NAC: 1g of clinical grade N-Acetylcysteine capsules, taken orally twice a day
Placebo: Coated Placebo capsules, manufactured to match appearance and taste, taken orally twice a day
Study 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
Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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
Primary outcome measure(s)
Caudate Atrophy Rate on volumetric MRI — Baseline through end of study (up to 3 years) Blinded assessment
Rate of motor phenoconversion — Baseline through end of study (up to 3 years) Defined by conversion to Diagnostic Confidence Level 4, upon blinded assessment using the UHDRS motor subscale
Trial sites (5)
Facility
City
Region
Status
Westmead Hospital
Westmead
New South Wales
Recruiting
The University of Queensland
Herston
Queensland
Not Yet Recruiting
Calvary Health Care Bethlehem
Parkdale
Victoria
Not Yet Recruiting
The Royal Melbourne Hospital
Parkville
Victoria
Not Yet Recruiting
Perron Institute
Nedlands
Western Australia
Not Yet Recruiting
More Western Sydney Local Health District trials in Australia
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
We use cookies to analyse site traffic and improve your experience. With your consent, we may also use cookies for advertising. You can change your choice at any time.