NCT02054949

Treatment Resistant Epilepsy and N-Acetyl Cysteine

Pilot Study of N-acetyl Cysteine for Refractory Generalized Epilepsy in Children With Autism

• Status: Withdrawn
• Phase: N/A
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Nationwide Children's Hospital · Academic / Other
• Sex: All
• Age: 6 Years – 21 Years
• Healthy volunteers: Not accepted

Summary

This is a study to find out whether the dietary amino acid supplement, N-acetyl cysteine (NAC) is helpful in reducing the frequency of seizures in children with autism and comorbid epilepsy whose seizures are not responding well to usual medical treatment. The study is also looking to see if this supplement is helpful for immediate and ongoing treatment of symptoms of irritability. Additionally, this study will also look to see if certain substances in the blood that measure a specific type of stress on cells in the body can help tell us how NAC may be helping.

Detailed description

One third of patients with idiopathic autism have treatment resistant epilepsy, associated with earlier onset of seizures. In addition to insufficient response to medical treatment, they also have poorer responses to surgical and VNS approaches (Sansa et al 2011). Novel approaches to reduce seizure burden and improve quality of life for the children and their caregivers are needed. There is a plethora of basic research documenting elevated oxidative stress in animal models of seizures. Several animal models have decreased oxidative stress through using compounds with antioxidant effects, but suprisingly, few human studies have been done to date. This small open-label pilot study will examine the use of N-acetyl cysteine, an inexpensive but readily available over-the-counter nutritional supplement, to reduce seizure frequency in 10 youth with autism who have not responded completely to conventional anticonvulsant therapy

Conditions

• Autistic Disorder
• Seizures
• Irritability

Interventions

Type	Name	Description
DRUG	N-Acetyl Cysteine (NAC)	NAC will be started at 500 mg by mouth twice daily for the first 2 weeks, then increased to 500 mg in the am and 1000 mg in the pm for week 3, and then increased to 1000 mg am and pm for weeks 4 through 8. Subjects will be maintained at the highest tolerated dose.

Timeline

• Start date: 2013-04-01
• Primary completion: 2015-01-01
• Completion: 2015-01-01
• First posted: 2014-02-04
• Last updated: 2015-10-09

Locations

1 site across 1 country: United States