Active — Not RecruitingNot Applicable

Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY

Italy111 participantsStarted 2025-02-24

Plain-language summary

This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics. The eligible participants are expected to participate in the study for a duration of 52 weeks of treatment.

Who can participate

Age range

2 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Adult patients, and minor patients ≥ 2 years old diagnosed with LGS, DS or TSC. * Clinical decision, taken by the physician, to initiate Epidyolex® * Adult participants, parents or legal representatives must be willing and able to give informed consent/assent for participation in the study. Exclusion Criteria: * Participants currently using or have used recreational, medicinal cannabis, or cannabinoid-based products within the three months prior to study entry and are unwilling to abstain from these products for the duration of the study. * Any reason, according to Investigator's judgment, able to compromise compliance with procedures outlined in the study There will not be any other specific exclusion criteria; however, contraindications, special warnings, and precautions for use as detailed in the Summary of Product Characteristics (SmPC) (particularly related to raising of aspartate aminotransferase \[AST\], alanine aminotransferase \[ALT\], and total bilirubin) will have to be considered by the treating physician.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Number of Participants Remaining on Therapy from Enrollment

Timeframe: Baseline up to Week 52 post-dose.

Trial details

NCT IDNCT05485831

SponsorJazz Pharmaceuticals

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2027-02

View on ClinicalTrials.gov More Lennox Gastaut Syndrome trials