12 Participants Needed

Fenfluramine for Dravet Syndrome

Recruiting at 2 trial locations
RR
Overseen ByRebecca Rochowiak
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

Dravet syndrome is a genetic epilepsy associated with pathogenic variants in SCN1A that codes for Nav1.1, a protein necessary for sodium channels. Children with Dravet syndrome classically present in the first year of life with prolonged seizures, often hemiclonic and in the setting of fever or temperature changes such as getting in or out of bath water. Many anti-seizure medications are sodium channel blockers and exacerbate seizures in this patient population. This creates some limitations in medication choices for this patient population. Recently fenfluramine was approved for use in Dravet syndrome for people 2 years and older. Randomized studies demonstrated a 74.9% reduction of convulsive motor seizures compared to 19.2% in the placebo group. Additionally, 16% of children treated with fenfluramine were seizure free. Fenfluramine is likely to be as effective in children under the age of 2 years. The current study has proposed a treatment protocol to allow access to fenfluramine for children under 24 months of age.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, it mentions that participants must have failed at least one anti-seizure medication that is not a sodium channel blocker, which might suggest some medications could be continued.

Is fenfluramine safe for humans?

Fenfluramine has been shown to be a safe treatment option for Dravet syndrome, although it was previously withdrawn from the market due to possible heart-related side effects. Recent studies and trials have demonstrated its safety and effectiveness for this condition, leading to its approval by health authorities.12345

How is the drug fenfluramine unique for treating Dravet syndrome?

Fenfluramine is unique for treating Dravet syndrome because it was originally developed as an appetite suppressant and works through a serotonergic mechanism (affecting serotonin levels in the brain), which is different from conventional antiepileptic drugs. It has shown promising results in reducing seizures in patients who do not respond well to other treatments.12346

What data supports the effectiveness of the drug fenfluramine for Dravet syndrome?

Research shows that fenfluramine, originally used as an appetite suppressant, has been found to be highly effective in reducing seizures in patients with Dravet syndrome, a severe form of epilepsy. Clinical trials and studies have demonstrated its safety and efficacy, leading to its approval by health authorities for this condition.12345

Who Is on the Research Team?

KK

Kelly Knupp, MD

Principal Investigator

University of Colorado, Denver

EW

Elaine Wirrel, MD

Principal Investigator

Mayo Clinic

JS

Joseph Sullivan, MD

Principal Investigator

University of California, San Francisco

Are You a Good Fit for This Trial?

This trial is for infants and toddlers under 24 months old with Dravet Syndrome, a severe form of epilepsy. To join, they must have the genetic markers associated with this condition and cannot be effectively treated with common anti-seizure medications that block sodium channels.

Inclusion Criteria

I have submitted my echocardiogram results for review.
Each subject will be reviewed by the multi-PIs to ensure agreement that the subject has Dravet syndrome
I have tried an anti-seizure medication other than lamotrigine, oxcarbazepine, carbamazepine, or eslicarbazepine without success.
See 3 more

Exclusion Criteria

I am not experiencing significant, unexplained weight loss or failure to thrive.
I do not have significant heart valve issues.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive fenfluramine with doses ranging from 0.2 mg/kg/day to 0.7 mg/kg/day, administered twice daily

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Fenfluramine
Trial Overview The trial tests fenfluramine's safety in young children with Dravet Syndrome. Fenfluramine has shown promise in older children by significantly reducing seizures. This study will explore if similar benefits occur in those under two years old.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: FenfluramineExperimental Treatment1 Intervention

Fenfluramine is already approved in United States, European Union for the following indications:

🇺🇸
Approved in United States as Fintepla for:
🇪🇺
Approved in European Union as Fintepla for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Colorado, Denver

Lead Sponsor

Trials
1,842
Recruited
3,028,000+

UCB Pharma

Industry Sponsor

Trials
345
Recruited
110,000+
Jean-Christophe Tellier profile image

Jean-Christophe Tellier

UCB Pharma

Chief Executive Officer since 2015

MD specialized in Rheumatology

Prof. Dr. Iris Löw-Friedrich profile image

Prof. Dr. Iris Löw-Friedrich

UCB Pharma

Chief Medical Officer since 2004

MD, PhD

Published Research Related to This Trial

Fenfluramine, originally developed as an appetite suppressant, has shown promising antiepileptic effects in treating seizures associated with Dravet syndrome, a severe form of epilepsy in infants.
Preliminary results from a Phase 3 trial indicate that fenfluramine may be a highly effective and well-tolerated treatment option for children with Dravet syndrome, who often experience frequent and severe seizures despite other medications.
Individualized treatment approaches: Fenfluramine, a novel antiepileptic medication for the treatment of seizures in Dravet syndrome.Polster, T.[2020]
In a clinical trial involving 119 patients with Dravet syndrome, fenfluramine at a dose of 0.7 mg/kg per day resulted in a significant 74.9% reduction in convulsive seizure frequency compared to a 19.2% reduction in the placebo group, demonstrating its efficacy as a treatment option.
Fenfluramine was generally well tolerated, with no evidence of serious heart issues like valvular heart disease or pulmonary arterial hypertension, indicating a favorable safety profile for patients.
Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: a randomised, double-blind, placebo-controlled trial.Lagae, L., Sullivan, J., Knupp, K., et al.[2022]
In a real-world study involving 149 patients with Dravet syndrome, fenfluramine treatment led to a significant reduction in monthly convulsive seizure frequency, with 62% of patients under 6 years, 53% aged 6-17, and 50% aged 18 and older achieving a ≥75% reduction after 3 months.
Fenfluramine was generally well-tolerated, with the most common side effects being loss of appetite (21%) and somnolence (16%), and no serious cardiovascular issues like valvular heart disease or pulmonary artery hypertension were reported, indicating a favorable safety profile.
An examination of the efficacy and safety of fenfluramine in adults, children, and adolescents with Dravet syndrome in a real-world practice setting: A report from the Fenfluramine European Early Access Program.Guerrini, R., Specchio, N., Aledo-Serrano, Á., et al.[2022]

Citations

Individualized treatment approaches: Fenfluramine, a novel antiepileptic medication for the treatment of seizures in Dravet syndrome. [2020]
Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: a randomised, double-blind, placebo-controlled trial. [2022]
An examination of the efficacy and safety of fenfluramine in adults, children, and adolescents with Dravet syndrome in a real-world practice setting: A report from the Fenfluramine European Early Access Program. [2022]
A critical evaluation of fenfluramine hydrochloride for the treatment of Dravet syndrome. [2022]
Successful use of fenfluramine as an add-on treatment for Dravet syndrome. [2022]
Fenfluramine for Treatment-Resistant Seizures in Patients With Dravet Syndrome Receiving Stiripentol-Inclusive Regimens: A Randomized Clinical Trial. [2023]
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