Clinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type II

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-UMIN000030962
• Date of registration: 24/01/2018
• Prospective Registration: Yes
• Primary sponsor: Hokkaido University Hospital
• Public title: Clinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type II
• Scientific title: Clinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type II - Study of sirolimus administration for FCDII type
• Date of first enrolment: 2018/02/28
• Target sample size: 2
• Recruitment status: Complete: follow-up continuing
• URL: https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000035162
• Study type: Interventional
• Study design: Single arm Non-randomized
• Phase: Not selected

Countries of recruitment

Japan

Contacts

• Name: Hideaki Shiraishi
• Affiliation: Hokkaido University Hospital Department of Pediatrics

• Name: Hideaki Shiraishi
• Affiliation: Hokkaido University Hospital Department of Pediatrics

Key inclusion & exclusion criteria

Inclusion criteria:
Exclusion 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.

Age minimum: 2years-old
Age maximum: 65years-old
Gender: Male and Female

Health Condition(s) or Problem(s) studied

Patients with epilepsy with focal cortical dysplasia type 2

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

Primary Outcome(s)

Safety in clinical research enforcement and presence or absence of occurrence of adverse events

Secondary Outcome(s)

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

Source(s) of Monetary Support

AMED

Ethics review

Status: YES
Approval date: 24/01/2018
Contact:

[e-mail]

Hokkaido University Clinical Research Review

011-706-7934

[e-mail]

Results

• Results available: Yes
• Date Completed: 31/03/2020