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Clinical study of sirolimus about safety for Japanese patients: Shizuoka 2019-1 (Sirolimus Shizuoka 2019-1)

Study about safety of sirolimus: Shizuoka 2019-1 ((Sirolimus Shizuoka 2019-1))

3

After completion of clinical trial (FCDS-01) in Shizuoka MINDs, Age, >= 6 years old; bodyweight over 20Kg. Male 2 patients, Female, 1 patient. Age at registration, from 11 to 21 years old. Three patients had FCD type 2.

Three patients were registered and treated with sirolimus. Two patients disagreed to continue this clinical study, and one patient moved to other clinical study conducted by Showa University (FCDS-02). We planned five patients including transfer from other hospitals, but no transfer was occurred.

No SAEs were observed. We observed mild adverse effect including fever related by chance infection, hairiness related with antiseizure medications, etc.

No SAEs were observed. We did not measure levels of sirolimus, because of no SAEs or aggravation of epileptic seizures.

Sirolimus was not effective in epileptic patients with FCD. No SAEs were observed.

Sept. 26, 2022

No

-

https://jrct.mhlw.go.jp/latest-detail/jRCTs041190059

Takahashi Yukitoshi

National Epilepsy Center, NHO Shizuoka Institute of Epilepsy and Neurological Disorders

886 Urushiyama Aoi-ku Shizuoka, Japan

takahashi-ped@umin.ac.jp

Takahashi Yukitoshi

National Epilepsy Center, NHO Shizuoka Institute of Epilepsy and Neurological Disorders

886 Urushiyama Aoi-ku Shizuoka, Japan

+81-54-245-5446

takahashi-ped@umin.ac.jp

Complete

Aug. 01, 2019

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1. After completion of clinical trial (FCDS-01) in Shizuoka MINDs, Age, 6-65 years old; bodyweight >= 20Kg
2. Improvement of epileptic seizures by FCDS-01
3. No severe adverse effect by sirolimus or antiepileptic drugs in preceding clinical trial (FCDS-01)
4. Informed consent by written agreement after precise explanation

1. Patients without accurate data of compliance, adverse event, seizure frequency, duration of seizure
2. Possible attendance another clinical trial
3. Prescription of PB or VGB
4. Possible ketogenic diet therapy
5. Pregnant patient or no agreement for contraception
6. Basic disease inflammation, degenerative diseases,
-7. Risk of suicide
8. History of alcoholic abuse
9. Among preceding clinical trial (FCDS-01), the following laboratory data were observed
9-1 AST or ALT >= 2.5 folds of standard value in our hospital
9-2 WBC < 3000 /mm3, Ht < 30%, Platelet < 80000/mm3, neutrophils < 1000/mm3
9-3 C cr < 50ml/min, eGFR < 30mL/min/1.73m2
9-4 HBsAg (+), HBs Ab(+) after HBV infection, HBc Ab (+)
9-5 Uncontrolled hyperlipidemia
10. Association of arrythmia or cardiac failure requiring treatment.
11. Association of immunodeficiency
12. Irregular visit to hospital
13. Decision of inappropriate patient for this study by responsible doctor

Both

Epileptic patients with focal cortical dysplasia type 2

sirolimus

Epilepsy, FCD type 2

adverse event

Dose & level of sirolimus around adverse event, continuation of sirolimus

+81-52-951-1111

July. 29, 2019

none