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

Fudan University

Status and phase: Enrolling; Phase 4

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

Treatments: Drug: Percutaneous sirolimus; Drug: Oral sirolimus

Study type: Interventional

Funder types: Other

Identifiers

NCT04921722

LK210106

Details and patient eligibility

About

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

Full 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.

Enrollment: 75 estimated patients

Sex: All

Ages: Under 18 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Single Blind

---

75 participants in 2 patient groups

Topical use of sirolimus

Experimental group

Description:

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.

Treatment:

Drug: Percutaneous sirolimus

Oral use of sirolimus

Active Comparator group

Description:

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.

Treatment:

Drug: Oral sirolimus