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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 130

Inclusion Criteria

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).

Exclusion Criteria

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.

+:If patients don't violate the exclusion criteria but have poor general condition, or it is difficult to determine the indication for treatment, the eligibility of this study should be discussed by the investigators.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Adverse effects and side effects

Secondary Outcome Measures

Name	Time	Method
Target lesion response rate determined by Independent Review Facility after 24, 52 weeks and every 24 weeks of treatments<br>Respiratory function after 24, 52 weeks and every 24 weeks of treatments<br>Evaluation of pleural effusion after 24, 52 weeks and every 24 weeks of treatments<br>Evaluation of ascites after 24, 52 weeks and every 24 weeks of treatments<br>Blood coagulation parameters after 5, 12, 24, 52 weeks and every 24 weeks of treatments<br>Bleeding after 24, 52 weeks and every 24 weeks of treatments<br>Pain after 24, 52 weeks and every 24 weeks of treatments<br>QOL improvement rates after 24 and 52 weeks of treatments<br>ADL improvement rates after 24, 52 weeks and every 24 weeks of treatments<br>Laboratory values<br>Vital signs<br>Pharmacokinetics

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