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

Launched by WESTERN SYDNEY LOCAL HEALTH DISTRICT · Aug 18, 2022

Keywords

ClinConnect Summary

The NAC-preHD trial is a research study looking at whether a medication called N-Acetyl Cysteine (NAC) can help people who have a gene that increases their risk of developing Huntington's disease but do not yet show clear symptoms. This study is for individuals aged 65 to 74 who have a specific genetic marker (39 or more CAG repeats) and are expected to develop Huntington's within the next 10 years. To participate, you need to be able to give informed consent and have a support person who can help provide your medical history.

Participants in this study will take NAC or a placebo (a pill with no active ingredient) for three years. They will be required to undergo regular MRI scans and blood tests to monitor their health and the effects of the treatment. It's important to note that individuals who currently show symptoms of Huntington's disease, are pregnant, or have certain medical conditions may not be eligible to join. Overall, this trial aims to gather information about NAC's potential to delay or prevent the onset of Huntington's symptoms in those at risk.

Gender

ALL

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