A Study of Oral N-Acetylcysteine in Children With Autism Spectrum Disorders
Condition: Autistic Disorder; Asperger Syndrome; Child Development Disorders, Pervasive
MeSH terms: Autistic Disorder; Autism Spectrum Disorder; Child Development Disorders, Pervasive; Asperger Syndrome; Developmental Disabilities
Intervention: N-acetylcysteine; Placebo
Intervention description: Capsules available in 300 mg or 600mg strength. Target dose of n-acetylcysteine will be 60mg/kg/day TID. Dosage will be increased to this target dose from week 1 to week 3 barring side effects. Dose reduction will be allowed at any time for adverse side effects. Maximum dose of n-acetylcysteine will be 4200mg/day.; Subjects randomized to placebo arm will receive placebo pill for duration of study.
Intervention MeSH terms: Acetylcysteine; N-monoacetylcystine
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Enrollment: 31.0
Sex: All
Minimum age: 4 Years
Maximum age: 12 Years
Healthy volunteers: No
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

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.
    

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.