Treatment for infantile spasms using fenfluramine
Official title: A Phase II Study of Fenfluramine for Treatment of Refractory Infantile Spasms
Run by Children's Hospital of Orange County · NCT04289467 · Data updated 2026-11-12
This study is testing if a medication called fenfluramine can help young children with hard-to-treat infantile spasms feel better and have fewer seizures.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Ages | 12 Months to 36 Months |
| Sex | All |
| Enrollment | 10 (estimated) |
| Sponsor | Children's Hospital of Orange County Academic / other |
| Locations | 2 sites (Los Angeles, California and 1 other locations) |
| Status | Recruiting |
| Started | June 16, 2023 |
| Main results expected | December 31, 2026 |
| Study ends | December 31, 2026 |
| Registry record updated | April 15, 2026 |
| Trial ID | NCT04289467 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates are children aged 12 to 36 months with a clinical diagnosis of infantile spasms who have not responded to ACTH and vigabatrin.
✕Probably not a fit
Patients with significant preexisting cardiovascular disease or those who have recently started other treatments for infantile spasms may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This phase II clinical trial aims to evaluate the efficacy, safety, and tolerability of fenfluramine in children aged 12 to 36 months with refractory infantile spasms, a severe form of epilepsy. Participants will undergo baseline video-EEG assessments before receiving fenfluramine treatment for 21 days, followed by repeat video-EEG to assess effectiveness. Those who respond favorably will have the option to continue treatment for up to six months. The study specifically targets children who have not responded to previous treatments with vigabatrin and ACTH.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for children suffering from refractory infantile spasms.
How similar studies have gone: While this approach is novel in the context of infantile spasms, similar studies using fenfluramine for other seizure disorders have shown promising results.
Who can join: the full rules
Show full inclusion / exclusion criteria
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
Where it's running
-
Children's Hospital of Orange County
Orange, California, United States Recruiting -
UCLA Health
Los Angeles, California, United States Not yet recruiting
Study contacts
- Principal investigator: Daniel Shrey, MD — Children's Hospital of Orange County
- Study coordinator: Kellie Bacon, MPH
- Email: kellie.bacon@choc.org
- Phone: 714-509-8972
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.