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

Primary Purpose

Autistic Disorder

Status

Completed

Phase

Phase 2

Locations

United States

Study Type

Interventional

Intervention

N-Acetyl Cysteine

About this trial

This is an interventional treatment trial for Autistic Disorder focused on measuring N-Acetyl Cysteine

Eligibility Criteria

3 Years - 12 Years (Child)All SexesDoes not accept healthy volunteers

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 on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation
Clinical Global Impression 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 N-Acetyl Cysteine trial

Exclusion Criteria:

Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified
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 glutathione prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks

Sites / Locations

Stanford University School of Medicine

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

N-Acetyl Cysteine

Arm Description

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

Outcomes

Primary Outcome Measures

Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES)

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)

Sensory Profile Questionnaire (SPQ)

Irritability Subscale of the Aberrant Behavior Checklist (ABC)

Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography

Full Information

NCT ID

NCT00676195

First Posted

May 7, 2008

Last Updated

February 8, 2017

Sponsor

Stanford University

1. Study Identification

Unique Protocol Identification Number

NCT00676195

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

February 2017

Overall Recruitment Status

Completed

Study Start Date

June 2008 (undefined)

Primary Completion Date

June 2010 (Actual)

Study Completion Date

June 2010 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Stanford University

4. Oversight

Data Monitoring Committee

Yes

5. 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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Autistic Disorder

Keywords

N-Acetyl Cysteine

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

24 (Actual)

8. Arms, Groups, and Interventions

Arm Title

N-Acetyl Cysteine

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

N-Acetyl Cysteine

Other Intervention Name(s)

NAC

Intervention Description

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

Primary Outcome Measure Information:

Title

Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES)

Description

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).

Time Frame

4, 8, and 12 weeks

Secondary Outcome Measure Information:

Title

Social Responsiveness Scale (SRS)

Time Frame

12 weeks

Title

Sensory Profile Questionnaire (SPQ)

Time Frame

12 weeks

Title

Irritability Subscale of the Aberrant Behavior Checklist (ABC)

Time Frame

4, 8, and 12 weeks

Title

Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography

Time Frame

12 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

3 Years

Maximum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility 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 on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation Clinical Global Impression 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 N-Acetyl Cysteine trial Exclusion Criteria: Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified 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 glutathione prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Antonio Hardan, MD

Organizational Affiliation

Stanford University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Stanford University School of Medicine

City

Stanford

State/Province

California

ZIP/Postal Code

94305

Country

United States

12. IPD Sharing Statement

Plan to Share IPD

Yes

Learn more about this trial

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

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