A Study to Prevent Infantile Spasms Relapse

Phase 2RecruitingNCT06819670

University of California, Los Angeles40 enrolled

Overview

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.

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

PREVENTION

Masking

QUADRUPLE

Enrollment

Conditions

Infantile Spasms Infantile Epileptic Spasms Syndrome West Syndrome

Interventions

PrednisoloneDRUG

active drug

FamotidineDRUG

active drug

PlaceboDRUG

non-active drug

Eligibility

Sex: ALLMin age: 2 MonthsMax age: 18 Months

Medical Language ↔ Plain English

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

Locations (1)

UCLA

Los Angeles, California, United States

RECRUITING

Outcomes

Primary Outcomes

Incidence of treatment-emergent adverse events

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.

Time frame: From enrollment to 5-month visit.

Incidence of epileptic spasms relapse

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.

Time frame: From enrollment to last evaluation at age 2 years

Secondary Outcomes

Incidence of autism spectrum disorder

At the final study visit, the investigators will use DSM-V criteria to determine if study participants fulfill diagnostic criteria for autism spectrum disorder.

Time frame: From enrollment to last evaluation at age 2 years

Developmental/Behavioral Level

At the final study visit at age 2 years, the investigators will measure adaptive behavior using the Vineland-3 questionnaire.

Time frame: From enrollment to last evaluation at age 2 years

Central Contacts

Shaun A. Hussain, MD, MS

CONTACT

310-206-7630 shussain@mednet.ucla.edu

Angela L. Martinez

CONTACT

310-206-7630 angelamartinez@mednet.ucla.edu

Data from ClinicalTrials.gov

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