Pilot Trial to Evaluate The Effect of Oral Methylprednisolone on Seizure Frequency in Children With Epilepsy

NCT04219995 | Unknown Phase 1 FDA Drug

• 1 other identifier: IRB# 19-179
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Intractable Epilepsy; Convulsive Seizures

Outcome Measures

Primary Outcomes (1)

• Seizure frequency

  The percentage of patients with 50% or more reduction in seizure frequency.

  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 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

  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

  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).

Study Arms (2)

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.

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.

Drug: methylprednisolone sodium succinate; Other: Placebo

Interventions

methylprednisolone sodium succinate DRUG

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.

Interventional start; Placebo start

Placebo OTHER

The placebo used in this study will be simple syrup.

Interventional start; Placebo start

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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
• a. 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

You may not qualify if:

• Patients with history of the following diagnoses:
• Traumatic brain injury
• Tuberous sclerosis
• Sturge Weber
• Cortical dysplasia
• Patients with known hereditary degenerative diseases as follows:
• 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

Sponsors & Collaborators

• Louisiana State University Health Sciences Center in New Orleans: lead

Study Sites (1)

Children's Hospital of New Orleans

New Orleans, Louisiana, 70118, United States

RECRUITING

MeSH Terms

Conditions

Drug Resistant Epilepsy; Seizures

Interventions

Methylprednisolone Hemisuccinate

Condition Hierarchy (Ancestors)

Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Methylprednisolone; Prednisolone; Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Study Officials

• Jeremy Toler, MD

  Louisiana State University Health Sciences Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xinran Maria Xiang, MD

CONTACT

5048969283; xxiang@lsuhsc.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 2, 2020
• First Posted: January 7, 2020
• Study Start: February 3, 2020
• Primary Completion: February 1, 2021
• Study Completion: February 1, 2021
• Last Updated: February 7, 2020

Record last verified: 2020-02

Locations

US (1)