What side effects are associated with this type of intervention?

Common treatments for Autism, such as N-acetylcysteine (NAC) and memantine, are generally well tolerated. NAC has shown promise in reducing symptom severity with minimal side effects. Memantine, while also well tolerated, has been associated with some worsening in communication measures in certain studies. Donepezil, another cognitive enhancer, has shown limited impact on cognitive functioning with no significant adverse effects noted in short-term use. Overall, these treatments appear to have a favorable safety profile, but individual responses may vary.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically for Autism Spectrum Disorder (ASD) that focus on antioxidant properties or modulation of glutamate levels, similar to N-acetylcysteine. While memantine, an NMDA receptor antagonist, has shown promise in trials for treating social deficits in intellectually capable adults with ASD, it is not yet FDA-approved for this indication. Other treatments like donepezil have been studied but have shown limited impact on cognitive functioning in ASD.

What other types of research exist?

Promising alternatives to N-acetylcysteine for autism treatment include omega-3 fatty acids, which have shown some potential benefits in small studies, though evidence is inconsistent. Coenzyme Q10 and vitamins E and A are other antioxidants that may be considered, but their efficacy in autism treatment is not well-established. Further research is needed to confirm their benefits.

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N-Acetylcysteine for Autism

Phase 2

Recruiting

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

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).

aged between 3 years and 12 years 11 months

Must not have

individuals taking antioxidant agents and glutathione prodrugs

presence of known genetic abnormalities associated with Autism Spectrum Disorder (e.g. Fragile X)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial aims to see if NAC, a safe nutritional supplement, can help reduce repetitive behaviors in children with autism by balancing brain chemicals. N-Acetylcysteine (NAC) has been studied for its potential benefits in treating obsessive-compulsive spectrum disorders and irritability in autism spectrum disorders.

Who is the study for?

This trial is for children aged 3 to almost 13 with Autism Spectrum Disorder, confirmed by specific diagnostic tests. They must be medically stable, prepubescent, and have moderate to severe repetitive behaviors. Kids can't join if they have metal in their body (unsafe for MRI), genetic abnormalities like Fragile X, or take certain antioxidants.

What is being tested?

The study is testing N-acetylcysteine (NAC), a nutritional supplement that might help reduce the severity of restricted and repetitive behaviors in autistic children. The research aims to understand how NAC affects these symptoms.

What are the potential side effects?

While not specified here, NAC is generally well-tolerated but could potentially cause side effects such as gastrointestinal discomfort, rash, headache or fatigue.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My autism diagnosis was confirmed with specific autism assessment tools.

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I am between 3 and 12 years old.

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My physical development shows I am in the earliest stage of puberty.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently taking antioxidants or glutathione supplements.

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I have a genetic condition linked to Autism.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2015 Phase 3 trial • 302 Patients • NCT01675661

Headache

Nausea

Diarrhea

Abdominal discomfort

Dyspepsia

Pruritis

Cellulitis

Alcohol abuse

Road traffic accident

Arthropod bite

Gastrointestinal disorder

Abdominal pain

Vomiting

Reflux disease

Dizziness

Insomnia

Rash

Energy increased

Groin Abscess

Flushing

Fatigue

Panic attack

Depression

Blood pressure increased

100%

80%

60%

40%

20%

Study treatment Arm

NAC Plus CM

Placebo Plus CM

Awards & Highlights

No Placebo-Only Group

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

Trial Design

2Treatment groups

Experimental Treatment

Group I: Placebo then N-acetylcysteineExperimental Treatment1 Intervention

Group II: N-acetylcysteine then PlaceboExperimental Treatment1 Intervention

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

N-Acetylcysteine

2013

Completed Phase 3

~1470

0 / 5Eligibility criteria met

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 being studied for Autism Spectrum Disorder (ASD) due to its antioxidant properties and its ability to modulate glutamate levels. NAC works by disrupting disulfide bonds, which helps to liquefy mucus and DNA, and by acting as an antioxidant, reducing oxidative stress. Additionally, NAC modulates glutamate levels, which is crucial because glutamate is a key neurotransmitter involved in brain function and its dysregulation is often observed in ASD. These mechanisms are important for ASD patients as they may help reduce symptom severity, including social impairment and aggressive behaviors, thereby potentially improving quality of life.

N-acetylcysteine for treatment of autism, a case report.