Autistic DisorderTRE-NACNCT02054949
Treatment Resistant Epilepsy and N-Acetyl Cysteine
A study testing N-Acetyl Cysteine (NAC) in people with Autistic Disorder and Seizures, run by Nationwide Children's Hospital at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 6 to 21 years
- Conditions the study is for
- Autistic Disorder, Seizures, Irritability
- What is being tested?
- N-Acetyl Cysteine (NAC)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2013to Jan 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Nationwide Children's Hospital
Columbus, Ohio, 43205
About this study
Official title: Pilot Study of N-acetyl Cysteine for Refractory Generalized Epilepsy in Children With Autism
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.
Read the 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
- Conditions
- Autistic Disorder, Seizures, Irritability
- Being tested
- N-Acetyl Cysteine (NAC)
- Keywords
- N-acetyl cysteine, Refractory Seizures
- Medical subject headings
- Autistic Disorder, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
N-Acetyl Cysteine (NAC)Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| N-Acetyl Cysteine (NAC) | Experimental | 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. Receives: Drug: N-Acetyl Cysteine (NAC) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Recruitment rate
screening to enrollment and retention rates will be measured
Measured: 1 year
Secondary outcomes (1)
seizure frequency
seizure count
Measured: up to 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2014 (estimated)
- Study startApr 2013
- Primary completionJan 2015
- Study completionJan 2015
Why it stopped: no eligible subjects located
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autistic DisorderStudies of the same condition, open and under way.
- More trials run by Nationwide Children's HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nationwide Children's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Barbara Gracious, Nationwide Children's Hospital
- Investigators
- Barbara Gracious, MD, Principal investigator, Nationwide Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02054949
- Sponsor's protocol ID
- IRB12-00325
- Acronym
- TRE-NAC
- Status last verified
- Oct 2015
- First submitted
- 3 Feb 2014
- Last updated
- 9 Oct 2015
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 9 Oct 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of N-acetyl Cysteine for Refractory Generalized Epilepsy in Children With Autism
- ClinicalTrials.gov ID
- NCT02054949
- Acronym / sponsor ID
- TRE-NAC / IRB12-00325
- Conditions
- Autistic Disorder, Seizures, Irritability
- Keywords
- N-acetyl cysteine, Refractory Seizures
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: no eligible subjects located
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Nationwide Children's HospitalUniversity, hospital or other
- Study start
- Apr 2013
- Primary completion
- Jan 2015 (actual)
- Study completion
- Jan 2015 (actual)
- First posted
- 4 Feb 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| N-Acetyl Cysteine (NAC) | Experimental | 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. |
Who may qualify
- Ages
- 6 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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) |
Primary outcome
Recruitment rate
Time frame: 1 year
screening to enrollment and retention rates will be measured
1 secondary outcome
seizure frequency
Time frame: up to 8 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Oct 2015.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Barbara Gracious, MD principal investigator
Nationwide Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing N-Acetyl Cysteine (NAC) in people with Autistic Disorder and Seizures, run by Nationwide Children's Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 9 Oct 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Recruitment rate (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Oct 2015.