Clinical trial NCT02054949: Treatment Resistant Epilepsy and N-Acetyl Cysteine
Official title: Pilot Study of N-acetyl Cysteine for Refractory Generalized Epilepsy in Children With Autism
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: NA
Conditions: Autistic Disorder
Seizures
Irritability
Keywords: N-acetyl cysteine
Refractory Seizures
Lead sponsor: Nationwide Children's Hospital
Sponsor class: OTHER
First posted: 2014-02-04
Enrollment: 0
Sex: ALL
Minimum age (years): 6
Maximum age (years): 21
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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

Eligibility criteria: 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.

Primary outcomes:
measure: Recruitment rate
timeFrame: 1 year
Secondary outcomes:
measure: seizure frequency
timeFrame: up to 8 weeks
Other outcomes:
measure: side effect rating form for children and adolescents
timeFrame: at baseline, 4, and 8 weeks
measure: Aberrant Behavior Checklist
timeFrame: baseline, 4, and 8 weeks
measure: Autism Spectrum Rating Scale
timeFrame: baseline, 4, and 8 weeks
measure: oxidative stress measures
timeFrame: baseline, 4, and 8 weeks
Locations:
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Nationwide Children's Hospital
name: Barbara Gracious, MD
role: PRINCIPAL_INVESTIGATOR
References: