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.
Study Type
OBSERVATIONAL
Enrollment
158
Administered as an oral solution
CHU Amiens-service adulte
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.
Time frame: Up to 24 months post-dose.
Percent Change From Baseline in Seizure Frequency (Average Per 28 Days)
Time frame: Up to 24 months post-dose.
50% Responder Rate
Time frame: Up to 24 months post-dose.
75% Responder Rate
Time frame: Up to 24 months post-dose.
100% Responder (Seizure Freedom) Rate
Time frame: Up to 24 months post-dose.
Number of Seizure-Free Days (Average Per 28 Days)
Time frame: Up to 24 months post-dose.
Longest Duration of Seizure-Free Days in the Last 28 Days
Time frame: 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).
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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
...and 19 more locations
Time frame: 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).
Time frame: 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.
Time frame: Baseline; Up to 24 months post-dose.
Modal Dose of Epidyolex Administered to Participants
Time frame: Up to 24 months post-dose.
Maximum Dose of Epidyolex Administered to Participants
Time frame: Up to 24 months post-dose.
Change from Baseline in Average Daily Dosage of Concomitant Anti-Epileptic Drugs (AEDs) and Other Medicines
Time frame: Up to 24 months post-dose.
Use of Rescue Medication Per Month
A month is defined as 28 days.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Up to 24 months post-dose.