Long-Term Follow-Up of a Cohort of Participants Prescribed Epidyolex in France in a Real-life Setting
- Registration Number
- NCT05772429
- Lead Sponsor
- Jazz Pharmaceuticals
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 158
- 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
- Previously initiated with Epidyolex before the start of the study (especially during the French early access program (EAP)).
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Epidyolex Epidiolex Participants will receive Epidyolex in accordance with their routine clinical practice as prescribed by their physician.
- Primary Outcome Measures
Name Time Method Retention Rate Percentage After Initiation of Treatment With Epidyolex Up to 24 months post-dose. Retention rate will be measured through assessing the frequency of seizures and the length of time on treatment before a seizure event.
- Secondary Outcome Measures
Name Time Method Percent Change From Baseline in Seizure Frequency (Average Per 28 Days) 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 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.
Maximum Dose of Epidyolex Administered to Participants 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 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. 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).
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. 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).
50% Responder Rate Up to 24 months post-dose. Modal 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.
75% Responder Rate Up to 24 months post-dose. 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.
Trial Locations
- Locations (29)
APHP Pitié Salpetriere- service adulte
🇫🇷Paris, France
APHP Robert Debré - service enfant
🇫🇷Paris, France
APHP Necker - service enfant
🇫🇷Paris, France
CHU Rennes - service adulte
🇫🇷Rennes, 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 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
APHM - Marseille - service adulte
🇫🇷Marseille, France
CHU Lille - service enfant
🇫🇷Lille, France
Hôpital Salengro - service adulte
🇫🇷Lille, France
Hôpital de la Timone - service enfant
🇫🇷Marseille, France
CHU Nancy - service adulte
🇫🇷Nancy, France
CHU Nancy - service enfant
🇫🇷Nancy, 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, France