Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study of N-Acetyl Cysteine in Children With Autism

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00627705
Recruitment Status : Completed
First Posted : March 3, 2008
Results First Posted : May 18, 2017
Last Update Posted : May 18, 2017
Sponsor:
Information provided by (Responsible Party):
Antonio Hardan, Stanford University

Tracking Information
First Submitted Date  ICMJE February 22, 2008
First Posted Date  ICMJE March 3, 2008
Results First Submitted Date  ICMJE August 12, 2016
Results First Posted Date  ICMJE May 18, 2017
Last Update Posted Date May 18, 2017
Study Start Date  ICMJE February 2008
Actual Primary Completion Date September 2010   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 10, 2017)
  • 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.
  • 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)
  • 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.
  • 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
Original Primary Outcome Measures  ICMJE
 (submitted: February 29, 2008)
  • The Aberrant Behavior Checklist total score (ABC)
  • Dosage Record and Treatment Emergent Symptom Scale (DOTES)
  • The Clinical Global Rating Scale (CGRS) Improvement subscale
  • GSH levels in peripheral blood, measured by state-of-the-art high-performance liquid chromatography (HPLC)
Change History Complete list of historical versions of study NCT00627705 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: April 10, 2017)
  • 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.
  • Social Responsiveness Scale (SRS) [ Time Frame: 12 weeks ]
    SRS total score (range 0-195); higher scores mean more social impairment
  • Sensory Profile Questionnaire (SPQ) [ Time Frame: 12 weeks ]
  • Glutathione (GSH) Metabolism Intermediates in Peripheral Blood [ Time Frame: 12 weeks ]
Original Secondary Outcome Measures  ICMJE
 (submitted: February 29, 2008)
  • Social Responsiveness Scale (SRS)
  • Sensory Profile Questionnaire (SPQ)
  • the Irritability subscale of the ABC
  • Yale-Brown Obsessive Compulsive scale (Y-BOCS)
  • GSH metabolism intermediates in peripheral blood measured by HPLC
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of N-Acetyl Cysteine in Children With Autism
Official Title  ICMJE Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.
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.

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Autistic Disorder
Intervention  ICMJE
  • 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 Name: NAC
  • 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 Name: Placebo
Study Arms  ICMJE
  • Active Comparator: N-Acetyl Cysteine
    active compound N-Acetyl Cysteine
    Intervention: Drug: N-Acetyl Cysteine
  • Placebo Comparator: Sugar pill
    Placebo or sugar pill
    Intervention: Other: Placebo - sugar pill
Publications * 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.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: April 10, 2017)
43
Original Enrollment  ICMJE
 (submitted: February 29, 2008)
40
Actual Study Completion Date  ICMJE September 2010
Actual Primary Completion Date September 2010   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 3 Years to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT00627705
Other Study ID Numbers  ICMJE SU-02012008-995
10142
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Responsible Party Antonio Hardan, Stanford University
Study Sponsor  ICMJE Stanford University
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Antonio Hardan, MD Stanford University
PRS Account Stanford University
Verification Date April 2017

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP