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

NCT03008889 | Completed Phase 2 Results FDA Drug

• 1 other identifier: IRB00088115
• Study Type: interventional
• Target: 8
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Autism Spectrum Disorder

Keywords

Repetitive Self-Injurious Behavior; N-acetylcysteine (NAC)

Outcome Measures

Primary Outcomes (5)

• Percentage of Participants Randomized

  Goal: randomize 1.75 participants per month

  12 months (throughout the duration of the study)

• Attrition Rate

  Attrition rate is defined as the percent of subjects who did not complete the study. Goal:less than 15% (to indicate that study was acceptable to participants and parents).

  12 months (throughout the duration of the study)

• Study Medication Compliance

  Goal: at least 70% treatment compliance (tablet counts and drug dairies).

  12 months (throughout the duration of the study)

• Successful Collection of Outcome Measures

  Goal: at least 80% collection of essential outcome data to demonstrate the feasibility of data collection procedures.

  12 months (throughout the duration of the study)

• Parent Satisfaction Rating

  Goal: at least 80% of parents will agree or strongly agree when asked in an anonymous survey that they would recommend the study and the study treatment to other parents of children with ASD and SIB.

  Week 9 (at the end of the study intervention)

Secondary Outcomes (5)

• Positive Predictive Value of Screening Method of Classifying Self-injurious Behavior (SIB) by Type.

  12 months (duration of the study)

• Aberrant Behavior Checklist Irritability Subscale Score at Baseline and 9 Weeks Post-intervention

  Baseline, Week 9

• Number of Self-Injurious Behavior Events

  Baseline, Week 9

• Change in Clinical Global Impression (CGI-I) Scale at 9 Weeks Post-intervention

  Week 9

• Change in Biomarkers and Possible Mechanisms of Action of NAC in Children With ASD.

  Baseline, Week 9

Study Arms (2)

Participants taking NAC

EXPERIMENTAL

Participants randomized to the active treatment study arm will receive gradually increasing doses of N-acetylcysteine (NAC) given as a dissolving tablet in juice or water. NAC is an over-the-counter oral dietary supplement that will be used a higher than usual doses in this study.

Drug: N-acetylcysteine

Participants taking Placebo

PLACEBO COMPARATOR

Participants randomized to placebo will receive dissolving tablets identical in size and appearance to the active treatment. Placebo capsules contain inactive ingredients.

Drug: Placebo

Interventions

N-acetylcysteine DRUG

Participants will start with taking 900mg of NAC once per day for one week (study days 1-7). At Day 7, parents (or primary caregiver) and subjects will return to the study site to review adverse events. In the absence of dose limiting adverse events attributable to the study drug, the dose will be gradually increased to 900 mg twice per day for study days 8-28. If this dose is well-tolerated, the dose will be increased to 900 three times per day for study days 29-63.

Also known as: NAC

Participants taking NAC

Placebo DRUG

Participants will start with taking 900mg of the placebo once per day for one week (study days 1-7). At Day 7, parents (or primary caregiver) and subjects will return to the study site to review adverse events. The dosing for the placebo will increase in the same fashion as the active treatment. In the absence of dose limiting adverse events attributable to the study drug, the dose will be gradually increased to 900 mg twice per day for study days 8-28. If this dose is well-tolerated, the dose will be increased to 900 three times per day for study days 29-63.

Participants taking Placebo

Eligibility Criteria

• Age: 5 Years - 12 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Emory University: lead

Study Sites (1)

Marcus Autism Center

Atlanta, Georgia, 30329, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

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

Results Point of Contact

• Title: Dr. Lawrence Scahill
• Organization: Emory University

lawrence.scahill@emory.edu

404-785-9336

Study Officials

• Lawrence Scahill, MSN, PhD

  Emory University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: December 28, 2016
• First Posted: January 4, 2017
• Study Start: July 5, 2018
• Primary Completion: September 12, 2019
• Study Completion: September 12, 2019
• Last Updated: January 26, 2021
• Results First Posted: December 16, 2020

Record last verified: 2020-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)