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

June 11, 2026 updated by: Jazz Pharmaceuticals

Long-term Follow-up of a Multicentre, Non-interventional, Prospective 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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
158

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • France
      • Amiens, France, 80054
        • CHU Amiens-service adulte
      • Amiens, France, 80054
        • CHU Amiens-service enfant
      • Angers, France, 49100
        • CHU Angers - service adulte
      • Angers, France, 49100
        • CHU Angers - service enfant
      • Bordeaux, France, 33000
        • CHU Bordeaux - service adulte
      • Bordeaux, France, 33000
        • CHU Bordeaux,neuropédiatrie
      • Bron, France, 69500
        • GH Est - Hôpital Femme Mère Enfant
      • Bron, France, 69677
        • HCL - Lyon - service adulte
      • Dijon, France, 21000
        • CHU Dijon - service neurophysiologie clinique
      • Grenoble, France, 38700
        • CHU Grenoble - service adulte
      • Grenoble, France, 38700
        • CHU Grenoble - service enfant
      • Le Kremlin-Bicêtre, France, 94270
        • APHP Kremlin Bicetre - service enfant
      • Lille, France, 59037
        • CHU Lille - service enfant
      • Lille, France, 59037
        • Hôpital Salengro - service adulte
      • Marseille, France, 13005
        • APHM - Marseille - service adulte
      • Marseille, France, 13005
        • Hôpital de la Timone - service enfant
      • Nancy, France, 54000
        • CHU Nancy - service adulte
      • Nancy, France, 54000
        • CHU Nancy - service enfant
      • Paris, France, 75013
        • APHP Pitié Salpetriere- service adulte
      • Paris, France, 75019
        • APHP Robert Debré - service enfant
      • Paris, France, 75743
        • APHP Necker - service enfant
      • Rennes, France, 35000
        • CHU Rennes - service adulte
      • Rennes, France, 35200
        • CHU Rennes - service enfant
      • Strasbourg, France, 67200
        • CHU Strasbourg - service enfant
      • Strasbourg, France, 67200
        • Hôpital de Hautepierre - service adulte
      • Toulouse, France, 31059
        • CHU Toulouse - service adulte
      • Toulouse, France, 31059
        • CHU Toulouse - service enfant
      • Tours, France, 37000
        • Hôpital Bretonneau - service adulte
      • Tours, France, 37044
        • Hôpital Clocheville - service enfant

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

2 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Participants for whom Epidyolex is prescribed according to the approved therapeutic indications and criteria of the summary of product Characteristics (SmPC). Participants will be recruited from approximately 15 facilities specializing in treating rare epilepsies in France.

Description

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)).

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
Up to 24 months post-dose.

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Percent Change From Baseline in Seizure Frequency (Average Per 28 Days)
Time Frame: Up to 24 months post-dose.
Up to 24 months post-dose.
50% Responder Rate
Time Frame: Up to 24 months post-dose.
Up to 24 months post-dose.
75% Responder Rate
Time Frame: Up to 24 months post-dose.
Up to 24 months post-dose.
100% Responder (Seizure Freedom) Rate
Time Frame: Up to 24 months post-dose.
Up to 24 months post-dose.
Number of Seizure-Free Days (Average Per 28 Days)
Time Frame: Up to 24 months post-dose.
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.
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
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.
Baseline; Up to 24 months post-dose.
Modal Dose of Epidyolex Administered to Participants
Time Frame: Up to 24 months post-dose.
Up to 24 months post-dose.
Maximum Dose of Epidyolex Administered to Participants
Time Frame: Up to 24 months post-dose.
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.
Up to 24 months post-dose.
Use of Rescue Medication Per Month
Time Frame: Up to 24 months post-dose.
A month is defined as 28 days.
Up to 24 months post-dose.
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.
Up to 24 months post-dose.
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.
Up to 24 months post-dose.
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.
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
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).
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
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).
Baseline; Up to 24 months post-dose.

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Jazz Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
April 17, 2023

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 1, 2026

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 6, 2023

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 6, 2023

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 16, 2023

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 15, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 11, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • GWEP20125

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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