Double-blind , Randomized, Placebo Controlled Study of N-Acetyl Cysteine in Autism.
Overview
- Phase
- Phase 2
- Intervention
- N-Acetyl Cysteine
- Conditions
- Autistic Disorder
- Sponsor
- Stanford University
- Enrollment
- 43
- Locations
- 1
- Primary Endpoint
- Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
- Status
- Completed
- Last Updated
- 8 years ago
Overview
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.
Investigators
Antonio Hardan
Professor
Stanford University
Eligibility Criteria
Inclusion Criteria
- •Outpatients between 3.0 and 12.11 years of age inclusive
- •Males and females who are physically healthy
- •diagnosis of autism based 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
Exclusion Criteria
- •DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder not otherwise specified
- •Prior adequate trial of N-Acetyl Cysteine
- •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
Arms & Interventions
N-Acetyl Cysteine
active compound N-Acetyl Cysteine
Intervention: N-Acetyl Cysteine
Sugar pill
Placebo or sugar pill
Intervention: Placebo - sugar pill
Outcomes
Primary Outcomes
Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES)
Time Frame: 4, 8, and 12 weeks
The Dosage Record and Treatment Emergent Symptom Scale (DOTES) provides information on the presence, frequency, and severity of side effects reported during the course of the trial.
The Clinical Global Rating Scale (CGRS) Improvement Subscale Score
Time Frame: 12 weeks
Score range 1-7 (lower score mean more improvement compared to baseline)
Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC)
Time Frame: 12 weeks
Data not collected. The laboratory was not able to measure Glutathione levels.
Irritability Subscale of the Aberrant Behavior Checklist (ABC)
Time Frame: baseline and 12 weeks
Aberrant Behavior Checklist (ABC) Irritability Subscale Score (range 0-45); higher scores mean higher irritability
Secondary Outcomes
- The Aberrant Behavior Checklist Total Score (ABC)(4, 8, and 12 weeks)
- Sensory Profile Questionnaire (SPQ)(12 weeks)
- Glutathione (GSH) Metabolism Intermediates in Peripheral Blood(12 weeks)
- Social Responsiveness Scale (SRS)(12 weeks)