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An Open-Label Study of N-Acetyl Cysteine in Children With Autism

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00676195
First Posted: May 12, 2008
Last Update Posted: March 29, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Antonio Hardan, Stanford University
  Purpose
The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism.

Condition Intervention Phase
Autistic Disorder Drug: N-Acetyl Cysteine Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Study of N-Acetyl Cysteine in Autism

Resource links provided by NLM:


Further study details as provided by Antonio Hardan, Stanford University:

Primary Outcome Measures:
  • Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) [ Time Frame: 4, 8, and 12 weeks ]
    Number of Participants with adverse events reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) during the course of the study as measured at time points (4, 8, and 12 weeks).


Secondary Outcome Measures:
  • Social Responsiveness Scale (SRS) [ Time Frame: 12 weeks ]
  • Sensory Profile Questionnaire (SPQ) [ Time Frame: 12 weeks ]
  • Irritability Subscale of the Aberrant Behavior Checklist (ABC) [ Time Frame: 4, 8, and 12 weeks ]
  • Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography [ Time Frame: 12 weeks ]

Enrollment: 24
Study Start Date: June 2008
Study Completion Date: June 2010
Primary Completion Date: June 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: N-Acetyl Cysteine

Dosage of orally administered N-Acetyl Cysteine is as follows:

Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day

Drug: N-Acetyl Cysteine

Dosage of orally administered N-Acetyl Cysteine is as follows:

Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day

Other Name: NAC

Detailed Description:
N-Acetyl Cysteine (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 N-Acetyl Cysteine 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.
  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   3 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects will be eligible for this study if they participated in the Double-blind, randomized, placebo controlled study of N-Acetyl Cysteine in Autism study at Stanford University and meet all of the following 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 on 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

Exclusion Criteria:

  1. Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified
  2. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)
  3. Pregnancy or sexually active females
  4. 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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00676195


Locations
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Antonio Hardan, MD Stanford University
  More Information

Responsible Party: Antonio Hardan, Associate Professor, Stanford University
ClinicalTrials.gov Identifier: NCT00676195     History of Changes
Other Study ID Numbers: SU-05062008-1139
First Submitted: May 7, 2008
First Posted: May 12, 2008
Results First Submitted: August 12, 2016
Results First Posted: March 29, 2017
Last Update Posted: March 29, 2017
Last Verified: February 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

Keywords provided by Antonio Hardan, Stanford University:
N-Acetyl Cysteine

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Acetylcysteine
N-monoacetylcystine
Antiviral Agents
Anti-Infective Agents
Expectorants
Respiratory System Agents
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antidotes