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

• Register: JPRN
• Last refreshed on: 17 October 2023
• Main ID: JPRN-UMIN000030522
• Date of registration: 22/12/2017
• Prospective Registration: No
• Primary sponsor: Gifu University
• Public title: A multicenter, single-arm, prospective study assessing efficacy and safety of the Sirolimus in the Treatment of intractable vascular anomalies
• Scientific title: 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
• Date of first enrolment: 2017/11/14
• Target sample size: 50
• Recruitment status: Recruiting
• URL: https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000034316
• Study type: Interventional
• Study design: Single arm Non-randomized
• Phase: Not selected

Countries of recruitment

Japan

Contacts

• Name: Michio Ozeki
• Affiliation: Gifu University Hospital Pediatrics

• Name: Ryuta Asada
• Affiliation: Gifu University Hospital Innovative and Clinical Research Promotion Center

Key inclusion & exclusion criteria

Inclusion 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

Age minimum: Not applicable
Age maximum: Not applicable
Gender: Male and Female

Health Condition(s) or Problem(s) studied

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

Intervention(s)

Body surface area (BSA) >= 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. Maximum dose of sirolimus is 4 mg per day. BSA < 1.0m2: an initial dose of sirolimus (1mg/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. Maximum dose of sirolimus is 4 mg per day.

Primary Outcome(s)

Adverse effects and side effects

Secondary Outcome(s)

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

Source(s) of Monetary Support

AMED

Ethics review

Status: YES
Approval date:
Contact: