In short
There is currently 1 ongoing clinical trial investigating treatment options for Epilepsy With Myoclonic-Atonic Seizures (also known as Myoclonic Astatic Epilepsy, Doose Syndrome). This trial is focused on evaluating the long-term safety of Fenfluramine as an add-on therapy for children and adolescents who have shown benefit from this medication in previous studies.
Clinical trial locations
- Germany
Long-Term Safety Study of Fenfluramine for Patients with Myoclonic Astatic Epilepsy (Doose Syndrome)
This clinical trial is being conducted in Germany to study the long-term safety of Fenfluramine (marketed as Fintepla 2.2 mg/ml oral solution) in young patients with this rare form of epilepsy. The condition is characterized by sudden, brief muscle jerks followed by loss of muscle tone, which can cause falls or dropping of objects. It typically begins in early childhood and can vary in how often and how severely seizures occur.
Main focus and goal: The study aims to monitor how safe and effective low doses of Fenfluramine are when used alongside other anti-epileptic medications over a long period. Researchers want to see if Fenfluramine can help reduce the number of seizures while ensuring it remains safe for extended use. The trial will run until approximately November 2026.
Investigational drug: Fenfluramine is a medication that works by increasing levels of serotonin in the brain, a chemical that can help reduce seizure activity. In this trial, it is given as an oral solution at doses ranging from 0.4 to 0.8 mg per kilogram of body weight per day, with a maximum of 30 mg per day. It is used as an add-on therapy, meaning patients continue taking their other epilepsy medications while also taking Fenfluramine.
Who can participate (inclusion criteria):
- Children and adolescents between 1 year (12 months) and 17 years old
- Currently enrolled in a previous study called FFA-MAE
- Must have shown meaningful benefit from Fenfluramine in the previous study, defined as at least a 50% reduction in total seizure count
- The study doctor must believe that continuing treatment with Fenfluramine will provide more benefits than risks
- Must be taking at least one other anti-epileptic drug in addition to Fenfluramine
- Written consent must be provided by the patient (when appropriate) and their parent or legal caregiver
Who cannot participate (exclusion criteria):
- Patients who do not have a diagnosis of myoclonic-astatic epilepsy
- Patients outside the specified age range of 1 to 17 years
- Patients considered part of a vulnerable population who may be at higher risk of harm or exploitation
- Patients who cannot take the study medication as an add-on therapy to their current treatment
Monitoring and safety: Throughout the study, participants will undergo regular health checks to ensure their safety. These include monitoring for any serious side effects, laboratory tests, vital signs checks, physical examinations, and heart health assessments using electrocardiograms (ECG) and echocardiograms (ECHO). Body weight will also be tracked regularly, and the number and frequency of seizures will be compared to baseline measurements taken before starting Fenfluramine treatment.
Summary
Currently, there is one active clinical trial for Epilepsy With Myoclonic-Atonic Seizures, taking place in Germany. This trial represents a continuation study for patients who have already participated in a previous Fenfluramine trial and shown positive results with at least a 50% reduction in seizures. The focus is specifically on long-term safety monitoring rather than initial efficacy testing, which suggests that earlier research has shown promising results. The trial is limited to children and adolescents aged 1 to 17 years and requires that participants continue taking their existing anti-epileptic medications while adding Fenfluramine to their treatment regimen. The study is expected to continue until late 2026, providing valuable information about the sustained safety profile of this medication for this rare form of childhood epilepsy.
