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Recruiting NCT05509153

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

Phase II Interventional Huntington Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: NAC, Placebo.
Who it may be relevant to
Registry conditions: Huntington Disease. Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Australia
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

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.

Interventions

  • 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

Primary outcome measures

  • Caudate Atrophy Rate on volumetric MRI [Time frame: Baseline through end of study (up to 3 years)]
  • Rate of motor phenoconversion [Time frame: Baseline through end of study (up to 3 years)]
Secondary outcome measures (11)
  • UHDRS motor subscale (total score) [Time frame: Baseline through end of study (up to 3 years)]
  • Stroop Word [Time frame: Baseline through end of study (up to 3 years)]
  • Trail Making Test [Time frame: Baseline through end study (up to 3 years)]
  • Montreal Cognitive Assessment [Time frame: Baseline through end of study (up to 3 years)]
  • Symbol Digit Modality Test [Time frame: Baseline through end of study (up to 3 years)]
  • Changes in Mood and Behavioural symptoms [Time frame: Baseline through end of study (up to 3 years)]
  • Changes in Daily Function [Time frame: Baseline through end of study (up to 3 years)]
  • Change to Quality of Life [Time frame: Baseline through end of study (up to 3 years)]
  • Study completion (Safety and Tolerability) [Time frame: Baseline through end of study (up to 3 years)]
  • Incidence of abnormal laboratory values and/or 12-lead ECG changes (Safety and Tolerability) [Time frame: Baseline through end of study (up to 3 years)]
  • Incidence of adverse and/or serious adverse events (Safety and Tolerability) [Time frame: Baseline through end of study (up to 3 years)]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

Australia · 5 centers
  • Westmead Hospital — Westmead
  • The University of Queensland — Herston
  • Calvary Health Care Bethlehem — Parkdale
  • The Royal Melbourne Hospital — Parkville
  • Perron Institute — Nedlands

Identifiers

NCT: NCT05509153 · 2021/ETH12013

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