An observational study in Lennox Gastaut Syndrome, Dravet Syndrome and Tuberous Sclerosis Complex, sponsored by Jazz Pharmaceuticals. Active, not recruiting at 18 sites in Italy. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2026-06-03.
Sponsored by Jazz Pharmaceuticals · Observational
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.
This observational study evaluates a Real-World population of children and adult participants affected by LGS, DS, and TSC and the effect of therapy with Epidyolex® administered according to clinical practice on epileptic symptoms. The study will assess specific scales, questionnaires, and the satisfaction of the caregivers and the participants/tutors.
68 studies on the registry are indexed under Lennox Gastaut Syndrome; 14 are open to participants now.
Browse Lennox Gastaut Syndrome studies →Jazz Pharmaceuticals is the lead sponsor of 173 studies on the registry; 18 are open to participants now.
Of its 42 completed or terminated interventional studies of FDA-regulated products, 32 (76%) have results posted.
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The study will comprise of 70-100 male and female participants, aged ≥ 2 years, diagnosed with LGS, DS, and TSC. The decision to prescribe Epidyolex® as adjunctive therapy for seizures associated with LGS, DS, and TSC will be entirely independent of including the participant in the study.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants ≥2 years of age diagnosed with LGS, DS, and TSC.
Drug: Epidiolex 100 mg/mL Oral Solution
Drug page: Cannabidiol
As prescribed in routine clinical practice in Italy.
Number of Participants Remaining on Therapy from Enrollment
Treatment retention will be evaluated through the proportion of participants remaining on therapy from the enrollment visit (baseline, V0) to each study visit (Weeks 4 \[V1\], 16 \[V2\], 28 \[V3\], 40 \[V4\], 52 \[V5\]).
Time frame: Baseline up to Week 52 post-dose.
Average Number of Seizure-Free Days in the Last 28 Days
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Longest Duration of Seizure Free Days in the Last 28 Days
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Average Maintenance Dose of Epidyolex®
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Maximum Maintenance Dose of Epidyolex®
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs)
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Change from Baseline to Week 28 (V3) in the Child Behavior Check List (CBCL)
The CBCL is a standardized form that parents fill out to describe their children's behavioral and emotional problems(e.g., anxiety, depression, social problems). The CBCL is also scored on (optional) competence scales for activities, social relations, school and total competence. The CBCL consists of 113 questions, scored on a three-point Likert scale (0=absent, 1= occurs sometimes, 2=occurs often). A higher score indicates a worse outcome.
Time frame: Baseline to week 28 (V3) post-dose.
Change from Baseline to Week 52 (V5) in the Child Behavior Check List (CBCL)
The CBCL is a standardized form that parents fill out to describe their children's behavioral and emotional problems(e.g., anxiety, depression, social problems). The CBCL is also scored on (optional) competence scales for activities, social relations, school and total competence. The CBCL consists of 113 questions, scored on a three-point Likert scale (0=absent, 1= occurs sometimes, 2=occurs often). A higher score indicates a worse outcome.
Time frame: Baseline to week 52 (V5) post-dose.
Change from Baseline to Week 28 (V3) in the Visual Analog Scale (VAS) of 10 Non-seizure Related Outcomes (NSRO)
VAS assessments are based on 10 NSRO (i.e., Alertness, Sleep Disorder, Motor Skills, Attention, Cognition, Communication, Behavior, Emotional/Social Functioning, Activities of Daily Living, and Participation). VAS is measured on a scale of 0-100; a score of 0 indicates a better outcome, and a score of 100 indicates a worse outcome.
Time frame: Baseline to week 28 (V3) post-dose.
Change from Baseline to Week 52 (V5) in the VAS of 10 NSRO
VAS assessments are based on 10 NSRO (i.e., Alertness, Sleep Disorder, Motor Skills, Attention, Cognition, Communication, Behavior, Emotional/Social Functioning, Activities of Daily Living, and Participation). VAS is measured on a scale of 0-100; a score of 0 indicates a better outcome, and a score of 100 indicates a worse outcome.
Time frame: Baseline to week 52 (V5) post-dose.
Average Caregiver Global Impression of Change (CGIC) Score at Week 28 (V3)
The CGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. The CGIC comprises of the following question: "Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment) using the scale below." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Time frame: Week 28 (V3) post-dose.
Average CGIC Score at Week 52 (V5)
The CGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. The CGIC comprises of the following question: "Since your child started treatment, please assess the status of your child's overall condition (comparing their condition now to their condition before treatment) using the scale below." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Time frame: Week 52 (V5) post-dose.
Average Caregiver Global Impression of Change in Seizure Duration (CGICSD) Score at Week 28 (V3)
The CGICSD comprises the following question to be rated on a three-point scale for each seizure subtype: Since the patient started treatment, please assess the average duration of the patient's seizures (comparing their condition now to their condition before treatment) using the scale below. The scale markers are: 1=Average duration of seizures has decreased; 2=Average duration of seizures has stayed the same; 3=Average duration of seizures has increased. Higher scores indicate a worse outcome.
Time frame: Week 28 (V3) post-dose.
Average CGICSD Score at Week 52 (V5)
The CGICSD comprises the following question to be rated on a three-point scale for each seizure subtype: Since the patient started treatment, please assess the average duration of the patient's seizures (comparing their condition now to their condition before treatment) using the scale below. The scale markers are: 1=Average duration of seizures has decreased; 2=Average duration of seizures has stayed the same; 3=Average duration of seizures has increased. Higher scores indicate a worse outcome.
Time frame: Week 52 (V5) post-dose.
Average Physician Global Impression of Change (PGIC) Score at Week 28 (V3)
The PGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. The PGIC comprises of the following question: "Please assess the change in the patient's general functional abilities since enrolment." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Time frame: Week 28 (V3) post-dose.
Average PGIC Score at Week 52 (V5)
The PGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. The PGIC comprises of the following question: "Please assess the change in the patient's general functional abilities since enrolment." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Time frame: Week 52 (V5) post-dose.
Plan to share: No
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Started Feb 2025 (actual). Primary completion is estimated for Feb 2027, 24 months after the start.
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This study is active, not recruiting, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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