臨床研究等提出・公開システム

A Singlecenter, single-arm, prospective study assessing efficacy and safety of Sirolimus granules twice daily administration in patients with intractuable vascular malformations ( Sirolimus granules twice daily administration for intractuable vascular malformations)

Efficacy and safety of Sirolimus granules twice daily administration in patients with intractuable vascular malformations (SITI study)

Mochizuki Shinji

National Center for Global Health and Medicine

1-21-1 Toyama, shinjuku,Tokyo

mochizuki.s@jihs.go.jp

Terada Junko

National Center for Global Health and Medicine

1-21-1 Toyama, shinjuku,Tokyo

+81-3-3202-7181

terada.j@jihs.go.jp

Not Recruiting

June. 19, 2020

Interventional

single arm study

open(masking not used)

uncontrolled control

single assignment

treatment purpose

1) one month and older
2) Patients with intractable vascular anomaly diagnosed by the investigator/subinvestigator
3) Have one or more measurable target lesions on MRI before treatment starts.
4) Severe disability or refractory symptoms depending on target disease
5) Have sufficient liver, kidney and heart functions at the time of registration
6) Written consent to participate in this clinical trial has been given by the subject in person or by a legal guardian (when the subject is younger than 20 years at consent).

1) Patients who have received a targeted drug related to the mTOR pathway within 8 weeks before the start of study drug administration.
2) Patients with infections that require systemic treatment.
3) Patients with a Karnofsky PS score of 30 or less (10 years or older) or Lansky play-PS of 30 or less (under 10 years) due to permanent sequelae due to cerebral disorders.
4) Patients with any of the following complications:
Uncontrolled diabetes, Uncontrolled hypertension, Uncontrolled hyperlipidemia, Severe liver disease, Severe renal disease
5) Patients receiving long-term immunosuppressive drugs (cyclosporine, tacrolimus, etc.) or steroids (4 weeks or more) at the time of registration.
6) Patients who require administration of a drug that affects CYP3A4 activity one week before starting sirolimus administration.
7) Patients with immunodeficiency such as HIV and primary immunodeficiency.
8) Patients who are carriers of hepatitis B virus and / or carriers of hepatitis C virus.
9) Patients who have undergone surgery (resection, sclerotherapy, endovascular treatment) for the target lesion within 2 weeks before obtaining consent.
10) Patients who have received a therapeutic drug (propranolol, Eppikajutsuto, Tokikenchuto, interferon, octreotide, bisphosphonate, denosumab, etc.) for the target disease within 2 weeks before obtaining consent.
11) Patients who received myelosuppressive chemotherapy, biologics, drugs not covered by insurance, etc. within 4 weeks before obtaining consent.
12) Patients who received radiotherapy for the target lesion within 24 weeks before obtaining consent.
13) Patients who meet any of the following.
may be pregnant or pregnant, lactating, Disagree with contraception during this study
14) If administering tablets, persons diagnosed with lymphangioma, lymphangiomatosis, or Gorham's disease.
15) In addition, patients whose investigator / assigning doctor determines that participation in this study is inappropriate.

Both

Intractable vascular malformations

Sirolimus granules are orally administered twice a day, with the starting dose determined according to body weight. Increase or decrease the amount of test drug by measuring blood levels.
After 24 weeks, subjects weighing 30 kg or more may switch to tablets.

Vascular disorders

D054079

Target lesion response rate determined by Independent Review Facility after 24 weeks of treatments

Response rates of target lesions 12, 52 weeks after administration.
Improvement of lesions other than target lesions (skin lesions) at 12, 24, and 52 weeks after administration
Respiratory function 24 and 52 weeks after administration
Pleural effusion 12, 24 and 52 weeks after administration.
Ascites 12, 24, and 52 weeks after administration
Anemia and blood coagulation parameters at 12, 24, and 52 weeks after
administration
Bleeding at 12, 24, and 52 weeks after administration
Pain at 12, 24 and 52 weeks after administration
QOL improvement after starting treatment
ADL improvement after starting treatment
Adverse events and side effects
Clinical test values vital signs
Pharmacokinetics

+81-3-3202-7181

May. 20, 2020

none