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Treatment Resistant Epilepsy and N-Acetyl Cysteine (TRE-NAC)

Primary Purpose

Autistic Disorder, Seizures, Irritability

Status
Withdrawn
Phase
Not Applicable
Locations
United States
Study Type
Interventional
Intervention
N-Acetyl Cysteine (NAC)
Sponsored by
Nationwide Children's Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autistic Disorder focused on measuring N-acetyl cysteine, Refractory Seizures

Eligibility Criteria

6 Years - 21 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Nationwide Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

N-Acetyl Cysteine (NAC)

Arm Description

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.

Outcomes

Primary Outcome Measures

Recruitment rate
screening to enrollment and retention rates will be measured

Secondary Outcome Measures

seizure frequency
seizure count

Full Information

First Posted
February 3, 2014
Last Updated
October 7, 2015
Sponsor
Nationwide Children's Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT02054949
Brief Title
Treatment Resistant Epilepsy and N-Acetyl Cysteine
Acronym
TRE-NAC
Official Title
Pilot Study of N-acetyl Cysteine for Refractory Generalized Epilepsy in Children With Autism
Study Type
Interventional

2. Study Status

Record Verification Date
October 2015
Overall Recruitment Status
Withdrawn
Why Stopped
no eligible subjects located
Study Start Date
April 2013 (undefined)
Primary Completion Date
January 2015 (Actual)
Study Completion Date
January 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Nationwide Children's Hospital

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autistic Disorder, Seizures, Irritability
Keywords
N-acetyl cysteine, Refractory Seizures

7. Study Design

Primary Purpose
Treatment
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)

8. Arms, Groups, and Interventions

Arm Title
N-Acetyl Cysteine (NAC)
Arm Type
Experimental
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
N-Acetyl Cysteine (NAC)
Intervention Description
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 Measure Information:
Title
Recruitment rate
Description
screening to enrollment and retention rates will be measured
Time Frame
1 year
Secondary Outcome Measure Information:
Title
seizure frequency
Description
seizure count
Time Frame
up to 8 weeks
Other Pre-specified Outcome Measures:
Title
side effect rating form for children and adolescents
Description
parent and child will be queried about changes in physical and mental health between study visits using a standardized form.
Time Frame
at baseline, 4, and 8 weeks
Title
Aberrant Behavior Checklist
Description
58 item parent-completed questionnaire
Time Frame
baseline, 4, and 8 weeks
Title
Autism Spectrum Rating Scale
Description
parent report form on symptoms and behaviors in autism
Time Frame
baseline, 4, and 8 weeks
Title
oxidative stress measures
Description
blood is collected and analyzed for oxidative changes in lipids, proteins.
Time Frame
baseline, 4, and 8 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
21 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Children and adolescents/young adults ages 6-21, male or female, all races and ethnicities. Autism IQ less than 80 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. Children with at least 1 grand mal/generalized seizure per week. Exclusion Criteria: Inability to swallow capsules In females, pregnancy or sexual activity Daily acetaminophen, glucocorticoid, or nonsteroidal anti-inflammatory drugs, or daily NAC or high-dose antioxidant vitamin supplements within 30 days of baseline. History of acute or chronic liver, renal, endocrine, infectious, autoimmune, hematologic, metabolic, or other disorder in the judgement of the study physician.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Barbara Gracious, MD
Organizational Affiliation
Nationwide Children's Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Nationwide Children's Hospital
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43205
Country
United States

12. IPD Sharing Statement

Learn more about this trial

Treatment Resistant Epilepsy and N-Acetyl Cysteine

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