RecruitingPhase 2NCT04289467

Treatment of Refractory Infantile Spasms With Fenfluramine

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


Sponsor

Children's Hospital of Orange County

Enrollment

10 participants

Start Date

Jun 16, 2023

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 12 MonthsMax Age: 36 Months

Inclusion Criteria3

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

Exclusion Criteria5

  • 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

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Interventions

DRUGFenfluramine

Open-label


Locations(2)

UCLA Health

Los Angeles, California, United States

Children's Hospital of Orange County

Orange, California, United States

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NCT04289467


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