Research of sirolimus administration for FCD II type

JRCT ID: jRCTs011180016

Registered date:19/03/2019

Basic Information

• Recruitment status: Complete
• Health condition(s) or Problem(s) studied: Patients with epilepsy with focal cortical s\dysplasia type 2
• Date of first enrollment: 28/02/2018
• Target sample size: 2
• Study type: Interventional
• Intervention(s): Prescribe the study medicine at the start of the gradual increase period (Visit 20 weeks) after the end of the preview phase. Administration of the study drug starts from the morning the following day of Visit 2. Take it once a day in the morning. For test drugs (1 mg tablets), weighing less than 20 kg of sirolimus 0.5 mg / day, body weight of less than 20 kg to less than 40 kg is 1 mg / day, body weight of 40 mg or more is orally administered 2 mg / day once daily in the morning. The upper limit of the daily dose is 4 mg. The dose was not changed in the first 4 weeks, the blood concentration of sirolimus was measured at the 4th week (visit 4), the next visit (4 weeks / visit 4-2 to 4-10) based on the examination result, From 0.5 mg / day for less than 20 kg to 1 mg / day for 20 kg or more, increase the dose and repeat the measurement of sirolimus blood concentration every 4 weeks until the trough concentration reaches 5 to 15 ng / mL, and the trough concentration. From the time when the target concentration is reached, the maintenance therapy period is entered. As a result of the blood concentration at Visit 4, when the trough blood concentration is within the range of 5 - 15 ng / mL, it will shift to the maintenance therapy period from Visit 5 (Week 8). Visit 5 (after week 8) is in the dose control phase and is fixed at the dose of sirolimus that has reached blood concentration of 5 - 15 ng / mL. The maintenance therapy period will be visited 4 weeks, 8 weeks, 12 weeks after the start of maintenance therapy and will continue for 12 weeks. The researcher who completed the procedure up to the end of the 12th week of maintenance therapy will complete the study

Outcome(s)

• Primary Outcome: Safety in clinical research enforcement and presence or absence of occurrence of adverse events
• Secondary Outcome: Percentage reduction in the incidence of epileptic seizures per week during fixed administration period, variable administration period (dose control period) of sirolimus, compared with the previous observation period without administration

Key inclusion & exclusion criteria

• Age minimum: >= 2age old
• Age maximum: < 65age old
• Gender: Both
• Include criteria: 1) Persons who are diagnosed pathologically by having localized cortical dysplasia type2 2) Patient who is treated for at least 1 year with two or more antiepileptic drugs after diagnosis with epilepsy. 3) Patients under treatment with 1 to 4 antiepileptic drugs. 4) Patients whose dose and usage of antiepileptic drug administration are constant from 28 days prior to the start of the preview period prior to obtaining consent. 5) Patients with two or more epileptic seizures in 28 days during the previewing period.
• Exclude criteria: 1) Patients taking oral administration of other test drugs (drugs used in other clinical studies / clinical trials) before the start of the study, just before the observation period. 2) Currently, patients taking sirolimus or everolimus internally or who have ever taken internal medicine. 3) Felbamate, Vigabatrin was taking within 6 months. 4) Patients who can not accurately record the number and time of epileptic seizures by the patient or substitute. 5) Patients who are suspected of progressive lesions with Computed Tomography (CT) or Magnetic Resonance Imaging (MRI) performed so far. 6) Patients who underwent cerebral surgery for epilepsy (cortical resection, functional hemisphere dissection, cerebral ablation, vagus nerve stimulation) within 180 days prior to obtaining consent. 7) Patients during ketogenic diet therapy. 8) Patients who have suicide attempts in the past. 9) Patients with a history or complication of substance abuse (including alcohol abuse). 10) Patients who are pregnant women who can not perform contraception during the study period (including partners), pregnant or lactating.

Related Information

• Primary Sponsor: shiraishi hideaki
• Secondary ID(s): UMIN000030962