Lennox Gastaut SyndromeEpiTALYNCT05485831
Epidyolex® in Lennox Gastaut, Dravet Syndrome and Tuberous Sclerosis Complex: an Observational Study in ITALY
An observational study of Lennox Gastaut Syndrome and Dravet Syndrome, run by Jazz Pharmaceuticals at 18 sites in Italy.
Trial summary
This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics. The eligible participants are expected to participate in the study for a duration of 52 weeks of treatment. Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Lennox Gastaut Syndrome, Dravet Syndrome, Tuberous Sclerosis Complex
- What is being tested?
- Epidiolex 100 mg/mL Oral Solution
- Type of treatment
- Pill or oral medicine
- How many people
- 111 participants
- Where
- 18 sitesItaly
- Study dates
- 24 Feb 2025to Feb 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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: The study will comprise of 70-100 male and female participants, aged ≥ 2 years, diagnosed with LGS, DS, and TSC. The decision to prescribe Epidyolex® as adjunctive therapy for seizures associated with LGS, DS, and TSC will be entirely independent of including the participant in the study.
Who may be able to join
- Adult patients, and minor patients ≥ 2 years old diagnosed with LGS, DS or TSC.
- Clinical decision, taken by the physician, to initiate Epidyolex®
- Adult participants, parents or legal representatives must be willing and able to give informed consent/assent for participation in the study.
Who cannot join
- Participants currently using or have used recreational, medicinal cannabis, or cannabinoid-based products within the three months prior to study entry and are unwilling to abstain from these products for the duration of the study.
- Any reason, according to Investigator's judgment, able to compromise compliance with procedures outlined in the study There will not be any other specific exclusion criteria; however, contraindications, special warnings, and precautions for use as detailed in the Summary of Product Characteristics (SmPC) (particularly related to raising of aspartate aminotransferase [AST], alanine aminotransferase [ALT], and total bilirubin) will have to be considered by the treating physician.
Trial locations and contacts
18 sites in 1 country.
18 sites in 1 country
Italy18 sites
Policlinico Sant'Orsola di Bologna
Bologna, 40138
NPI AOU Mater Domini Università Magna Graecia
Catanzaro, 88100
Università degli studi "G. D'annunzio" Chieti
Chieti, 66100
Azienda Ospedaliera Universitaria Meyer Neurologia Pediatrica
Florence, 50139
Istituto Gaslini Neurologia Pediatrica e Malattie Muscolari
Genova, 16147
AOU Gaetano Martino
Messina, 98124
Centro Regionale Epilessia - ASST Santi Paolo e Carlo
Milan, 20142
ASST Grande Ospedale metropolitano Niguarda
Milan, 20162
Azienda Ospedaliera Universitaria Federico II
Naples, 80131
AOU Maggiore della Carità di Novara
Novara, 28100
AOU di Padova
Padova, 35128
Policlinico Tor Vergata
Roma, 00133
IRCCS Ospedale Pediatrico Bambin Gesù
Roma, 00165
Fondazione Policlinico Universitario Agostino Gemelli - IRCCS-NPI
Roma, 00168
Azienda Ospedaliero Universitaria Sant'Andrea
Roma, 00189
Ospedale Casa del sollievo e della sofferenza
San Giovanni Rotondo, 71013
AOU Città della Salute PO Molinette
Torino, 10126
Azienda Ospedaliero Universitaria delle Marche
Torrette, 60126
About this study
Official title: Observational, Prospective, Multicenter Study of Epidyolex® (Cannabidiol CBD 100 mg/ml) Oral Solution, as Adjunctive Treatment for Seizures Associated With Lennox- Gastaut Syndrome (LGS), Dravet Syndrome (DS) and Tuberous Sclerosis Complex (TSC)
This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics. The eligible participants are expected to participate in the study for a duration of 52 weeks of treatment.
Read the detailed description
This observational study evaluates a Real-World population of children and adult participants affected by LGS, DS, and TSC and the effect of therapy with Epidyolex® administered according to clinical practice on epileptic symptoms. The study will assess specific scales, questionnaires, and the satisfaction of the caregivers and the participants/tutors.
