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

Study Overview
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Brief Summary
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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.

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

Conditions
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Seizures

Intervention / Treatment
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* Drug: Epidiolex


Participation Criteria
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Eligibility Criteria
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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)).

Ages Eligible for Study
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Minimum Age: 2 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Epidyolex<br>Participants will receive Epidyolex in accordance with their routine clinical practice as prescribed by their physician. | Drug: Epidiolex<br>* Administered as an oral solution<br>* Other names: Epidyolex;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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 | 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. | 
| 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. | 
| 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. | 
| 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 | A month is defined as 28 days. | Up to 24 months post-dose. | 
| 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. | 
| 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. | 
| 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. | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Seizure Therapy, Cannabidiol, GW CBD, Epidyolex, Epidiolex, Jazz CBD