A Study of N-Acetyl Cysteine in Children With Autism

Autistic Disorder Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00627705
• Other study ID #: SU-02012008-995
• Secondary ID: 10142
• Status: Completed
• Phase: Phase 2
• First received: February 22, 2008
• Last updated: July 20, 2011
• Start date: February 2008
• Est. completion date: September 2010

Study information

• Verified date: July 2011
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 40
• Est. completion date: September 2010
• Est. primary completion date: September 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years to 12 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autistic Disorder

Intervention

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:
• 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).

Locations

Country	Name	City	State
United States	Stanford University School of Medicine	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Stanford University

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The Aberrant Behavior Checklist total score (ABC)		4, 8, and 12 weeks	No
Primary	Dosage Record and Treatment Emergent Symptom Scale (DOTES)		4, 8, and 12 weeks	Yes
Primary	: The Clinical Global Rating Scale (CGRS) Improvement subscale		4, 8, and 12 weeks	Yes
Primary	GSH levels in peripheral blood, measured by state-of-the-art high-performance liquid chromatography (HPLC)		12 weeks	No
Secondary	Social Responsiveness Scale (SRS)		12 weeks	No
Secondary	Sensory Profile Questionnaire (SPQ)		12 weeks	No
Secondary	Irritability subscale of the ABC		4, 8, and 12 weeks	No
Secondary	GSH metabolism intermediates in peripheral blood measured by HPLC		12 weeks	No