Dravet Syndrome Level: PT Classification code 10054859
Conditions
Interventions
Trade Name: stiripentolo
Pharmaceutical Form: Capsule, hard
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Trade Name: TOPAMAX
Pharmaceutical Form: Capsule,
Sponsors
ISTITUTO C. MONDINO
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). - At least 2 clonic or tonic-clonic seizures and/or status epilepticus during the baseline period (a status epilepticus is defined as a clonic or tonic-clonic seizure lasting more than 15 minutes) 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: - Patients currently treated or treated in the past with STP or TPM. - Patients treated with any other AED that VPA and CLB in the months preceding the inclusion visit, with the exception of diazepam (VALIUM) 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 complete crisis diary. - Ongoing pregnancy in a female patient.
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 Dravet?s syndrome not adequately controlled with clobazam and valproate, compared to CLB used at the maximal tolerated dosage;Secondary Objective: - Evaluate the efficacy and safety of STP and TPM, used in addition to VPA and CLB used at usual dosages, on status epilepticus in Dravet's syndrome, compared to CLB at the maximal tolerated dosage (MTD) - Compare the efficacy and safety of STP to that of TPM, used in addition to VPA and CLB at usual dosages, on the clonic or tonic-clonic seizures and status epilepticus in Dravet's syndrome. - Evaluate the efficacy of STP, TPM and CLB at the MTD in patients with mutations in the alpha subunit of the gene coding for the SCN1A sodium channel, compared to patients without mutations. - Evaluate the influence of genetic polymorphisms of CYP 2C19*2 and *3 on the efficacy and safety of STP in addition to VPA and CLB in Dravet syndrome and of genetic polymorphisms of Pro385Ser of the alpha subunit of the GABA A receptor and of P-glycoprotein (MDR1, C3435T, G2677T and G1199A genes) on the susceptibility to CLB or STP associated to STP or CLB and VPA in Dravet syndrome;Primary end point(s): - Percentage of change in the number of clonic or tonic-clonic seizures during the last month of the comparison period compared to the baseline | — |
Countries
Italy
Outcome results
None listed