Treatment of Refractory Infantile Spasms With Fenfluramine

Children's Health of Orange County

Status and phase: Enrolling; Phase 2

Conditions: Infantile Spasm

Treatments: Drug: Fenfluramine

Study type: Interventional

Funder types: Other

Identifiers

NCT04289467

ZX008-IS

Details and patient eligibility

About

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.

Enrollment: 10 estimated patients

Sex: All

Ages: 12 to 36 months old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Fenfluramine treatment

Experimental group

Description:

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.

Treatment:

Drug: Fenfluramine