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SeizuresOPERANCT05772429

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

An observational study of Seizures, run by Jazz Pharmaceuticals at 29 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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… Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Seizures
What is being tested?
Epidiolex
Type of treatment
Pill or oral medicine
How many people
158 participants
Where
29 sitesFrance
Study dates
17 Apr 2023to 25 Aug 2026
Phase
Observational
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

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.

Who may be able to join

  • 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.

Who cannot join

  • Previously initiated with Epidyolex before the start of the study (especially during the French early access program (EAP)).

Trial locations and contacts

29 sites in 1 country.

29 sites in 1 country

France29 sites
  • CHU Amiens-service adulte

    Amiens, 80054

  • CHU Amiens-service enfant

    Amiens, 80054

  • CHU Angers - service adulte

    Angers, 49100

  • CHU Angers - service enfant

    Angers, 49100

  • CHU Bordeaux - service adulte

    Bordeaux, 33000

  • CHU Bordeaux,neuropédiatrie

    Bordeaux, 33000

  • GH Est - Hôpital Femme Mère Enfant

    Bron, 69500

  • HCL - Lyon - service adulte

    Bron, 69677

  • CHU Dijon - service neurophysiologie clinique

    Dijon, 21000

  • CHU Grenoble - service adulte

    Grenoble, 38700

  • CHU Grenoble - service enfant

    Grenoble, 38700

  • APHP Kremlin Bicetre - service enfant

    Le Kremlin-Bicêtre, 94270

  • CHU Lille - service enfant

    Lille, 59037

  • Hôpital Salengro - service adulte

    Lille, 59037

  • APHM - Marseille - service adulte

    Marseille, 13005

  • Hôpital de la Timone - service enfant

    Marseille, 13005

  • CHU Nancy - service adulte

    Nancy, 54000

  • CHU Nancy - service enfant

    Nancy, 54000

  • APHP Pitié Salpetriere- service adulte

    Paris, 75013

  • APHP Robert Debré - service enfant

    Paris, 75019

  • APHP Necker - service enfant

    Paris, 75743

  • CHU Rennes - service adulte

    Rennes, 35000

  • CHU Rennes - service enfant

    Rennes, 35200

  • CHU Strasbourg - service enfant

    Strasbourg, 67200

  • Hôpital de Hautepierre - service adulte

    Strasbourg, 67200

  • CHU Toulouse - service adulte

    Toulouse, 31059

  • CHU Toulouse - service enfant

    Toulouse, 31059

  • Hôpital Bretonneau - service adulte

    Tours, 37000

  • Hôpital Clocheville - service enfant

    Tours, 37044

Other trials in France

About this study

Official title: 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.

Conditions
Seizures
Being tested
Epidiolex
Keywords
Seizure Therapy, Cannabidiol, GW CBD, Epidyolex, Epidiolex, Jazz CBD
Medical subject headings
Seizures

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • EpidiolexDrug

    Also called Cannabidiol, Epidyolex

    Administered as an oral solution

Study groups in NCT05772429
Study groupRoleWhat they receive
EpidyolexParticipants will receive Epidyolex in accordance with their routine clinical practice as prescribed by their physician.
Receives: Drug: Epidiolex

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Up to 24 months post-dose.

Secondary outcomes (16)
  1. Percent Change From Baseline in Seizure Frequency (Average Per 28 Days)

    Measured: Up to 24 months post-dose.

  2. 50% Responder Rate

    Measured: Up to 24 months post-dose.

  3. 75% Responder Rate

    Measured: Up to 24 months post-dose.

  4. 100% Responder (Seizure Freedom) Rate

    Measured: Up to 24 months post-dose.

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

    Measured: Up to 24 months post-dose.

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

    Measured: 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

    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).

    Measured: Baseline; Up to 24 months post-dose.

  8. 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).

    Measured: Baseline; Up to 24 months post-dose.

  9. 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.

    Measured: Baseline; Up to 24 months post-dose.

  10. Modal Dose of Epidyolex Administered to Participants

    Measured: Up to 24 months post-dose.

  11. Maximum Dose of Epidyolex Administered to Participants

    Measured: Up to 24 months post-dose.

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

    Measured: Up to 24 months post-dose.

  13. Use of Rescue Medication Per Month

    A month is defined as 28 days.

    Measured: Up to 24 months post-dose.

  14. 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.

    Measured: Up to 24 months post-dose.

  15. 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.

    Measured: Up to 24 months post-dose.

  16. 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.

    Measured: Up to 24 months post-dose.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Mar 2023
  2. Study start17 Apr 2023
  3. Primary completion25 Aug 2026
  4. Study completion25 Aug 2026

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Observational
Observational model
Cohort
Time perspective
Prospective
Enrolment
158 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05772429
Sponsor's protocol ID
GWEP20125
Acronym
OPERA
Status last verified
Nov 2026
First submitted
6 Mar 2023
Last updated
3 Nov 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

An observational study of Seizures, run by Jazz Pharmaceuticals at 29 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 3 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Retention Rate Percentage After Initiation of Treatment With Epidyolex (time frame: Up to 24 months post-dose.). It is what the study was designed to measure, not a finding.

Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Nov 2026.

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