Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies

NCT ID: NCT04921722

Last Updated: 2024-03-15

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE4
• Total Enrollment: 75 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-22
• Study Completion Date: 2025-11-01

Brief Summary

In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.

Detailed Description

Vascular anomaly is a kind of rare disease. According to histology, biological behavior and clinical manifestations, it can be divided into two categories: vascular tumor and vascular malformation.

mTOR inhibitors are proved with the properties of anti-proliferation and anti-angiogenesis. Therefore, they have been used in the treatment of vascular anomalies. Sirolimus, by its ability to prevent downstream protein synthesis and subsequent cell proliferation and angiogenesis, has become a novel and effective treatment. However, after the children reach complete response, there may still be skin manifestations that affect the appearance and cause psychological shadows. Therefore, intervention is required.

Studies have reported that topical sirolimus is effective in treating Kaposiform Hemangioendothelioma (KHE). It is absorbed through the skin, avoiding the first pass elimination effect of the liver. Fewer adverse reactions have been observed. In this study, we investigate the efficacy and safety of percutaneous administration of sirolimus in the treatment of superficial complicated vascular anomalies.

Conditions

Kaposiform Hemangioendothelioma; Tufted Angioma; Superficial Vascular Anomalies; Superficial Lymphatic Malformations

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: SINGLE

Study Groups

Topical use of sirolimus

Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months.

Group Type: EXPERIMENTAL

Percutaneous sirolimus

Intervention Type: DRUG

We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In experimental group, we administrate percutaneous sirolimus. Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months.

Oral use of sirolimus

Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml.

Group Type: ACTIVE_COMPARATOR

Oral sirolimus

Intervention Type: DRUG

We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In active comparator group, we administrate oral sirolimus. Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml.

Interventions

Percutaneous sirolimus

We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In experimental group, we administrate percutaneous sirolimus. Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months.

Intervention Type: DRUG

Oral sirolimus

We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In active comparator group, we administrate oral sirolimus. Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml.

Intervention Type: DRUG

Other Intervention Names

Rapamycin; Rapamycin

Eligibility Criteria

Inclusion Criteria

• Participant clinically or pathologically diagnosed with KHE, TA or complicated superficial vascular anomolies involving lymphatic components.

1. The case is initial, with a relatively limited superficial lesion.

2. The participant has residual surface lesions after oral medication.

• Participant with no use of other medication or surgical treatment
• Participant with detailed medical records of the disease at the time of screening
• Participant with signed and dated informed consent from the guardian(s)

Exclusion Criteria

• Participants with Kasabach-Merritt Phenomenon, with platelets <50×10 9 /L.
• Participants with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
• Participants with other hematological diseases or solid tumor.
• Participants allergic to sirolimus or dressing.

• Minimum Eligible Age: 0 Years
• Maximum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Children's Hospital of Fudan University

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Children's Hospital of Fudan University

Shanghai, , China

Site Status: RECRUITING

Countries

China

Facility Contacts

Kai Li, MD, PhD

Role: primary

likai2727@163.com

+86 02164931114

Other Identifiers

LK210106

Identifier Type: -

Identifier Source: org_study_id