Treatment of Refractory Infantile Spasms With Fenfluramine

Infantile Spasm Clinical Trial

Official title:

A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04289467
• Other study ID #: ZX008-IS
• Status: Recruiting
• Phase: Phase 2
• Start date: June 16, 2023
• Est. completion date: December 31, 2025

Study information

• Verified date: February 2024
• Source: Children's Hospital of Orange County
• Contact: Angelyque Lorenzana, MA
• Phone: 714-509-8972
• Email: alorenzana[@]choc.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 10
• Est. completion date: December 31, 2025
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Months to 36 Months

Eligibility

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

Related Conditions & MeSH terms

• Infantile Spasm
• Spasm
• Spasms, Infantile

Intervention

Drug:
• Fenfluramine
Open-label

Locations

Country	Name	City	State
United States	UCLA Health	Los Angeles	California
United States	Children's Hospital of Orange County	Orange	California

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Orange County

Country where clinical trial is conducted

United States, 

Outcome

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