Intractable EpilepsyPhase 1/2NCT04219995
Pilot Trial to Evaluate The Effect of Oral Methylprednisolone on Seizure Frequency in Children With Epilepsy
A Phase 1/2 study testing methylprednisolone sodium succinate in people with Intractable Epilepsy and Convulsive Seizures, run by Louisiana State University Health Sciences Center in New Orleans at 1 site in United States.
Trial summary
Although corticosteroids have been shown to be beneficial anecdotally for refractory epilepsy, the effects of corticosteroids on pediatric epilepsy have primarily been studied retrospectively amongst a heterogeneous patient population. Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Intractable Epilepsy, Convulsive Seizures
- What is being tested?
- methylprednisolone sodium succinate
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 3 Feb 2020to Feb 2021
- Phase
- Phase 1/2
- 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 2 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients age 2 -18 years of age
- Patients who have at least 4 convulsive (generalized tonic or tonic-clonic) seizures per month on 2 or more anti-epileptic drugs (AEDs) at therapeutic doses
- Epilepsy diagnosed by historical clinical evidence
- Family's ability to understand and willingness to sign a written informed consent document for patients under 18.
- Willingness to complete seizure diary for duration of study
- Willingness to present to all study visits
Who cannot join
- Patients with history of the following diagnoses:
- Traumatic brain injury
- Tuberous sclerosis
- Sturge Weber
- Cortical dysplasia
- Patients with known hereditary degenerative diseases as follows:
Show all 42Show fewer
- Adrenoleukodystrophy
- Neuronal ceroid lipofuscinosis
- Leigh Syndrome
- Myoclonic epilepsy with ragged red fibers (MERRF)
- Rett Syndrome
- Patients with the following epilepsy syndromes
- Infantile spasms
- West Syndrome
- Progressive myoclonic epilepsy
- Dravet syndrome
- Doose syndrome
- Ohtahara syndrome
- Rasmussen's encephalitis
- Patients with the following metabolic disorders
- Phenylketonuria
- Maple syrup urine disease
- Organic acidemias
- Galactosemia
- Peroxismal disorders (e.g. Zellwegers)
- Lysosomal disorders
- Urea cycle disorders
- Patients with history of immunodeficiency
- Patients with the following infections
- HIV/AIDS
- Active or latent TB
- Active or suspected bacterial infection
- Active, latent or suspected fungemia
- Active or suspected parasitic infection
- Patients with history of malignancy
- Patients with history of or active myopathy
- Patients with degenerative neuromuscular disorders
- Patients with history of hypersensitivity or allergic reactions to corticosteroids
- Patients with history of psychosis
- Patients with diabetes mellitus
- Pregnancy
- Any underlying predisposition to gastrointestinal bleeding (peptic ulcer disease, gastritis, colitis)
Trial locations and contacts
1 site in 1 country.
United States1 site, 1 recruiting
- Recruiting
Children's Hospital of New Orleans
New Orleans, Louisiana, 70118
Xinran Xiang, MD, 504-896-9283, xxiang@lsuhsc.edu
Jeremy Toler, MD
About this study
Official title: Pilot Randomized, Placebo-Controlled Trial to Evaluate The Effect of Oral Pulsed Methylprednisolone on Seizure Frequency in Pediatric Patients With Idiopathic Intractable Convulsive Epilepsy
Although corticosteroids have been shown to be beneficial anecdotally for refractory epilepsy, the effects of corticosteroids on pediatric epilepsy have primarily been studied retrospectively amongst a heterogeneous patient population. The objective of this prospective cross-over study is to determine the effect of oral steroids on convulsive seizure frequency and evaluate the tolerability of pulsed oral steroids. Participants will be prospectively enrolled from pediatric neurology clinic at Children's Hospital of New Orleans, and baseline seizure frequency will be assessed. Participants will then be randomized to receive either the study drug, methylprednisolone, or placebo during the first month, followed by a one-month wash-out period. During the third month of the study, participants will enter the cross-over phase of the study where those who received methylprednisolone will receive placebo, and those who received placebo will receive methylprednisolone. The primary outcome will be the percentage of patients with 50% or more reduction in seizure frequency following one course of oral methylprednisolone. frequency following 1 course of oral methylprednisolone.
- Conditions
- Intractable Epilepsy, Convulsive Seizures
- Being tested
- methylprednisolone sodium succinate, Placebo
- Medical subject headings
- Drug Resistant Epilepsy, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
methylprednisolone sodium succinateDrug
Methylprednisolone sodium succinate will be re-constituted in simple syrup in a concentration of 80mg/mL and will be administered orally at 20mg/kg (max 1000mg) for days 1, 2, and 3 of the intervention phase of the study.
PlaceboOther
The placebo used in this study will be simple syrup.
| Study group | Role | What they receive |
|---|---|---|
| Interventional start | Experimental | Patients who randomize to the interventional start arm will receive the study drug, methylprednisolone sodium succinate, in the first month of the study, followed by placebo in the cross-over phase of the study. Receives: Drug: methylprednisolone sodium succinate; Other: Placebo |
| Placebo start | Placebo comparator | Patients who randomize to the placebo start arm will receive placebo in the first month of the study, followed by the study drug, methylprednisolone sodium succinate, in the cross-over phase of the study. Receives: Drug: methylprednisolone sodium succinate; Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Seizure frequency
The percentage of patients with 50% or more reduction in seizure frequency.
Measured: The number of seizures per month will be assessed at 1 month (following placebo/intervention), 2 months (following wash-out), and 3 months (following cross-over phase of placebo/intervention). Change compared to baseline will be calculated.