- Being tested
- Epidiolex 100 mg/mL Oral Solution
- Keywords
- seizures, Epidyolex, cannabidiol oral solution
- Medical subject headings
- Lennox Gastaut Syndrome, Epilepsies, Myoclonic, Tuberous Sclerosis, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Epidiolex 100 mg/mL Oral SolutionDrug
As prescribed in routine clinical practice in Italy.
| Study group | Role | What they receive |
|---|---|---|
| Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex | Participants ≥2 years of age diagnosed with LGS, DS, and TSC. Receives: Drug: Epidiolex 100 mg/mL Oral Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Remaining on Therapy from Enrollment
Treatment retention will be evaluated through the proportion of participants remaining on therapy from the enrollment visit (baseline, V0) to each study visit (Weeks 4 [V1], 16 [V2], 28 [V3], 40 [V4], 52 [V5]).
Measured: Baseline up to Week 52 post-dose.
Secondary outcomes (16)
Average Number of Seizure-Free Days in the Last 28 Days
Measured: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Longest Duration of Seizure Free Days in the Last 28 Days
Measured: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Average Maintenance Dose of Epidyolex®
Measured: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Maximum Maintenance Dose of Epidyolex®
Measured: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs)
Measured: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy
Measured: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Change from Baseline to Week 28 (V3) in the Child Behavior Check List (CBCL)
The CBCL is a standardized form that parents fill out to describe their children's behavioral and emotional problems(e.g., anxiety, depression, social problems). The CBCL is also scored on (optional) competence scales for activities, social relations, school and total competence. The CBCL consists of 113 questions, scored on a three-point Likert scale (0=absent, 1= occurs sometimes, 2=occurs often). A higher score indicates a worse outcome.
Measured: Baseline to week 28 (V3) post-dose.
Change from Baseline to Week 52 (V5) in the Child Behavior Check List (CBCL)
The CBCL is a standardized form that parents fill out to describe their children's behavioral and emotional problems(e.g., anxiety, depression, social problems). The CBCL is also scored on (optional) competence scales for activities, social relations, school and total competence. The CBCL consists of 113 questions, scored on a three-point Likert scale (0=absent, 1= occurs sometimes, 2=occurs often). A higher score indicates a worse outcome.
Measured: Baseline to week 52 (V5) post-dose.
Change from Baseline to Week 28 (V3) in the Visual Analog Scale (VAS) of 10 Non-seizure Related Outcomes (NSRO)
VAS assessments are based on 10 NSRO (i.e., Alertness, Sleep Disorder, Motor Skills, Attention, Cognition, Communication, Behavior, Emotional/Social Functioning, Activities of Daily Living, and Participation). VAS is measured on a scale of 0-100; a score of 0 indicates a better outcome, and a score of 100 indicates a worse outcome.
Measured: Baseline to week 28 (V3) post-dose.
Change from Baseline to Week 52 (V5) in the VAS of 10 NSRO
VAS assessments are based on 10 NSRO (i.e., Alertness, Sleep Disorder, Motor Skills, Attention, Cognition, Communication, Behavior, Emotional/Social Functioning, Activities of Daily Living, and Participation). VAS is measured on a scale of 0-100; a score of 0 indicates a better outcome, and a score of 100 indicates a worse outcome.
Measured: Baseline to week 52 (V5) post-dose.
Average Caregiver Global Impression of Change (CGIC) Score at Week 28 (V3)
The CGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. 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) using the scale below." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Measured: Week 28 (V3) post-dose.
Average CGIC Score at Week 52 (V5)
The CGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. 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) using the scale below." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Measured: Week 52 (V5) post-dose.
Average Caregiver Global Impression of Change in Seizure Duration (CGICSD) Score at Week 28 (V3)
The CGICSD comprises the following question to be rated on a three-point scale for each seizure subtype: Since the patient started treatment, please assess the average duration of the patient's seizures (comparing their condition now to their condition before treatment) using the scale below. The scale markers are: 1=Average duration of seizures has decreased; 2=Average duration of seizures has stayed the same; 3=Average duration of seizures has increased. Higher scores indicate a worse outcome.
