N-Acetylcysteine for Autism
Recruiting in Palo Alto (17 mi)
Age: < 18
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 2
Recruiting
Sponsor: Stanford University
Must not be taking: Antioxidants, Glutathione prodrugs
Disqualifiers: Genetic abnormalities, Severe psychiatric disorder, others
No Placebo Group
Prior Safety Data
Approved in 4 Jurisdictions
Trial Summary
What is the purpose of this trial?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.
Will I have to stop taking my current medications?
The trial requires that participants stop taking antioxidant agents and glutathione prodrugs.
What data supports the effectiveness of the drug N-Acetylcysteine for autism?
Research suggests that N-Acetylcysteine (NAC) may help reduce some symptoms of autism, such as social impairment and irritability. In one case report, a child's social interaction improved and aggressive behaviors decreased after taking NAC, and other studies have explored its potential benefits when used alongside other medications.
12345Is N-Acetylcysteine safe for humans?
Research on N-Acetylcysteine (NAC) for autism suggests it is generally safe for humans, as studies have focused on its safety and tolerability in children and adolescents with autism spectrum disorder.
12367What makes the drug N-acetylcysteine unique for treating autism?
N-acetylcysteine (NAC) is unique for treating autism because it targets oxidative stress and neuroinflammation, which are believed to play a role in autism, and it acts as an antioxidant by providing cystine, a precursor for glutathione, an important brain antioxidant. Unlike other treatments, NAC may help improve social interaction and reduce irritability by modulating brain glutamate levels, which are often dysregulated in autism.
12348Eligibility Criteria
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.Inclusion Criteria
My autism diagnosis was confirmed with specific autism assessment tools.
passes MR safety screening (e.g., no metal in the body).
I am between 3 and 12 years old.
+4 more
Exclusion Criteria
I am currently taking antioxidants or glutathione supplements.
You have been diagnosed with a severe psychiatric disorder such as schizophrenia either currently or in the past.
You are not willing or able to swallow the study medication during the screening visit.
+3 more
Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Treatment
Participants receive a single dose of N-acetylcysteine or placebo to study its effects on restricted and repetitive behaviors in children with autism
1 day
Follow-up
Participants are monitored for safety and effectiveness after the single-dose treatment
4 weeks
Participant Groups
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.
2Treatment groups
Experimental Treatment
Group I: Placebo then N-acetylcysteineExperimental Treatment1 Intervention
Group II: N-acetylcysteine then PlaceboExperimental Treatment1 Intervention
N-Acetylcysteine is already approved in United States, European Union, Canada, Australia for the following indications:
🇺🇸 Approved in United States as Mucomyst for:
- Mucolytic agent
- Acetaminophen overdose
🇪🇺 Approved in European Union as Fluimucil for:
- Mucolytic agent
- Respiratory tract disorders
🇨🇦 Approved in Canada as N-Acetylcysteine for:
- Mucolytic agent
- Acetaminophen overdose
🇦🇺 Approved in Australia as N-Acetylcysteine for:
- Mucolytic agent
- Respiratory tract disorders
- Paracetamol overdose
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Stanford University School of MedicineStanford, CA
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Who Is Running the Clinical Trial?
Stanford UniversityLead Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)Collaborator
References
A randomized placebo-controlled pilot study of N-acetylcysteine in youth with autism spectrum disorder. [2018]Social impairment is a defining feature of autism spectrum disorder (ASD) with no demonstrated effective pharmacologic treatments. The goal of this study was to evaluate efficacy, safety, and tolerability of oral N-acetylcysteine (NAC), an antioxidant whose function overlaps with proposed mechanisms of ASD pathophysiology, targeting core social impairment in youth with ASD.
Effectiveness of N-acetylcysteine in autism spectrum disorders: A meta-analysis of randomized controlled trials. [2022]Label="OBJECTIVE">Currently, pharmaceutical treatment options for autism spectrum disorder are limited. Brain glutaminergic dysregulation is observed in autism spectrum disorder. N-acetylcysteine, which can be converted to glutathione and subsequently release glutamate into the extracellular space, and thus reduce glutamatergic neurotransmission at synapses, is considered a potential drug for autism spectrum disorder treatment. Here, we analyzed the treatment effects of N-acetylcysteine on autism spectrum disorder in randomized controlled trials.
N-acetylcysteine for treatment of autism, a case report. [2021]There are a limited number of Food and Drug Administration (FDA)-approved medications for the treatment of autism. Meanwhile, oxidative stress and neuroinflammation are supposed to play a causative role in autism. N-acetylcysteine may provide cystine, a precursor for glutathione (GSH), which is an important antioxidant factor in the brain. We here report a child with autism, whose symptoms were markedly decreased after taking oral N-acetylcysteine 800 mg/day, in three divided doses. His social interaction was significantly increased. The score of social impairment on a visual analog scale decreased from 10 to 6 in the two-month trial. The aggressive behaviors decreased from 10 to 3. This case suggests that N-acetylcysteine may decrease some symptoms of autism.
N-acetylcysteine as an adjunctive therapy to risperidone for treatment of irritability in autism: a randomized, double-blind, placebo-controlled clinical trial of efficacy and safety. [2022]According to the proposed interference of N-acetylcysteine (NAC) with pathophysiologic processes of autistic disorders (ADs), we aimed to assess the effectiveness and safety of NAC as an adjunct to risperidone in the treatment of ADs in a randomized, double-blind, clinical trial.
A randomized double blind placebo controlled clinical trial of N-Acetylcysteine added to risperidone for treating autistic disorders. [2022]This study examined the efficacy and safety of N-acetylcysteine (NAC) augmentation for treating irritability in children and adolescents with autism spectrum disorders (ASD).
Acetylcysteine for treatment of autism spectrum disorder symptoms. [2019]Successful use of acetylcysteine to control irritability and aggressive behaviors in a hospitalized adolescent patient with autism spectrum disorder (ASD) is described.
A randomized controlled pilot trial of oral N-acetylcysteine in children with autism. [2022]An imbalance in the excitatory/inhibitory systems with abnormalities in the glutamatergic pathways has been implicated in the pathophysiology of autism. Furthermore, chronic redox imbalance was also recently linked to this disorder. The goal of this pilot study was to assess the feasibility of using oral N-acetylcysteine (NAC), a glutamatergic modulator and an antioxidant, in the treatment of behavioral disturbance in children with autism.
The Impact of N-acetylcysteine on Major Depression: Qualitative Observation and Mixed Methods Analysis of Participant Change during a 12-week Randomised Controlled Trial. [2023]Label="Objective" NlmCategory="UNASSIGNED"> N-acetylcysteine (NAC) is a novel therapeutic agent with multiple mechanisms of action in the central nervous system and a favourable side effect profile. Clinical evidence indicates that adjunctive NAC may reduce the severity of depressive symptoms in individuals with major depressive disorder (MDD).