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

Mochizuki Shinji0 sites11 target enrollmentJune 19, 2020

Overview

No summary available.

Registry

who.int

Start Date

June 19, 2020

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Sex

All

Sponsor

Mochizuki Shinji

•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.