We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Epidyolex® in Lennox Gastaut and Dravet Syndrome: an Observational Study in ITALY (EpiTALY)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05485831
Recruitment Status : Not yet recruiting
First Posted : August 3, 2022
Last Update Posted : December 2, 2022
Sponsor:
Collaborator:
Evidilya S.r.l.
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Brief Summary:
This is a prospective, observational study on approximately 70 Real World participants affected by LGS or DS, 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 56 weeks of treatment.

Condition or disease Intervention/treatment
Lennox Gastaut Syndrome Dravet Syndrome Drug: Epidiolex 100 mg/mL Oral Solution

Detailed Description:
This observational study evaluates a Real-World population of children and adolescent participants affected by LGS and DS and the effect of therapy with Epidyolex® administered according to clinical practice on epileptic symptoms. The study will assess specific scales, questionnaires, the QoL of the participants, and the satisfaction of the caregivers and the participants/tutors.

Layout table for study information
Study Type : Observational
Estimated Enrollment : 70 participants
Observational Model: Cohort
Time Perspective: Prospective
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) and Dravet Syndrome (DS)
Estimated Study Start Date : January 2023
Estimated Primary Completion Date : October 2023
Estimated Study Completion Date : October 2023


Group/Cohort Intervention/treatment
Lennox Gastaut and Dravet Syndrome
Participants aged 6-17 years of age diagnosed with LGS and DS.
Drug: Epidiolex 100 mg/mL Oral Solution
As prescribed in routine clinical practice in Italy.




Primary Outcome Measures :
  1. Change from Baseline to Study Visit in Proportion of Participants Remaining on Therapy from Enrollment [ Time Frame: Baseline up to Week 56 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], 56 [V5]).


Secondary Outcome Measures :
  1. Percentage Change from Baseline in Frequency of LGS/DS Associated Seizures (average per 28 days) [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) post-dose. ]
    LGS associated seizures are defined as drop seizures which include tonic-clonic, tonic or atonic seizures. DS associated seizures are defined as convulsive seizures which include tonic, clonic, atonic, tonic-clonic.

  2. Percentage Change from Baseline in Total Seizure Frequency (average per 28 days) [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
    Total seizures are defined as all countable seizures.

  3. Percentage of Participants Achieving a ≥25% Reduction in Seizure Frequency from Baseline [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  4. Percentage of Participants Achieving a ≥50% Reduction in Seizure Frequency from Baseline [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  5. Percentage of Participants Achieving a ≥75% Reduction in Seizure Frequency from Baseline [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  6. Average Number of Seizure-Free Days in the Last 28 Days [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  7. Longest Duration of Seizure Free Days in the Last 28 Days [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  8. Number of Events of Status Epilepticus [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  9. Average Maintenance Dose of Epidyolex® [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  10. Maximum Maintenance Dose of Epidyolex® [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  11. Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs) [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  12. Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy [ Time Frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 56 (V5) ]
  13. Change from Baseline to Week 28 (V3) in the Child Behavior Check List (CBCL) [ Time Frame: Baseline to week 28 (v3) post-dose. ]
    The CBCL is a standardized form that parents fill out to describe their adolescent and 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.

  14. Change from Baseline to Week 56 (V5) in the Child Behavior Check List (CBCL) [ Time Frame: Baseline to week 56 (V5) post-dose. ]
    The CBCL is a standardized form that parents fill out to describe their adolescent and 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.

  15. Change from Baseline to Week 28 (V3) in the Sleep Disturbance Scale for Children (SDSC) [ Time Frame: Baseline to week 28 (v3) post-dose. ]
    The SDSC assesses sleep behavior and disturbances during the previous 6 mo. The questionnaire consists of two sections: the first one is used to obtain demographic, behavioral and clinical data, information about previous illnesses and present medical status with specific questions regarding pathology that could affect sleep; the second is made up of 27 items in a Likert-type scale with values 1 (never) to 5 (always [daily]) with the wording arranged so that higher scores reflect a greater clinical severity of symptoms and indicate more acute sleep disturbances.

  16. Change from Baseline to Week 56 (V5) in the Sleep Disturbance Scale for Children (SDSC) [ Time Frame: Baseline to week 56 (v5) post-dose. ]
    The SDSC assesses sleep behavior and disturbances during the previous 6 mo. The questionnaire consists of two sections: the first one is used to obtain demographic, behavioral and clinical data, information about previous illnesses and present medical status with specific questions regarding pathology that could affect sleep; the second is made up of 27 items in a Likert-type scale with values 1 (never) to 5 (always [daily]) with the wording arranged so that higher scores reflect a greater clinical severity of symptoms and indicate more acute sleep disturbances.

  17. Change from Baseline to Week 28 (V3) in the Social Communication Questionnaire (SCQ) [ Time Frame: Baseline to week 28 (v3) post-dose. ]
    The SCQ is a parent report screening measure for autism spectrum disorders (ASDs) based on the Autism Diagnostic Interview-Revised (ADI-R). This brief instrument helps evaluate communication skills and social functioning in children who may have autism or autism spectrum disorders. It is available in two forms-Lifetime and Current-each composed of just 40 yes-or-no questions. Scores above the cutoff of 15 suggest the individual is likely to be on the autism spectrum and a more extended evaluation should be undertaken.

