Infantile Epileptic Spasms Syndrome, Infantile Spasms, West Syndrome
Conditions
Keywords
Relapse, Infantile Spasms, Prednisolone
Brief summary
After initially successful treatment, many children with infantile spasms unfortunately have a relapse, and relapse is linked to poor long-term outcomes such as autism and other forms of epilepsy. The aim of this study is to determine if treatment with low-dose prednisolone is safe, well tolerated, and effective in reducing the risk of relapse.
Detailed description
In this study, the investigators will enroll infants who have responded to standard therapy for treatment of Infantile Epileptic Spasms Syndrome (IESS). Patients will be randomized to receive either low-dose prednsiolone or placebo for 4 months. During the first 7 months, patients will be evaluated monthly, with clinic visits and electroencephalography (EEG). Patients will then return for a final visit at age 2 years. Key outcomes will be determination of IESS relapse, emergence of other types of seizures/epilepsy, and evaluation of developmental/behavioral status at age 2 years. We will attempt to determine whether or not the prednisolone intervention reduces the risk of any adverse outcome, but this may not be possible given the study design. We will also evaluate the feasibility of the intervention, study procedures, and recruitment.
Interventions
DRUGPrednisolone
active drug
DRUGFamotidine
active drug
DRUGPlacebo
non-active drug
Sponsors
Pediatric Epilepsy Research Foundation
University of California, Los Angeles
Study design
Allocation
RANDOMIZED
Intervention model
FACTORIAL
Primary purpose
PREVENTION
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
Prednisolone (1 mg/kg/day) or placebo AND famotidine (1 mg/kg/day) or placebo. Both medications will be administered daily for 4 months, followed by a 1-month taper
Eligibility
Sex/Gender
ALL
Age
2 Months to 18 Months
Healthy volunteers
No
Inclusion criteria
1. Age 2 to 18 months, inclusive 2. Clinical diagnosis of infantile spasms syndrome, with EEG-confirmed complete response to standard treatment (prednisolone, ACTH, and/or vigabatrin)
Exclusion criteria
1. Presence of clinically significant hypertension, infection, or any other diagnosis which poses unreasonable risk in the setting of extended corticosteroid therapy, in the view of the study physician 2. Exposure to any artisanal cannabinoid product within 14 days of screening 3. Ongoing therapy with the ketogenic diet 4. Implantation of a vagal nerve stimulator within 3 months of screening, or any change in stimulation parameters within 1 month of screening 5. Treatment of IESS via epilepsy surgery
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of treatment-emergent adverse events | From enrollment to 5-month visit. | The investigators will tabulate of adverse events and determine whether any adverse event is associated with prednisolone treatment during the first 5 months of the study. |
| Incidence of epileptic spasms relapse | From enrollment to last evaluation at age 2 years | The investigators will record whether or not study participant experience a relapse of epileptic spasms during the study. Relapse is classified as present or absent and based on interpretation of EEG. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of autism spectrum disorder | From enrollment to last evaluation at age 2 years | At the final study visit, the investigators will use DSM-V criteria to determine if study participants fulfill diagnostic criteria for autism spectrum disorder. |
| Developmental/Behavioral Level | From enrollment to last evaluation at age 2 years | At the final study visit at age 2 years, the investigators will measure adaptive behavior using the Vineland-3 questionnaire. |
Countries
United States
Contacts
Primary ContactShaun A. Hussain, MD, MS
Backup ContactAngela L. Martinez
Outcome results
None listed