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New treatment option for Huntington Disease
Official title A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
ClinicalTrials.gov ID: NCT05509153
What this study is testing
What is NAC?
NAC is an investigational medicine, given as a twice-daily pill taken by mouth, being studied as a potential treatment for huntington disease.
Also referred to as N-Acetylcysteine.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
- You might receive a placebo (an inactive treatment) instead of the study drug, decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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
You likely can't join if
- 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
See the full eligibility 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
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Westmead, New South Wales, Australia
- Herston, Queensland, Australia
- Parkdale, Victoria, Australia
- Parkville, Victoria, Australia
- Nedlands, Western Australia, Australia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Westmead, New South Wales, Australia; Herston, Queensland, Australia; Parkdale, Victoria, Australia; Parkville, Victoria, Australia; Nedlands, Western Australia, Australia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.