A Study of N-Acetyl Cysteine in Children With Autism

April 10, 2017 updated by: Antonio Hardan, Stanford University

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

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

43

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

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

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

3 years to 12 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion 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 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. DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified
  2. Prior adequate trial of N-Acetyl Cysteine
  3. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)
  4. Pregnancy or sexually active females
  5. 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
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).

Other Names:
  • NAC
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).

Other Names:
  • Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Time Frame: 4, 8, and 12 weeks
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.
4, 8, and 12 weeks
The Clinical Global Rating Scale (CGRS) Improvement Subscale Score
Time Frame: 12 weeks
Score range 1-7 (lower score mean more improvement compared to baseline)
12 weeks
Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)
Time Frame: 12 weeks
Data not collected. The laboratory was not able to measure Glutathione levels.
12 weeks
Irritability Subscale of the Aberrant Behavior Checklist (ABC)
Time Frame: baseline and 12 weeks
Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability
baseline and 12 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The Aberrant Behavior Checklist Total Score (ABC)
Time Frame: 4, 8, and 12 weeks
Total score was not analyzed since we analyzed the sub scales. Additionally, the authors of the instrument do not recommend analyzing the total score.
4, 8, and 12 weeks
Social Responsiveness Scale (SRS)
Time Frame: 12 weeks
SRS total score (range 0-195); higher scores mean more social impairment
12 weeks
Sensory Profile Questionnaire (SPQ)
Time Frame: 12 weeks
12 weeks
Glutathione (GSH) Metabolism Intermediates in Peripheral Blood
Time Frame: 12 weeks
12 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Stanford University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

February 1, 2008

Primary Completion (Actual)

September 1, 2010

Study Completion (Actual)

September 1, 2010

Study Registration Dates

First Submitted

February 22, 2008

First Submitted That Met QC Criteria

February 29, 2008

First Posted (Estimate)

March 3, 2008

Study Record Updates

Last Update Posted (Actual)

May 18, 2017

Last Update Submitted That Met QC Criteria

April 10, 2017

Last Verified

April 1, 2017

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SU-02012008-995
  • 10142 (DAIDS ES)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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