A Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Fenfluramine (Hydrochloride) in Infants 1 Year to Less Than 2 Years of Age With Dravet Syndrome
This research study is focused on a medicine called fenfluramine for babies and toddlers aged 12 to 23 months who have Dravet syndrome. Dravet syndrome is a rare and severe form of epilepsy. The main goal of the study is to carefully look at how safe the medicine is and how well these very young children handle it at different dose levels. Researchers will also be checking how the medicine moves through and stays in the body. The hope is to find out if fenfluramine could be a helpful treatment option for this specific age group suffering from Dravet syndrome, as there are often limited effective treatments for such young children.
At a glance
What is this study about?
This research study is about a medicine called fenfluramine, which is being investigated for use in very young children with Dravet syndrome. Dravet syndrome is a serious and rare form of epilepsy that begins in infancy and causes frequent, prolonged seizures that are often difficult to control with standard medications.
The main purpose of this study is to see how safe fenfluramine is for babies and toddlers aged 1 to just under 2 years old. Researchers will be giving different doses of the medicine and closely watching to see if there are any side effects, and how well the children tolerate it. They will also look at how the body processes the medicine, which is called pharmacokinetics.
Finding effective and safe treatments for very young children with Dravet syndrome is really important because their condition can be very challenging to manage. This study aims to gather important information that could help doctors understand if fenfluramine can be a safe and useful option for this specific age group.
Key takeaways
- The study is for children aged 1 to under 2 years old with Dravet syndrome.
- It aims to check the safety and how well fenfluramine is tolerated at different doses.
- Children will continue their usual anti-seizure medicines during the study.
- Participation requires ongoing visits and careful monitoring, including heart checks.
- The decision to join is completely voluntary, and you can withdraw at any time.
Who may be eligible?
To join this study, your child must be between 1 and just under 2 years old. They must have received a clear diagnosis of Dravet syndrome from their doctor. Your child should already be taking at least one other anti-seizure medicine at a stable dose, and this medicine should have been used for at least four weeks before the study starts.
Importantly, your child's seizures must not be fully controlled by their current medicines. This means they should have tried at least two other seizure medicines that haven't stopped their seizures completely. To qualify, your child must also have experienced at least one specific type of motor seizure (like a generalized tonic-clonic or focal motor seizure) in the month before the study begins. They also need to weigh at least 8 kilograms.
Children cannot join if they are allergic to fenfluramine or any of its ingredients. They also cannot participate if they have certain heart or lung problems, especially if they have high blood pressure in the arteries of their lungs (pulmonary hypertension) or another serious ongoing medical condition.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child aged between 12 and 23 months?
- Does your child have a confirmed diagnosis of Dravet syndrome?
- Is your child currently taking at least one anti-seizure medicine?
- Are your child's seizures not fully controlled by their current medicines?
- Does your child weigh at least 8 kg (about 17.6 pounds)?
- Does your child have any known severe heart or lung conditions?
What does participation involve?
If your child takes part in this study, they will receive the study medicine, fenfluramine, at a specific dose determined by the study doctors. They will be closely monitored throughout the study to check for safety and how well they are tolerating the medicine. This will involve regular visits to the clinic, where doctors and nurses will perform examinations, take measurements, and possibly some blood samples. Your child will continue to take their existing anti-seizure medicines as well.
The study will involve several visits over a period of time, and each visit may include different assessments like checking vital signs, listening to your child's heart, and potentially performing heart scans (echocardiograms) or electrical heart tests (ECGs). You will be asked to keep a record of your child's seizures. The total length of the study for each child will vary, but it's important to understand the commitment involved.
Potential risks and benefits
Locations (12)
- Ep0213 105Verified postcodeMemphis, United States
- Ep0213 107Verified postcodeDallas, United States
- Ep0213 103Verified postcodeSeattle, United States
- Ep0213 502Verified postcodeBrussels, Belgium
- Ep0213 501Verified postcodeEdegem, Belgium
- Ep0213 303Verified postcodeBielefeld, Germany
- Ep0213 202Verified postcodeFlorence, Italy
- Ep0213 203Verified postcodeGenova, Italy
- Ep0213 201Verified postcodeRoma, Italy
- Ep0213 204Verified postcodeRoma, Italy
- Ep0213 403Verified postcodeGlasgow, United Kingdom
- Ep0213 401Verified postcodeLondon, United Kingdom
Common questions
What is Dravet syndrome?
Dravet syndrome is a rare and severe type of epilepsy that usually starts in babies, causing frequent and often long-lasting seizures.
What is fenfluramine?
Fenfluramine is a medicine being tested to see if it can help control seizures in children with Dravet syndrome.
How old does my child need to be to join?
Your child must be between 12 months (1 year) and 23 months (just under 2 years) old.
Will my child stop their current seizure medicine?
No, your child will continue to take their existing seizure medicines alongside the study medicine, fenfluramine.
Is this medicine available to everyone now?
No, this is a 'Phase 3' study, which means the medicine is still being tested and is not yet generally available for this age group.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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