A Study of N-Acetyl Cysteine in Children With Autism - Full Text View

Sponsor:	Stanford University
Information provided by:	Stanford University
ClinicalTrials.gov Identifier:	NCT00627705

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 Other: Placebo - sugar pill	Phase II

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.

Resource links provided by NLM:

MedlinePlus related topics: Antioxidants Autism

Drug Information available for: Cysteine Acetylcysteine Cysteine hydrochloride

U.S. FDA Resources

Further study details as provided by Stanford University:

Primary Outcome Measures:

The Aberrant Behavior Checklist total score (ABC) [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: No ]
Dosage Record and Treatment Emergent Symptom Scale (DOTES) [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: Yes ]
: The Clinical Global Rating Scale (CGRS) Improvement subscale [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: Yes ]
GSH levels in peripheral blood, measured by state-of-the-art high-performance liquid chromatography (HPLC) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

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 ]

Estimated Enrollment:	40
Study Start Date:	February 2008
Study Completion Date:	September 2010
Primary Completion Date:	September 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Active Comparator: N-Acetyl Cysteine active compound N-Acetyl Cysteine	Drug: N-Acetyl Cysteine 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).
Placebo Comparator: Sugar pill Placebo or sugar pill	Other: Placebo - sugar pill 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).

Eligibility

Ages Eligible for Study:	3 Years to 12 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion 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:

DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder NOS
Prior adequate trial of NAC
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 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: NCT00627705

Locations

United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305

Sponsors and Collaborators

Stanford University

Investigators

Principal Investigator:	Antonio Hardan	Stanford University
Sub-Investigator:	Rabin Tirouvanziam PhD	Stanford University

More Information

No publications provided

Responsible Party:	Antonio Hardan, Stanford University School of Medicine
ClinicalTrials.gov Identifier:	NCT00627705 History of Changes
Other Study ID Numbers:	SU-02012008-995, 10142
Study First Received:	February 22, 2008
Last Updated:	July 20, 2011
Health Authority:	United States: Food and Drug Administration; United States: Institutional Review Board

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 February 09, 2012