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

NCT ID: NCT05664789

Last Updated: 2025-11-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

144 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-04-26

Study Completion Date

2026-01-31

Brief Summary

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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 and the effects of NAC on altering restricted and repetitive behavior symptom severity in children with autism spectrum disorder. This is a 12-week double-blind, randomized, placebo-controlled trial of NAC.

Detailed Description

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Conditions

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Autism Spectrum Disorder

Keywords

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Magnetic resonance imaging N-acetyl cysteine Restricted and repetitive behaviors Electroencephalography

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Double-blind, randomized, placebo-controlled trial 2 arms, N-acetylcysteine and placebo
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
The placebo compound will match the active N-acetylcysteine in appearance, odor, taste, and packaging.

Study Groups

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N-acetylcysteine

12 week administration of active study compound

Group Type ACTIVE_COMPARATOR

N acetyl cysteine

Intervention Type DRUG

N-acetylcysteine (NAC) is an over-the-counter dietary supplement that is relatively well tolerated and exhibits minimal side effects, even at high dosages. N

Placebo

12 week administration of matched placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

matched placebo

Interventions

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N acetyl cysteine

N-acetylcysteine (NAC) is an over-the-counter dietary supplement that is relatively well tolerated and exhibits minimal side effects, even at high dosages. N

Intervention Type DRUG

Placebo

matched placebo

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* children between 3 years and 12 years 11 months
* diagnosis of autism spectrum disorder confirmed with the Autism Diagnostic Interview- Revised and the Autism Diagnostic Observation Schedule-2, Brief Observation of Symptoms of Autism, or Childhood Autism Rating Scale
* at least moderate severity of restricted and repetitive behaviors defined by a Children's Yale-Brown Obsessive Compulsive Scale for Autism Spectrum Disorder score ≥ 11
* physical development indicative of prepubescence as defined by criteria for Tanner Stage 1
* if home address is within 300 miles of the primary study site, participants must pass MR safety screening (e.g., no metal in the body) and attempt baseline neuroimaging (MRI or EEG), otherwise baseline neuroimaging is not required for participation
* have stable medication regimens (≥ 30 days) and psychosocial treatments (≥ 60 days) prior to randomization with no anticipated changes during the trial

Exclusion Criteria

* presence of known genetic abnormalities associated with ASD (e.g. Fragile X)
* current or life-time diagnosis of severe psychiatric disorder (e.g., schizophrenia)
* presence of significant medical problems
* the inability of at least one caregiver to speak and read English to a sufficient level
* participants taking glutathione agents/prodrugs
* history of any adverse effects to glutathione agents/prodrugs
* the inability to drink a sample study compound dissolved in liquid
Minimum Eligible Age

3 Years

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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

NIH

Sponsor Role collaborator

Stanford University

OTHER

Sponsor Role lead

Responsible Party

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John Patrick Hegarty

Clinical Assistant Professor

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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John Hegarty, PhD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

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Department of Psychiatry and Behavioral Sciences

Palo Alto, California, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Brianna Alconcher

Role: CONTACT

Phone: (650)723-7845

Email: [email protected]

John Hegarty, PhD

Role: CONTACT

Phone: (650)723-7845

Email: [email protected]

Facility Contacts

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Brianna Alconcher

Role: primary

Other Identifiers

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4R00HD101702-03

Identifier Type: NIH

Identifier Source: secondary_id

View Link

IRB-68353

Identifier Type: -

Identifier Source: org_study_id