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

Observational, Prospective, Multicenter Study of Epidyolex® (Cannabidiol CBD 100 mg/ml) Oral Solution, as Adjunctive Treatment for Seizures Associated With Lennox- Gastaut Syndrome (LGS), Dravet Syndrome (DS) and Tuberous Sclerosis Complex (TSC)

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Tuberous Sclerosis Complex; Dravet Syndrome; Lennox Gastaut Syndrome
• Intervention / Treatment: Drug: Epidiolex 100 mg/mL Oral Solution

Detailed Description

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.

• Study Type: Observational
• Enrollment (Actual): 111

Contacts and Locations

Study Locations

• Italy
  • Bologna, Italy, 40138
    • Policlinico Sant'Orsola di Bologna
  • Catanzaro, Italy, 88100
    • NPI AOU Mater Domini Università Magna Graecia
  • Chieti, Italy, 66100
    • Università degli studi "G. D'annunzio" Chieti
  • Florence, Italy, 50139
    • Azienda Ospedaliera Universitaria Meyer Neurologia Pediatrica
  • Genova, Italy, 16147
    • Istituto Gaslini Neurologia Pediatrica e Malattie Muscolari
  • Messina, Italy, 98124
    • AOU Gaetano Martino
  • Milan, Italy, 20162
    • ASST Grande Ospedale Metropolitano Niguarda
  • Milan, Italy, 20142
    • Centro Regionale Epilessia - ASST Santi Paolo e Carlo
  • Naples, Italy, 80131
    • Azienda Ospedaliera Universitaria Federico II
  • Novara, Italy, 28100
    • AOU Maggiore della Carità di Novara
  • Padova, Italy, 35128
    • AOU di Padova
  • Roma, Italy, 00133
    • Policlinico Tor Vergata
  • Roma, Italy, 00165
    • IRCCS Ospedale Pediatrico Bambin Gesù
  • Roma, Italy, 00168
    • Fondazione Policlinico Universitario Agostino Gemelli - IRCCS-NPI
  • Roma, Italy, 00189
    • Azienda Ospedaliero Universitaria Sant'Andrea
  • San Giovanni Rotondo, Italy, 71013
    • Ospedale Casa del sollievo e della sofferenza
  • Torino, Italy, 10126
    • AOU Città della Salute PO Molinette
  • Torrette, Italy, 60126
    • Azienda Ospedaliero Universitaria delle Marche

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 17 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex; Participants ≥2 years of age diagnosed with LGS, DS, and TSC.	Drug: Epidiolex 100 mg/mL Oral Solution; As prescribed in routine clinical practice in Italy.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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]).	Baseline up to Week 52 post-dose.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Average Number of Seizure-Free Days in the Last 28 Days		Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Longest Duration of Seizure Free Days in the Last 28 Days		Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Average Maintenance Dose of Epidyolex®		Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Maximum Maintenance Dose of Epidyolex®		Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs)		Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy		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.	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.	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.	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.	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.	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.	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.	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.	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.	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.	Baseline to week 52 (V5) post-dose.

Collaborators and Investigators

• Sponsor: Jazz Pharmaceuticals
• Collaborators: Advice Pharma Group srl

Study record dates

Study Major Dates

• Study Start (Actual): February 24, 2025
• Primary Completion (Estimated): February 1, 2027
• Study Completion (Estimated): February 1, 2027

Study Registration Dates

• First Submitted: July 29, 2022
• First Submitted That Met QC Criteria: August 1, 2022
• First Posted (Actual): August 3, 2022

Study Record Updates

• Last Update Posted (Actual): June 3, 2026
• Last Update Submitted That Met QC Criteria: June 1, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: seizures; Epidyolex; cannabidiol oral solution
• Additional Relevant MeSH Terms: Epileptic Syndromes; Neurologic Manifestations; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neoplasms; Genetic Diseases, Inborn; Neurodegenerative Diseases; Congenital Abnormalities; Epilepsy, Generalized; Epilepsy; Heredodegenerative Disorders, Nervous System; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Hamartoma; Neoplasms, Multiple Primary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Tuberous Sclerosis; Seizures; Epilepsies, Myoclonic; Lennox Gastaut Syndrome; Organic Chemicals; Pharmaceutical Preparations; Hydrocarbons; Terpenes; Cannabinoids; Cannabidiol; Solutions
• Other Study ID Numbers: JZP926-525

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No