Treatment Resistant Epilepsy and N-Acetyl Cysteine

Seizures Clinical Trial

— TRE-NAC  

Official title:

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

Clinical Trial Details — Status: Withdrawn

Administrative data

• NCT number: NCT02054949
• Other study ID #: IRB12-00325
• Status: Withdrawn
• Phase: N/A
• First received: February 3, 2014
• Last updated: October 7, 2015
• Start date: April 2013
• Est. completion date: January 2015

Study information

• Verified date: October 2015
• Source: Nationwide Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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

Recruitment information / eligibility

• Status: Withdrawn
• Enrollment: 0
• Est. completion date: January 2015
• Est. primary completion date: January 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 6 Years to 21 Years

Eligibility

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

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autistic Disorder
• Irritability
• Seizures

Intervention

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.

Locations

Country	Name	City	State
United States	Nationwide Children's Hospital	Columbus	Ohio

Sponsors (1)

Lead Sponsor	Collaborator
Nationwide Children's Hospital

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	side effect rating form for children and adolescents	parent and child will be queried about changes in physical and mental health between study visits using a standardized form.	at baseline, 4, and 8 weeks	Yes
Other	Aberrant Behavior Checklist	58 item parent-completed questionnaire	baseline, 4, and 8 weeks	No
Other	Autism Spectrum Rating Scale	parent report form on symptoms and behaviors in autism	baseline, 4, and 8 weeks	No
Other	oxidative stress measures	blood is collected and analyzed for oxidative changes in lipids, proteins.	baseline, 4, and 8 weeks	No
Primary	Recruitment rate	screening to enrollment and retention rates will be measured	1 year	No
Secondary	seizure frequency	seizure count	up to 8 weeks	Yes