UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000033504 |
|---|---|
| Receipt number | R000038203 |
| Scientific Title | An uncontrolled open-label study on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type-II |
| Date of disclosure of the study information | 2018/07/25 |
| Last modified on | 2021/08/11 14:37:47 |
* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments
Basic information
Public title
An uncontrolled open-label study on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type-II
Acronym
An investigator-initiated clinical trial on the efficacy and safety of sirolimus for epileptic seizures associated with FCD type-II
Scientific Title
An uncontrolled open-label study on the efficacy and safety of sirolimus for epileptic seizures associated with focal cortical dysplasia type-II
Scientific Title:Acronym
An investigator-initiated clinical trial on the efficacy and safety of sirolimus for epileptic seizures associated with FCD type-II
Region
| Japan |
Condition
Condition
focal cortical dysplasia (type-II)
Classification by specialty
| Neurology | Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
To assess the efficacy, safety, and pharmacokinetics of sirolimus against epileptic seizures in focal cortical dysplasia type-II
Basic objectives2
Efficacy
Basic objectives -Others
Trial characteristics_1
Exploratory
Trial characteristics_2
Developmental phase
Phase II
Assessment
Primary outcomes
A reduction rate of incidence of partial seizures (including secondarily generalized seizures) per 28 days during maintenance therapy from the observation period
Key secondary outcomes
- 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
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
The investigational drug (sirolimus) is orally administered once a day with the following doses:
<Dose adjustment period>
Initial amount: 1 mg/day for body weight of less than 40 kg, and 2 mg/day for 40 kg or more.
Dose adjustment: to increase as necessary to adjust the trough concentration of sirolimus to the range of 5-15 ng/ml, and then, to shift to the maintenance therapy period.
<Maintenance therapy period>
To adopt dosage regimen and dose of sirolimus at trough concentration of 5-15 ng/ml.
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. Diagnosed with pathologically localized cortical dysplasia type-II by neuroimaging findings in head MRI within 156 weeks prior to enrollment or brain pathology examination before enrollment.
2. Aged 6 to 65 years at the time of informed consent.
3. Diagnosed with a partial seizure (a simple partial seizure with motor signs which can be objectively diagnosed, a complex partial seizure, or a secondarily generalized seizure) based on the 1981 epileptic seizure type international classification of the International League Against Epilepsy (ILAE).
4. Having treated for at least 52 weeks with two or more antiepileptic drugs after diagnosis with epilepsy.
5. Under treatment with 1 to 4 antiepileptic drugs.
6. Whose dose and regimen of antiepileptic drug are constant from the 8th week before enrollment, or whose stimulation condition is constant from the 8th week before enrollment in case of performing vagus nerve stimulation therapy.
7. With two or more partial seizures (including secondarily generalized seizures) during the baseline observation period of 28 days.
8. Provided written consent by themselves/parent or legal representatives.
Key exclusion criteria
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.
Target sample size
15
Research contact person
Name of lead principal investigator
| 1st name | Mitsuhiro |
| Middle name | |
| Last name | Kato |
Organization
Showa University Hospital
Division name
Pediatrics
Zip code
142-8555
Address
1-5-8 Hatanodai, Shinagawa-ku, Tokyo 142-8555, Japan
TEL
03-3784-8000
ktmt-hro@umin.ac.jp
Public contact
Name of contact person
| 1st name | Kenji |
| Middle name | |
| Last name | Nagaya |
Organization
National Hospital Organization Nagoya Medical Center
Division name
Department of Clinical Research Planning and Management, Clinical Research Center
Zip code
460-0001
Address
4-1-1 Sannomaru, Naka-ku, Nagoya, Aichi 460-0001, Japan
TEL
052-951-1111
Homepage URL
study.office@nnh.go.jp
Sponsor or person
Institute
Hokkaido University Hospital
Nishi-Niigata Chuo National Hospital
National Center Hospital, NCNP
Shizuoka Institute of Epilepsy and Neurological Disorders
Okayama University Hospital
Institute
Department
Personal name
Funding Source
Organization
AMED
Organization
Division
Category of Funding Organization
Government offices of other countries
Nationality of Funding Organization
Japan
Other related organizations
Co-sponsor
Name of secondary funder(s)
IRB Contact (For public release)
Organization
NHO Shizuoka Institute of Epilepsy and Neurological Disorders
Address
Urushiyama 886, Aoi-ku, Shizuoka 420-8688, Japan
Tel
054-245-5680
yamamoty@shizuokamind.org
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
北海道大学病院(北海道)
国立病院機構西新潟中央病院(新潟県)
国立精神・神経医療研究センター病院(東京都)
国立病院機構静岡てんかん・神経医療センター(静岡県)
岡山大学病院(岡山県)
Other administrative information
Date of disclosure of the study information
| 2018 | Year | 07 | Month | 25 | 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
16
Results
Results date posted
Results Delayed
Results Delay Reason
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
Completed
Date of protocol fixation
| 2018 | Year | 07 | Month | 13 | Day |
Date of IRB
| 2018 | Year | 08 | Month | 24 | Day |
Anticipated trial start date
| 2018 | Year | 10 | Month | 01 | Day |
Last follow-up date
| 2020 | Year | 09 | Month | 30 | Day |
Date of closure to data entry
| 2020 | Year | 10 | Month | 30 | Day |
Date trial data considered complete
| 2020 | Year | 12 | Month | 15 | Day |
Date analysis concluded
| 2021 | Year | 03 | Month | 12 | Day |
Other
Other related information
Management information
Registered date
| 2018 | Year | 07 | Month | 25 | Day |
Last modified on
| 2021 | Year | 08 | Month | 11 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000038203
| Research Plan | |
|---|---|
| Registered date | File name |
| Research case data specifications | |
|---|---|
| Registered date | File name |
| Research case data | |
|---|---|
| Registered date | File name |
