Targeting the Neurobiology of Restricted and Repetitive Behaviors in Children With Autism Using N-acetylcysteine

Sponsor

Stanford University (Other)

Overall Status

Recruiting

CT.gov ID

NCT04278898

Collaborator

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (NIH)

Enrollment

Location

Arms

34.6

Anticipated Duration (Months)

0.7

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The goal of this study is to target the neurobiology of restricted and repetitive behaviors in children with autism spectrum disorder using N-acetylcysteine (NAC), a well-tolerated nutritional supplement that has shown promise for reducing symptom severity in recent small-scale trials. The findings from this research will shed light on the mechanisms of action underlying the clinical benefits of NAC.

Condition or Disease	Intervention/Treatment	Phase
Autism Spectrum Disorder	Drug: N-Acetylcysteine	Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

24 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

Targeting the Neurobiology of Restricted and Repetitive Behaviors in Children With Autism Using N-acetylcysteine: a Single-dose Challenge Study

Actual Study Start Date :

Feb 12, 2021

Anticipated Primary Completion Date :

Aug 1, 2023

Anticipated Study Completion Date :

Jan 1, 2024

Arms and Interventions

Arm	Intervention/Treatment
Experimental: N-acetylcysteine then Placebo	Drug: N-Acetylcysteine N-acetylcysteine Single Dose 2700 mg taken orally
Experimental: Placebo then N-acetylcysteine	Drug: N-Acetylcysteine N-acetylcysteine Single Dose 2700 mg taken orally

Arm

Intervention/Treatment

Experimental: N-acetylcysteine then Placebo

Drug: N-Acetylcysteine

N-acetylcysteine Single Dose 2700 mg taken orally

Experimental: Placebo then N-acetylcysteine

Drug: N-Acetylcysteine

N-acetylcysteine Single Dose 2700 mg taken orally

Outcome Measures

Primary Outcome Measures

Change in glutamatergic neurometabolites (Glx) measured by proton spectroscopy Magnetic Resonance Imaging (MRI) [1 hour after single dose]

Secondary Outcome Measures

Change in Gamma band amplitude and synchronization measured by electroencephalography [1 hour after single dose]

Eligibility Criteria

Criteria

Ages Eligible for Study:

3 Years to 12 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

aged between 3 years and 12 years 11 months,
diagnosis of Autism Spectrum Disorder confirmed with the Autism Diagnostic Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule-2 (ADOS-2), or Brief Observation of Symptoms of Autism (BOSA), or Childhood Autism Rating Scale- Second Edition (CARS-2).
at least moderate Restricted and Repetitive Behaviors severity defined by a Children's Yale-Brown Obsessive Compulsive Scale for children with autism spectrum disorder score ≥ 11,
physical development indicative of prepubescence as defined by the criteria for Tanner Stage 1,
medically stable,
passes MR safety screening (e.g., no metal in the body).

Exclusion Criteria:

presence of known genetic abnormalities associated with Autism Spectrum Disorder (e.g. Fragile X),
current or life-time diagnosis of severe psychiatric disorder (e.g., schizophrenia),
presence of significant medical problems that would interfere with participation,
the inability of at least one caregiver to speak/read English to a sufficient level to complete study requirements and materials,
individuals taking antioxidant agents and glutathione prodrugs, or
the inability/unwillingness to swallow an agent during the screening visit.