Measured: Week 28 (V3) post-dose.
Average CGICSD Score at Week 52 (V5)
The CGICSD comprises the following question to be rated on a three-point scale for each seizure subtype: Since the patient started treatment, please assess the average duration of the patient's seizures (comparing their condition now to their condition before treatment) using the scale below. The scale markers are: 1=Average duration of seizures has decreased; 2=Average duration of seizures has stayed the same; 3=Average duration of seizures has increased. Higher scores indicate a worse outcome.
Measured: Week 52 (V5) post-dose.
Average Physician Global Impression of Change (PGIC) Score at Week 28 (V3)
The PGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. The PGIC comprises of the following question: "Please assess the change in the patient's general functional abilities since enrolment." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Measured: Week 28 (V3) post-dose.
Average PGIC Score at Week 52 (V5)
The PGIC evaluates efficacy and quality of life. At enrollment the investigator will be asked to write a brief description of the patient's overall condition as a memory aid for assessment at each subsequent appointment. The PGIC comprises of the following question: "Please assess the change in the patient's general functional abilities since enrolment." The questionnaire is rated on a seven-point scale: "Very Much Improved" (1); "Much Improved" (2);Slightly Improved" (3); "No Change" (4); "Slightly Worse" (5); "Much Worse" (6); "Very Much Worse" (7). A score of 1 indicates a better outcome and a score of 7 indicates a worse outcome.
Measured: Week 52 (V5) post-dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2022
- Study start24 Feb 2025
- Primary completionFeb 2027 (estimated)
- Study completionFeb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lennox Gastaut SyndromeStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- 111 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Advice Pharma Group srl
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05485831
- Sponsor's protocol ID
- JZP926-525
- Acronym
- EpiTALY
- Status last verified
- Jun 2026
- First submitted
- 29 Jul 2022
- Last updated
- 3 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Observational, Prospective, Multicenter Study of Epidyolex® (Cannabidiol CBD 100 mg/ml) Oral Solution, as Adjunctive Treatment for Seizures Associated With Lennox- Gastaut Syndrome (LGS), Dravet Syndrome (DS) and Tuberous Sclerosis Complex (TSC)
- ClinicalTrials.gov ID
- NCT05485831
- Acronym / sponsor ID
- EpiTALY / JZP926-525
- Keywords
- seizures, Epidyolex, cannabidiol oral solution
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 111 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Advice Pharma Group srl
- Study start
- 24 Feb 2025 (actual)
- Primary completion
- Feb 2027 (estimated)
- Study completion
- Feb 2027 (estimated)
- First posted
- 3 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective, observational study on approximately 70-100 Real World participants affected by LGS, DS, or TSC treated with Epidyolex® as prescribed in the summary of product characteristics. The eligible participants are expected to participate in the study for a duration of 52 weeks of treatment.
Detailed description
This observational study evaluates a Real-World population of children and adult participants affected by LGS, DS, and TSC and the effect of therapy with Epidyolex® administered according to clinical practice on epileptic symptoms. The study will assess specific scales, questionnaires, and the satisfaction of the caregivers and the participants/tutors.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex | – | Drug: Epidiolex 100 mg/mL Oral SolutionParticipants ≥2 years of age diagnosed with LGS, DS, and TSC. |
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study will comprise of 70-100 male and female participants, aged ≥ 2 years, diagnosed with LGS, DS, and TSC. The decision to prescribe Epidyolex® as adjunctive therapy for seizures associated with LGS, DS, and TSC will be entirely independent of including the participant in the study.
Key inclusion criteria (3 listed)
- Adult patients, and minor patients ≥ 2 years old diagnosed with LGS, DS or TSC.
- Clinical decision, taken by the physician, to initiate Epidyolex®
- Adult participants, parents or legal representatives must be willing and able to give informed consent/assent for participation in the study.
Key exclusion criteria (2 listed)
- Participants currently using or have used recreational, medicinal cannabis, or cannabinoid-based products within the three months prior to study entry and are unwilling to abstain from these products for the duration of the study.
