Study on Cannabidiol for Reducing Seizures in Children and Young Adults with Severe Epilepsy Associated with Rare Diseases

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What is this study about?

This clinical trial is focused on studying the effects of a treatment for severe epilepsy associated with rare diseases in children and young adults. The treatment being tested is an oral solution containing cannabidiol, also known as CBD. Cannabidiol is a chemical substance that is being used in addition to the current anti-seizure medications that participants are already taking. The main goal of the study is to see if adding cannabidiol can help reduce the number and severity of seizures experienced by the participants.

Participants in the study will take the cannabidiol oral solution for a period of 24 weeks. During this time, researchers will monitor the frequency and severity of seizures to determine if there is any improvement. The study will also look at the safety and tolerability of cannabidiol, which means checking for any side effects or health changes in the participants. Additionally, the study will assess how cannabidiol interacts with other anti-seizure medications that participants are taking.

Throughout the study, various aspects of the participants’ health and well-being will be evaluated, including any changes in their cognitive abilities, behavior, and overall quality of life. The study aims to provide valuable information on whether cannabidiol can be an effective and safe addition to existing treatments for severe epilepsy in young people with rare diseases.

1 initial visit

Upon joining the study, an initial visit is conducted. During this visit, a detailed medical history is taken, and a physical examination is performed. This helps to establish a baseline for your current health status.

You will be asked to provide information about your current medications and any previous treatments for epilepsy. This includes any anti-seizure medications you are currently taking.

2 baseline period

A baseline period of 4 weeks is observed to record the frequency and severity of seizures. This information is crucial for comparing changes during the treatment period.

During this time, you will continue with your current anti-seizure medications without any changes.

3 treatment initiation

The treatment phase begins with the administration of cannabidiol oral solution, known as Epidyolex 100 mg/ml. This is taken orally as an additional treatment to your current anti-seizure medications.

The dosage and frequency of the cannabidiol solution will be determined by the study team and adjusted as needed based on your response and any side effects.

4 treatment period

The treatment period lasts for 24 weeks. During this time, regular visits are scheduled to monitor your health and the effects of the treatment.

You will be required to maintain a diary to record seizure activity, any side effects, and any changes in your condition.

5 regular assessments

Throughout the treatment period, regular assessments are conducted. These include physical and neurological examinations, as well as monitoring of vital signs and body weight.

Blood tests may be performed every 4 weeks to check the levels of cannabidiol and other medications in your system.

6 EEG evaluations

Electroencephalogram (EEG) evaluations are conducted at specific intervals to assess any changes in brain activity. These are compared to the baseline EEG to determine any improvements or changes.

The EEG results are reviewed by experienced specialists to provide a score indicating any changes in seizure activity.

7 final evaluation

At the end of the 24-week treatment period, a final evaluation is conducted. This includes a comprehensive review of your health, seizure activity, and any changes observed during the study.

The study team will discuss the results with you and provide guidance on the next steps for your treatment.

Who Can Join the Study?

Must be a male or female aged between 2 and 25 years old.
Must have a rare disease-associated severe epilepsy. This means the epilepsy is linked to a rare disease recognized by the National Health System.
Must experience at least 4 motor seizures per month. Motor seizures can be generalized (affecting the whole brain), focal (affecting part of the brain), or both.
Must have tried at least 2 anti-seizure medications (ASMs) in the past and currently be taking between one and four ASMs. The treatment should have been stable for the last 4 weeks. This can include a special diet called the ketogenic diet or a treatment called vagal nerve stimulation.
The parent or caregiver must be informed about the study and must give informed consent. This means they agree to participate after understanding the study details.
The parent or caregiver must be willing and able to keep a diary, follow the visit schedule, and manage the study medication as instructed by the study team.
Surgical treatment for epilepsy must have either failed or not be an option.

Who Cannot Join the Study?

Patients who are not diagnosed with epilepsy. Epilepsy is a condition that affects the brain and causes repeated seizures.
Patients who are not currently taking anti-seizure medications. These are medicines used to help control seizures.
Patients who are not children or young adults. The study is focused on these age groups.
Patients who do not have severe epilepsy associated with a rare disease. This means the epilepsy is linked to a less common health condition and is more serious.

Where you can join this trial?

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Other Sites

Site Name	City	Country	Status
Azienda Ospedaliera Universitaria Meyer IRCCS	Florence	Italy

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	01.06.2023

Trial locations

Investigated drugs:

Cannabidiol

Cannabidiol is a medication being tested in this clinical trial. It is an oral solution that is used as an additional treatment for children and young adults who have severe epilepsy associated with rare diseases. The goal of using Cannabidiol in this study is to see if it can help reduce the number or severity of seizures when used alongside the medications that patients are already taking to control their seizures. Cannabidiol is known for its potential to help with seizure control, and this trial aims to understand how effective and safe it is for young patients with challenging forms of epilepsy.

Investigated diseases:

Epilepsy

Epilepsy – Epilepsy is a neurological disorder characterized by recurrent, unprovoked seizures. These seizures are episodes of disturbed brain activity that can cause changes in attention or behavior. The condition can manifest as generalized seizures, affecting both sides of the brain, or focal seizures, affecting a specific part of the brain. The progression of epilepsy varies, with some individuals experiencing frequent seizures and others having long periods without any. Seizures can range from brief and nearly undetectable to long periods of vigorous shaking. Over time, epilepsy can impact cognitive, behavioral, and physical functions, depending on the frequency and severity of the seizures.

Trial ID:

2023-503709-12-00

Protocol code:

CBD_RE

NCT ID:

NCT05803434

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Cannabidiol for Reducing Seizures in Children and Young Adults with Severe Epilepsy Associated with Rare Diseases

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?