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Phase 2 N=2 Randomized Quadruple-blind Treatment

A Feasibility Study of N-acetylcysteine for Self-injurious Behavior in Children With Autism Spectrum Disorder

Autism Spectrum Disorder

Enrolled (actual)
2
Serious AEs
0.0%
Results posted
Dec 2020
Primary outcome: Primary: Percentage of Participants Randomized — 100; 100 percentage of participants randomized

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
N-acetylcysteine (Drug); Placebo (Drug)
Age
Pediatric · 5+ yrs
Sex
All
Sponsor
Emory University
Primary completion
Sep 2019

Outcome Measures

OutcomeResultp-value
PRIMARY
Percentage of Participants Randomized
100; 100
PRIMARY
Attrition Rate
0; 0
PRIMARY
Study Medication Compliance
1; 1
PRIMARY
Successful Collection of Outcome Measures
1; 1
PRIMARY
Parent Satisfaction Rating
1; 1
SECONDARY
Positive Predictive Value of Screening Method of Classifying Self-injurious Behavior (SIB) by Type.
3
SECONDARY
Aberrant Behavior Checklist Irritability Subscale Score at Baseline and 9 Weeks Post-intervention
31; 28; 23; 29
SECONDARY
Number of Self-Injurious Behavior Events
219; 93; 59; 655
SECONDARY
Change in Clinical Global Impression (CGI-I) Scale at 9 Weeks Post-intervention
2; 4
SECONDARY
Change in Biomarkers and Possible Mechanisms of Action of NAC in Children With ASD.

Summary

The purpose of this study is to demonstrate the feasibility of a 9-week, randomized trial of N-acetylcysteine (NAC) compared to placebo in 14 children (age 5 to 12 years) with Autism Spectrum Disorder (ASD) and a moderate level of repetitive self-injurious behavior (SIB). Additional aims are to evaluate the positive predictive value of a screening method to classify children with automatically maintained self-injurious behavior; to evaluate the preliminary efficacy of NAC for reducing repetitive SIB in children with ASD; and to evaluate biomarkers and possible mechanisms of action of NAC in children with ASD.

Eligibility Criteria

Inclusion Criteria

  • Confirmed diagnosis of Autism Spectrum Disorder (ASD)
  • Confirmed presence of moderate Self Injurious Behavior (SIB)
  • Score > 16 on the parent-rated Aberrant Behavior Checklist Irritability subscale (moderate level of disruptive behavior)
  • Classified as having automatically maintained SIB (determined during screening by a detailed functional analysis)

Exclusion Criteria

  • On a stable medication dose for less than 4 weeks
  • Planned change in medication during the 9-week trial
  • Had one or more seizures in the last 6 months
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03008889). 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.

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