A Study of N-Acetyl Cysteine in Children With Autism

Phase 2

Completed

• Conditions: Autistic Disorder
• Interventions: Drug: N-Acetyl Cysteine; Other: Placebo - sugar pill

• Registration Number: NCT00627705
• Lead Sponsor: Stanford University

Brief Summary

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.

Detailed Description

Not available

Basic Information
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Recruitment & Eligibility
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Study & Design
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Trial Locations

Study Timeline

• Study Start: 2008-02
• Study First Submitted: 2008-02-22
• Study First Submitted QC: 2008-02-29
• Study First Posted: 2008-03-03
• Primary Completion: 2010-09
• Study Completion: 2010-09
• Results First Submitted: 2016-08-12
• Status Verified: 2017-04
• Results First Submitted QC: 2017-04-10
• Last Update Submitted: 2017-04-10
• Results First Posted: 2017-05-18
• Last Update Posted: 2017-05-18

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 43

Inclusion Criteria

1. Outpatients between 3.0 and 12.11 years of age inclusive
2. Males and females who are physically healthy
3. diagnosis of autism based Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation
4. Clinical Global Impression Severity rating of 4
5. Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis
6. Ability of subject to swallow the compound
7. Stable concomitant medications for at least 2 weeks
8. No planned changes in psychosocial interventions during the open-label N-Acetyl Cysteine trial

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Exclusion Criteria

1. DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified
2. Prior adequate trial of N-Acetyl Cysteine
3. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)
4. Pregnancy or sexually active females
5. Subjects taking antioxidant agents and glutathione prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
N-Acetyl Cysteine	N-Acetyl Cysteine	active compound N-Acetyl Cysteine
Sugar pill	Placebo - sugar pill	Placebo or sugar pill

Primary Outcome Measures

Name	Time	Method
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)	4, 8, and 12 weeks	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.
The Clinical Global Rating Scale (CGRS) Improvement Subscale Score	12 weeks	Score range 1-7 (lower score mean more improvement compared to baseline)
Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)	12 weeks	Data not collected. The laboratory was not able to measure Glutathione levels.
Irritability Subscale of the Aberrant Behavior Checklist (ABC)	baseline and 12 weeks	Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability

Secondary Outcome Measures

Name	Time	Method
The Aberrant Behavior Checklist Total Score (ABC)	4, 8, and 12 weeks	Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score.
Sensory Profile Questionnaire (SPQ)	12 weeks
Glutathione (GSH) Metabolism Intermediates in Peripheral Blood	12 weeks
Social Responsiveness Scale (SRS)	12 weeks	SRS total score (range 0-195); higher scores mean more social impairment

Trial Locations

Locations (1)

Stanford University School of Medicine; 🇺🇸 Stanford, California, United States