Clinical trial NCT00453180: A Study of Oral N-Acetylcysteine in Children With Autism Spectrum Disorders
Official title: A Pilot Study of Oral N-Acetylcysteine in Children With Autism Spectrum Disorders
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autistic Disorder
Asperger Syndrome
Child Development Disorders, Pervasive
Keywords: Autistic Disorder
Asperger's
PDD NOS
Pervasive Developmental Disorder
Autism
N-acetylcysteine
acetylcysteine
NAC
antioxidant
treatment
core symptoms
Autism Spectrum Disorders
Asperger's Disorder
Pervasive Developmental Disorder Not Otherwise Specified
Pervasive Developmental Disorders
Lead sponsor: Indiana University
Sponsor class: OTHER
Collaborators: name: National Alliance for Autism Research
First posted: 2007-03-28
Enrollment: 31
Sex: ALL
Minimum age (years): 4
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine whether treatment with oral N-acetylcysteine (NAC) will improve behavior problems often associated with autism spectrum disorders.

Detailed description: Autism is increasingly being recognized as a common disorder with enormous public health significance. The core symptoms of autism include severe deficits in social relatedness and communication, and interfering repetitive behavior. No medications have been shown to consistently improve any of these symptoms.

The central hypothesis of this study is that NAC will improve behavioral manifestations of autism which may include core or associated symptoms. We plan to test our hypothesis and complete the objectives of this project by pursuing the following specific aims:

* Evaluate the efficacy of oral NAC in a 12-week, double-blind, placebo-controlled study involving 32 children and adolescents with autism spectrum disorders.
* Evaluate the safety and tolerability of oral NAC in 32 children and adolescents with autism spectrum disorders.

Eligibility criteria: Inclusion Criteria:

* Age 4 to 12 years.
* Diagnosis of autistic disorder, Asperger's disorder, or PDD NOS.
* If taking concomitant psychotropic medications, the medication must be at a constant dose for 60 days with no dose changes planned for the duration of the trial.
* Able to swallow capsules.

Exclusion Criteria:

* Presence of any medical condition that significantly increases risk or hampers assessment (e.g., unstable hypertension or cardiac disease, unstable asthma, kidney disease, unstable seizure disorder, pregnancy or any other medical condition as determined by the investigator).
* Weight \< 15 kg.
* Subjects taking concomitant medications or supplements known for their glutamatergic effects (e.g., dextromethorphan, D-cycloserine, amantadine, memantine, lamotrigine, riluzole) or antioxidant properties (high dose vitamin supplements, DMG, TMG, many alternative treatments) within 30 days of the baseline visit with the exception of short term use of dextromethorphan as needed as a cough suppressant. The use of this medicine must be stopped at least 7 days prior to the baseline visit. Regular multivitamins will be allowed.
* Subjects taking daily acetaminophen or nonsteroidal anti-inflammatory drugs within 30 days of the baseline visit.
* Profound mental retardation as evidenced by a mental age below 18 months.
* Subjects taking concomitant medications with the potential for pharmacokinetic or pharmacodynamic drug-drug interactions (e.g., carbamazepine) within 30 days of the baseline visit.
* Subjects who are likely to experience significant changes in their ongoing psychosocial or medical treatments for autism over the course of the trial (e.g., initiation of new behavioral therapy, initiation of new medication or alternative treatment \[e.g., chelation\]). Minor changes in ongoing treatment (e.g., missed therapy sessions due to holiday/vacation; planned break in therapy due to school holidays) will not be considered significant.
* History of prior treatment with NAC.
* Evidence of hypersensitivity/allergy to NAC.
* Presence of certain neurodevelopmental disorders such as Fragile X Syndrome, Tuberous Sclerosis, or other neurological disorders known to be associated with autism or autistic features.
* Diagnosis of Rett's disorder, childhood disintegrative disorder, schizophrenia, bipolar disorder, another psychotic disorder, or substance abuse disorder.

Primary outcomes:
measure: Clinical Global Impression - Severity
timeFrame: Week 12
measure: Clinical Global Impression - Improvement
timeFrame: Week 12
Secondary outcomes:
measure: Aberrant Behavior Checklist
timeFrame: Week 12
measure: Social Responsiveness Scale
timeFrame: Week 12
measure: Pervasive Developmental Disorder Behavior Index
timeFrame: Week 12
measure: Vineland Adaptive Behavior Scales-II (VABS-II)
timeFrame: Week 12
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Riley Hospital, Riley Child and Adolescent Psychiatry Clinic
state: Indiana
Study officials:
affiliation: Indiana University School of Medicine
name: Martin H. Plawecki, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: Wink LK, Adams R, Wang Z, Klaunig JE, Plawecki MH, Posey DJ, McDougle CJ, Erickson CA. A randomized placebo-controlled pilot study of N-acetylcysteine in youth with autism spectrum disorder. Mol Autism. 2016 Apr 21;7:26. doi: 10.1186/s13229-016-0088-6. eCollection 2016.