An Open-Label Study of N-Acetyl Cysteine in Children With Autism

NCT00676195 | Completed Phase 2 Results DMC

• 1 other identifier: SU-05062008-1139
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to test the tolerability and efficacy of N-Acetyl Cysteine (NAC) in children with Autism.

Conditions

Autistic Disorder

Keywords

N-Acetyl Cysteine

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events Reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES)

  Number of Participants with adverse events reported on the Dosage Record and Treatment Emergent Symptom Scale (DOTES) during the course of the study as measured at time points (4, 8, and 12 weeks).

  4, 8, and 12 weeks

Secondary Outcomes (4)

• Social Responsiveness Scale (SRS)

  12 weeks

• Sensory Profile Questionnaire (SPQ)

  12 weeks

• Irritability Subscale of the Aberrant Behavior Checklist (ABC)

  4, 8, and 12 weeks

• Glutathione Metabolism Intermediates in Peripheral Blood Measured by High-performance Liquid Chromatography

  12 weeks

Study Arms (1)

N-Acetyl Cysteine

EXPERIMENTAL

Dosage of orally administered N-Acetyl Cysteine is as follows: Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day

Drug: N-Acetyl Cysteine

Interventions

N-Acetyl Cysteine DRUG

Dosage of orally administered N-Acetyl Cysteine is as follows: Days 1-30: 900 mg, once per day Days 31-60: 900 mg, twice per day Days 61-90: 900 mg, three times per day

Also known as: NAC

N-Acetyl Cysteine

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Subjects will be eligible for this study if they participated in the Double-blind, randomized, placebo controlled study of N-Acetyl Cysteine in Autism study at Stanford University and meet all of the following criteria:
• Outpatients between 3.0 and 12.11 years of age inclusive
• Males and females who are physically healthy
• diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation
• Clinical Global Impression Severity rating of 4
• Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis
• Ability of subject to swallow the compound
• Stable concomitant medications for at least 2 weeks
• No planned changes in psychosocial interventions during the open-label N-Acetyl Cysteine trial

You may not qualify if:

• Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified
• Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology)
• Pregnancy or sexually active females
• 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

Sponsors & Collaborators

• Stanford University: lead

Study Sites (1)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

MeSH Terms

Conditions

Autistic Disorder

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Limitations and Caveats

This is an open-label trial with a small sample size.

Results Point of Contact

• Title: Antonio Hardan, MD
• Organization: Stanford University School of Medicine

hardanay@stanford.edu

650-736-1235

Study Officials

• Antonio Hardan, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: May 7, 2008
• First Posted: May 12, 2008
• Study Start: June 1, 2008
• Primary Completion: June 1, 2010
• Study Completion: June 1, 2010
• Last Updated: March 29, 2017
• Results First Posted: March 29, 2017

Record last verified: 2017-02

Data Sharing

• IPD Sharing: Will share

Locations

US (1)