Recruiting
Phase 2

Fenfluramine

Sponsor:

Children's Hospital of Orange County

Code:

NCT04289467

Conditions

Infantile Spasm

Eligibility Criteria

Sex: All

Age: 1 - 3

Healthy Volunteers: Not accepted

Interventions

Fenfluramine

Study Details

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

Study ID

NCT04289467

Start date

Jun 16, 2023

Status verified date

Apr, 2026

Completion date

Dec 31, 2026

Anticipated

Primary completion date

Dec 31, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 3

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

10 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Fenfluramine treatment

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.

Interventions

Fenfluramine

Open-label

Primary outcome measure

  • Electroclinical response (Efficacy) [ Time Frame: 12 months ]

Central Contacts and Locations

Central contacts

Kellie Bacon, MPH

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

Virginia Allhusen, PhD

vallhusen@choc.org

Locations

Children's Hospital of Orange County

Recruiting

Orange, California, United States, 92868

Contacts

Daniel Shrey, MD

dshrey@choc.org

Angelyque Lorenzana

714-509-8972alorenzana@choc.org

More Information

Sponsor

Children's Hospital of Orange County

Last update posted

Apr 15, 2026

Last verified

Apr, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Children's Hospital of Orange County on 2026-04-15.