  18. Change from Baseline to Week 56 (V5) in the Social Communication Questionnaire (SCQ) [ Time Frame: Baseline to week 56 (v5) post-dose. ]
    The SCQ is a parent report screening measure for autism spectrum disorders (ASDs) based on the Autism Diagnostic Interview-Revised (ADI-R). This brief instrument helps evaluate communication skills and social functioning in children who may have autism or autism spectrum disorders. It is available in two forms-Lifetime and Current-each composed of just 40 yes-or-no questions. Scores above the cutoff of 15 suggest the individual is likely to be on the autism spectrum and a more extended evaluation should be undertaken.

  19. Change from Baseline to Week 28 (V3) in the Pediatric Quality of Life (Peds-QoL) -Epilepsy [ Time Frame: Baseline to week 28 (v3) post-dose. ]
    The Parent Report for Toddlers (ages 2-4) of the PedsQLTM 3.0 Epilepsy Module is composed of 22 items comprising 5 dimensions. The dimensions consist of impact (6 items), cognitive functioning (5 items), sleep/rest (2 items), executive functioning (4 items), and mood/behavior (5 items). The items are rated on a 5-point Likert scale: 0 never a problem, 1 almost never a problem, 2 sometime a problem, 3 often a problem, 4 almost always a problem. Scores are transformed to a 0 to 100 scale. Higher scores indicate lower problems and a better outcome.

  20. Change from Baseline to Week 56 (V5) in the Pediatric Quality of Life (Peds-QoL) -Epilepsy [ Time Frame: Baseline to week 56 (v5) post-dose. ]
    The Parent Report for Toddlers (ages 2-4) of the PedsQLTM 3.0 Epilepsy Module is composed of 22 items comprising 5 dimensions. The dimensions consist of impact (6 items), cognitive functioning (5 items), sleep/rest (2 items), executive functioning (4 items), and mood/behavior (5 items). The items are rated on a 5-point Likert scale: 0 never a problem, 1 almost never a problem, 2 sometime a problem, 3 often a problem, 4 almost always a problem. Scores are transformed to a 0 to 100 scale. Higher scores indicate lower problems and a better outcome.

  21. Caregiver Global Impression of Change (CGIC) at Week 28 (V3) [ Time Frame: Week 28 (V3) post-dose. ]
    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.

  22. Caregiver Global Impression of Change (CGIC) at Week 56 (V5) [ Time Frame: Week 56 (V5) post-dose. ]
    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.

  23. Caregiver Global Impression of Change in Seizure Duration (CGICSD) at Week 28 (V3) [ Time Frame: Week 28 post-dose. ]
    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.

  24. Caregiver Global Impression of Change in Seizure Duration (CGICSD) at Week 56 (V5) [ Time Frame: Week 56 post-dose. ]
    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.

  25. Physician Global Impression of Change (PGIC) at Week 28 (V3) [ Time Frame: Week 28 (V3) post-dose. ]
    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.

  26. Physician Global Impression of Change at Week 56 (V5) [ Time Frame: Week 56 (V5) post-dose. ]
    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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Sampling Method:   Probability Sample
Study Population
The study groups will comprise of 70 male and female participants, aged 6-17 years, satisfying the inclusion and exclusion criteria and agreeing to take part in this observational study. The decision to prescribe Epidyolex® as adjunctive therapy for seizures associated with LGS or DS will be entirely independent of including the participant in the study.
Criteria

Inclusion Criteria:

  • Paediatric participants aged 6-17 years, diagnosed with LGS or DS
  • Clinical decision, taken by the physician, to initiate Epidyolex®
  • Availability of a diary with the number and type of epileptic seizures that occurred at least 28 days before the start of the study
  • Parents or legal representatives must be willing and able to give informed consent/assent for participation in the study.

Exclusion Criteria:

  • 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.
  • This study will not include participants who have already been prescribed Epidyolex® before the start 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05485831


Contacts
Layout table for location contacts
Contact: Clinical Trial Disclosure & Transparency 215-832-3750 ClinicalTrialDisclosure@JazzPharma.com

Locations
Layout table for location information
Italy
Ospedale Pediatrico Bambino Gesù (OPBG)
Roma, Italy, 00152
Sponsors and Collaborators
Jazz Pharmaceuticals
Evidilya S.r.l.
Layout table for additonal information
Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT05485831    
Other Study ID Numbers: GWPIT001
First Posted: August 3, 2022    Key Record Dates
Last Update Posted: December 2, 2022
Last Verified: December 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Keywords provided by Jazz Pharmaceuticals:
seizures
Epidyolex
cannabidiol oral solution
Additional relevant MeSH terms:
Layout table for MeSH terms
Epilepsies, Myoclonic
Lennox Gastaut Syndrome
Syndrome
Disease
Pathologic Processes
Epilepsy, Generalized
Epilepsy
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Epileptic Syndromes
Genetic Diseases, Inborn
Cannabidiol
Anticonvulsants