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

Stanford University

Status and phase: Enrolling; Phase 2

Conditions: Autism Spectrum Disorder

Treatments: Drug: N-Acetylcysteine

Study type: Interventional

Funder types: Other; NIH

Identifiers

NCT04278898

IRB-54931

1K99HD101702 (U.S. NIH Grant/Contract)

Details and patient eligibility

About

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.

Enrollment: 24 estimated patients

Sex: All

Ages: 3 to 12 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 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).

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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24 participants in 2 patient groups

N-acetylcysteine then Placebo

Experimental group

Treatment:

Drug: N-Acetylcysteine

Placebo then N-acetylcysteine

Experimental group

Treatment:

Drug: N-Acetylcysteine