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

Primary Purpose

Autism Spectrum Disorder

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
N acetyl cysteine
Placebo
Sponsored by
Stanford University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Magnetic resonance imaging, N-acetyl cysteine, Restricted and repetitive behaviors, Electroencephalography

Eligibility Criteria

3 Years - 12 Years (Child)All SexesDoes not accept healthy volunteers

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 passes MR safety screening (e.g., no metal in the body) and attempts baseline neuroimaging (MRI or EEG) 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

Sites / Locations

  • Department of Psychiatry and Behavioral SciencesRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

N-acetylcysteine

Placebo

Arm Description

12 week administration of active study compound

12 week administration of matched placebo

Outcomes

Primary Outcome Measures

Change in Children's Yale Brown-Obsessive Compulsive Scale for Autism Spectrum Disorder (CYBOCS-ASD) at 12 weeks
Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors
Change in Glutamatergic neurometabolites at 12 weeks
Glu and Glx measured by magnetic resonance imaging

Secondary Outcome Measures

Change in Restricted and Repetitive Behavior Scale Revised (RBS-R) at 12 weeks
Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors
Change in Gamma band activity at 12 weeks
Electrical activity between 30-48 Hz measured by electroencephalography

Full Information

First Posted
December 7, 2022
Last Updated
August 14, 2023
Sponsor
Stanford University
Collaborators
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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1. Study Identification

Unique Protocol Identification Number
NCT05664789
Brief Title
Targeting the Neurobiology of RRB in Autism Using N-acetylcysteine: Trial
Official Title
Targeting the Neurobiology of Restricted and Repetitive Behaviors in Children With Autism Using N-acetylcysteine: A Randomized, Controlled Trial
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
April 26, 2023 (Actual)
Primary Completion Date
January 31, 2026 (Anticipated)
Study Completion Date
January 31, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Stanford University
Collaborators
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. 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 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorder
Keywords
Magnetic resonance imaging, N-acetyl cysteine, Restricted and repetitive behaviors, Electroencephalography

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Model Description
Double-blind, randomized, placebo-controlled trial 2 arms, N-acetylcysteine and placebo
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
The placebo compound will match the active N-acetylcysteine in appearance, odor, taste, and packaging.
Allocation
Randomized
Enrollment
48 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
N-acetylcysteine
Arm Type
Active Comparator
Arm Description
12 week administration of active study compound
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
12 week administration of matched placebo
Intervention Type
Drug
Intervention Name(s)
N acetyl cysteine
Intervention Description
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
Intervention Name(s)
Placebo
Intervention Description
matched placebo
Primary Outcome Measure Information:
Title
Change in Children's Yale Brown-Obsessive Compulsive Scale for Autism Spectrum Disorder (CYBOCS-ASD) at 12 weeks
Description
Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors
Time Frame
Screening and week 12
Title
Change in Glutamatergic neurometabolites at 12 weeks
Description
Glu and Glx measured by magnetic resonance imaging
Time Frame
Baseline and week 12
Secondary Outcome Measure Information:
Title
Change in Restricted and Repetitive Behavior Scale Revised (RBS-R) at 12 weeks
Description
Severity of restricted and repetitive behaviors, higher scores indicate more severe behaviors
Time Frame
Baseline and week 12
Title
Change in Gamma band activity at 12 weeks
Description
Electrical activity between 30-48 Hz measured by electroencephalography
Time Frame
Baseline and week 12

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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 passes MR safety screening (e.g., no metal in the body) and attempts baseline neuroimaging (MRI or EEG) 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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Katie Hodges, BS
Phone
(650)723-7845
Email
autismresearch@stanford.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Robin Libove, BA
Phone
(650) 736-1235
Email
rlibove@stanford.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
John Hegarty, PhD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Psychiatry and Behavioral Sciences
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Maddy Clark
Phone
650-736-1235
Email
autismresearch@stanford.edu

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
De-Identified data will be shared with the National Database for Autism Research (NDAR)
IPD Sharing Time Frame
The data will be shared on January 15th and July 15th each year
IPD Sharing Access Criteria
Researchers with access to the National Database for Autism Research (NDAR) will be able to obtain the submitted data
IPD Sharing URL
http://nda.nih.gov

Learn more about this trial

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

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