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.
Study Type
OBSERVATIONAL
Enrollment
100
As prescribed in routine clinical practice in Italy.
Policlinico Sant'Orsola di Bologna
Bologna, Italy
RECRUITINGNumber 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 \[V5\]).
Time frame: Baseline up to Week 52 post-dose.
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)
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.
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NPI AOU Mater Domini Università Magna Graecia
Catanzaro, Italy
RECRUITINGUniversità degli studi "G. D'annunzio" Chieti
Chieti, Italy
RECRUITINGAzienda Ospedaliera Universitaria Meyer Neurologia Pediatrica
Florence, Italy
RECRUITINGIstituto Gaslini Neurologia Pediatrica e Malattie Muscolari
Genova, Italy
RECRUITINGAOU Gaetano Martino
Messina, Italy
RECRUITINGCentro Regionale Epilessia - ASST Santi Paolo e Carlo
Milan, Italy
RECRUITINGASST Grande Ospedale metropolitano Niguarda
Milan, Italy
RECRUITINGAzienda Ospedaliera Universitaria Federico II
Napoli, Italy
RECRUITINGAOU Maggiore della Carità di Novara
Novara, Italy
RECRUITING...and 8 more locations
Time frame: 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.
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)
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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: 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.
Time frame: Week 52 (V5) post-dose.