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

• ClinicalTrials.gov Identifier: NCT00676195
• Recruitment Status: Completed
• First Posted: May 12, 2008
• Results First Posted: March 29, 2017
• Last Update Posted: March 29, 2017
• Sponsor: Stanford University
• Information provided by (Responsible Party): Antonio Hardan, Stanford University

Study Description

Brief Summary:

The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism.

Condition or disease	Intervention/treatment	Phase
Autistic Disorder	Drug: N-Acetyl Cysteine	Phase 2

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.

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 24 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: An Open-Label Study of N-Acetyl Cysteine in Autism
• Study Start Date: June 2008
• Actual Primary Completion Date: June 2010
• Actual Study Completion Date: June 2010

Arms and Interventions

Arm	Intervention/treatment
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

Outcome Measures

Primary Outcome Measures :

1. 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 :

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

Eligibility Criteria

• 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

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
• Other Study ID Numbers: SU-05062008-1139
• First Posted: May 12, 2008
• 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; Autism Spectrum 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