Long-Term Follow-Up of a Cohort of Participants Prescribed Epidyolex in France in a Real-life Setting

Seizures Clinical Trial

— OPERA  

Official title:

Long-term Follow-up of a Multicentre, Non-interventional, Prospective Cohort of Participants Prescribed Epidyolex in France in a Real-life Setting

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05772429
• Other study ID #: GWEP20125
• Status: Recruiting
• Start date: April 17, 2023
• Est. completion date: March 1, 2025

Study information

• Verified date: February 2024
• Source: Jazz Pharmaceuticals
• Contact: Clinical Trial Disclosure & Transparency
• Phone: 215-832-3750
• Email: ClinicalTrialDisclosure[@]JazzPharma.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: March 1, 2025
• Est. primary completion date: March 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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)).

Related Conditions & MeSH terms

• Seizures

Intervention

Drug:
• Epidiolex
Administered as an oral solution

Locations

Country	Name	City	State
France	CHU Amiens-service adulte	Amiens
France	CHU Amiens-service enfant	Amiens
France	CHU Angers - service adulte	Angers
France	CHU Angers - service enfant	Angers
France	CHU Bordeaux - service adulte	Bordeaux
France	CHU Bordeaux,neuropédiatrie	Bordeaux
France	GH Est - Hôpital Femme Mère Enfant	Bron
France	HCL - Lyon - service adulte	Bron
France	CHU Clermont-Ferrand - service enfant	Clermont-Ferrand
France	CHU Gabriel Montpied - service neurologie	Clermont-Ferrand
France	CHU Dijon - service neurophysiologie clinique	Dijon
France	CHU Grenoble - service adulte	Grenoble
France	CHU Grenoble - service enfant	Grenoble
France	APHP Kremlin Bicetre - service enfant	Le Kremlin-Bicêtre
France	CHU Lille - service enfant	Lille
France	Hôpital Salengro - service adulte	Lille
France	APHM - Marseille - service adulte	Marseille
France	Hôpital de la Timone - service enfant	Marseille
France	CHU Nancy - service adulte	Nancy
France	CHU Nancy - service enfant	Nancy
France	APHP Necker - service enfant	Paris
France	APHP Pitié Salpetriere- service adulte	Paris
France	APHP Robert Debré - service enfant	Paris
France	CHU Rennes - service adulte	Rennes
France	CHU Rennes - service enfant	Rennes
France	CHU Strasbourg - service enfant	Strasbourg
France	Hôpital de Hautepierre - service adulte	Strasbourg
France	CHU Toulouse - service adulte	Toulouse
France	CHU Toulouse - service enfant	Toulouse
France	Hôpital Bretonneau - service adulte	Tours
France	Hôpital Clocheville - service enfant	Tours

Sponsors (1)

Lead Sponsor	Collaborator
Jazz Pharmaceuticals

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Retention Rate Percentage After Initiation of Treatment With Epidyolex	Retention rate will be measured through assessing the frequency of seizures and the length of time on treatment before a seizure event.	Up to 24 months post-dose.
Secondary	Percent Change From Baseline in Seizure Frequency (Average Per 28 Days)		Up to 24 months post-dose.
Secondary	50% Responder Rate		Up to 24 months post-dose.
Secondary	75% Responder Rate		Up to 24 months post-dose.
Secondary	100% Responder (Seizure Freedom) Rate		Up to 24 months post-dose.
Secondary	Number of Seizure-Free Days (Average Per 28 Days)		Up to 24 months post-dose.
Secondary	Longest Duration of Seizure-Free Days in the Last 28 Days		Up to 24 months post-dose.
Secondary	Change From Baseline in Behavior Rating Inventory of Executive Function, Preschool Version (BRIEF-P) Score in Participants Aged =5 Years	The BRIEF-P is a 63-item questionnaire which consists of a single Rating Form used by parents, teachers, and day care providers to assess a child's executive functions within the context of their everyday environments--home and preschool. High scores obtained on the BRIEF-P suggest a higher level of dysfunction in a specific domain of executive functions. The BRIEF rating scores is an ordinal set of a technically summarized degree of disorder in functionality: Never = 1 (Minimum), Sometimes = 2 and Often = 3 (Maximum).	Baseline; Up to 24 months post-dose.
Secondary	Change From Baseline in Behavior Rating Inventory of Executive Function (BRIEF) Score in Participants Aged 6 to 18 Years	BRIEF is an 86-item questionnaire used to assess the executive function behaviors at home and at school for children and adolescents ages 5-18. High scores obtained on the BRIEF suggest a higher level of dysfunction in a specific domain of executive functions. The BRIEF rating scores is an ordinal set of a technically summarized degree of disorder in functionality: Never = 1 (Minimum), Sometimes = 2 and Often = 3 (Maximum).	Baseline; Up to 24 months post-dose.
Secondary	Change From Baseline in Behavior Rating Inventory of Executive Function, Adult Version (BRIEF- A) Score in Participants Aged >18 Years	BRIEF-A is a patient-reported scale to measure various aspects of adult executive functioning and self-regulation in the person's everyday environment. It is a 75-item questionnaire that participants rate on a 3-point Likert scale (1 = behavior is never observed to 3 = behavior is often observed). Higher scores indicate greater impairment in executive functioning.	Baseline; Up to 24 months post-dose.
Secondary	Modal Dose of Epidyolex Administered to Participants		Up to 24 months post-dose.
Secondary	Maximum Dose of Epidyolex Administered to Participants		Up to 24 months post-dose.
Secondary	Change from Baseline in Average Daily Dosage of Concomitant Anti-Epileptic Drugs (AEDs) and Other Medicines		Up to 24 months post-dose.
Secondary	Use of Rescue Medication Per Month	A month is defined as 28 days.	Up to 24 months post-dose.
Secondary	Physician Global Clinical Impression of Change (PGIC) Score	The PGIC comprises the following question: "Please assess the change in the patient's general functional abilities since enrollment," scored on a seven-point scale from 1 (Very Much Improved) to 7 (Very Much Worse). Lower scores on the scale indicate a better quality of life.	Up to 24 months post-dose.
Secondary	Caregiver Global Impression of Change (CGIC) Score	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)," scored on a seven-point scale from 1 (Very Much Improved) to 7 (Very Much Worse). Lower scores on the scale indicate a better quality of life.	Up to 24 months post-dose.
Secondary	Change from Baseline in Health Utility Index Mark II (HUI-2) Score	The HUI-2 is a generic measure of health status and health-related quality of life. There are 15 questions in the questionnaire with 1-week recall completed by the caregiver. Answers to the questionnaire are mapped into a classification system of 7 sets of utility scores (sensation, mobility, emotion, cognition, self-care, pain, and fertility) with a scoring scale of a minimum score of 0.00 (lack of functional capacity or most disabled) to a maximum of 1.00 (full function or no disability). A higher score indicates a better outcome.	Up to 24 months post-dose.