Treatment Resistant Epilepsy and N-Acetyl Cysteine

Not Applicable

Withdrawn

• Conditions: Autistic Disorder; Irritability; Seizures
• Interventions: Drug: N-Acetyl Cysteine (NAC)

• Registration Number: NCT02054949
• Lead Sponsor: Nationwide Children's Hospital

Brief 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

Study Timeline

• Study Start: 2013-04
• Study First Submitted: 2014-02-03
• Study First Submitted QC: 2014-02-03
• Study First Posted: 2014-02-04
• Primary Completion: 2015-01
• Study Completion: 2015-01
• Status Verified: 2015-10
• Last Update Submitted: 2015-10-07
• Last Update Posted: 2015-10-09

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. Children and adolescents/young adults ages 6-21, male or female, all races and ethnicities.
2. Autism
3. IQ less than 80
4. 10 subjects with treatment resistant generalized epilepsy as defined by trials of 3 or more anticonvulsants with continued seizure frequency of at least 1 per week, need to use a helmet, or vagal nerve stimulator placement.
5. Children with at least 1 grand mal/generalized seizure per week.

Exclusion Criteria

1. Inability to swallow capsules
2. In females, pregnancy or sexual activity
3. Daily acetaminophen, glucocorticoid, or nonsteroidal anti-inflammatory drugs, or daily NAC or high-dose antioxidant vitamin supplements within 30 days of baseline.
4. History of acute or chronic liver, renal, endocrine, infectious, autoimmune, hematologic, metabolic, or other disorder in the judgement of the study physician.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
N-Acetyl Cysteine (NAC)	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.

Primary Outcome Measures

Name	Time	Method
Recruitment rate	1 year	screening to enrollment and retention rates will be measured

Secondary Outcome Measures

Name	Time	Method
seizure frequency	up to 8 weeks	seizure count

Trial Locations

Locations (1)

Nationwide Children's Hospital; 🇺🇸 Columbus, Ohio, United States