UMIN-CTR Clinical Trial

Basic information
Public title	Clinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type II
Acronym	Study of sirolimus administration for FCDII type
Scientific Title	Clinical study on efficacy and safety of sirolimus against epileptic seizures of focal cortical dysplasia type II
Scientific Title:Acronym	Study of sirolimus administration for FCDII type
Region	Japan

Condition
Condition	Patients with epilepsy with focal cortical dysplasia type 2
Classification by specialty	Neurology Pediatrics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Clinical trial of sirolimus for patients with focal cortical dysplasia type 2. Confirm safety in administration of this drug.
Basic objectives2	Safety
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Safety in clinical research enforcement and presence or absence of occurrence of adverse events
Key secondary outcomes	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

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	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
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	2 years-old <=
Age-upper limit	65 years-old >
Gender	Male and Female
Key inclusion criteria	Persons who are diagnosed pathologically by having localized cortical dysplasia type2 Patient who is treated for at least 1 year with two or more antiepileptic drugs after diagnosis with epilepsy. Patients under treatment with 1 to 4 antiepileptic drugs. 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. Patients with two or more epileptic seizures in 28 days during the previewing period.
Key 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.
Target sample size	2

Research contact person
Name of lead principal investigator	1st name Hideaki Middle name Last name Shiraishi
Organization	Hokkaido University Hospital
Division name	Department of Pediatrics
Zip code	060-8648
Address	North 14, West 5, Kita-ku, Sapporo
TEL	011-706-5954
Email	siraisi@med.hokudai.ac.jp

Public contact
Name of contact person	1st name Hideaki Middle name Last name Shiraishi
Organization	Hokkaido University Hospital
Division name	Department of Pediatrics
Zip code	060-8648
Address	North 14, West 5, Kita-ku, Sapporo
TEL	011-706-5954
Homepage URL
Email	siraisi@med.hokudai.ac.jp

Sponsor
Institute	Hokkaido University Hospital
Institute
Department

Funding Source
Organization	AMED
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization	Japan

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization	Hokkaido University Clinical Research Review
Address	North 14, West 5, Kitaku, Sapporo
Tel	011-706-7934
Email	recjimu@huhp.hokudai.ac.jp

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2018 Year 01 Month 24 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled	2
Results
Results date posted
Results Delayed	Delay expected
Results Delay Reason	Under review
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	No longer recruiting
Date of protocol fixation	2017 Year 12 Month 11 Day
Date of IRB	2018 Year 01 Month 24 Day
Anticipated trial start date	2018 Year 02 Month 28 Day
Last follow-up date	2020 Year 03 Month 31 Day
Date of closure to data entry	2020 Year 03 Month 31 Day
Date trial data considered complete	2020 Year 03 Month 31 Day
Date analysis concluded	2021 Year 03 Month 31 Day

Other
Other related information

Management information
Registered date	2018 Year 01 Month 24 Day
Last modified on	2022 Year 01 Month 27 Day

Link to view the page
URL(English)	https://center6.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000035162