Study on Radiprodil for Seizures and Behavioral Symptoms in Patients with Tuberous Sclerosis Complex or Focal Cortical Dysplasia Type II

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Radiprodil in patients with two specific conditions: Tuberous Sclerosis Complex and Focal Cortical Dysplasia Type II. These conditions can cause seizures and behavioral symptoms, and the study aims to understand how Radiprodil can help manage these issues. Radiprodil is given as an oral suspension, which means it is a liquid medicine taken by mouth.

The purpose of the study is to assess the safety and tolerability of Radiprodil, as well as how the body processes the medication. Participants will receive multiple doses of Radiprodil that are adjusted individually. The study will also look at how the medication affects seizure frequency and behavioral symptoms over time. Participants will be monitored for any side effects, changes in vital signs, and other health indicators throughout the study.

The study is open-label, meaning both the participants and the researchers know which treatment is being administered. It will involve regular check-ups and assessments to track the effects of Radiprodil on the participants’ conditions. The study will also use tools like electronic diaries to record seizure activity and other symptoms. The goal is to gather comprehensive data on how Radiprodil can benefit patients with these specific neurological conditions.

1 beginning of the trial

Upon joining the study, you will receive an initial assessment to confirm your eligibility. This includes reviewing your medical history and ensuring you meet the criteria for participation.

You will be asked to sign a consent form, and if applicable, provide assent. This confirms your understanding and agreement to participate in the study.

2 baseline period

During this period, your current health status and seizure frequency will be recorded. This involves keeping a daily record of seizures using an electronic diary, known as an eDiary.

You will continue your existing treatment regimen, which should remain stable for 28 days before starting the study medication.

3 treatment initiation

You will begin taking the study medication, radiprodil, as an oral suspension. The dosage will be individually adjusted to find the most suitable amount for you.

The medication is taken by mouth, and the frequency and duration will be determined by the study team based on your response.

4 monitoring and assessments

Throughout the study, regular check-ups will be conducted to monitor your health and the effects of the medication. This includes physical exams, blood tests, and heart monitoring using an ECG (electrocardiogram).

You will continue to record your seizures and any changes in behavior in the eDiary.

5 end of treatment

At the end of the treatment period, a final assessment will be conducted to evaluate the effects of the medication on your seizures and overall health.

You will be asked to complete questionnaires about your quality of life and any changes you have experienced during the study.

6 follow-up

After completing the treatment, there will be a follow-up period to monitor your health and any lasting effects of the medication.

You may be required to attend additional visits or provide information about your health status during this time.

Who Can Join the Study?

Age range: Must be at least 6 months old and not older than 18 years at the start of the study.
Must have tried at least 2 different anti-seizure medications without success, even when taken at the right doses and for the right amount of time.
Must have a diagnosis of Focal Cortical Dysplasia (FCD) Type II confirmed by symptoms and a positive MRI scan, or a diagnosis of Tuberous Sclerosis Complex (TSC) confirmed by clinical or genetic criteria.
Must have had at least 8 countable motor seizures in a 4-week period before the study, with at least 1 seizure in at least 3 of those 4 weeks.
All treatments for epilepsy or behavior, including special diets or devices, should be stable for 28 days before the study starts, with no more than 6 days per month using rescue medication. Must stay on a stable treatment plan during the study.
The participant or their caregiver must have signed a consent form, and the participant must have signed an assent form if applicable.
The participant or their caregiver must be willing and able to fill out a daily electronic diary.
Must have had an MRI scan within 12 months before the first dose of the study drug.
If the participant is of childbearing potential, they must not be pregnant or breastfeeding and must use an acceptable method of birth control, such as abstinence, hormonal methods, or an intrauterine device, from the start of the study until 90 days after the last dose. If male, must be willing to use a condom during the same period.

Who Cannot Join the Study?

Patients with medical conditions other than Focal Cortical Dysplasia (FCD) Type II or Tuberous Sclerosis Complex (TSC) cannot participate.
Patients who are not within the specified age range for the study cannot participate.
Patients who are part of a vulnerable population cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
Katholieke Universiteit te Leuven	Leuven	Belgium

Other Sites

Site Name	City	Country
IRCCS Istituto Giannina Gaslini	Genoa	Italy
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Instytut Pomnik Centrum Zdrowia Dziecka	Warsaw	Poland
Uniwersytecki Szpital Kliniczny W Poznaniu	Poznan	Poland
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy
Hospital Sant Joan De Deu Barcelona	Esplugues De Llobregat	Spain
Hospital Ruber Internacional	Madrid	Spain
Centrum Medyczne Plejady Magdalena Celinska Loewenhoff Michal Zolnowski sp.k.	Cracow	Poland
Hospital La Milagrosa S.A.	Madrid	Spain
Ospedale Pediatrico Bambino Gesu’	Rome	Italy
Uqnandbnoksdmk Cjgcnka Kiqxfslcw	Gdansk	Poland
Uopjezwvnd Op Awvcoya	Edegem	Belgium
Harhwxjg Vvja dsqhdeay	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Belgium	Not recruiting	15.12.2023
Italy	Not recruiting	15.12.2023
Poland	Not recruiting	15.12.2023
Spain	Not recruiting	15.12.2023
The Netherlands	Not recruiting	15.12.2023

Trial locations

Investigated drugs:

Radiprodil

Radiprodil is a medication being studied for its potential to help people with certain brain conditions, specifically Tuberous Sclerosis Complex (TSC) and Focal Cortical Dysplasia (FCD) Type II. These conditions can cause seizures and behavioral problems. The study aims to see how safe and tolerable radiprodil is when given in different amounts to each person. Researchers are also looking at how the body processes the medication and how much of it ends up in the blood. This information will help determine if radiprodil can effectively reduce seizures and improve behavior in patients with these conditions.

Focal Cortical Dysplasia (FCD) Type II – Focal Cortical Dysplasia Type II is a brain malformation characterized by abnormal development of the cerebral cortex. It often leads to epilepsy, with seizures being a common symptom. The condition is caused by disorganized layers of neurons in the brain, which disrupt normal electrical activity. Over time, the abnormal brain tissue can lead to increased seizure frequency and severity. The progression of the disease can vary, with some individuals experiencing more frequent seizures as they age. The condition is typically identified in childhood or adolescence.

Tuberous Sclerosis Complex (TSC) – Tuberous Sclerosis Complex is a genetic disorder that causes non-cancerous tumors to form in various organs, primarily the brain, skin, kidneys, heart, and lungs. The disease is characterized by a wide range of symptoms, including seizures, developmental delays, and skin abnormalities. As the condition progresses, the tumors can grow larger and potentially affect organ function. Neurological symptoms, such as seizures and cognitive impairment, are common and may worsen over time. The severity and progression of symptoms can vary significantly among individuals. TSC is often diagnosed in early childhood due to the presence of seizures or developmental issues.

Trial ID:

2023-506301-20-00

Protocol code:

RAD-GRIN-201

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Radiprodil for Seizures and Behavioral Symptoms in Patients with Tuberous Sclerosis Complex or Focal Cortical Dysplasia Type II

What is this study about?

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