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

Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorWestern Sydney Local Health District

About this trial

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

Qualifiers

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

Disqualifiers

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

Trial design

Treatments tested in this trial

  • NAC
  • Placebo

Treatment groups

160 Participants
are divided into 2 treatment groups

Sponsors and collaborators

Western Sydney Local Health District

Lead sponsor

Deakin University

Collaborator

Monash University

Collaborator

Royal Perth Hospital

Collaborator

The University of Queensland

Collaborator

University of Melbourne

Collaborator