Clinical trial NCT05772429: Long-Term Follow-Up of a Cohort of Participants Prescribed Epidyolex in France in a Real-life Setting
Official title: Long-term Follow-up of a Multicentre, Non-interventional, Prospective Cohort of Participants Prescribed Epidyolex in France in a Real-life Setting
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Seizures
Keywords: Seizure Therapy
Cannabidiol
GW CBD
Epidyolex
Epidiolex
Jazz CBD
Lead sponsor: Jazz Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2023-03-16
Enrollment: 158
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants for whom Epidyolex is prescribed according to the approved therapeutic indications and criteria of the summary of product Characteristics (SmPC). Participants will be recruited from approximately 15 facilities specializing in treating rare epilepsies in France.

Brief summary: This is a multi-center, non-interventional and prospective study of patients receiving Epidyolex as part of standard clinical practice in France. The study will state an overview of patient characteristics and clinical history (including age, sex, diagnosis, duration of epilepsy, predominant seizure type, previous medications, current co-medications and Epidyolex dose), and an evaluation of retention rates, safety profile, seizure activity, changes in executive function and quality of life measured in a 2-year follow-up period.


Eligibility criteria: Key Inclusion Criteria:

* Individuals for whom treatment with Epidyolex has been initiated by a physician with experience in the treatment of epilepsy.
* The participant and/or parent(s)/legal representative is willing and able to give informed consent/assent for participation in the study.

Key Exclusion Criteria:

* Previously initiated with Epidyolex before the start of the study (especially during the French early access program (EAP)).

Primary outcomes:
measure: Retention Rate Percentage After Initiation of Treatment With Epidyolex
timeFrame: Up to 24 months post-dose.
Secondary outcomes:
measure: Percent Change From Baseline in Seizure Frequency (Average Per 28 Days)
timeFrame: Up to 24 months post-dose.
measure: 50% Responder Rate
timeFrame: Up to 24 months post-dose.
measure: 75% Responder Rate
timeFrame: Up to 24 months post-dose.
measure: 100% Responder (Seizure Freedom) Rate
timeFrame: Up to 24 months post-dose.
measure: Number of Seizure-Free Days (Average Per 28 Days)
timeFrame: Up to 24 months post-dose.
measure: Longest Duration of Seizure-Free Days in the Last 28 Days
timeFrame: Up to 24 months post-dose.
measure: Change From Baseline in Behavior Rating Inventory of Executive Function, Preschool Version (BRIEF-P) Score in Participants Aged ≤5 Years
timeFrame: Baseline; Up to 24 months post-dose.
measure: Change From Baseline in Behavior Rating Inventory of Executive Function (BRIEF) Score in Participants Aged 6 to 18 Years
timeFrame: Baseline; Up to 24 months post-dose.
measure: Change From Baseline in Behavior Rating Inventory of Executive Function, Adult Version (BRIEF- A) Score in Participants Aged >18 Years
timeFrame: Baseline; Up to 24 months post-dose.
measure: Modal Dose of Epidyolex Administered to Participants
timeFrame: Up to 24 months post-dose.
measure: Maximum Dose of Epidyolex Administered to Participants
timeFrame: Up to 24 months post-dose.
measure: Change from Baseline in Average Daily Dosage of Concomitant Anti-Epileptic Drugs (AEDs) and Other Medicines
timeFrame: Up to 24 months post-dose.
measure: Use of Rescue Medication Per Month
timeFrame: Up to 24 months post-dose.
measure: Physician Global Clinical Impression of Change (PGIC) Score
timeFrame: Up to 24 months post-dose.
measure: Caregiver Global Impression of Change (CGIC) Score
timeFrame: Up to 24 months post-dose.
measure: Change from Baseline in Health Utility Index Mark II (HUI-2) Score
timeFrame: Up to 24 months post-dose.
Other outcomes:
Locations:
city: Amiens
country: France
facility: CHU Amiens-service adulte
city: Amiens
country: France
facility: CHU Amiens-service enfant
city: Angers
country: France
facility: CHU Angers - service adulte
city: Angers
country: France
facility: CHU Angers - service enfant
city: Bordeaux
country: France
facility: CHU Bordeaux - service adulte
city: Bordeaux
country: France
facility: CHU Bordeaux,neuropédiatrie
city: Bron
country: France
facility: GH Est - Hôpital Femme Mère Enfant
city: Bron
country: France
facility: HCL - Lyon - service adulte
city: Dijon
country: France
facility: CHU Dijon - service neurophysiologie clinique
city: Grenoble
country: France
facility: CHU Grenoble - service adulte
city: Grenoble
country: France
facility: CHU Grenoble - service enfant
city: Le Kremlin-Bicêtre
country: France
facility: APHP Kremlin Bicetre - service enfant
city: Lille
country: France
facility: CHU Lille - service enfant
city: Lille
country: France
facility: Hôpital Salengro - service adulte
city: Marseille
country: France
facility: APHM - Marseille - service adulte
city: Marseille
country: France
facility: Hôpital de la Timone - service enfant
city: Nancy
country: France
facility: CHU Nancy - service adulte
city: Nancy
country: France
facility: CHU Nancy - service enfant
city: Paris
country: France
facility: APHP Pitié Salpetriere- service adulte
city: Paris
country: France
facility: APHP Robert Debré - service enfant
city: Paris
country: France
facility: APHP Necker - service enfant
city: Rennes
country: France
facility: CHU Rennes - service adulte
city: Rennes
country: France
facility: CHU Rennes - service enfant
city: Strasbourg
country: France
facility: CHU Strasbourg - service enfant
city: Strasbourg
country: France
facility: Hôpital de Hautepierre - service adulte
city: Toulouse
country: France
facility: CHU Toulouse - service adulte
city: Toulouse
country: France
facility: CHU Toulouse - service enfant
city: Tours
country: France
facility: Hôpital Bretonneau - service adulte
city: Tours
country: France
facility: Hôpital Clocheville - service enfant
Study officials:
References: