The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism. NAC is a compound that increases the levels of Glutathione, the body's main antioxidant. Glutathione is a compound in the blood that is part of a natural defense system (the antioxidant system). Anti-oxidants protect the body from damage caused by internal toxins called "free radicals." It is possible that children with Autism tend to have lower levels of glutathione, an important compound in our bodies that helps combat the effects of toxic free radicals. We hope that by studying the antioxidant system in more detail, we will increase our understanding of the reasons why people develop Autism so that we can design better ways to treat individuals with this condition. This study is meant to test the safety tolerability of NAC and its effectiveness in the treatment of behavioral difficulties in children with autism. It will also examine the possible benefit of this agent in improving the core deficits in autism such as social deficits.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
43
Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks Entire intervention lasts for 12 weeks (drug administration is continuous).
Phase 1: Oral, 900 mg daily for 4 weeks Phase 2: Oral, 900 mg twice daily 4 weeks Phase 3: Oral, 900 mg three times daily for 4 weeks Entire intervention lasts for 12 weeks (drug administration is continuous).
Stanford University School of Medicine
Stanford, California, United States
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
The Dosage Record and Treatment Emergent Symptom Scale (DOTES) provides information on the presence, frequency, and severity of side effects reported during the course of the trial.
Time frame: 4, 8, and 12 weeks
The Clinical Global Rating Scale (CGRS) Improvement Subscale Score
Score range 1-7 (lower score mean more improvement compared to baseline)
Time frame: 12 weeks
Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)
Data not collected. The laboratory was not able to measure Glutathione levels.
Time frame: 12 weeks
Irritability Subscale of the Aberrant Behavior Checklist (ABC)
Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability
Time frame: baseline and 12 weeks
The Aberrant Behavior Checklist Total Score (ABC)
Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score.
Time frame: 4, 8, and 12 weeks
Social Responsiveness Scale (SRS)
SRS total score (range 0-195); higher scores mean more social impairment
Time frame: 12 weeks
Sensory Profile Questionnaire (SPQ)
Time frame: 12 weeks
Glutathione (GSH) Metabolism Intermediates in Peripheral Blood
Time frame: 12 weeks
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