Treatment of Refractory Infantile Spasms With Fenfluramine

NCT04289467 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ZX008-IS
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 2

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.

Conditions

Infantile Spasm

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (2)

• Computational electroencephalography response (Efficacy)

  12 months

• Incidence of treatment emergent adverse events (Safety and tolerability)

  12 months

Study Arms (1)

Fenfluramine treatment

EXPERIMENTAL

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.

Drug: Fenfluramine

Interventions

Fenfluramine DRUG

Open-label

Also known as: Fenfluramine Hydrochloride

Fenfluramine treatment

Eligibility Criteria

• Age: 12 Months - 36 Months
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital of Orange County: lead

Study Sites (2)

UCLA Health

Los Angeles, California, 90095, United States

NOT YET RECRUITING

Children's Hospital of Orange County

Orange, California, 92868, United States

RECRUITING

MeSH Terms

Conditions

Spasms, Infantile

Interventions

Fenfluramine

Condition Hierarchy (Ancestors)

Epilepsy, Generalized; Epilepsy; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epileptic Syndromes

Intervention Hierarchy (Ancestors)

Phenethylamines; Ethylamines; Amines; Organic Chemicals

Study Officials

• Daniel Shrey, MD

  Children's Hospital of Orange County

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Kellie Bacon, MPH

CONTACT

714-509-8972; kellie.bacon@choc.org

Virginia Allhusen, PhD

CONTACT

vallhusen@choc.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: February 21, 2020
• First Posted: February 28, 2020
• Study Start: June 16, 2023
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: April 15, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)