RecruitingPhase 2NCT05509153

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

Sponsor

Western Sydney Local Health District

Enrollment

160 participants

Start Date

Jun 1, 2024

Study Type

INTERVENTIONAL

Conditions

Huntington Disease

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

Min Age: 18 Years

Inclusion Criteria12

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 Criteria12

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