A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
NCT ID: NCT05509153
Last Updated: 2025-02-28
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
160 participants
INTERVENTIONAL
2024-06-01
2027-05-01
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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NAC
1g N-Acetylcysteine capsules, taken orally twice a day.
NAC
1g of clinical grade N-Acetylcysteine capsules, taken orally twice a day
Placebo
Coated Placebo capsules, taken orally twice a day
Placebo
Coated Placebo capsules, manufactured to match appearance and taste, taken orally twice a day
Interventions
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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
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
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Western Sydney Local Health District
OTHER
Deakin University
OTHER
Monash University
OTHER
Royal Perth Hospital
OTHER
The University of Queensland
OTHER
University of Melbourne
OTHER
Responsible Party
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Clement Loy
Director and Neurologist, Huntington Disease Service
Principal Investigators
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Clement Loy
Role: PRINCIPAL_INVESTIGATOR
University of Sydney
Yenni Lie
Role: PRINCIPAL_INVESTIGATOR
Calvary Health Care Bethlehem
Dennis Velakoulis
Role: PRINCIPAL_INVESTIGATOR
Melbourne Health
Carolyn Orr
Role: PRINCIPAL_INVESTIGATOR
Perron Institute
John O'Sullivan
Role: PRINCIPAL_INVESTIGATOR
The University of Queensland
Rob Adam
Role: PRINCIPAL_INVESTIGATOR
The University of Queensland
Locations
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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
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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2021/ETH12013
Identifier Type: -
Identifier Source: org_study_id
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