Clinical trial NCT00627705: A Study of N-Acetyl Cysteine in Children With Autism
Official title: Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autistic Disorder
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2008-03-03
Enrollment: 43
Sex: ALL
Minimum age (years): 3
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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

Primary outcomes:
measure: Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
timeFrame: 4, 8, and 12 weeks
measure: The Clinical Global Rating Scale (CGRS) Improvement Subscale Score
timeFrame: 12 weeks
measure: Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)
timeFrame: 12 weeks
measure: Irritability Subscale of the Aberrant Behavior Checklist (ABC)
timeFrame: baseline and 12 weeks
Secondary outcomes:
measure: The Aberrant Behavior Checklist Total Score (ABC)
timeFrame: 4, 8, and 12 weeks
measure: Social Responsiveness Scale (SRS)
timeFrame: 12 weeks
measure: Sensory Profile Questionnaire (SPQ)
timeFrame: 12 weeks
measure: Glutathione (GSH) Metabolism Intermediates in Peripheral Blood
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University School of Medicine
state: California
Study officials:
affiliation: Stanford University
name: Antonio Hardan, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.