Clinical trial NCT06819670: A Study to Prevent Infantile Spasms Relapse
Official title: A Multicenter Pilot Clinical Trial to Prevent Infantile Spasms Relapse
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Infantile Spasms
Infantile Epileptic Spasms Syndrome
West Syndrome
Keywords: Relapse
Infantile Spasms
Prednisolone
Lead sponsor: University of California, Los Angeles
Sponsor class: OTHER
Collaborators: name: Pediatric Epilepsy Research Foundation
First posted: 2025-02-11
Enrollment: 40
Sex: ALL
Minimum age (years): 2
Maximum age (years): 18
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: FACTORIAL
Intervention model description: 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
Primary purpose: PREVENTION

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.

Eligibility criteria: 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

Primary outcomes:
measure: Incidence of treatment-emergent adverse events
timeFrame: From enrollment to 5-month visit.
measure: Incidence of epileptic spasms relapse
timeFrame: From enrollment to last evaluation at age 2 years
Secondary outcomes:
measure: Incidence of autism spectrum disorder
timeFrame: From enrollment to last evaluation at age 2 years
measure: Developmental/Behavioral Level
timeFrame: From enrollment to last evaluation at age 2 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA
state: California
Study officials:
affiliation: University of California, Los Angeles
name: Shaun A. Hussain, MD, MS
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.