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

Sponsor

Jazz Pharmaceuticals (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05772429

Collaborator

(none)

150

Enrollment

Locations

Anticipated Duration (Months)

7.1

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Seizures	Drug: Epidiolex

Study Design

Study Type:

Observational

Anticipated Enrollment :

150 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Anticipated Study Start Date :

Mar 1, 2023

Anticipated Primary Completion Date :

Mar 1, 2025

Anticipated Study Completion Date :

Mar 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Epidyolex Participants will receive Epidyolex in accordance with their routine clinical practice as prescribed by their physician.	Drug: Epidiolex Administered as an oral solution Other Names: Cannabidiol Epidyolex

Arm

Intervention/Treatment

Epidyolex

Participants will receive Epidyolex in accordance with their routine clinical practice as prescribed by their physician.

Drug: Epidiolex

Administered as an oral solution

Other Names:

Cannabidiol

Epidyolex

Outcome Measures

Primary Outcome Measures

Retention Rates After Initiation of Treatment With Epidyolex [Up to 24 months post-dose.]

Secondary Outcome Measures

Percent Change From Baseline in Seizure Frequency (Average Per 28 Days) [Up to 24 months post-dose.]
50% Responder Rate [Up to 24 months post-dose.]
75% Responder Rate [Up to 24 months post-dose.]
100% Responder (Seizure Freedom) Rate [Up to 24 months post-dose.]
Number of Seizure-Free Days (Average Per 28 Days) [Up to 24 months post-dose.]
Longest Duration of Seizure-Free Days in the Last 28 Days [Up to 24 months post-dose.]
Change From Baseline in Behavior Rating Inventory of Executive Function, Preschool Version (BRIEF-P) Score in Participants Aged ≤5 Years [Baseline; Up to 24 months post-dose.]
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.
Change From Baseline in Behavior Rating Inventory of Executive Function (BRIEF) Score in Participants Aged 6 to 18 Years [Baseline; Up to 24 months post-dose.]
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.
Change From Baseline in Behavior Rating Inventory of Executive Function, Adult Version (BRIEF- A) Score in Participants Aged >18 Years [Baseline; Up to 24 months post-dose.]
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.
Modal Dose of Epidyolex Administered to Participants [Up to 24 months post-dose.]
Maximum Dose of Epidyolex Administered to Participants [Up to 24 months post-dose.]
Change from Baseline in Average Daily Dosage of Concomitant Anti-Epileptic Drugs (AEDs) and Other Medicines [Up to 24 months post-dose.]
Use of Rescue Medication Per Month [Up to 24 months post-dose.]
A month is defined as 28 days.
Physician Global Clinical Impression of Change (PGIC) Score [Up to 24 months post-dose.]
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.
Caregiver Global Impression of Change (CGIC) Score [Up to 24 months post-dose.]
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.
Change from Baseline in Health Utility Index Mark II (HUI-2) Score [Up to 24 months post-dose.]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

2 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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)).

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	CHU Angers - service adulte	Angers	France	49100
2	CHU Angers - service enfant	Angers	France	49100
3	CHU Bordeaux	Bordeaux	France	33000
4	CHU Bordeaux - service adulte	Bordeaux	France	33076
5	GH Est - Hôpital Femme Mère Enfant	Bron	France	69500
6	HCL - Lyon - service adulte	Bron	France	69677
7	APHP Kremlin Bicetre - service adulte	Le Kremlin-Bicêtre	France	94270
8	APHP Kremlin Bicetre - service enfant	Le Kremlin-Bicêtre	France	94270
9	CHU Lille - service enfant	Lille	France	59037
10	Hôpital Salengro - service adulte	Lille	France	59037
11	APHP Pitié Salpetriere- service adulte	Paris	France	75013
12	APHP Robert Debré - service enfant	Paris	France	75019
13	APHP Necker - service enfant	Paris	France	75743
14	CHU Rennes - service adulte	Rennes	France	35000
15	CHU Rennes - service enfant	Rennes	France	35200
16	CHU Strasbourg - service enfant	Strasbourg	France	67200
17	Hôpital de Hautepierre - service adulte	Strasbourg	France	67200
18	CHU Toulouse - service adulte	Toulouse	France	31059
19	CHU Toulouse - service enfant	Toulouse	France	31059
20	Hôpital Bretonneau - service adulte	Tours	France	37000
21	Hôpital Clocheville - service enfant	Tours	France	37044