A Study to Prevent Infantile Spasms Relapse

A Multicenter Pilot Clinical Trial to Prevent Infantile Spasms Relapse

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.

Study Overview

• Status: Recruiting
• Conditions: Infantile Spasms; West Syndrome; Infantile Epileptic Spasms Syndrome
• Intervention / Treatment: Drug: Prednisolone; Drug: Famotidine; Drug: Placebo

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 Type: Interventional
• Enrollment (Estimated): 40
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Angela L. Martinez; Phone Number: 310-206-7630; Email: angelamartinez@mednet.ucla.edu
• Study Contact Backup: Name: Shaun A. Hussain, MD, MS; Phone Number: 310-206-7630; Email: shussain@mednet.ucla.edu

Study Locations

• United States
  • California
    • Los Angeles, California, United States, 90095
      • Recruiting
      • UCLA
      • Contact: Angela Martinez; Phone Number: 3102067630; Email: angelamartinez@mednet.ucla.edu
      • Principal Investigator: Shaun Hussain, MD, MS

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: Randomized
• Interventional Model: Factorial Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Low-dose prednisolone; Prednisolone and famotidine.	Drug: Prednisolone; active drug; Drug: Famotidine; active drug
Placebo Comparator: Placebo; Placebo (prednisolone) and placebo (famotidine)	Drug: Placebo; non-active drug

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	From enrollment to last evaluation at age 2 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	From enrollment to last evaluation at age 2 years

Collaborators and Investigators

• Sponsor: University of California, Los Angeles
• Collaborators: Pediatric Epilepsy Research Foundation
• Investigators: Principal Investigator: Shaun A. Hussain, MD, MS, University of California, Los Angeles

Publications and helpful links

General Publications

• Hussain SA. Treatment of infantile spasms. Epilepsia Open. 2018 Oct 23;3(Suppl Suppl 2):143-154. doi: 10.1002/epi4.12264. eCollection 2018 Dec.

Helpful Links

• Related Info

Study record dates

Study Major Dates

• Study Start (Actual): May 5, 2025
• Primary Completion (Estimated): February 28, 2028
• Study Completion (Estimated): June 30, 2028

Study Registration Dates

• First Submitted: January 29, 2025
• First Submitted That Met QC Criteria: February 4, 2025
• First Posted (Actual): February 11, 2025

Study Record Updates

• Last Update Posted (Actual): May 8, 2025
• Last Update Submitted That Met QC Criteria: May 5, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: Prednisolone; Relapse; Infantile Spasms
• Additional Relevant MeSH Terms: Epileptic Syndromes; Neurologic Manifestations; Musculoskeletal Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Manifestations; Pathologic Processes; Disease Attributes; Disease; Epilepsy, Generalized; Epilepsy; Syndrome; Recurrence; Spasm; Muscle Cramp; Spasms, Infantile; Antineoplastic Agents; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Inflammatory Agents; Gastrointestinal Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Histamine Antagonists; Histamine Agents; Neurotransmitter Agents; Anti-Ulcer Agents; Histamine H2 Antagonists; Prednisolone; Famotidine
• Other Study ID Numbers: 22-001710; 20224426 (Other Grant/Funding Number: Pediatric Epilepsy Research Foundation)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: Deidentified study data will be shared with other researchers upon reasonable request, after publication of results.
• IPD Sharing Time Frame: IPD will be shared upon reasonable request after publication of study results.
• IPD Sharing Access Criteria: Deidentified patient data, study protocol, statistical analysis plan, informed consent form, clinical study report, and analytic code.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; ANALYTIC_CODE; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No