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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Antonio Hardan, Stanford University
ClinicalTrials.gov Identifier:
NCT00676195
First received: May 7, 2008
Last updated: June 17, 2013
Last verified: June 2013
  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: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: 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 Stanford University:

Primary Outcome Measures:
  • Dosage Record and Treatment Emergent Symptom Scale [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Social Responsiveness Scale (SRS) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Sensory Profile Questionnaire (SPQ) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Irritability subscale of the ABC [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: No ]
  • GSH metabolism intermediates in peripheral blood measured by HPLC [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

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 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


Detailed Description:

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.

  Eligibility

Ages Eligible for Study:   3 Years to 12 Years
Genders Eligible for Study:   Both
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 DSM-IV- TR criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation
  4. CGI 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 NAC trial

Exclusion Criteria:

  1. DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder NOS
  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 GSH prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: Antonio Hardan, Associate Professor, Stanford University
ClinicalTrials.gov Identifier: NCT00676195     History of Changes
Other Study ID Numbers: SU-05062008-1139
Study First Received: May 7, 2008
Last Updated: June 17, 2013
Health Authority: United States: Food and Drug Administration

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

ClinicalTrials.gov processed this record on July 29, 2014