Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Single-dose

NCT04278898 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: IRB-549311K99HD101702
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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.

Conditions

Autism Spectrum Disorder

Keywords

Magnetic Resonance Imaging; N-acetylcysteine; Restricted and Repetitive Behaviors; electroencephalography

Outcome Measures

Primary Outcomes (1)

• Change in glutamatergic neurometabolites (Glx) measured by proton spectroscopy Magnetic Resonance Imaging (MRI)

  1 hour after single dose

Secondary Outcomes (1)

• Change in Gamma band amplitude and synchronization measured by electroencephalography

  1 hour after single dose

Study Arms (2)

N-acetylcysteine then Placebo

EXPERIMENTAL

Drug: N-Acetylcysteine

Placebo then N-acetylcysteine

EXPERIMENTAL

Drug: N-Acetylcysteine

Interventions

N-Acetylcysteine DRUG

N-acetylcysteine Single Dose 2700 mg taken orally

N-acetylcysteine then Placebo; Placebo then N-acetylcysteine

Eligibility Criteria

• Age: 3 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• children between 3 years and 12 years 11 months at the time of consent
• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Stanford University: lead
• Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD): collaborator

Study Sites (1)

Stanford University School of Medicine

Stanford, California, 94305, United States

RECRUITING

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

Study Officials

• John Hegarty, PhD

  Stanford University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Brianna Alconcher

CONTACT

(650) 723-7845; autismresearch@stanford.edu

John Hegarty, PhD

CONTACT

(650) 723-7845; hegartyj@stanford.edu

Study Design

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

Study Record Dates

• First Submitted: February 18, 2020
• First Posted: February 20, 2020
• Study Start: February 12, 2021
• Primary Completion (Estimated): July 31, 2028
• Study Completion (Estimated): January 31, 2029
• Last Updated: January 20, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will share
• Time Frame: The data will be submitted on January 15th and July 15th each year.
• Access Criteria: Researchers with access to the National Database for Autism Research (NDAR) will be able to obtain the submitted data.

Locations

US (1)