Dravet Syndrome MedDRA version: 14.1 Level: PT Classification code 10054859 Term: Myoclonic epilepsy System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Trade Name: stiripentolo
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Other antiepileptics
Concentration unit: mg milligram(s)
Concentration number: 250-
Trade Name: TOPAMAX
Pharmaceutical
Sponsors
Inserm-ISP Pole Recherches cliniques et thérapeutiques
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: - Aged between 6 months and 15 years. - A signed Consent Form collection from parents or legal guardian. - Proven diagnosis of Dravet`s syndrome *. - Treated with VPA and CLB at usual dosages (at the appreciation of the investigator). - Clonic or tonic-clonic seizures not adequately controlled with VPA and CLB at usual dosages (at the appreciation of the investigator). * Criteria for Â?confirmed diagnosis of Dravet syndromeÂ? - First seizure before the age of 1 year - Seizure types: convulsive seizures (clonic or tonic-clonic) - generalized or hemibody seizures - usually prolonged ((> 15 minutes), - febrile and afebrile - Later on (from the age fo1year to 2 years) seizure types possible: myoclonia, absences, partial seizures - Normal psychomotor development before the first seizure - Later on, severe mental retardation - EEG usually normal before the first seizure - Later on, abnormalities usually present *Diagnostic criteria for DravetÂ?s syndrome in patients between 6 months and 1 year - At least two clonic or tonic-clonic seizures, either generalized or unilateral or followed by a unilateral deficit - At least one afebrile seizure - At least one prolonged seizure (> 15 minutes), - In case of unilateral seizures or seizures followed by a unilateral deficit, both sides having been involved at least once - Normal psychomotor development - No pathological perinatal antecedent Are the trial subjects under 18? yes Number of subjects for this age range: 0 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: - Patients currently treated or history of treatment [in the past] with STP or TPM, - Patients treated with any other AED than VPA and CLB within one month before screening visit, with the exception of diazepam [(VALIUM)], midazolam, lorazepam and clonazepam [(RIVOTRIL)] if used [only] occasionally as emergency treatment for epileptic seizures, - Patients treated with medications known as inhibitors of the CYP3A4 (macrolides, azol antifungal agents, theophylline) or with oral anti-coagulants, - Parents or legal guardian unable to follow the study protocol and or complete the subjectâ??s seizures diary, - Contra indications to study treatments : o Hypersensitivity to the active substance or to any of the excipients. o History of psychoses in the form of episodes of delirium o Renal and/or hepatic function disorder o Glucose and galactose malabsorption o Congenital intolerance to fructose o patients with myasthenia gravis o severe respiratory insufficiency o sleep apnoea syndrome - ongoing pregnancy or breastfeeding female, - childbearing potential female not willing to use an effective mean of contraception, - parents or legual guardians unable to give their consent.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: evaluate the efficacy of STP and TPM, used as an add-on therapy in addition to VPA and CLB used at usual dosages, on the clonic or tonic-clonic seizures in paediatric patients with DravetÂ?s syndrome not adequately controlled with clobazam and valproate, compared to CLB used at the maximal tolerated dosage;Secondary Objective: - Evaluate the efficacy of STP and TPM, used as an add-on therapy in addition to VPA and CLB used at usual dosages, on convulsive status epilepticus (a status epilepticus is defined as a clonic or tonic-clonic seizure lasting more than 15 minutes) in Dravet`s syndrome, compared to CLB used at the maximal tolerated dosage. - Compare the efficacy of STP to that of TPM, used as an add-on therapy in addition to VPA and CLB used at usual dosages, on the clonic or tonic-clonic seizures and convulsive status epilepticus in Dravet`s syndrome. - Evaluate the efficacy of STP and TPM, used as an add-on therapy in addition to VPA and CLB used at usual dosages in other seizure types other than clonic or tonic-clonic, compared to CLB used at the maximal tolerated dosage. - Evaluate the safety of STP and TPM (defined as their toxicity), used as an add-on therapy in addition to VPA and CLB used at usual dosages, in Dravet`s syndrome, compared to CLB used at the maximal tolerated dosage.;Primary end point(s): Absolute variation of the monthly number of clonic or tonic-clonic seizures and status epilepticus (defined as clonic or tonic-clonic seizures > 15 minutes), formulated for 30 days in the double blind comparison period (total number of seizures over the three months divided by 3) compared to the screening period ( total number of seizures over the 5 weeks * 4/5). | — |
Countries
Italy
Outcome results
None listed