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NCT Number: NCT05772429

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

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.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Age range

2 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

CHU Amiens-service adulte, Amiens, France

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)).

Treatment and study plan

Epidiolex

Drug

Administered as an oral solution

Other names: Cannabidiol, Epidyolex

Primary outcomes

  1. Retention Rate Percentage After Initiation of Treatment With Epidyolex

    Time frame: 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 outcomes

  1. Percent Change From Baseline in Seizure Frequency (Average Per 28 Days)

    Time frame: Up to 24 months post-dose.

  2. 50% Responder Rate

    Time frame: Up to 24 months post-dose.

  3. 75% Responder Rate

    Time frame: Up to 24 months post-dose.

  4. 100% Responder (Seizure Freedom) Rate

    Time frame: Up to 24 months post-dose.

  5. Number of Seizure-Free Days (Average Per 28 Days)

    Time frame: Up to 24 months post-dose.

  6. Longest Duration of Seizure-Free Days in the Last 28 Days

    Time frame: Up to 24 months post-dose.

  7. Change From Baseline in Behavior Rating Inventory of Executive Function, Preschool Version (BRIEF-P) Score in Participants Aged ≤5 Years

    Time frame: 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).

  8. Change From Baseline in Behavior Rating Inventory of Executive Function (BRIEF) Score in Participants Aged 6 to 18 Years

    Time frame: 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).

  9. Change From Baseline in Behavior Rating Inventory of Executive Function, Adult Version (BRIEF- A) Score in Participants Aged >18 Years

    Time frame: 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.

  10. Modal Dose of Epidyolex Administered to Participants

    Time frame: Up to 24 months post-dose.

  11. Maximum Dose of Epidyolex Administered to Participants

    Time frame: Up to 24 months post-dose.

  12. Change from Baseline in Average Daily Dosage of Concomitant Anti-Epileptic Drugs (AEDs) and Other Medicines

    Time frame: Up to 24 months post-dose.

  13. Use of Rescue Medication Per Month

    Time frame: Up to 24 months post-dose.

    A month is defined as 28 days.

  14. Physician Global Clinical Impression of Change (PGIC) Score

    Time frame: 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.

  15. Caregiver Global Impression of Change (CGIC) Score

    Time frame: 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.

  16. Change from Baseline in Health Utility Index Mark II (HUI-2) Score

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Jazz Pharmaceuticals

Industry

Registry information

Official study title

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

Acronym: OPERA

Important dates

Study start
2023
Primary completion
2026
Study completion
2026
First posted
Mar 16, 2023
Registry last updated
Jun 15, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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