Phase 2
Completed N=33
A Study of N-Acetyl Cysteine in Children With Autism
Autistic Disorder
Source: ClinicalTrials.gov NCT00627705 ↗
Enrolled (actual)
33
Serious AEs
0.0%
Results posted
May 2017
Primary outcomePrimary: Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES) — 11; 7; 3; 2 participants
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.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Total Number of Subjects With Reported Side Effects as Assessed by Dosage Record and Treatment Emergent Symptom Scale (DOTES) |
11; 7; 3; 2; 6; 3 | — |
| PRIMARY The Clinical Global Rating Scale (CGRS) Improvement Subscale Score |
3.2; 2.9 | 0.449 |
| PRIMARY Glutathione (GSH) Levels in Peripheral Blood, Measured by State-of-the-art High-performance Liquid Chromatography (HPLC) |
— | — |
| PRIMARY Irritability Subscale of the Aberrant Behavior Checklist (ABC) |
16.9; 14.8; 7.2; 13.1 | <.001 sig |
| SECONDARY The Aberrant Behavior Checklist Total Score (ABC) |
— | — |
| SECONDARY Social Responsiveness Scale (SRS) |
111.9; 104.7; 93.8; 98.5 | .141 |
| SECONDARY Sensory Profile Questionnaire (SPQ) |
— | — |
| SECONDARY Glutathione (GSH) Metabolism Intermediates in Peripheral Blood |
— | — |
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
Data sourced from ClinicalTrials.gov (NCT00627705). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.