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Evaluating fenfluramine for infants with Dravet syndrome

Open-Label, Single-Arm, Phase 3 Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome

Phase 3 Interventional UCB Pharma · NCT06118255

This study is testing if a medication called fenfluramine is safe and well-tolerated for infants with Dravet syndrome who are between 1 and 2 years old.

Quick facts

PhasePhase 3
Study typeInterventional
Enrollment20 (estimated)
Ages1 Year to 23 Months
SexAll
SponsorUCB Pharma Industry-sponsored
Locations19 sites (Orange, California and 18 other locations)
Trial IDNCT06118255 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and tolerability of fenfluramine hydrochloride in infants aged 1 to less than 2 years who have Dravet syndrome. Participants will receive doses ranging from 0.2 to 0.8 mg/kg/day while continuing their current anti-seizure medications. The study aims to determine how well the drug is tolerated and its pharmacokinetics in this young population. The research is crucial for understanding treatment options for drug-resistant epilepsy in infants.

Who should consider this trial

Good fit: Ideal candidates are infants aged 1 to less than 2 years with a documented diagnosis of Dravet syndrome and drug-resistant epilepsy.

Not a fit: Patients who do not have Dravet syndrome or those who are not experiencing drug-resistant epilepsy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option for infants suffering from Dravet syndrome, potentially improving seizure control.

How similar studies have performed: Other studies have shown promise in using fenfluramine for treating Dravet syndrome, indicating a potential for success in this approach.

Eligibility criteria

Show full inclusion / exclusion criteria 
Inclusion Criteria:

* Participant is ≥1 to \<2 years of age as of the day of the first administration of study drug
* Participant has a documented diagnosis or likely diagnosis of Dravet syndrome according to the International League Against Epilepsy (ILAE) criteria and as agreed by the Epilepsy Study Consortium (ESC)
* Participant must be currently receiving ≥1 concomitant anti seizure medication (ASM) at a stable dose for ≥4 weeks prior to the Screening Visit and is expected to remain stable throughout the study. Rescue medications for seizures are not counted towards the total number of ASMs
* Participant must have drug resistant epilepsy as defined as a history of failure of adequate trials of 2 tolerated, appropriately chosen and used antiepileptic drug schedules (whether as monotherapies or in combination) to achieve sustained seizure freedom
* Participants must have ≥1 countable motor seizures (CMS) during the Baseline Period. The CMS include distinct seizures of generalized tonic-clonic, bilateral clonic, focal motor, bilateral tonic, atonic (drop), bilateral tonic/atonic, or focal to bilateral tonic-clonic type. If the participant fails to have ≥1 qualifying seizures in 28 days, the Baseline Period may be extended by an additional 14 days with Sponsor approval. Participants with an extended Baseline Period must still have ≥1 CMS in the 28 days immediately prior to the day of the first administration of study drug
* Body weight is ≥8 kg
* Males and females

Exclusion Criteria:

* Participant has a known hypersensitivity to fenfluramine hydrochloride (HCl) or any of the excipients in the study drug
* Participant has an exclusionary cardiovascular or cardiopulmonary abnormality based on echocardiogram (ECHO), electrocardiogram (ECG), or physical examination and is not approved for entry by the central cardiac reader
* Participant has a diagnosis of pulmonary arterial hypertension
* Participant has a clinically significant medical condition, including chronic obstructive pulmonary disease, interstitial lung disease, or portal hypertension, or has had clinically relevant symptoms or a clinically significant illness currently or in the 4 weeks prior to the Screening Visit, other than epilepsy, that in the opinion of the Investigator would negatively impact study participation, collection of study data, or pose a risk to the participant
* Participant has current or past history of cardiovascular or cerebrovascular disease, such as cardiac valvulopathy, myocardial infarction or stroke, severe ventricular arrhythmias, or clinically significant structural cardiac abnormality, including but not limited to mitral valve prolapse, atrial or ventricular septal defects, patent ductus arteriosus, and patent foramen ovale with reversal of shunt. (Note: Patent foramen ovale or a bicuspid aortic valve are not considered exclusionary.)
* Participant has a current or past history of glaucoma
* Participant has moderate to severe hepatic impairment, assessed based on the Child-Pugh classification system
* Participant has moderate to severe renal impairment (estimated glomerular filtration rate \<50 mL/min/1.73 m\^2 calculated with the updated Bedside Schwartz equation for children
* QT interval corrected (QTc) \>450 msec
* Participant is taking \>4 concomitant ASMs
* Participant is receiving concomitant treatment with cannabidiol other than Epidiolex/Epidyolex or is being actively treated with tetrahydrocannabinol (THC) or any marijuana product for any condition
* Participant is receiving concomitant therapy with any of the following: centrally-acting anorectic agents; monoamine-oxidase inhibitors; any centrally-acting compound with clinically appreciable amount of serotonin agonist or antagonist properties, including serotonin reuptake inhibition; other centrally-acting noradrenergic agonists, including atomoxetine; or cyproheptadine. Disallowed medications are subject to washout of ≥5 half-lives before the first day of study drug administration
* Participant is currently receiving another investigational product(s) or has received another investigational product within 30 days or within \<5 times the half-life of that investigational product, whichever is longer, prior to the Screening Visit
* Participant has previously been treated with Fintepla (fenfluramine HCl) prior to the Screening Visit

Where this trial is running

Orange, California and 18 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Dravet SyndromeDravet syndromepediatricsFinteplafenfluramine
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.