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| Name | Class |
|---|---|
| Advice Pharma Group srl | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Lennox Gastaut, Dravet Syndrome, and Tuberous Sclerosis Complex | Participants ≥2 years of age diagnosed with LGS, DS, and TSC. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Epidiolex 100 mg/mL Oral Solution | Drug | As prescribed in routine clinical practice in Italy. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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]). | Baseline up to Week 52 post-dose. |
| Measure | Description | Time Frame |
|---|---|---|
| Average Number of Seizure-Free Days in the Last 28 Days | Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5) | |
| Longest Duration of Seizure Free Days in the Last 28 Days | Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5) |
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Inclusion Criteria:
Exclusion Criteria:
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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.
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Policlinico Sant'Orsola di Bologna | Bologna | 40138 | Italy | |||
| NPI AOU Mater Domini Università Magna Graecia |
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| Average Maintenance Dose of Epidyolex® | Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5) |
| Maximum Maintenance Dose of Epidyolex® | Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5) |
| Type, Dosage, and Frequency of Concomitant Anti-Seizure Medications (ASMs) | Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5) |
| Number of Participants Reducing Number/Dosage of Concomitant Medication Related to Epilepsy | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | Week 52 (V5) post-dose. |
| Catanzaro |
| 88100 |
| Italy |
| Università degli studi "G. D'annunzio" Chieti | Chieti | 66100 | Italy |
| Azienda Ospedaliera Universitaria Meyer Neurologia Pediatrica | Florence | 50139 | Italy |
| Istituto Gaslini Neurologia Pediatrica e Malattie Muscolari | Genova | 16147 | Italy |
| AOU Gaetano Martino | Messina | 98124 | Italy |
| Centro Regionale Epilessia - ASST Santi Paolo e Carlo | Milan | 20142 | Italy |
| ASST Grande Ospedale metropolitano Niguarda | Milan | 20162 | Italy |
| Azienda Ospedaliera Universitaria Federico II | Naples | 80131 | Italy |
| AOU Maggiore della Carità di Novara | Novara | 28100 | Italy |
| AOU di Padova | Padova | 35128 | Italy |
| Policlinico Tor Vergata | Roma | 00133 | Italy |
| IRCCS Ospedale Pediatrico Bambin Gesù | Roma | 00165 | Italy |
| Fondazione Policlinico Universitario Agostino Gemelli - IRCCS-NPI | Roma | 00168 | Italy |
| Azienda Ospedaliero Universitaria Sant'Andrea | Roma | 00189 | Italy |
| Ospedale Casa del sollievo e della sofferenza | San Giovanni Rotondo | 71013 | Italy |
| AOU Città della Salute PO Molinette | Torino | 10126 | Italy |
| Azienda Ospedaliero Universitaria delle Marche | Torrette | 60126 | Italy |
| ID | Term |
|---|---|
| D065768 | Lennox Gastaut Syndrome |
| D004831 | Epilepsies, Myoclonic |
| D014402 | Tuberous Sclerosis |
| D012640 | Seizures |
| ID | Term |
|---|---|
| D000073376 | Epileptic Syndromes |
| D004827 | Epilepsy |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D004829 | Epilepsy, Generalized |
| D006222 | Hamartoma |
| D009369 | Neoplasms |
| D009378 | Neoplasms, Multiple Primary |
| D009386 | Neoplastic Syndromes, Hereditary |
| D065703 | Malformations of Cortical Development, Group I |
| D054220 | Malformations of Cortical Development |
| D009421 | Nervous System Malformations |
| D020752 | Neurocutaneous Syndromes |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D000013 | Congenital Abnormalities |
| D009461 | Neurologic Manifestations |
| D012816 | Signs and Symptoms |
| D013568 | Pathological Conditions, Signs and Symptoms |
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| ID | Term |
|---|---|
| D002185 | Cannabidiol |
| D012996 | Solutions |
| ID | Term |
|---|---|
| D002186 | Cannabinoids |
| D013729 | Terpenes |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D004364 | Pharmaceutical Preparations |
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