A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies - Sirolimus for intractable vascular anomalies

Ozeki Michio0 sites130 target enrollmentMarch 15, 2019

Overview

No summary available.

Registry

who.int

Start Date

March 15, 2019

End Date

TBD

Last Updated

2 years ago

Study Type

Interventional

Sex

All

Sponsor

Ozeki Michio

•1\) Patients with intractable vascular anomaly diagnosed by the investigator/subinvestigator
•2\) Patients must have vascular anomalies that have severe complication or intractable symptoms.\#
•3\) Normal liver, renal, and cardiac function at entry
•Total bilirubin \< 3 x ULN for age
•CRE \< 3 x ULN for age
•4\) 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).
•\#: Severe complications or intractable synptoms on this study are conditions that consist systemically or locally lesions of vascular anomalies and require treatments. If they don't perform treatment, it will cause obvious problems or they will have poor outcomes treated with conventional therapy (surgery, sclerotherapy).

•1\) Patients who currently have an uncontrolled infection
•2\) Uncontrolled diabetes, uncontrolled hypertension, uncontrolled hyperlipidemia, chronic liver disease, or chronic renal disease and bleeding that needs frequent transfusion
•3\) History of allergy to sirolimus, or additive substance
•4\) Patients with primary or secondary immunodeficiency, or patients who may have a risk of severe infection after administration of sirolimus\*
•5\) Patients who have undergone surgical resection or interventional radiology procedures for target lesions within 2 weeks
•6\) Pregnant, probably pregnant, or breast\-feeding woman.
•Patients who do not agree birth control during clinical trial.
•7\) Patient who is judged inappropriate to participate in this study by the investigators
•8\) Less than PS score 20
•\*: The immunodeficiency refer to condition in which there is a congenital or secondary immunodeficiency state, which may be worsened by sirolimus administration or clearly increase the risk of infection. Specifically, if the white blood cell count or immunoglobulin count is low due to malnutrition, immunosuppressive therapy, protein leakage at administration of sirolimus. Or the patients have a histry of sepsis or infection.