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

NCT ID: NCT04219995

Last Updated: 2020-02-07

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2020-02-03
• Study Completion Date: 2021-02-28

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

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

Interventional start

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.

Group Type: EXPERIMENTAL

methylprednisolone sodium succinate

Intervention Type: 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.

Placebo

Intervention Type: OTHER

The placebo used in this study will be simple syrup.

Placebo start

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.

Group Type: PLACEBO_COMPARATOR

methylprednisolone sodium succinate

Intervention Type: 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.

Placebo

Intervention Type: OTHER

The placebo used in this study will be simple syrup.

Interventions

methylprednisolone sodium succinate

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.

Intervention Type: DRUG

Placebo

The placebo used in this study will be simple syrup.

Intervention Type: OTHER

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)

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Louisiana State University Health Sciences Center in New Orleans

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Jeremy Toler, MD

Role: PRINCIPAL_INVESTIGATOR

Louisiana State University Health Sciences Center

Locations

Children's Hospital of New Orleans

New Orleans, Louisiana, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Xinran Maria Xiang, MD

Role: CONTACT

xxiang@lsuhsc.edu

5048969283

Facility Contacts

Xinran Xiang, MD

Role: primary

xxiang@lsuhsc.edu

504-896-9283

Other Identifiers

IRB# 19-179

Identifier Type: -

Identifier Source: org_study_id