Clinical trial NCT05485831: Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
Official title: 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)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Lennox Gastaut Syndrome
Dravet Syndrome
Tuberous Sclerosis Complex
Keywords: seizures
Epidyolex
cannabidiol oral solution
Lead sponsor: Jazz Pharmaceuticals
Sponsor class: INDUSTRY
Collaborators: name: Advice Pharma Group srl
First posted: 2022-08-03
Enrollment: 100
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

Brief 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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants Remaining on Therapy from Enrollment
timeFrame: Baseline up to Week 52 post-dose.
Secondary outcomes:
measure: Average Number of Seizure-Free Days in the Last 28 Days
timeFrame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
measure: Longest Duration of Seizure Free Days in the Last 28 Days
timeFrame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
measure: Average Maintenance Dose of Epidyolex®
timeFrame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
measure: Maximum Maintenance Dose of Epidyolex®
timeFrame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
measure: Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs)
timeFrame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
measure: Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy
timeFrame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
measure: Change from Baseline to Week 28 (V3) in the Child Behavior Check List (CBCL)
timeFrame: Baseline to week 28 (V3) post-dose.
measure: Change from Baseline to Week 52 (V5) in the Child Behavior Check List (CBCL)
timeFrame: Baseline to week 52 (V5) post-dose.
measure: Change from Baseline to Week 28 (V3) in the Visual Analog Scale (VAS) of 10 Non-seizure Related Outcomes (NSRO)
timeFrame: Baseline to week 28 (V3) post-dose.
measure: Change from Baseline to Week 52 (V5) in the VAS of 10 NSRO
timeFrame: Baseline to week 52 (V5) post-dose.
measure: Average Caregiver Global Impression of Change (CGIC) Score at Week 28 (V3)
timeFrame: Week 28 (V3) post-dose.
measure: Average CGIC Score at Week 52 (V5)
timeFrame: Week 52 (V5) post-dose.
measure: Average Caregiver Global Impression of Change in Seizure Duration (CGICSD) Score at Week 28 (V3)
timeFrame: Week 28 (V3) post-dose.
measure: Average CGICSD Score at Week 52 (V5)
timeFrame: Week 52 (V5) post-dose.
measure: Average Physician Global Impression of Change (PGIC) Score at Week 28 (V3)
timeFrame: Week 28 (V3) post-dose.
measure: Average PGIC Score at Week 52 (V5)
timeFrame: Week 52 (V5) post-dose.
Other outcomes:
Locations:
city: Bologna
country: Italy
facility: Policlinico Sant'Orsola di Bologna
city: Catanzaro
country: Italy
facility: NPI AOU Mater Domini Università Magna Graecia
city: Chieti
country: Italy
facility: Università degli studi "G. D'annunzio" Chieti
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Meyer Neurologia Pediatrica
city: Genova
country: Italy
facility: Istituto Gaslini Neurologia Pediatrica e Malattie Muscolari
city: Messina
country: Italy
facility: AOU Gaetano Martino
city: Milano
country: Italy
facility: Centro Regionale Epilessia - ASST Santi Paolo e Carlo
city: Milano
country: Italy
facility: ASST Grande Ospedale metropolitano Niguarda
city: Novara
country: Italy
facility: AOU Maggiore della Carità di Novara
city: Padova
country: Italy
facility: AOU di Padova
city: Roma
country: Italy
facility: Policlinico Tor Vergata
city: Roma
country: Italy
facility: IRCCS Ospedale Pediatrico Bambin Gesù
city: Roma
country: Italy
facility: Fondazione Policlinico Universitario Agostino Gemelli - IRCCS-NPI
city: San Giovanni Rotondo
country: Italy
facility: Ospedale Casa del sollievo e della sofferenza
city: Torino
country: Italy
facility: AOU Città della Salute PO Molinette
Study officials:
References: