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

Autism Spectrum Disorder Clinical Trial

Official title:

Targeting the Neurobiology of Restricted and Repetitive Behaviors in Children With Autism Using N-acetylcysteine: an Open Label Extension

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05494398
• Other study ID #: IRB-64292
• Secondary ID: K99HD101702
• Status: Enrolling by invitation
• Phase: Phase 2/Phase 3
• Start date: November 15, 2022
• Est. completion date: September 2028

Study information

• Verified date: December 2023
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 48
• Est. completion date: September 2028
• Est. primary completion date: September 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 13 Years

Eligibility

Inclusion Criteria:

• Inclusion criteria: successful completion of IRB protocols 54931 or 68353.

Inclusion for 54931 include:

• individuals between 3 years and 13 years and 11 months at the time of consent
• 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 in ASD score = 11
• physical development indicative of prepubescence as defined by criteria for Tanner Stage 1
• medically stable
• passes MR safety screening (e.g., no metal in the body).

Inclusion for 65821:

• same criteria as above for 54931
• have stable medication regimens (= 30 days) and psychosocial treatments (= 60 days) prior to randomization with no anticipated changes during the trial
• successfully completes baseline neuroimaging (MRI or EEG).

Exclusion Criteria:

• significant adverse affects that led to withdrawal or early termination from IRB 54931 or 65821.

Exclusion criteria for 54931 and 65821 include:

• 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, or (6) the inability to drink a dissolved agent.

Related Conditions & MeSH terms

• Autism Spectrum Disorder
• Autistic Disorder

Intervention

Drug:
• N-acetylcysteine
12 week administration of N-Acetylcysteine to target restricted repetitive behavior severity.

Locations

Country	Name	City	State
United States	Stanford University School of Medicine	Stanford	California

Sponsors (2)

Lead Sponsor	Collaborator
Stanford University	Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in glutamatergic neurometabolites (Glx) measured by proton spectroscopy Magnetic Resonance Imaging (MRI)		Baseline and Week 12 of treatment
Primary	Change in restricted and repetitive behaviors as measured by Children's Yale Brown Obsessive Compulsive Scale - Autism Spectrum Disorder (CYBOCS-ASD)	Higher scores on the Children's Yale Brown Obsessive Compulsive Scale - Autism Spectrum Disorder mean higher levels of restricted and repetitive behaviors (Raw Score Total Range: 0-20)	Screening, Week 8, week 12
Secondary	Change in Gamma band amplitude and synchronization measured by electroencephalography (EEG)		Baseline, Week 12
Secondary	Change in restricted repetitive behavior subtypes as measured by the Restricted Behavior Scale - Revised (RBS-R)	Higher scores on the Repetitive Behavior Scale- Revised mean higher levels of repetitive and restricted behaviors. (Raw Score Total Range: 0 - 129)	Baseline, week 8, week 12