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

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ClinicalTrials.gov Identifier: NCT02054949
Recruitment Status : Withdrawn (no eligible subjects located)
First Posted : February 4, 2014
Last Update Posted : October 9, 2015
Sponsor:
Information provided by (Responsible Party):
Barbara Gracious, 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.

Condition or disease Intervention/treatment Phase
Autistic Disorder Seizures Irritability Drug: N-Acetyl Cysteine (NAC) Not Applicable

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


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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Pilot Study of N-acetyl Cysteine for Refractory Generalized Epilepsy in Children With Autism
Study Start Date : April 2013
Actual Primary Completion Date : January 2015
Actual Study Completion Date : January 2015

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Epilepsy Seizures

Arm Intervention/treatment
Experimental: 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.
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.




Primary Outcome Measures :
  1. Recruitment rate [ Time Frame: 1 year ]
    screening to enrollment and retention rates will be measured


Secondary Outcome Measures :
  1. seizure frequency [ Time Frame: up to 8 weeks ]
    seizure count


Other Outcome Measures:
  1. side effect rating form for children and adolescents [ Time Frame: at baseline, 4, and 8 weeks ]
    parent and child will be queried about changes in physical and mental health between study visits using a standardized form.

  2. Aberrant Behavior Checklist [ Time Frame: baseline, 4, and 8 weeks ]
    58 item parent-completed questionnaire

  3. Autism Spectrum Rating Scale [ Time Frame: baseline, 4, and 8 weeks ]
    parent report form on symptoms and behaviors in autism

  4. oxidative stress measures [ Time Frame: baseline, 4, and 8 weeks ]
    blood is collected and analyzed for oxidative changes in lipids, proteins.



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02054949


Locations
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United States, Ohio
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Sponsors and Collaborators
Nationwide Children's Hospital
Investigators
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Principal Investigator: Barbara Gracious, MD Nationwide Children's Hospital

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Responsible Party: Barbara Gracious, Principal Investigator, Nationwide Children's Hospital
ClinicalTrials.gov Identifier: NCT02054949     History of Changes
Other Study ID Numbers: IRB12-00325
First Posted: February 4, 2014    Key Record Dates
Last Update Posted: October 9, 2015
Last Verified: October 2015

Keywords provided by Barbara Gracious, Nationwide Children's Hospital:
N-acetyl cysteine
Refractory Seizures

Additional relevant MeSH terms:
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Acetylcysteine
N-monoacetylcystine
Epilepsy
Seizures
Autistic Disorder
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Neurologic Manifestations
Signs and Symptoms
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Antiviral Agents
Anti-Infective Agents
Expectorants
Respiratory System Agents
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antidotes