Treatment of Refractory Infantile Spasms With Fenfluramine

Phase 2

Recruiting

• Conditions: Infantile Spasm
• Interventions: Drug: Fenfluramine

• Registration Number: NCT04289467
• Lead Sponsor: Children's Hospital of Orange County

Brief Summary

This is a phase II clinical trial in which children with refractory infantile spasms (also called epileptic spasms or West syndrome) will be treated with fenfluramine, to evaluate efficacy, safety, and tolerability. Patients with infantile spasms that have not responded to treatment with vigabatrin and ACTH we will be invited to participate. Study participants will undergo baseline video-EEG, receive treatment with fenfluramine for 21 days, and then undergo repeat video-EEG to determine effectiveness. Patients with favorable response will have the opportunity to continue treatment for up to 6 months.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-02-21
• Study First Submitted QC: 2020-02-26
• Study First Posted: 2020-02-28
• Study Start: 2023-06-16
• Status Verified: 2024-02
• Last Update Submitted: 2024-02-05
• Last Update Posted: 2024-02-07
• Primary Completion: 2024-12-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Children ages 12 to 36 months, inclusive
• Clinical diagnosis of infantile spasms
• Continued epileptic spasms despite adequate treatment with ACTH and vigabatrin.

Exclusion Criteria

• Significant preexisting cardiovascular disease
• Exposure to any cannabinoid product within 14 days of screening
• Initiation or dose-titration of any second-line treatment for infantile spasms in the 14 days prior to screening.
• Implantation of a vagal nerve simulator within 14 days of screening
• Initiation and maintenance of the ketogenic diet within 3 months of screening

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Fenfluramine treatment	Fenfluramine	Open label treatment with fenfluramine. Dosage will be titrated to 0.8 mg/kg/day, for an initial duration of 21 days. Patients with favorable response will have an option to continue treatment for up to 6 months.

Primary Outcome Measures

Name	Time	Method
Electroclinical response (Efficacy)	12 months	Number of participants with resolution of epileptic spasms and hypsarrhythmia (if present at baseline) after 21 days of treatment, as determined by overnight video-electroencephalography (EEG) evaluation and caregiver seizure diary.

Secondary Outcome Measures

Name	Time	Method
Incidence of treatment emergent adverse events (Safety and tolerability)	12 months	Detailed accounting of all treatment-emergent adverse events, including number of participants with clinically-significant valvulopathy and/or pulmonary hypertension, as determined by echocardiography.
Computational electroencephalography response (Efficacy)	12 months	Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden.

Trial Locations

Locations (2)

UCLA Health; 🇺🇸 Los Angeles, California, United States

Children's Hospital of Orange County; 🇺🇸 Orange, California, United States