- Any reason, according to Investigator's judgment, able to compromise compliance with procedures outlined in the study There will not be any other specific exclusion criteria; however, contraindications, special warnings, and precautions for use as detailed in the Summary of Product Characteristics (SmPC) (particularly related to raising of aspartate aminotransferase [AST], alanine aminotransferase [ALT], and total bilirubin) will have to be considered by the treating physician.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Epidiolex 100 mg/mL Oral SolutionAs prescribed in routine clinical practice in Italy. | Drug | Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex |
Primary outcome
Number of Participants Remaining on Therapy from Enrollment
Time frame: Baseline up to Week 52 post-dose.
Treatment retention will be evaluated through the proportion of participants remaining on therapy from the enrollment visit (baseline, V0) to each study visit (Weeks 4 [V1], 16 [V2], 28 [V3], 40 [V4], 52 [V5]).
16 secondary outcomes
Average Number of Seizure-Free Days in the Last 28 Days
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Longest Duration of Seizure Free Days in the Last 28 Days
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Average Maintenance Dose of Epidyolex®
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Maximum Maintenance Dose of Epidyolex®
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs)
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Change from Baseline to Week 28 (V3) in the Child Behavior Check List (CBCL)
Time frame: Baseline to week 28 (V3) post-dose.
Change from Baseline to Week 52 (V5) in the Child Behavior Check List (CBCL)
Time frame: Baseline to week 52 (V5) post-dose.
Change from Baseline to Week 28 (V3) in the Visual Analog Scale (VAS) of 10 Non-seizure Related Outcomes (NSRO)
Time frame: Baseline to week 28 (V3) post-dose.
Change from Baseline to Week 52 (V5) in the VAS of 10 NSRO
Time frame: Baseline to week 52 (V5) post-dose.
Average Caregiver Global Impression of Change (CGIC) Score at Week 28 (V3)
Time frame: Week 28 (V3) post-dose.
Average CGIC Score at Week 52 (V5)
Time frame: Week 52 (V5) post-dose.
Average Caregiver Global Impression of Change in Seizure Duration (CGICSD) Score at Week 28 (V3)
Time frame: Week 28 (V3) post-dose.
Average CGICSD Score at Week 52 (V5)
Time frame: Week 52 (V5) post-dose.
Average Physician Global Impression of Change (PGIC) Score at Week 28 (V3)
Time frame: Week 28 (V3) post-dose.
Average PGIC Score at Week 52 (V5)
Time frame: Week 52 (V5) post-dose.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2026.
- Individual participant dataSharing planned: No
Related trials for Lennox Gastaut Syndrome
RecruitingPhase 3
Investigate Efficacy and Safety of Carisbamate as Adjunctive Treatment for Seizures Associated With LGS in Children and Adults
Seizures, Lennox Gastaut Syndrome
Not yet recruitingPhase 4
A Study to Investigate the Effect of Gradual Titration to Optimize Cannabidiol Treatment in Adults With LGS
Lennox-Gastaut Syndrome
Enrolling by invitationPhase 3
A Study to Investigate LP352 in Children and Adults With Developmental and Epileptic Encephalopathy (DEE)
Developmental and Epileptic Encephalopathy
RecruitingPhase 3
An Efficacy and Safety Study of Clemizole HCl in Patients With Lennox-Gastaut Syndrome
Lennox Gastaut Syndrome
Enrolling by invitation
Clinical Utility of Reduced EEG Home Monitoring in Fenfluramine Titration for Dravet and LGS
Dravet Syndrome (DS), Lennox Gastaut Syndrome (LGS)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
18 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Lennox Gastaut Syndrome and Dravet Syndrome, run by Jazz Pharmaceuticals at 18 sites in Italy.
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 3 inclusion and 2 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: under way, not looking for participants, as of the sponsor's last update on 3 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Italy. 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?
This study is still under way. Results are not available in the source record yet.
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?
Number of Participants Remaining on Therapy from Enrollment (time frame: Baseline up to Week 52 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 Jun 2026.