Clinical trial NCT04219995: Pilot Trial to Evaluate The Effect of Oral Methylprednisolone on Seizure Frequency in Children With Epilepsy
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
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
PHASE2
Conditions: Intractable Epilepsy
Convulsive Seizures
Lead sponsor: Louisiana State University Health Sciences Center in New Orleans
Sponsor class: OTHER
First posted: 2020-01-07
Enrollment: 10
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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. 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.


Eligibility criteria: Inclusion Criteria:

1. Patients age 2 -18 years of age
2. 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

   a. Epilepsy diagnosed by historical clinical evidence
3. Family's ability to understand and willingness to sign a written informed consent document for patients under 18.
4. Willingness to complete seizure diary for duration of study
5. Willingness to present to all study visits

Exclusion Criteria:

1. Patients with history of the following diagnoses:

   1. Traumatic brain injury
   2. Tuberous sclerosis
   3. Sturge Weber
   4. Cortical dysplasia
2. Patients with known hereditary degenerative diseases as follows:

   1. Adrenoleukodystrophy
   2. Neuronal ceroid lipofuscinosis
   3. Leigh Syndrome
   4. Myoclonic epilepsy with ragged red fibers (MERRF)
   5. Rett Syndrome
3. Patients with the following epilepsy syndromes

   1. Infantile spasms
   2. West Syndrome
   3. Progressive myoclonic epilepsy
   4. Dravet syndrome
   5. Doose syndrome
   6. Ohtahara syndrome
   7. Rasmussen's encephalitis
4. Patients with the following metabolic disorders

   1. Phenylketonuria
   2. Maple syrup urine disease
   3. Organic acidemias
   4. Galactosemia
   5. Peroxismal disorders (e.g. Zellwegers)
   6. Lysosomal disorders
   7. Urea cycle disorders
5. Patients with history of immunodeficiency
6. Patients with the following infections

   1. HIV/AIDS
   2. Active or latent TB
   3. Active or suspected bacterial infection
   4. Active, latent or suspected fungemia
   5. Active or suspected parasitic infection
7. Patients with history of malignancy
8. Patients with history of or active myopathy
9. Patients with degenerative neuromuscular disorders
10. Patients with history of hypersensitivity or allergic reactions to corticosteroids
11. Patients with history of psychosis
12. Patients with diabetes mellitus
13. Pregnancy
14. Any underlying predisposition to gastrointestinal bleeding (peptic ulcer disease, gastritis, colitis)

Primary outcomes:
measure: Seizure frequency
timeFrame: 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:
measure: Seizure freedom
timeFrame: 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).
measure: Adverse events
timeFrame: 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).
measure: Drop out percentage
timeFrame: 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).
Other outcomes:
Locations:
city: New Orleans
country: United States
facility: Children's Hospital of New Orleans
state: Louisiana
Study officials:
affiliation: Louisiana State University Health Sciences Center
name: Jeremy Toler, MD
role: PRINCIPAL_INVESTIGATOR
References: