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

Kaposiform Hemangioendothelioma Clinical Trial

Official title:

Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies: a Randomized Controlled Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04921722
• Other study ID #: LK210106
• Status: Recruiting
• Phase: Phase 4
• Start date: October 22, 2021
• Est. completion date: November 1, 2025

Study information

• Verified date: March 2024
• Source: Children's Hospital of Fudan University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 75
• Est. completion date: November 1, 2025
• Est. primary completion date: November 1, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• Congenital Abnormalities
• Hemangioendothelioma
• Kaposiform Hemangioendothelioma
• Lymphangioma
• Lymphatic Abnormalities
• Tufted Angioma
• Vascular Malformations

Intervention

Drug:
• 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.
• 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.

Locations

Country	Name	City	State
China	Children's Hospital of Fudan University	Shanghai

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Fudan University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Effective rate	Achauer BM et al. made the four-level standard as follows. Grade I: tumor size and skin lesion color regression = 25%; grade II: tumor size and skin lesion color regression 25%-50%; grade III: tumor size and skin lesion color regression 50-75%; grade IV: tumor size and skin lesion color regression =75%. After 6 months of treatment, the pzrticipant will be evaluated. Grade I will be viewed as invalid. Grade II and grade III will be viewed as effective, and grade IV will be viewed as very effective. Those in grade ?, ? or ? will be calculated in effectiveness rate.	From admission to follow-up six months
Secondary	Changes of resistance coefficient	Measured by ultrosonic doppler flowmetery at follow-up	From admission to follow-up six months
Secondary	Incidence of adverse events	Adverse events will be reported according to Common Terminology Criteria for Adverse Events, version 4.0 (CTCAE v4.0). Incidence of complications such as oral ulcers, abnormal liver enzymes, infections will be recorded. It is defined as occurring if individual subject has any of the above complications during the 6-month intervention	From admission to follow-up six months
Secondary	Platelet count	Platelet count is one of the major indicators of response to treatment. It is supposed to be greater than 100×10^9/L.	From admission to follow-up six months
Secondary	Changes of peak blood flow	Measured by ultrosonic doppler flowmetery at follow-up	From admission to follow-up six months