Overview
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
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