NCT04775173

Efficacy and Safety of Different Concentrations of Sirolimus in the Treatment of Kaposiform Hemangioendothelioma.

Efficacy and Safety of High- vs Low-Dose Sirolimus in Patients With Kaposiform Hemangioendothelioma: A Trial Protocol

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 79 (actual)
• Sponsor: West China Hospital · Academic / Other
• Sex: All
• Age: 1 Day – 14 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to compare the efficacy and safety of different concentration gradients of sirolimus in the treatment of Kaposiform hemangioendothelioma.

Detailed description

Kaposiform hemangioendothelioma (KHE) is a rare aggressive vascular neoplasm that occurs predominantly in infancy or early childhood, with an incidence of approximately 0.71/100,000. Currently, sirolimus is a promising treatment modality for KHE. Most scholars consider sirolimus blood concentration of 5-15 ng/ml to be an effective therapeutic concentration. However, long-term higher dose sirolimus treatment can cause some common complications such as oral mucositis which affects the quality of life of the patient. Finer control of the plasma concentration of sirolimus may contribute to the efficacy of treatment and reduce the incidence of complications. Therefore, we conducted this study to see if low-dose sirolimus is beneficial to the prognosis of patients.

Conditions

• Kaposiform Hemangioendothelioma

Interventions

Type	Name	Description
DRUG	Sirolimus	Use of different doses of the same drug

Timeline

• Start date: 2021-02-17
• Primary completion: 2023-08-10
• Completion: 2023-08-10
• First posted: 2021-03-01
• Last updated: 2024-05-07

Locations

1 site across 1 country: China