Testing N-Acetyl Cysteine for people at risk of Huntington's Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase II randomized controlled trial investigates the effects of oral N-Acetyl Cysteine (NAC) on premanifest carriers of the Huntington gene. Participants will be monitored for clinical and radiological outcomes over a three-year period. The study aims to determine whether NAC can delay the onset of Huntington's Disease symptoms in individuals who are genetically predisposed but currently asymptomatic. The trial includes a placebo group for comparison to assess the efficacy of NAC.

Why it matters

Who can join: the full rules

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

Where it's running

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

Study contacts

• Principal investigator: Clement Loy — University of Sydney
• Study coordinator: Clement Loy
• Email: clement.loy@sydney.edu.au
• Phone: 001164 4 8890 3560

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Pridopidine for Huntington's Disease: a 1-year, randomized, placebo-controlled study

  This trial tests whether pridopidine can slow the progression of Huntington's disease in adults compared with placebo over one year.

  Recruiting at 17 sites Phase 3 Prilenia · Birmingham, Alabama and 65 other locations · NCT07609108

• Low-intensity focused ultrasound targeting the globus pallidus to improve movement in Huntington's disease

  This trial tests whether different pulse frequencies of low-intensity focused ultrasound (LIFU) aimed at a deep brain target can safely reduce chorea and other motor symptoms in adults with Huntington's disease.

  Recruiting First Affiliated Hospital of Fujian Medical University · Fuzhou, Fujian · NCT07513844

• Safety and tolerability of human neural stem cell implants for Huntington's disease

  This research will test whether an implant of hNSC-01 human neural stem cells is safe and tolerable for adults with Huntington's disease.

  Recruiting Phase 1/2 University of California, Irvine · Irvine, California · NCT07451613

• SKY-0515 for adults with Huntington's disease

  This trial will test whether the oral drug SKY-0515 can lower harmful huntingtin protein and improve symptoms in adults aged 25 and older with genetically confirmed Huntington's disease.

  Recruiting at 19 sites Phase 2/3 Skyhawk Therapeutics, Inc. · Englewood, Colorado and 30 other locations · NCT07378644

• Votoplam for adults with early symptomatic Huntington's disease

  This trial will test whether votoplam (HTT227) is safe and can slow disease progression in adults with early symptomatic Huntington's disease.

  Recruiting at 117 sites Phase 3 Novartis · Birmingham, Alabama and 116 other locations · NCT07326709

• Comparing three social cognition tests in adults with early Huntington disease

  This project will test whether the DMASC-MC and two established social cognition tests can detect social thinking problems in adults with early Huntington disease.

  Recruiting Region Skane · Lund · NCT07253038

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.