Developmental and Epileptic Encephalopathy (DEE) MedDRA version: 20.0 Level: PT Classification code 10077380 Term: Epileptic encephalopathy System Organ Class: 10029205 - Nervous system disorders MedDRA version: 20.0 Level: PT Classification code 10077380 Term: Epileptic encephalopathy System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: Epidiolex
Product Name: Epidiolex
Product Code: [Epidiolex]
Pharmaceutical Form: Oral solution
INN or Proposed INN: Cannabidiolo
CAS Number: 13956-29-1
Current Sponsor code: CBD
Other desc
Sponsors
IRCCS, OSPEDALE PEDIATRICO BAMBINO GESÙ DI ROMA
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: o Male or female; o Aged between 2 and 7 years; o A documented history of DEE [7]; o Currently taking at least 1 other AED between one and four AEDs, with a stable antiseizure treatment for the previous 4 weeks (including ketogenic diet and vagus nerve stimulation); o Inadequately seizure control with 2 or more current or prior AEDs; o Written informed consent provided by the participant and/or parent(s)/caregiver(s). Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: o Clinically significant unstable medical or psychiatric conditions that may place patient’s safety at risk; o Clinically significant liver disease or serum aminotransferase (ALT or AST) >3 times the upper limit of the normal range or total bilirubin >2 times the upper limit of the normal range or international normalized ratio (INR) >1.5; o Known or suspected intolerance or hypersensitivity to cannabinoids or any of the excipients of the medicinal product such as sesame oil; o Current use of ACTH or other systemic steroids; o Current use (or use in the previous 2 months) of medicinal cannabis, or synthetic cannabinoid-based medications; o Inadequate supervision by parent(s)/caregiver(s) in the judgment of investigators o Stable felbamate dosing = 1 year; o Subjects who have been part of a clinical trial involving another investigational product in the previous six months; o Subjects with severe renal impairment: estimated glomerular filtration rate (eGFR) calculated as crCL < 30 ml/min, according to Schwartz equation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of the study is to demonstrate that CBD therapy reduces the number and/or severity of seizures and reduces or solves EEG abnormalities in child with DEE when taken in addition to current anti-epileptic drugs (AEDs);Secondary Objective: The secondary objective of the study is to assess safety and tolerability of CBD and to assess whether there is a psychomotor or cognitive improvement (depending on age) with CBD therapy;Primary end point(s): • Assess the percentage change per 28 days from the 4-week baseline period in motor seizure frequency during the 24-week treatment period; • Assess EEG improvement from baseline during treatment period in a blind senior epileptologists evaluation procedure; a score will be established for each patient, based on review and comparison of all baseline-EEG/8-weeks control-EEG and baseline-EEG/14-weeks control-EEG, with values ranging from 0 (= worsened EEG), to a maximum of 2 (= improved); 1 will be assigned if the EEG trace is unmodified.;Timepoint(s) of evaluation of this end point: • 28 days within period from week 4 to week 24; • from baseline to 8 and 14 weeks; | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Assess the safety and tolerability of CBD as an adjunctive therapy based on safety variables (adverse events, laboratory tests, ECG, vital signs, body weight, physical examination, neurological examination); • Number of subjects considered treatment responders, defined as those with a =25%, =50% or =75% reduction in convulsive seizures from baseline; • Number of subjects who are convulsive seizure free • Change in number of seizures by subtype • Change in severity of seizures • Change in number of seizure-free days • Change from baseline in cognitive score • Change from baseline in adaptive score • Change from baseline in Behaviour score • Change from baseline in quality of life score • Change from baseline in parent evaluation • Change from baseline in Investigator evaluation;Timepoint(s) of evaluation of this end point: 20 weeks | — |
Countries
Italy
Contacts
Public ContactCRO
Contract Research Organization Ospedale Pediatrico Bambino Gesù
Outcome results
None listed