Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12610000635066
Ethics application status
Approved
Date submitted
9/12/2009
Date registered
3/08/2010
Date last updated
31/08/2015
Type of registration
Prospectively registered

Titles & IDs
Public title
Efficacy Of N-Acetyl Cysteine In Autism: A Double-Blind, Placebo-Controlled Randomised Trial
Scientific title
Efficacy Of N-Acetyl Cysteine on Social Skills, Communication and Behaviour In Autistic Children: A Double-Blind, Placebo-Controlled Randomised Trial
Secondary ID [1] 1541 0
None
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Autism 252367 0
Condition category
Condition code
Mental Health 256549 256549 0 0
Autistic spectrum disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Oral N-acetyl cysteine (500 mg once a day) for a total of 24 weeks
Intervention code [1] 255670 0
Treatment: Drugs
Comparator / control treatment
Placebo - 500 mg sugar pill tablets once a day for a total of 24 weeks
Control group
Placebo

Outcomes
Primary outcome [1] 253443 0
Improvement in social skills (e.g. social awareness, capacity for reciprocal social communication) as measured by the Social Responsiveness Scale
Timepoint [1] 253443 0
Baseline, 1, 3, 6, 12 and 18 months.
Primary outcome [2] 258191 0
Improvement in verbal and non-verbal communication, as measured by the Children's Communication Checklist
Timepoint [2] 258191 0
Baseline, 1, 3, 6, 12 and 18 months.
Primary outcome [3] 258192 0
Improvement in repetitive and stereotypic behaviour as measured by the Repetitive Behavior Scale
Timepoint [3] 258192 0
Baseline, 1, 3, 6, 12 and 18 months.
Secondary outcome [1] 262573 0
Improvement in behavioural and emotional problems as measured by the Developmental Behaviour Checklist (Parent and Teacher Versions)
Timepoint [1] 262573 0
Baseline, 1, 3, 6, 12 and 18 months.
Secondary outcome [2] 262574 0
Improvement in everyday functioning, as measured by the Vineland Adaptive Behaviour Scales
Timepoint [2] 262574 0
Baseline, 6, 12 and 18 months.
Secondary outcome [3] 262576 0
Improvement in overall functioning, as assessed by the clinician using the Clinical Global Impression-Severity and Improvement (CGI-S and CGI-I)
Timepoint [3] 262576 0
Baseline, 1, 3, 6, 12 and 18 months.
Secondary outcome [4] 262579 0
Improvement in cognitive functioning as measured by the Wechsler Pre-School Scales of Intelligence or Wechsler Intelligence Scales for Children - 4th Edition or Psychoeducational Profile
Timepoint [4] 262579 0
Baseline, 6, 12 and 18 months.
Secondary outcome [5] 263835 0
Improvement in communication skills and social functioning, as measured by the Social Communication Questionnaire
Timepoint [5] 263835 0
Baseline, 1, 3, 6, 12 and 18 months.

Eligibility
Key inclusion criteria
Meet Diagnostic and Statistical Manual, Fourth Edition, Text Revision (DSM-IV-TR) criteria for Autistic Disorder. If on pharmacological therapies, participants must be on stable therapy for 2 weeks prior to commencing the trial
Minimum age
3 Years
Maximum age
10 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Individuals with a primary diagnosis of Asperger’s Disorder, or other Pervasive Developmental Disorders. Children with a known or suspected clinically relevant systemic medical disorder. Children who have had a prior sensitivity or allergy to N-acetyl cysteine (NAC) or gluten (corn starch). Inability to comply with the treatment protocol, for example fussy restricted diet that may lead to refusal to take NAC. Parental non-fluency in English. History of epilepsy. Clinically relevant biochemical or haematological abnormalities at baseline.

