Study on the Effects of Full Spectrum Cannabis Extract (Dronabinol, Cannabidiol) for Patients with Hard-to-Treat Epilepsy in Tuberous Sclerosis Complex

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What is this study about?

This clinical trial is focused on studying a condition known as refractory epilepsy, which is a type of epilepsy that is difficult to control with standard treatments. This condition is associated with a genetic disorder called Tuberous Sclerosis Complex (TSC). The trial will explore the effects of a treatment using a full spectrum cannabis extract, which contains two active substances: dronabinol and cannabidiol. The treatment being tested is referred to by the code name YCJ-01. Some participants in the study will receive a placebo, which is a substance with no active medication.

The purpose of the study is to evaluate how effective and safe the treatment is in reducing the number of epileptic seizures in patients with refractory epilepsy due to TSC. Participants will be given either the YCJ-01 treatment or a placebo over a period of time. The study will monitor changes in the frequency of seizures and any side effects that may occur. The treatment is administered orally, meaning it is taken by mouth in the form of an oil.

Throughout the study, participants will have regular check-ups to assess their response to the treatment. The trial aims to determine if the YCJ-01 treatment can significantly decrease the number of seizures compared to the baseline period before the treatment started. The study will also compare the results between those receiving the actual treatment and those receiving the placebo to understand the treatment’s effectiveness better. The trial is expected to continue until the end of 2026.

1 initial assessment

Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes verifying age, diagnosis of tuberous sclerosis complex (TSC), and the presence of refractory epilepsy.

The assessment ensures stability in current treatments, such as anti-epileptic drugs (AEDs) and any dietary or device-based interventions, for at least four weeks prior to starting the trial.

2 baseline period

During the baseline period, the frequency of epileptic seizures is recorded. This period helps establish a reference point for evaluating the effectiveness of the treatment.

Patients or caregivers are required to maintain a seizure diary to document the number and type of seizures experienced.

3 treatment initiation

The treatment phase begins with the administration of the investigational product, full spectrum cannabis extract, or a placebo. The extract contains active substances such as dronabinol and cannabidiol.

The product is administered orally in the form of oil. The dosage and frequency are determined by the study protocol and are adjusted based on individual response and tolerance.

4 treatment monitoring

Throughout the treatment phase, regular monitoring is conducted to assess the number of seizures and any changes in seizure patterns.

The occurrence of any adverse events, changes in vital signs, and laboratory values are also monitored to ensure safety.

5 evaluation of treatment efficacy

The primary goal is to evaluate the change in the number of monthly seizures compared to the baseline period. This is assessed at various points during the trial.

Secondary evaluations include the proportion of patients achieving significant reductions in seizure frequency and the overall response rate to the treatment.

6 completion of trial

Upon completion of the trial, a final assessment is conducted to evaluate the long-term effects of the treatment.

Participants are monitored for any lasting changes in seizure frequency and severity, as well as any delayed adverse effects.

Who Can Join the Study?

Patients of any sex between 2 and 65 years of age (both included).
Diagnosis confirmed by the TSC investigator, which means a doctor has confirmed the condition using clinical criteria or a genetic study.
Having refractory epilepsy due to Tuberous Sclerosis Complex (TSC), which means epilepsy that does not respond well to traditional treatments like anti-epileptic drugs (AEDs), a special diet called the ketogenic diet, a device called a vagal nerve stimulator, or surgery.
Experiencing at least 4 epileptic seizures within 4 weeks before starting the trial treatment, with noticeable signs like loss of consciousness or movements.
No changes in the doses of AEDs, ketogenic diet, or settings of the vagal nerve stimulator for at least 4 weeks before starting the trial treatment.
Currently taking 3 or fewer AEDs when signing the informed consent. Clobazam, a specific medication, is not counted as an AED for this purpose.
Patients or their caregivers/family members (if the patient is a minor or under legal guardianship) must be willing to keep a diary of seizures.
Women of child-bearing age must agree to use contraceptive measures from the time of signing the informed consent until three months after stopping the trial medication, for safety reasons.
Patients must have signed the informed consent themselves or through a legal representative.

Who Cannot Join the Study?

Patients who do not have refractory epilepsy related to tuberous sclerosis complex cannot participate. Refractory epilepsy means seizures that are difficult to control with medication.
Patients who are not within the specified age range for the study cannot participate. The age range is typically defined by the study but is not specified here.
Patients who are not male or female cannot participate, as the study includes both genders.
Patients who are not considered part of a vulnerable population cannot participate. A vulnerable population includes groups that may need special protection, like children or those with certain health conditions.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country
Hospital Ruber Internacional	Madrid	Spain
Hospital Universitario 12 De Octubre	Madrid	Spain
Hfyczmeq Dk Lm Sdans Cziz I Sxef Puw	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Spain	Recruiting	12.06.2024

Trial locations

Investigated drugs:

YCJ-01 is a full-spectrum cannabis extract being studied for its potential to reduce the number of epileptic seizures in patients with refractory epilepsy associated with Tuberous Sclerosis Complex. The trial aims to evaluate how effective this treatment is in managing seizures and to assess its safety for patients with this condition.

Investigated diseases:

Tuberous sclerosis complex

Refractory Epilepsy in Tuberous Sclerosis Complex – This condition is characterized by frequent and persistent seizures that do not respond well to standard treatments. It occurs in individuals with Tuberous Sclerosis Complex, a genetic disorder that causes non-cancerous tumors to form in various organs. The seizures can vary in type and severity, often impacting daily life and development. Over time, the frequency and intensity of seizures may fluctuate, making management challenging. The condition requires ongoing monitoring to assess changes in seizure patterns.

Trial ID:

2024-520171-27-00

Protocol code:

EstuEla2022

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on the Effects of Full Spectrum Cannabis Extract (Dronabinol, Cannabidiol) for Patients with Hard-to-Treat Epilepsy in Tuberous Sclerosis Complex

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?