A Study of N-Acetyl Cysteine in Children With Autism

NCT ID: NCT00627705

Last Updated: 2017-05-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 43 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2008-02-29
• Study Completion Date: 2010-09-30

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.

Conditions

Autistic Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

N-Acetyl Cysteine

active compound N-Acetyl Cysteine

Group Type: ACTIVE_COMPARATOR

N-Acetyl Cysteine

Intervention Type: DRUG

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).

Sugar pill

Placebo or sugar pill

Group Type: PLACEBO_COMPARATOR

Placebo - sugar pill

Intervention Type: OTHER

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).

Interventions

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).

Intervention Type: DRUG

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).

Intervention Type: OTHER

Other Intervention Names

NAC; Placebo

Eligibility 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 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

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Antonio Hardan

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Antonio Hardan, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University School of Medicine

Stanford, California, United States

Countries

United States

References

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.

Reference Type: RESULT

PMID: 22342106 (View on PubMed)

Other Identifiers

10142

Identifier Type: -

Identifier Source: secondary_id

SU-02012008-995

Identifier Type: -

Identifier Source: org_study_id