N-Acetylcysteine study in children in the works for ASD.

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

This study is currently recruiting patients.
Verified by Indiana University School of Medicine July 2007Sponsors and Collaborators: Indiana University School of Medicine
National Alliance for Autism Research
Information provided by: Indiana University School of Medicine
ClinicalTrials.gov Identifier: NCT00453180



Purpose
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. Condition Intervention Phase
Autistic Disorder
Asperger Syndrome
Child Development Disorders, Pervasive Drug: N-acetylcysteine Phase II


MedlinePlus related topics: Asperger's Syndrome; Autism; Mental Health

Study Type: Interventional
Study Design: Treatment, Randomized, Double-Blind, Placebo Control, Parallel Assignment, Safety/Efficacy Study

Official Title: A Pilot Study of Oral N-Acetylcysteine in Children With Autism Spectrum Disorders
Further study details as provided by Indiana University School of Medicine:
Primary Outcome Measures:
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 Measures:
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

Total Enrollment: 32

Study start: March 2007

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
Ages Eligible for Study: 4 Years - 12 Years, Genders Eligible for Study: 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.

Location and Contact Information
Please refer to this study by ClinicalTrials.gov identifier NCT00453180

Jennifer J McCarthy, BS 317-274-1218 [email protected]
Jennifer M Wilkerson, BSW 317-274-1221 [email protected]

United States, Indiana
Riley Hospital for Children - Christian Sarkine Autism Treatment Center, Indianapolis, Indiana, 46202, United States; Recruiting
Jennifer M Wilkerson, BSW 317-274-1218 [email protected]
Jennifer J McCarthy, BS 317-274-1218 [email protected]

Study chairs or principal investigators

David J Posey, M.D., M.S., Principal Investigator, Indiana University School of Medicine

More Information
Study ID Numbers: 0611-10
Last Updated: July 11, 2007
Record first received: March 27, 2007
ClinicalTrials.gov Identifier: NCT00453180
Health Authority: United States: Institutional Review Board
ClinicalTrials.gov processed this record on July 25, 2007

What is wrong with using acetyl-L-carnitine instead? It is used for fixing some of the worst symptoms. Acetylcysteine has been used for the same things, but is not as popular. On Google, for memory, Acetylcysteine gets only about half as many hits.

Steve

1: J Autism Dev Disord. 2004 Dec;34(6):615-23.
Links
Relative carnitine deficiency in autism.
Filipek PA, Juranek J, Nguyen MT, Cummings C, Gargus JJ.

Department of Pediatrics, College of Medicine, University of California, Irvine, CA, USA. [email protected]

A random retrospective chart review was conducted to document serum carnitine levels on 100 children with autism. Concurrently drawn serum pyruvate, lactate, ammonia, and alanine levels were also available in many of these children. Values of free and total carnitine (p < 0.001), and pyruvate (p = 0.006) were significantly reduced while ammonia and alanine levels were considerably elevated (p < 0.001) in our autistic subjects. The relative carnitine deficiency in these patients, accompanied by slight elevations in lactate and significant elevations in alanine and ammonia levels, is suggestive of mild mitochondrial dysfunction. It is hypothesized that a mitochondrial defect may be the origin of the carnitine deficiency in these autistic children.

PMID: 15679182 [PubMed - indexed for MEDLINE]