Study on Cannabidiol as Add-on Therapy for Seizures in Patients Aged 1-65 with Tuberous Sclerosis Complex

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What is this study about?

This clinical trial is focused on studying the effects of a medication called Epidyolex, which contains the active ingredient cannabidiol, on individuals with a condition known as Tuberous Sclerosis Complex (TSC). TSC is a genetic disorder that causes non-cancerous tumors to form in various organs, and it often leads to seizures and behavioral challenges. The purpose of this study is to explore how adding Epidyolex to the current treatment plan affects behavior and other related outcomes in people with TSC who experience seizures.

Participants in this study will be between the ages of 1 and 65. The study will involve taking Epidyolex as an additional therapy to their existing treatment. The study will last for a period of up to 56 weeks, during which participants will receive the medication and be monitored for changes in their behavior and seizure activity. The study will also look at how the medication impacts the quality of life and family functioning, as well as any changes in sleep patterns and overall symptom severity.

Throughout the study, participants will have regular check-ins to assess their progress and any side effects they may experience. The study aims to provide valuable insights into how Epidyolex can help manage the symptoms of TSC, particularly focusing on the most challenging behaviors identified by caregivers. This research could potentially lead to improved treatment options for individuals living with TSC and their families.

1 joining the study

Upon joining the study, the participant will be assessed to confirm eligibility. This includes verifying age, medical history, and current health status.

The participant must have a confirmed diagnosis of tuberous sclerosis complex with a history of seizures and exhibit certain behavioral characteristics.

2 baseline assessment

A baseline assessment will be conducted to evaluate the participant’s current health and behavior. This includes measuring the most problematic behavior score, which must be 6 or higher.

The participant’s caregiver will also be involved in this assessment to provide additional insights into the participant’s behavior.

3 treatment initiation

The participant will begin treatment with Epidyolex 100 mg/ml oral solution, which contains cannabidiol. This medication is administered orally or through a nasogastric or gastrostomy tube.

The dosage and frequency of administration will be determined by the study protocol and adjusted as necessary based on the participant’s response.

4 ongoing assessments

Throughout the study, the participant will undergo regular assessments at weeks 4, 13, 26, and 52 to monitor changes in behavior and seizure frequency.

These assessments will include evaluations of sleep characteristics, quality of life, and overall severity of symptoms.

5 end of study

The study is expected to conclude by January 29, 2026. At the end of the study, a final assessment will be conducted to evaluate the overall impact of the treatment on the participant’s behavior and seizure control.

The participant’s experience and any changes in health status will be documented for further analysis.

Who Can Join the Study?

The participant must be within the required age range at the time of signing the consent form:
- In the US: 1 to 65 years old.
- Outside the US: 2 to 65 years old.
The participant’s parent or legal representative must agree to notify the responsible authorities about the participant’s involvement in the study if required by local law.
The participant’s parent or legal representative must agree to inform the participant’s primary care doctor and any consultant about the study participation if they are different from the study investigator.
The participant must have a dedicated caregiver to help with the study.
The participant must have a confirmed diagnosis of Tuberous Sclerosis Complex (TSC) with a history of seizures, according to specific medical criteria.
The participant must have behaviors like aggression, impulsivity, or mood swings that are considered moderate or severe. At the start of the study, these behaviors must have a score of 6 or higher to be eligible.
The participant must not have used CBD-OS treatment recently or must have stopped using it at least 3 months before the study starts.
The participant must agree to keep any factors that might affect seizures stable, like alcohol use, smoking, or other medications.
The participant can be male or female:
- Male participants must agree to use a condom and another form of birth control with female partners who can become pregnant, and not donate semen during the study and for a short period after.
- Female participants must not be pregnant or breastfeeding and must use a highly effective birth control method during the study and for a short period after.
The participant or their parent/legal representative must be able to give signed informed consent, agreeing to follow the study’s rules and requirements.
The participant and their caregiver must be willing and able to follow all study requirements, as judged by the study investigator.

Who Cannot Join the Study?

Individuals who do not have a diagnosis of Tuberous Sclerosis Complex (TSC) cannot participate. TSC is a genetic disorder that causes non-cancerous tumors to form in different parts of the body.
Participants who do not experience seizures related to TSC are excluded. Seizures are sudden, uncontrolled electrical disturbances in the brain.
Children under the age of 3 and adults over the age of 65 are not eligible to participate.
Participants who are not able to provide informed consent or do not have a caregiver to provide consent on their behalf are excluded.
Individuals who are pregnant or breastfeeding cannot take part in the study.
Participants who are currently enrolled in another clinical trial are not eligible.
Individuals with other significant medical conditions that could interfere with the study are excluded.

Where you can join this trial?

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Other Sites

Site Name	City	Country
Centrum Medyczne Plejady Magdalena Celinska Loewenhoff Michal Zolnowski sp.k.	Cracow	Poland
Mkcddqv Ufpsfgxusx Oa Gworzw	Gdansk	Poland
Mhfdlnl Ultvsxwsoo Oy Shcgmhd Kygamcdk Pbaxhu	Katowice	Poland

Trial status

Country	Status	Recruitment Start
Poland	Not recruiting	01.03.2024

Trial locations

Investigated drugs:

Cannabidiol

EPIDIOLEX: This medication is used as an add-on therapy for individuals with seizures associated with Tuberous Sclerosis Complex (TSC). It is designed to help reduce the frequency and severity of seizures. The study aims to assess how this medication can improve behavioral and other related outcomes in patients aged 1 to 65 years old.

Investigated diseases:

Tuberous sclerosis complex

Tuberous Sclerosis Complex – Tuberous Sclerosis Complex (TSC) is a genetic disorder characterized by the growth of non-cancerous tumors in various parts of the body, including the brain, skin, kidneys, and heart. These tumors can lead to a range of symptoms, such as seizures, developmental delays, and skin abnormalities. The severity and progression of symptoms can vary widely among individuals. In some cases, TSC may also cause behavioral issues and learning difficulties. The condition is caused by mutations in either the TSC1 or TSC2 genes, which are responsible for regulating cell growth. TSC is a lifelong condition, and its manifestations can change over time.

Trial ID:

2023-507426-17-00

Protocol code:

JZP926-402

NCT ID:

NCT05864846

Trial Phase:

Human Pharmacology (Phase I) – Other

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Study on Cannabidiol as Add-on Therapy for Seizures in Patients Aged 1-65 with Tuberous Sclerosis Complex

What is this study about?

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