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

Status: Recruiting
Location: See all (5) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
SUMMARY

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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Australia
The University of Queensland
NOT_YET_RECRUITING
Herston
Perron Institute
NOT_YET_RECRUITING
Nedlands
Calvary Health Care Bethlehem
NOT_YET_RECRUITING
Parkdale
The Royal Melbourne Hospital
NOT_YET_RECRUITING
Parkville
Westmead Hospital
RECRUITING
Westmead
Contact Information
Primary
Clement Loy
clement.loy@sydney.edu.au
001164 4 8890 3560
Backup
Sarah Samperi
sarah.samperi@health.nsw.gov.au
001164 2 8890 9146
Time Frame
Start Date: 2024-06-01
Estimated Completion Date: 2027-05-01
Participants
Target number of participants: 160
Treatments
Experimental: NAC
1g N-Acetylcysteine capsules, taken orally twice a day.
Placebo_comparator: Placebo
Coated Placebo capsules, taken orally twice a day
Sponsors
Collaborators: Monash University, The University of Queensland, Royal Perth Hospital, Deakin University, University of Melbourne
Leads: Western Sydney Local Health District

This content was sourced from clinicaltrials.gov