An uncontrolled open-label study on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type-II
- Conditions
- focal cortical dysplasia (type-II)
- Registration Number
- JPRN-UMIN000033504
- Lead Sponsor
- Hokkaido University Hospital Nishi-Niigata Chuo National Hospital National Center Hospital, NCNP Shizuoka Institute of Epilepsy and Neurological Disorders Okayama University Hospital
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete: follow-up complete
- Sex
- All
- Target Recruitment
- 16
Not provided
1. Those participated in other trials within 12 weeks before the time of consent. 2. Having used sirolimus or everolimus within 52 weeks before enrollment. 3. Who cannot accurately record the number and time of epileptic seizures by themselves/representatives. 4. Suspected of progressive lesions in CT or MRI. 5. Having underwent cerebral surgery for epilepsy within 28 weeks prior to enrollment. 6. Taking Felbamate or Vigabatrine or having taken it within 28 weeks before enrollment. 7. Under ketogenic diet therapy. 8. Having suicide attempts in the past. 9. With a history or complications of substance abuse including alcohol abuse. 10. Those possibly pregnant and cannot perform contraception during the study period (including partners), pregnant or lactating. 11. Falling under any of the following in clinical examination during baseline observation period: - At least 2.5 times the reference value of AST or ALT - WBC count of less than 3,000/micro L, Ht of less than 30%, platelets of less than 80,000/micro L, or neutrophil counts of less than 1,000/ micro L - Ccr of less than 50 ml/min - HBs antigen-positive, HBs antibody-positive except after vaccination with hepatitis B vaccine, or HBc antibody-positive. Or active hepatitis C excluding inactive cases with normal liver function. - In poor control of dyslipidemia with serum triglycerides of 500 mg/dL or more, or LDL cholesterol of 190 mg/dL or more despite being treated for dyslipidaemia. - With renal dysfunction. (eGFR of less than 30 ml/min/1.73 m2) 12. With complications of arrhythmia requiring treatment. 13. With complications of heart/renal failure that may affect hemodynamics. 14. With immunodeficiency complications. 15. Having underwent surgery (surgery requiring invasion into a body cavity or surgery requiring suture of three or more needles including biopsy) within 12 weeks before enrollment. 16. Judged inappropriate for participating in this study by the principal investigator/sub-investigators.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method A reduction rate of incidence of partial seizures (including secondarily generalized seizures) per 28 days during maintenance therapy from the observation period
- Secondary Outcome Measures
Name Time Method - Change in incidence of general seizures per 28 days during maintenance therapy from the observation period - Change in incidence epileptic spasm per 28 days during maintenance therapy from the observation period - Change in incidence of heavy-weight attacks per 28 days during maintenance period from the observation period - A response rate (proportion of cases in which incidence of seizures during maintenance therapy decreased by 50% or more from the observation period) - Proportion of resolution of partial seizures during maintenance therapy - A decrease rate and a response rate of incidence of partial seizures at 4 weeks, 8 weeks, and 12 weeks in the maintenance therapy from the observation period - Adverse events