A Study to Prevent Infantile Spasms Relapse

Phase 2

Recruiting

• Conditions: Infantile Spasms; Infantile Epileptic Spasms Syndrome; West Syndrome
• Interventions: Drug: Placebo; Drug: Prednisolone; Drug: Famotidine

• Registration Number: NCT06819670
• Lead Sponsor: University of California, Los Angeles

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.

Study Timeline

• Study First Submitted: 2025-01-29
• Study First Submitted QC: 2025-02-04
• Study First Posted: 2025-02-11
• Status Verified: 2025-05
• Study Start: 2025-05-05
• Last Update Submitted: 2025-05-05
• Last Update Posted: 2025-05-08
• Primary Completion: 2028-02-28
• Study Completion: 2028-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 40

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: FACTORIAL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Placebo (prednisolone) and placebo (famotidine)
Low-dose prednisolone	Prednisolone	Prednisolone and famotidine.
Low-dose prednisolone	Famotidine	Prednisolone and famotidine.

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.

Trial Locations

Locations (1)

UCLA; 🇺🇸 Los Angeles, California, United States