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NCT Number: NCT04289467

Treatment of Refractory Infantile Spasms With Fenfluramine

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.

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Key information

Conditions

Infantile Spasm Brain Diseases Central Nervous System Diseases Epilepsy Epilepsy, Generalized Epileptic Syndromes Nervous System Diseases Spasms, Infantile

Age range

12 month–36 month

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

UCLA Health, Los Angeles, California, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

fenfluramine

Drug

Open-label

Other names: Fenfluramine Hydrochloride

Primary outcomes

  1. Electroclinical response (Efficacy)

    Time frame: 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 outcomes

  1. Computational electroencephalography response (Efficacy)

    Time frame: 12 months

    Median and range of response quantified using the probability-weighted response index (PWRI), a novel computational electroencephalography measure of hypsarrhythmia burden.

  2. Incidence of treatment emergent adverse events (Safety and tolerability)

    Time frame: 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.

Study contacts

Contact information is provided by the study sponsor or research team.

Kellie Bacon, MPH

CONTACT

[email protected]

714-509-8972

Virginia Allhusen, PhD

CONTACT

[email protected]

Sponsors and collaborators

Lead sponsor

Children's Hospital of Orange County

Other

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Feb 28, 2020
Registry last updated
Apr 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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