Epidiolex 100 mg/mL Oral Solution
As prescribed in routine clinical practice in Italy.
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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.
Locally preserved from ClinicalTrials.govOpen ClinicalTrials.govlast source update 2026-06-03
What this record can show
This locally preserved record separates the registered protocol from source-reported registry results. Neither is a treatment recommendation.
Registry facts
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.
Linked local records
Local links use governed condition terms or exact source-reported intervention names. They are navigation candidates, not efficacy claims.
Interventions
As prescribed in routine clinical practice in Italy.
Eligibility
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.
primary outcomes
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 \[V5\]).
secondary outcomes
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Time frame: Weeks 4 (V1), 16 (V2), 28 (V3), 40 (V4) and 52 (V5)
Time frame: Baseline to week 28 (V3) post-dose.
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.
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.
Publications
No exact PMID-linked public article is currently readable locally.
Time frame: Baseline to week 28 (V3) post-dose.
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.
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: 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.
Time frame: Week 52 (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.
Time frame: Week 28 (V3) 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.
Time frame: Week 52 (V5) 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.
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.
Time frame: Week 52 (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.