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Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000033504
Receipt No. 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 2018/10/15

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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
Middle name
Last name Mitsuhiro Kato
Organization Showa University Hospital
Division name Pediatrics
Zip code
Address 1-5-8 Hatanodai, Shinagawa-ku, Tokyo 142-8555, Japan
TEL 03-3784-8000
Email ktmt-hro@umin.ac.jp

Public contact
Name of contact person
1st name
Middle name
Last name Kenji Nagaya
Organization National Hospital Organization Nagoya Medical Center
Division name Department of Clinical Research Planning and Management, Clinical Research Center
Zip code
Address 4-1-1 Sannomaru, Naka-ku, Nagoya, Aichi 460-0001, Japan
TEL 052-951-1111
Homepage URL
Email study.office@nnh.go.jp

Sponsor
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

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
Address
Tel
Email

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
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 Open public recruiting
Date of protocol fixation
2018 Year 07 Month 13 Day
Date of IRB
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
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2018 Year 07 Month 25 Day
Last modified on
2018 Year 10 Month 15 Day


Link to view the page
URL(English) https://upload.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


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