Not recruitingRare disease

Study on the Safety and Effects of Fenfluramine in Infants Aged 1 to Less Than 2 Years with Dravet 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 safety and effects of a medication called fenfluramine hydrochloride in infants aged 1 to less than 2 years who have Dravet syndrome. Dravet syndrome is a rare and severe form of epilepsy that begins in infancy and is characterized by frequent and prolonged seizures. The medication being tested, known by the code name ZX008, is given as an oral solution and is intended to help manage seizures associated with this condition.

The purpose of the study is to evaluate how safe and tolerable fenfluramine hydrochloride is for young children with Dravet syndrome. Participants in the study will receive the medication for up to 52 weeks. During this time, researchers will monitor the children to see how the medication moves through their bodies and how it affects their health, including any changes in seizure frequency and overall well-being. The study will also look at any potential side effects, such as changes in heart function or growth patterns.

Throughout the study, the children will have regular check-ups to assess their health and development. These visits will help researchers gather important information about the medication's impact on the participants. The study aims to provide valuable insights into the use of fenfluramine hydrochloride for treating Dravet syndrome in very young children, potentially offering a new option for managing this challenging condition.

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
Initial administration
The study begins with the first administration of the medication Fintepla, which is an oral solution containing fenfluramine hydrochloride.
The dosage ranges from 0.2 to 0.8 mg per kilogram of body weight per day. The exact dosage will be determined based on individual needs and medical assessment.
Step 2
Treatment period
The treatment period extends from Week 1 through the end of the study or early termination. During this time, the medication is taken daily as prescribed.
Regular monitoring of health parameters, including heart function and growth measurements, is conducted to ensure safety and effectiveness.
Step 3
Monitoring and assessments
Throughout the study, assessments are conducted to monitor changes in seizure frequency and overall health.
Primary assessments include changes in heart function and body growth, while secondary assessments focus on seizure frequency and overall improvement as perceived by caregivers and investigators.
Step 4
End of treatment
At the end of the treatment period, a final evaluation is conducted to assess the overall impact of the medication.
This includes a review of any changes in health parameters and seizure activity since the beginning of the study.

Who can join the trial?

6 criteria

The participant must be at least 1 year old but less than 2 years old on the day they first receive the study drug.
The participant must have a documented or likely diagnosis of Dravet syndrome, which is a specific type of epilepsy, according to certain medical criteria.
The participant must currently be taking at least one antiseizure medication (ASM) at a stable dose for at least 4 weeks before the screening visit and should continue this stable dose throughout the study. Medications used only during a seizure emergency are not counted in this requirement.
The participant must have at least one countable motor seizure during the baseline period. Motor seizures include specific types such as generalized tonic-clonic, bilateral clonic, focal motor, bilateral tonic, atonic (drop), bilateral tonic/atonic, or focal to bilateral tonic-clonic seizures. If the participant does not have at least one qualifying seizure in 28 days, the baseline period may be extended by 14 days with approval. Even with an extension, the participant must have at least one motor seizure in the 28 days before starting the study drug.
The participant must weigh at least 8 kilograms.
Both male and female participants are eligible.

Who cannot join the trial?

6 criteria

Infants who are not diagnosed with Dravet syndrome cannot participate. Dravet syndrome is a rare form of epilepsy that starts in infancy.
Infants who are younger than 1 year or older than 2 years cannot participate.
Infants who have any other medical conditions that might interfere with the study cannot participate.
Infants who are taking medications that might interfere with the study cannot participate.
Infants who have had a serious allergic reaction to similar medications cannot participate.
Infants who have any other health issues that the study doctors think might make it unsafe for them to participate cannot participate.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Italy
Responsive
Hospital Ruber Internacional
Madrid, Spain
Verified
Hospital Universitario De Navarra
Pamplona, Spain

Trial locations

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Investigated drugs

Fenfluramine is a medication being studied for its safety and how well it is tolerated in infants with Dravet syndrome. Dravet syndrome is a rare form of epilepsy that begins in infancy. This study is focused on understanding how the body processes the medication and ensuring it is safe for young children.

Fenfluramine Hydrochloride

What is already known about the treatment

Fenfluramine – This medication is administered orally and is currently being studied in clinical trials for its safety and tolerability in infants with Dravet syndrome. It is primarily used to help manage seizures associated with this condition. Fenfluramine works by increasing the levels of serotonin in the brain, which helps to reduce seizure activity. It is classified as a serotonin-releasing agent.

Investigated diseases

Dravet syndrome – Dravet syndrome is a rare, severe form of epilepsy that begins in infancy. It is characterized by prolonged seizures that are often triggered by fever or hot temperatures. As the child grows, other types of seizures may develop, including myoclonic and absence seizures. The condition can also lead to developmental delays, speech impairment, and motor difficulties. Children with Dravet syndrome may experience behavioral challenges and have an increased risk of sudden unexplained death in epilepsy (SUDEP). The syndrome is typically caused by mutations in the SCN1A gene, which affects the function of sodium channels in the brain.