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)
Participants will be sequentially randomised to the trial. An independent researcher, who will not be directly involved with assessment of participants, will establish the randomisation code. Treatment and placebo will be randomly assigned and both participants and trial clinicians will be blinded.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
A block sequence will be generated by a computer program designed to generate clinical trial intervention logs.
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Date of first participant enrolment
Anticipated
1/06/2011
Actual
1/06/2011
Date of last participant enrolment
Anticipated
Actual
30/04/2013
Date of last data collection
Anticipated
Actual
Sample size
Target
80
Accrual to date
Final
98
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Funding source category [1] 256149 0
Charities/Societies/Foundations
Name [1] 256149 0
Australian Rotary Health Fund
Country [1] 256149 0
Australia
Funding source category [2] 291946 0
Charities/Societies/Foundations
Name [2] 291946 0
Simons Foundation Autism Research Initiative (SFARI)
Country [2] 291946 0
United States of America
Primary sponsor type
University
Name
University of Melbourne
Address
Department of Clinical and Biomedical Sciences: Barwon Health. P.O. Box 281, Geelong, VIC 3220
Country
Australia
Secondary sponsor category [1] 251492 0
None
Name [1] 251492 0
Address [1] 251492 0
Country [1] 251492 0
Other collaborator category [1] 993 0
University
Name [1] 993 0
Monash University
Address [1] 993 0
246 Clayton Road, Clayton, VIC 3168
Country [1] 993 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 258251 0
Barwon Health Research Ethics Committee
Ethics committee address [1] 258251 0
Barwon Health, P.O. Box 281, Geelong, VIC 3220
Ethics committee country [1] 258251 0
Australia
Date submitted for ethics approval [1] 258251 0
09/11/2009
Approval date [1] 258251 0
10/06/2010
Ethics approval number [1] 258251 0
1/09/0141

Summary
Brief summary
This study aims to investigate the antioxidant precursor, N-acetyl cysteine (NAC) as a treatment for children with autism. Recent evidence has shown children with autism to have imbalances in their antioxidant defences. The primary antioxidant in the brain, glutathione, has been shown to be decreased in these children. This study aims to increase the amount of available glutathione by administering NAC. The trial will be conducted in a total of 80 children, 40 of which will take the NAC treatment and the remaining 40 will take a matched placebo. The study will run over six months after which there will be a 12 and 18 month post-baseline follow up to determine the children’s symptoms following the trial. Standard rating scales will be used to assess the children at baseline, 1, 3, 6 and subsequently 12 and 18 months. Children will remain on any treatment regime they are currently taking part in. Treatment options are limited at present for children with autism and it is hoped that this trial will be efficacious and provide more options for these children.
Trial website
Trial related presentations / publications
Dean, O., Gray, K.M., Villagonzalo, K., Dodd, S., Mohebbi, M., Vick, T., Tonge, B.J., and Berk, M. (2015). N-acetylcysteine in children with autism: A randomised, double-blind, placebo controlled trial. Manuscript submitted to Lancet Psychiatry.
Public notes

Contacts
Principal investigator
Name 30597 0
Prof Michael Berk
Address 30597 0
Swanston Centre, Barwon Health, PO Box 281, Geelong, VIC 3220, Australia
Country 30597 0
Australia
Phone 30597 0
+61 3 4215 3330
Fax 30597 0
Email 30597 0
[email protected]
Contact person for public queries
Name 13844 0
Dr Olivia Dean
Address 13844 0
Swanston Centre, Barwon Health, P.O. Box 281, Geelong VIC 3220
Country 13844 0
Australia
Phone 13844 0
+61 3 52603088
Fax 13844 0
Email 13844 0
[email protected]
Contact person for scientific queries
Name 4772 0
Dr Olivia Dean
Address 4772 0
Swanston Centre, Barwon Health, P.O. Box 281, Geelong VIC 3220
Country 4772 0
Australia
Phone 4772 0
+ 61 3 4215 3300
Fax 4772 0
Email 4772 0
[email protected]

No information has been provided regarding IPD availability


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
SourceTitleYear of PublicationDOI
EmbaseA randomised, double blind, placebo-controlled trial of a fixed dose of N -acetyl cysteine in children with autistic disorder.2017https://dx.doi.org/10.1177/0004867416652735
N.B. These documents automatically identified may not have been verified by the study sponsor.