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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05509153




Registration number
NCT05509153
Ethics application status
Date submitted
8/08/2022
Date registered
19/08/2022
Date last updated
24/08/2022

Titles & IDs
Public title
A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
Scientific title
A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
Secondary ID [1] 0 0
2021/ETH12013
Universal Trial Number (UTN)
Trial acronym
NAC-preHD
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Huntington Disease 0 0
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - NAC
Treatment: Drugs - Placebo

Experimental: NAC - 1g N-Acetylcysteine capsules, taken orally twice a day.

Placebo Comparator: Placebo - Coated Placebo capsules, taken orally twice a day


Treatment: Drugs: NAC
1g of clinical grade N-Acetylcysteine capsules, taken orally twice a day

Treatment: Drugs: Placebo
Coated Placebo capsules, manufactured to match appearance and taste, taken orally twice a day
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Caudate Atrophy Rate on volumetric MRI
Timepoint [1] 0 0
Baseline through end of study (up to 3 years)
Primary outcome [2] 0 0
Rate of motor phenoconversion
Timepoint [2] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [1] 0 0
UHDRS motor subscale (total score)
Timepoint [1] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [2] 0 0
Stroop Word
Timepoint [2] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [3] 0 0
Trail Making Test
Timepoint [3] 0 0
Baseline through end study (up to 3 years)
Secondary outcome [4] 0 0
Montreal Cognitive Assessment
Timepoint [4] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [5] 0 0
Symbol Digit Modality Test
Timepoint [5] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [6] 0 0
Changes in Mood and Behavioural symptoms
Timepoint [6] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [7] 0 0
Changes in Daily Function
Timepoint [7] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [8] 0 0
Change to Quality of Life
Timepoint [8] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [9] 0 0
Study completion (Safety and Tolerability)
Timepoint [9] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [10] 0 0
Incidence of abnormal laboratory values and/or 12-lead ECG changes (Safety and Tolerability)
Timepoint [10] 0 0
Baseline through end of study (up to 3 years)
Secondary outcome [11] 0 0
Incidence of adverse and/or serious adverse events (Safety and Tolerability)
Timepoint [11] 0 0
Baseline through end of study (up to 3 years)

Eligibility
Key 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
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
1/11/2022
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/05/2027
Actual
Sample size
Target
160
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,VIC,WA
Recruitment hospital [1] 0 0
Westmead Hospital - Westmead
Recruitment hospital [2] 0 0
The University of Queensland - Herston
Recruitment hospital [3] 0 0
Calvary Health Care Bethlehem - Parkdale
Recruitment hospital [4] 0 0
The Royal Melbourne Hospital - Parkville
Recruitment hospital [5] 0 0
Perron Institute - Nedlands
Recruitment postcode(s) [1] 0 0
2145 - Westmead
Recruitment postcode(s) [2] 0 0
4029 - Herston
Recruitment postcode(s) [3] 0 0
3195 - Parkdale
Recruitment postcode(s) [4] 0 0
3050 - Parkville
Recruitment postcode(s) [5] 0 0
6009 - Nedlands

Funding & Sponsors
Primary sponsor type
Other
Name
Western Sydney Local Health District
Address
Country
Other collaborator category [1] 0 0
Other
Name [1] 0 0
Deakin University
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Other
Name [2] 0 0
Monash University
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
Royal Perth Hospital
Address [3] 0 0
Country [3] 0 0
Other collaborator category [4] 0 0
Other
Name [4] 0 0
The University of Queensland
Address [4] 0 0
Country [4] 0 0
Other collaborator category [5] 0 0
Other
Name [5] 0 0
University of Sydney
Address [5] 0 0
Country [5] 0 0
Other collaborator category [6] 0 0
Other
Name [6] 0 0
University of Melbourne
Address [6] 0 0
Country [6] 0 0

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/ct2/show/NCT05509153
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Clement Loy
Address 0 0
University of Sydney
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Clement Loy
Address 0 0
Country 0 0
Phone 0 0
001164 4 8890 3560
Fax 0 0
Email 0 0
clement.loy@sydney.edu.au
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05509153