief

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.

Key facts

Study ID

NCT05509153

Run by

Western Sydney Local Health District

People needed

160

Starts

2024-06-01

Expected to finish

2027-05-01

Last updated by the study team

2025-02-28

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Westmead Hospital — Westmead, New South Wales, Australia (enrolling)
• The University of Queensland — Herston, Queensland, Australia
• Calvary Health Care Bethlehem — Parkdale, Victoria, Australia
• The Royal Melbourne Hospital — Parkville, Victoria, Australia
• Perron Institute — Nedlands, Western Australia, Australia

Full record on ClinicalTrials.gov

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