What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) that involve antioxidants and glutamate modulation, such as N-acetylcysteine (NAC), have shown promise but also come with potential side effects. NAC, for instance, has been associated with gastrointestinal issues, such as nausea and diarrhea, and in rare cases, severe reactions like status epilepticus. Other pharmacotherapies for ASD, including GABAergic agents like riluzole and vigabatrin, may cause sedation, dizziness, and potential long-term side effects. Overall, while these treatments can be beneficial, their side effects necessitate careful monitoring and consultation with healthcare providers.

What prior FDA approvals exist?

Currently, the FDA has approved two medications for the treatment of irritability associated with Autism Spectrum Disorder (ASD): risperidone and aripiprazole. These medications primarily target behavioral symptoms rather than core symptoms of ASD. While N-acetylcysteine (NAC) is being studied for its potential benefits in reducing restricted and repetitive behaviors in ASD due to its antioxidant and glutamate modulation properties, there are no FDA-approved treatments specifically targeting these mechanisms for ASD at this time. Further research and clinical trials are needed to explore the efficacy and safety of NAC and similar treatments.

What other types of research exist?

Promising novel alternatives to N-acetylcysteine (NAC) for autism treatment include L-Carnosine, which has been studied for its potential benefits in children with autism spectrum disorders, and GlyNAC (a combination of glycine and N-acetylcysteine), which has shown improvements in oxidative stress and mitochondrial function in older adults and may have potential applications in autism. Additionally, emerging pharmacotherapies targeting neurodevelopmental disorders, such as those restoring neurogenesis and cognitive performance, could be considered.

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Nutritional Supplement

N-acetylcysteine for Autism

Phase 2 & 3

Waitlist Available

Led By John Hegarty, PhD

Research Sponsored by Stanford University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline, week 8, week 12

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a nutritional supplement called N-acetylcysteine (NAC) to help children with autism spectrum disorder. NAC has shown promise in smaller studies for reducing repetitive behaviors. The study aims to understand how NAC works and its effects on these behaviors. NAC has been studied for its potential to reduce irritability and self-injurious behaviors in children with autism spectrum disorder (ASD).

Who is the study for?

This trial is for children aged 3 to under 13 with autism spectrum disorder, confirmed by specific diagnostic tools. They must be prepubescent, medically stable, and have no metal in their body that would interfere with MRI or EEG tests. Participants should be on a steady medication and treatment plan for at least 30 days before the trial starts and not expected to change during the trial.

What is being tested?

The study is testing N-acetylcysteine (NAC), a nutritional supplement thought to help reduce symptoms of autism, particularly restricted and repetitive behaviors. The research aims to understand how NAC works in the brain of children with autism.

What are the potential side effects?

While not specified here, common side effects of N-acetylcysteine may include nausea, vomiting, rash, and fever. As it's well-tolerated generally, severe side effects are rare but can occur.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline, week 8, week 12

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline, week 8, week 12

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline, week 8, week 12 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change in restricted and repetitive behaviors as measured by Children's Yale Brown Obsessive Compulsive Scale - Autism Spectrum Disorder (CYBOCS-ASD)

Secondary study objectives

Change in restricted repetitive behavior subtypes as measured by the Restricted Behavior Scale - Revised (RBS-R)

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: N-AcetylcysteineExperimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

N-acetylcysteine

2008

Completed Phase 4

~1290

Do I qualify?Apply below to find out

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

N-acetylcysteine (NAC) is a treatment for Autism that works primarily through its antioxidant properties and its ability to modulate glutamate levels in the brain. As an antioxidant, NAC helps reduce oxidative stress, which is thought to be elevated in individuals with Autism. By modulating glutamate, a key neurotransmitter involved in excitatory signaling, NAC may help balance neural activity and reduce symptoms such as irritability and repetitive behaviors. These mechanisms are significant for Autism patients because they target underlying biological processes that may contribute to the disorder, potentially leading to improvements in behavior and overall functioning.

A randomised, double blind, placebo-controlled trial of a fixed dose of N-acetyl cysteine in children with autistic disorder.N-acetylcysteine for treatment of autism, a case report.