Now Available for Public Comment: Notice of Proposed Rulemaking (NPRM) for FDAAA 801 and NIH Draft Reporting Policy for NIH-Funded Trials

A Study of N-Acetyl Cysteine in Children With Autism

This study has been completed.
Sponsor:
Information provided by:
Stanford University
ClinicalTrials.gov Identifier:
NCT00627705
First received: February 22, 2008
Last updated: July 20, 2011
Last verified: July 2011
  Purpose

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.


Condition Intervention Phase
Autistic Disorder
Drug: N-Acetyl Cysteine
Other: Placebo - sugar pill
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.

Resource links provided by NLM:


Further study details as provided by Stanford University:

Primary Outcome Measures:
  • The Aberrant Behavior Checklist total score (ABC) [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: No ]
  • Dosage Record and Treatment Emergent Symptom Scale (DOTES) [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: Yes ]
  • : The Clinical Global Rating Scale (CGRS) Improvement subscale [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: Yes ]
  • GSH levels in peripheral blood, measured by state-of-the-art high-performance liquid chromatography (HPLC) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Social Responsiveness Scale (SRS) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Sensory Profile Questionnaire (SPQ) [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
  • Irritability subscale of the ABC [ Time Frame: 4, 8, and 12 weeks ] [ Designated as safety issue: No ]
  • GSH metabolism intermediates in peripheral blood measured by HPLC [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 40
Study Start Date: February 2008
Study Completion Date: September 2010
Primary Completion Date: September 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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).

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).


  Eligibility

Ages Eligible for Study:   3 Years to 12 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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 DSM-IV- TR criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation
  4. CGI 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 NAC trial

Exclusion Criteria:

  1. DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder NOS
  2. Prior adequate trial of NAC
  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 GSH prodrugs will be excluded from the study except if they have been off these compounds for at least 4 weeks
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00627705

Locations
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Antonio Hardan Stanford University
Sub-Investigator: Rabin Tirouvanziam PhD Stanford University
  More Information

No publications provided by Stanford University

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Antonio Hardan, Stanford University School of Medicine
ClinicalTrials.gov Identifier: NCT00627705     History of Changes
Other Study ID Numbers: SU-02012008-995, 10142
Study First Received: February 22, 2008
Last Updated: July 20, 2011
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Mental Disorders
Mental Disorders Diagnosed in Childhood
Acetylcysteine
N-monoacetylcystine
Anti-Infective Agents
Antidotes
Antioxidants
Antiviral Agents
Expectorants
Free Radical Scavengers
Molecular Mechanisms of Pharmacological Action
Pharmacologic Actions
Physiological Effects of Drugs
Protective Agents
Respiratory System Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on November 25, 2014