A Study of Oral N-Acetylcysteine in Children With Autism Spectrum Disorders

Condition(s) targeted: Autistic Disorder; Asperger Syndrome; Child Development Disorders, Pervasive

Intervention: N-acetylcysteine (Drug); Placebo (Drug)

Phase: Phase 2

Status: Recruiting

Sponsored by: Indiana University School of Medicine

Official(s) and/or principal investigator(s):
David J Posey, M.D., M.S., Principal Investigator, Affiliation: Indiana University School of Medicine

Overall contact:
Jennifer Mullett, RN, CCRP, Phone: 317-274-1981, Email: mullettj@iupui.edu

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.

Official title: A Pilot Study of Oral N-Acetylcysteine in Children With Autism Spectrum Disorders

Study design: Treatment, Randomized, Double Blind (Subject, Caregiver, Investigator), Placebo Control, Parallel Assignment, Safety/Efficacy Study

Primary outcome:

Clinical Global Impression - Severity done at Screen, Baseline, and at the end of week 12 of treatment

Clinical Global Impression - Improvement done at the end of weeks 4, 8, and 12 of treatment

Secondary outcome:

Aberrant Behavior Checklist done at Baseline and end of weeks 4, 8, and 12

Social Responsiveness Scale done at Baseline and end of weeks 4, 8, and 12

Pervasive Developmental Disorder Behavior Index done at Baseline and end of week 12

Vineland Adaptive Behavior Scales done at Baseline and end of week 12

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.

Minimum age: 4 Years. Maximum age: 12 Years. Gender(s): Both.

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.

Jennifer Mullett, RN, CCRP, Phone: 317-274-1981, Email: mullettj@iupui.edu

Riley Hospital for Children - Christian Sarkine Autism Treatment Center, Indianapolis, Indiana 46202, United States; Recruiting
Clinic email, Email: kidpsych@iupui.edu

Starting date: March 2007
Ending date: July 2009
Last updated: July 31, 2009