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A 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

Placebo - sugar pill

About this trial

This is an interventional treatment trial for Autistic Disorder

Eligibility Criteria

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

Inclusion Criteria:

Outpatients between 3.0 and 12.11 years of age inclusive
Males and females who are physically healthy
diagnosis of autism based 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:

DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified
Prior adequate trial of N-Acetyl Cysteine
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 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

N-Acetyl Cysteine

Sugar pill

Arm Description

active compound N-Acetyl Cysteine

Placebo or sugar pill

Outcomes

Primary Outcome Measures

Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)

The Dosage Record and Treatment Emergent Symptom Scale (DOTES) provides information on the presence, frequency, and severity of side effects reported during the course of the trial.

The Clinical Global Rating Scale (CGRS) Improvement Subscale Score

Score range 1-7 (lower score mean more improvement compared to baseline)

Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)

Data not collected. The laboratory was not able to measure Glutathione levels.

Irritability Subscale of the Aberrant Behavior Checklist (ABC)

Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability

Secondary Outcome Measures

The Aberrant Behavior Checklist Total Score (ABC)

Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score.

Social Responsiveness Scale (SRS)

SRS total score (range 0-195); higher scores mean more social impairment

Sensory Profile Questionnaire (SPQ)

Glutathione (GSH) Metabolism Intermediates in Peripheral Blood

Full Information

NCT ID

NCT00627705

First Posted

February 22, 2008

Last Updated

April 10, 2017

Sponsor

Stanford University

1. Study Identification

Unique Protocol Identification Number

NCT00627705

Brief Title

A Study of N-Acetyl Cysteine in Children With Autism

Official Title

Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.

Study Type

Interventional

2. Study Status

Record Verification Date

April 2017

Overall Recruitment Status

Completed

Study Start Date

February 2008 (undefined)

Primary Completion Date

September 2010 (Actual)

Study Completion Date

September 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. 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.

6. Conditions and Keywords

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

Autistic Disorder

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

43 (Actual)

8. Arms, Groups, and Interventions

Arm Title

N-Acetyl Cysteine

Arm Type

Active Comparator

Arm Description

active compound N-Acetyl Cysteine

Arm Title

Sugar pill

Arm Type

Placebo Comparator

Arm Description

Placebo or sugar pill

Intervention Type

Drug

Intervention Name(s)

N-Acetyl Cysteine

Other Intervention Name(s)

NAC

Intervention Description

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).

Intervention Type

Other

Intervention Name(s)

Placebo - sugar pill

Other Intervention Name(s)

Placebo

Intervention Description

Primary Outcome Measure Information:

Title

Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)

Description

The Dosage Record and Treatment Emergent Symptom Scale (DOTES) provides information on the presence, frequency, and severity of side effects reported during the course of the trial.

Time Frame

4, 8, and 12 weeks

Title

The Clinical Global Rating Scale (CGRS) Improvement Subscale Score

Description

Score range 1-7 (lower score mean more improvement compared to baseline)

Time Frame

12 weeks

Title

Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)

Description

Data not collected. The laboratory was not able to measure Glutathione levels.

Time Frame

12 weeks

Title

Irritability Subscale of the Aberrant Behavior Checklist (ABC)

Description

Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability

Time Frame

baseline and 12 weeks

Secondary Outcome Measure Information:

Title

The Aberrant Behavior Checklist Total Score (ABC)

Description

Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score.

Time Frame

4, 8, and 12 weeks

Title

Social Responsiveness Scale (SRS)

Description

SRS total score (range 0-195); higher scores mean more social impairment

Time Frame

12 weeks

Title

Sensory Profile Questionnaire (SPQ)

Time Frame

12 weeks

Title

Glutathione (GSH) Metabolism Intermediates in Peripheral Blood

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: Outpatients between 3.0 and 12.11 years of age inclusive Males and females who are physically healthy diagnosis of autism based 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: DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified Prior adequate trial of N-Acetyl Cysteine 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

Citations:

PubMed Identifier

22342106

Citation

Hardan AY, Fung LK, Libove RA, Obukhanych TV, Nair S, Herzenberg LA, Frazier TW, Tirouvanziam R. A randomized controlled pilot trial of oral N-acetylcysteine in children with autism. Biol Psychiatry. 2012 Jun 1;71(11):956-61. doi: 10.1016/j.biopsych.2012.01.014. Epub 2012 Feb 18.

Results Reference

result

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A Study of N-Acetyl Cysteine in Children With Autism

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