Intractable vascular anomalies: Cystic lymphatic malformation, Lymphangiomatosis (Generalized lymphatic anomaly, Kaposiform lymphangiomatosis), Gorham-Stout disease, Kaposiform hemangioendothelioma and Tuffted angioma with Kasabach-Merritt phenomenon, Nenous malformation, Arteriovenous malformation, Klippel-Trenaunay-Weber syndrome,Bluerubber bleb nevus syndrome, Complex-combined vascular malformations
Conditions
Interventions
= 1.0m2: an initial dose of sirolimus (2mg/day) is single orally administered under fed or fasting condition. Subsequently, the sirolimus dosage is adjusted to achieve trough levels between 5-15 ng/mL
Sponsors
Gifu University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Patients with intractable vascular anomaly diagnosed by the investigator/subinvestigator 2) Patients must have vascular anomalies that have potential to cause significant morbidity. 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).
Exclusion criteria
Exclusion criteria: 1)Patients who currently have an uncontrolled infection 2) Uncontrolled diabetes, uncontrolled hypertension, uncontrolled hyperlipidemia, chronic liver disease, or chronic renal disease 3) History of allergy to sirolimus, or additive substance 4) Known history of HIV seropositivity or known immunodeficiency 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse effects and side effects | — |
Secondary
| Measure | Time frame |
|---|---|
| Target lesion response rate determined by Independent Review Facility after 24 and 52 weeks of treatments Respiratory function after 24 and 52 weeks of treatments Evaluation of pleural effusion after 24 and 52 weeks of treatments Evaluation of ascites after 24 and 52 weeks of treatments Blood coagulation parameters after 5, 12, 24 and 52 weeks of treatments Bleeding after 24 and 52 weeks of treatments Pain after 24 and 52 weeks of treatments QOL improvement rates after 24 and 52 weeks of treatments ADL improvement rates after 24 and 52 weeks of treatments Laboratory values Vital signs Pharmacokinetics | — |
Countries
Japan
Contacts
Public ContactRyuta Asada
Gifu University Hospital Innovative and Clinical Research Promotion Center
Outcome results
None listed