A Study to Prevent Infantile Spasms Relapse

NCT06819670 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 22-00171020224426
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Infantile Spasms; Infantile Epileptic Spasms Syndrome; West Syndrome

Keywords

Relapse; Infantile Spasms; Prednisolone

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (2)

• Incidence of autism spectrum disorder

  From enrollment to last evaluation at age 2 years

• Developmental/Behavioral Level

  From enrollment to last evaluation at age 2 years

Study Arms (2)

Low-dose prednisolone

ACTIVE COMPARATOR

Prednisolone and famotidine.

Drug: Prednisolone; Drug: Famotidine

Placebo

PLACEBO COMPARATOR

Placebo (prednisolone) and placebo (famotidine)

Drug: Placebo

Interventions

Prednisolone DRUG

active drug

Low-dose prednisolone

Famotidine DRUG

active drug

Low-dose prednisolone

Placebo DRUG

non-active drug

Placebo

Eligibility Criteria

• Age: 2 Months - 18 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age 2 to 18 months, inclusive
• Clinical diagnosis of infantile spasms syndrome, with EEG-confirmed complete response to standard treatment (prednisolone, ACTH, and/or vigabatrin)

You may not qualify if:

• 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
• Exposure to any artisanal cannabinoid product within 14 days of screening
• Ongoing therapy with the ketogenic diet
• Implantation of a vagal nerve stimulator within 3 months of screening, or any change in stimulation parameters within 1 month of screening
• Treatment of IESS via epilepsy surgery

Sponsors & Collaborators

• University of California, Los Angeles: lead
• Pediatric Epilepsy Research Foundation: collaborator

Study Sites (1)

UCLA

Los Angeles, California, 90095, United States

RECRUITING

Related Publications (1)

• 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.

  PMID: 30564773 BACKGROUND

Related Links

• Related Info

MeSH Terms

Conditions

Spasms, Infantile; Recurrence

Interventions

Prednisolone; Famotidine

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

Pregnadienetriols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Thiazoles; Sulfur Compounds; Organic Chemicals; Azoles; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds

Study Officials

• Shaun A. Hussain, MD, MS

  University of California, Los Angeles

  PRINCIPAL INVESTIGATOR

Central Study 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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: FACTORIAL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: 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

Study Record Dates

• First Submitted: January 29, 2025
• First Posted: February 11, 2025
• Study Start: May 5, 2025
• Primary Completion (Estimated): February 28, 2028
• Study Completion (Estimated): June 30, 2028
• Last Updated: May 8, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: IPD will be shared upon reasonable request after publication of study results.
• Access Criteria: Deidentified patient data, study protocol, statistical analysis plan, informed consent form, clinical study report, and analytic code.

Locations

US (1)