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Completed

NCT Number: NCT00627705

A Study of N-Acetyl Cysteine in Children With Autism

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.

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Key information

Conditions

Autistic Disorder Autism Spectrum Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

3 year–12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Stanford University School of Medicine

Stanford, California, 94305, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

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

Exclusion criteria

  • DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified
  • Prior adequate trial of N-Acetyl Cysteine
  • Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)
  • Pregnancy or sexually active females
  • 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

Treatment and study plan

N-acetyl cysteine

Drug

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).

Other names: NAC

Placebo - sugar pill

Other

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).

Other names: Placebo

Primary outcomes

  1. Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)

    Time frame: 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.

  2. The Clinical Global Rating Scale (CGRS) Improvement Subscale Score

    Time frame: 12 weeks

    Score range 1-7 (lower score mean more improvement compared to baseline)

  3. Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)

    Time frame: 12 weeks

    Data not collected. The laboratory was not able to measure Glutathione levels.

  4. Irritability Subscale of the Aberrant Behavior Checklist (ABC)

    Time frame: baseline and 12 weeks

    Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability

Secondary outcomes

  1. The Aberrant Behavior Checklist Total Score (ABC)

    Time frame: 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.

  2. Social Responsiveness Scale (SRS)

    Time frame: 12 weeks

    SRS total score (range 0-195); higher scores mean more social impairment

  3. Sensory Profile Questionnaire (SPQ)

    Time frame: 12 weeks

  4. Glutathione (GSH) Metabolism Intermediates in Peripheral Blood

    Time frame: 12 weeks

Sponsors and collaborators

Lead sponsor

Stanford University

Other

Registry information

Official study title

Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.

Important dates

Study start
2008
Primary completion
2010
Study completion
2010
First posted
Mar 3, 2008
Registry last updated
May 18, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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