A Study to Prevent Infantile Spasms Relapse

University of California, Los Angeles (UCLA)

Status and phase: Enrolling; Phase 2

Conditions: Infantile Epileptic Spasms Syndrome; Infantile Spasms; West Syndrome

Treatments: Drug: Placebo; Drug: Prednisolone; Drug: Famotidine

Study type: Interventional

Funder types: Other

Identifiers

NCT06819670

20224426 (Other Grant/Funding Number)

22-001710

Details and patient eligibility

About

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.

Full 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.

Enrollment: 40 estimated patients

Sex: All

Ages: 2 to 18 months old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Factorial Assignment

Masking: Quadruple Blind

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40 participants in 2 patient groups, including a placebo group

Low-dose prednisolone

Active Comparator group

Description:

Prednisolone and famotidine.

Treatment:

Drug: Famotidine

Drug: Prednisolone

Placebo

Placebo Comparator group

Description:

Placebo (prednisolone) and placebo (famotidine)

Treatment:

Drug: Placebo