UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000030797 |
|---|---|
| Receipt number | R000034192 |
| Scientific Title | Uncontrolled open label study of sirolimus about efficacy for epileptic seizures and safety in patients with focal cortical dysplasia type 2 |
| Date of disclosure of the study information | 2018/01/13 |
| Last modified on | 2023/01/16 14:43:08 |
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Basic information
Public title
Uncontrolled open label study of sirolimus about efficacy for epileptic seizures and safety in patients with focal cortical dysplasia type 2
Acronym
Clinical research-sirolimus FCD Shizuoka2017-1
Scientific Title
Uncontrolled open label study of sirolimus about efficacy for epileptic seizures and safety in patients with focal cortical dysplasia type 2
Scientific Title:Acronym
Clinical research-sirolimus FCD Shizuoka2017-1
Region
| Japan |
Condition
Condition
focal cortical dysplasia type 2
Classification by specialty
| Neurology | Pediatrics | Neurosurgery |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To reveal the efficacy of sirolimus for epileptic seizures, safety and pharmacokinetics in patients with focal cortical dysplasia type 2
Basic objectives2
Pharmacokinetics
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Developmental phase
Phase II
Assessment
Primary outcomes
Reduction rate of partial seizures (including secondary GTC) among 28 days from observation period to maintenance dose period
Key secondary outcomes
Reduction rate of other seizures than partial seizures among 28 days from observation period to maintenance dose period
Base
Study type
Interventional
Study design
Basic design
Single arm
Randomization
Non-randomized
Randomization unit
Blinding
Open -no one is blinded
Control
Uncontrolled
Stratification
NO
Dynamic allocation
NO
Institution consideration
Institution is not considered as adjustment factor.
Blocking
NO
Concealment
No need to know
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Period: 8 weeks to 2 years
Initial dose:
1mg/day for boy weight from 20 to 40Kg
2mg/day for body weight above 40Kg
Increasing dose: 1mg/day until the level of 5-15ng/ml (sirolimus)
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
| 6 | years-old | <= |
Age-upper limit
| 65 | years-old | >= |
Gender
Male and Female
Key inclusion criteria
1. Diagnossi of FCD type 2 by MRI and /or histological exaamination.
2. Age (6 years to 65 years old), body weitht more than 20 Kg.
3. partial seizures difined by classification of ILAE.
4. Seizure continued, in spite of treatment for one year, by more than 2 AEDS.
5. Current AEDs:1-4 AEDs
6. No change of prescription of AEDs from obserbational stage.
7. Written informed consent from gurdians or patients.
8. Seizure frequency: more than two times among obserbational stage.
Key exclusion criteria
1. Other clinical trials in less than one month before obsebational stage.
2. Experience of eberolimus or sirolimus treatment.
3. Imposible counting of seizure frequency and duration.
4. Possible inflammation and /or degeneration suggested by CT , MRI, etc.
5. No epilepsy surgery among 6 months before obserbational stage. Partients with VNS before 6 months of obserbational stge, stimulation frequency is consistent.
6. Treatment with PB or VGB in 6 months before obserbational stage.
7. Current ketogenic diet.
8. History of suicide.
9. History or comorbidity of addiction including alcohol.
10. Pregnant mowen and breast-feedin women.
11. active hepatitis.
12. arhythmia with treatment.
13. heart failure with treatment.
14. immunodeficiency
15. surgical intervention in 8 weeks before obserbational stage.
16. Judgement of contraindication by reserchers.
Target sample size
1
Research contact person
Name of lead principal investigator
| 1st name | Yukitoshi |
| Middle name | |
| Last name | Takahashi |
Organization
National Epilepsy Center,
Shizuoka Institute of Epilepsy and Neurological Disorders, NHO
Division name
Pediatrics
Zip code
420 8688
Address
Shizuoka
TEL
054-245-5446
takahashi-ped@umin.ac.jp
Public contact
Name of contact person
| 1st name | Yukitoshi |
| Middle name | |
| Last name | Takahashi |
Organization
National Epilepsy Center,
Division name
Pediatrics
Zip code
420 8688
Address
886 Urushiyama Aoi-ku Shizuoka 420-8688, Japan
TEL
054-245-5446
Homepage URL
takahashi-ped@umin.ac.jp
Sponsor or person
Institute
National Epilepsy Center,
Shizuoka Institute of Epilepsy and Neurological Disorders, NHO
Institute
Department
Personal name
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
National Epilepsy Center,
Address
886 Urushiyama Aoi-ku Shizuoka 420-8688, Japan
Tel
054-245-5446
takahashi-ped@umin.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
国立病院機構静岡てんかん・神経医療センター
Other administrative information
Date of disclosure of the study information
| 2018 | Year | 01 | Month | 13 | Day |
Related information
URL releasing protocol
in progress
Publication of results
Unpublished
Result
URL related to results and publications
in progress
Number of participants that the trial has enrolled
1
Results
Effectiveness for seizure outcome was not confirmed.
Results date posted
| 2023 | Year | 01 | Month | 16 | Day |
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
adolescent female
Participant flow
one patient
Adverse events
nothing
Outcome measures
seizure outcome
Plan to share IPD
IPD sharing Plan description
Progress
Recruitment status
Completed
Date of protocol fixation
| 2017 | Year | 10 | Month | 31 | Day |
Date of IRB
| 2017 | Year | 10 | Month | 31 | Day |
Anticipated trial start date
| 2017 | Year | 12 | Month | 01 | Day |
Last follow-up date
| 2020 | Year | 09 | Month | 30 | Day |
Date of closure to data entry
| 2020 | Year | 10 | Month | 31 | Day |
Date trial data considered complete
| 2020 | Year | 10 | Month | 31 | Day |
Date analysis concluded
| 2020 | Year | 10 | Month | 31 | Day |
Other
Other related information
Management information
Registered date
| 2018 | Year | 01 | Month | 13 | Day |
Last modified on
| 2023 | Year | 01 | Month | 16 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034192
| Research Plan | |
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| Registered date | File name |
| Research case data specifications | |
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| Registered date | File name |
| Research case data | |
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| Registered date | File name |
