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

Sponsored by:	Stanford University
Information provided by:	Stanford University
ClinicalTrials.gov Identifier:	NCT00676195

Purpose

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.

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

MedlinePlus related topics: Antioxidants Autism

Drug Information available for: Acetylcysteine Cysteine Cysteine hydrochloride

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety/Efficacy Study
Official Title:	An Open-Label Study of N-Acetyl Cysteine in Autism

Further study details as provided by Stanford University:

Primary Outcome Measures:

Dosage Record and Treatment Emergent Symptom Scale (DOTES)
The Clinical Global Rating Scale (CGRS) Improvement subscale
GSH levels in peripheral blood, measured by state-of-the-art high-performance liquid chromatography (HPLC)
The Aberrant Behavior Checklist total score (ABC)

Secondary Outcome Measures:

Social Responsiveness Scale (SRS)
Sensory Profile Questionnaire (SPQ)
The Irritability subscale of the ABC
Yale-Brown Obsessive Compulsive scale (Y-BOCS)
GSH metabolism intermediates in peripheral blood measured by HPLC

Estimated Enrollment:	40
Study Start Date:	June 2008

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:

Outpatients between 3.0 and 12.11 years of age inclusive
Males and females who are physically healthy
diagnosis of autism based DSM-IV- TR criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation
CGI 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 NAC trial Exclusion Criteria:
- a. DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder NOS
- b. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)
- c. Pregnancy or sexually active females
- d. 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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00676195

Contacts

Contact: Robin Libove

650-736-1235

rlibove@stanford.edu

Locations

United States, California
Stanford University School of Medicine	Recruiting
Stanford, California, United States, 94305
Contact: Robin Libove 650-736-1235 rlibove@stanford.edu
Principal Investigator: Antonio Hardan
Sub-Investigator: Rabin Tirouvanziam PhD

Sponsors and Collaborators

Stanford University

Investigators

Principal Investigator:

Antonio Hardan

Stanford University

More Information

Study ID Numbers:	SU-05062008-1139, SU eProtocol: 12181
Study First Received:	May 7, 2008
Last Updated:	May 9, 2008
ClinicalTrials.gov Identifier:	NCT00676195
Health Authority:	United States: Food and Drug Administration

Study placed in the following topic categories:

Developmental Disabilities
Child Development Disorders, Pervasive
Mental Disorders
Autistic Disorder

Mental Disorders Diagnosed in Childhood
Acetylcysteine
N-monoacetylcystine

Additional relevant MeSH terms:

Anti-Infective Agents
Respiratory System Agents
Antioxidants
Molecular Mechanisms of Pharmacological Action
Therapeutic Uses
Expectorants

Physiological Effects of Drugs
Free Radical Scavengers
Protective Agents
Antiviral Agents
Pharmacologic Actions
Antidotes

ClinicalTrials.gov processed this record on January 14, 2009