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RECRUITINGPhase 2INTERVENTIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - Able to provide willing to sign a consent form - 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 Who Should NOT Join This Trial: - 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 Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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

Treatments Being Tested

DRUG

NAC

1g of clinical grade N-Acetylcysteine capsules, taken orally twice a day

DRUG

Placebo

Coated Placebo capsules, manufactured to match appearance and taste, taken orally twice a day

Locations (5)

Westmead Hospital
Westmead, New South Wales, Australia
The University of Queensland
Herston, Queensland, Australia
Calvary Health Care Bethlehem
Parkdale, Victoria, Australia
The Royal Melbourne Hospital
Parkville, Victoria, Australia
Perron Institute
Nedlands, Western Australia, Australia