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Long-Term Safety Study of Fenfluramine for Patients with Myoclonic Astatic Epilepsy (Doose Syndrome)

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the long-term safety of a medication called Fenfluramine in patients with a type of epilepsy known as Myoclonic Astatic Epilepsy, also referred to as Doose Syndrome. The medication being tested is called Fintepla 2.2 mg/ml oral solution, which is taken by mouth. The purpose of the study is to monitor how safe and effective low doses of Fenfluramine are when used alongside other treatments for this condition.

Participants in the study will receive Fenfluramine as an additional therapy to their current treatment plan. The study will observe the effects of Fenfluramine over a long period, with doses ranging from 0.4 to 0.8 mg per kilogram of body weight per day, up to a maximum of 30 mg per day. The study aims to see if Fenfluramine can help reduce the number of seizures experienced by patients while ensuring that it remains safe for long-term use.

Throughout the study, various health checks will be conducted to ensure the safety of the participants. These checks include monitoring for any serious side effects, conducting laboratory tests, checking vital signs, and performing physical examinations. Additionally, heart health will be assessed using tests like the 12-lead electrocardiogram (ECG) and Doppler echocardiogram (ECHO). The study will also track changes in body weight to ensure the overall well-being of the participants.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, you will be informed about the nature of the study. Written informed consent must be obtained from you and your legally responsible parent or caregiver.
You must be between 1 and 17 years old and currently enrolled in the FFA-MAE study. The investigator will determine if you are a candidate for continued treatment with Fenfluramine based on previous benefits observed.
Step 2
Medication administration
You will receive Fenfluramine as an add-on therapy. The dosage will be either 0.4 or 0.8 mg per kilogram of body weight per day, with a maximum of 30 mg per day.
The medication is provided as an oral solution, which means you will take it by mouth. The exact dosage and frequency will be determined by the study team based on your specific needs.
Step 3
Monitoring and assessments
Throughout the study, your health and response to the medication will be closely monitored. This includes tracking the number and frequency of seizures compared to your baseline before starting Fenfluramine treatment.
Regular assessments will include checking for any adverse events, laboratory measurements, vital signs, physical examinations, and heart tests such as ECGs and ECHOs. Your body weight will also be monitored.
Step 4
Duration of the study
The study is designed to monitor long-term safety and efficacy, with an estimated end date of November 1, 2026.
Your participation will continue as long as the benefits of the treatment outweigh any potential risks, as determined by the study team.

Who can join the trial?

6 criteria

The person must be currently enrolled in the FFA-MAE study.
The person and their parents or caregiver must have been informed about the study and have given written consent to participate.
The person must be between 1 year (12 months) and 17 years old.
In the opinion of the study doctor, the person must be suitable for continued treatment with Fenfluramine for a longer period. This means the person has shown a clinically meaningful benefit from Fenfluramine in the previous study (FFA-MAE), and the benefits of continuing treatment are greater than the potential risks.
Clinically meaningful benefit is defined as at least a 50% reduction in the total number of seizures compared to the start of the FFA-MAE study.
The person must be taking at least one other anti-epileptic drug (AED) in addition to Fenfluramine.

Who cannot join the trial?

4 criteria

Patients who are not diagnosed with myoclonic-astatic epilepsy cannot participate. This is a specific type of epilepsy characterized by sudden muscle jerks and loss of muscle tone.
Patients who are not within the specified age range for the study cannot participate. The study is designed for a specific age group.
Patients who are part of a vulnerable population cannot participate. This means individuals who may be at a higher risk of harm or exploitation.
Patients who are not able to take the study medication as an add-on therapy cannot participate. This means the medication is meant to be taken in addition to other treatments.

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Verified sites

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Universitaetsklinikum Schleswig-Holstein AöR
Kiel, Germany

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Investigated drugs

Fenfluramine is a medication used in this clinical trial as an additional treatment for patients with Myoclonic Astatic Epilepsy, also known as Doose Syndrome. This medication is being studied to see how safe it is for long-term use and how well it works when added to the patient's current treatment plan. Fenfluramine is believed to help reduce the frequency and severity of seizures in patients with this type of epilepsy. The trial aims to ensure that patients can use this medication safely over a long period while monitoring any potential side effects or improvements in their condition.

Fenfluramine Hydrochloride

What is already known about the treatment

Fenfluramine – Fenfluramine is administered orally in the form of a low-dose medication, typically as an add-on therapy for patients with Myoclonic Astatic Epilepsy, also known as Doose Syndrome. Currently, it is being studied in clinical trials to assess its long-term safety and effectiveness. The main therapeutic use of Fenfluramine is to help control seizures in epilepsy patients. At the molecular level, it works by increasing the levels of serotonin, a chemical in the brain that can help reduce seizure activity. Fenfluramine is classified as a serotonin-releasing agent in pharmacology.

Investigated diseases

Myoclonic-astatic epilepsy – Myoclonic-astatic epilepsy is a type of epilepsy characterized by sudden, brief muscle jerks followed by a loss of muscle tone. These episodes can cause the person to fall or drop objects they are holding. The condition typically begins in early childhood and can vary in frequency and severity. Over time, the pattern of seizures may change, with some individuals experiencing fewer myoclonic jerks and more astatic (drop) attacks. The progression of the disease can lead to a combination of different seizure types. It is considered a rare form of epilepsy.