Secondary outcomes (3)
Seizure freedom
The percentage of participants who become seizure free over 1 month
Measured: The number of seizures per month will be assessed at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).
Adverse events
Percentage of participants who reports adverse events and the severity of the adverse events.
Measured: Adverse events will be recorded at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).
Drop out percentage
Percentage of participants who drop out of the study due to adverse events
Measured: The number of participants who drop out will be counted and recorded at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2020
- Study start3 Feb 2020
- Primary completionFeb 2021 (estimated)
- Study completionFeb 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Intractable EpilepsyStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Louisiana State University Health Sciences Center in New Orleans
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeremy Toler, MD, Principal investigator, Louisiana State University Health Sciences Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04219995
- Sponsor's protocol ID
- IRB# 19-179
- Status last verified
- Feb 2020
- First submitted
- 2 Jan 2020
- Last updated
- 7 Feb 2020
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 7 Feb 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Randomized, Placebo-Controlled Trial to Evaluate The Effect of Oral Pulsed Methylprednisolone on Seizure Frequency in Pediatric Patients With Idiopathic Intractable Convulsive Epilepsy
- ClinicalTrials.gov ID
- NCT04219995
- Acronym / sponsor ID
- IRB# 19-179
- Conditions
- Intractable Epilepsy, Convulsive Seizures
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Louisiana State University Health Sciences Center in New OrleansUniversity, hospital or other
- Study start
- 3 Feb 2020 (actual)
- Primary completion
- Feb 2021 (estimated)
- Study completion
- Feb 2021 (estimated)
- First posted
- 7 Jan 2020 (actual)
- 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.
Although corticosteroids have been shown to be beneficial anecdotally for refractory epilepsy, the effects of corticosteroids on pediatric epilepsy have primarily been studied retrospectively amongst a heterogeneous patient population. The objective of this prospective cross-over study is to determine the effect of oral steroids on convulsive seizure frequency and evaluate the tolerability of pulsed oral steroids. Participants will be prospectively enrolled from pediatric neurology clinic at Children's Hospital of New Orleans, and baseline seizure frequency will be assessed. Participants will then be randomized to receive either the study drug, methylprednisolone, or placebo during the first month, followed by a one-month wash-out period. During the third month of the study, participants will enter the cross-over phase of the study where those who received methylprednisolone will receive placebo, and those who received placebo will receive methylprednisolone. The primary outcome will be the percentage of patients with 50% or more reduction in seizure frequency following one course of oral methylprednisolone. frequency following 1 course of oral methylprednisolone.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Interventional start | Experimental | Drug: methylprednisolone sodium succinate, Other: PlaceboPatients who randomize to the interventional start arm will receive the study drug, methylprednisolone sodium succinate, in the first month of the study, followed by placebo in the cross-over phase of the study. |
| Placebo start | Placebo comparator | Drug: methylprednisolone sodium succinate, Other: PlaceboPatients who randomize to the placebo start arm will receive placebo in the first month of the study, followed by the study drug, methylprednisolone sodium succinate, in the cross-over phase of the study. |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Patients age 2 -18 years of age
- Patients who have at least 4 convulsive (generalized tonic or tonic-clonic) seizures per month on 2 or more anti-epileptic drugs (AEDs) at therapeutic doses
- Family's ability to understand and willingness to sign a written informed consent document for patients under 18.
- Willingness to complete seizure diary for duration of study
- Willingness to present to all study visits
Key exclusion criteria (42 listed)
- Patients with history of the following diagnoses:
- Patients with known hereditary degenerative diseases as follows:
- Patients with the following epilepsy syndromes
- Patients with the following metabolic disorders
- Patients with history of immunodeficiency
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 |
|---|---|---|
| methylprednisolone sodium succinateMethylprednisolone sodium succinate will be re-constituted in simple syrup in a concentration of 80mg/mL and will be administered orally at 20mg/kg (max 1000mg) for days 1, 2, and 3 of the intervention phase of the study. | Drug | Interventional start, Placebo start |
| PlaceboThe placebo used in this study will be simple syrup. | Other | Interventional start, Placebo start |
Primary outcome
Seizure frequency
Time frame: The number of seizures per month will be assessed at 1 month (following placebo/intervention), 2 months (following wash-out), and 3 months (following cross-over phase of placebo/intervention). Change compared to baseline will be calculated.
The percentage of patients with 50% or more reduction in seizure frequency.
3 secondary outcomes
Seizure freedom
Time frame: The number of seizures per month will be assessed at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).
Adverse events
Time frame: Adverse events will be recorded at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).
Drop out percentage
Time frame: The number of participants who drop out will be counted and recorded at 1 month (following placebo/intervention), 2 months (following wash-out period), and 3 months (following cross-over phase of placebo/intervention).
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Feb 2020.
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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.
Jeremy Toler, MD principal investigator
Louisiana State University Health Sciences Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing methylprednisolone sodium succinate in people with Intractable Epilepsy and Convulsive Seizures, run by Louisiana State University Health Sciences Center in New Orleans 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 6 inclusion and 42 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: status not confirmed, as of the sponsor's last update on 7 Feb 2020. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Xinran Maria Xiang, MD (5048969283, xxiang@lsuhsc.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Seizure frequency (time frame: The number of seizures per month will be assessed at 1 month (following placebo/intervention), 2 months (following wash-out), and 3 months (following cross-over phase of placebo/intervention). Change compared to baseline will be calculated.). 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 7 Feb 2020.