NCT06673290

Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations

• Status: Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 150 (estimated)
• Sponsor: West China Hospital · Academic / Other
• Sex: All
• Age: 1 Year – 18 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 cystic lymphatic malformation.

Detailed description

Cystic lymphatic malformation is a disease caused by abnormal development of the lymphatic system, characterized by abnormal dilation and/or structural disorder of lymphatic vessels. These abnormalities may lead to accumulation of lymphatic fluid, causing local swelling, pain, and even affecting organ function. Cystic lymphatic malformation can occur anywhere in the body, including skin, soft tissue, internal organs, etc. Sirolimus, also known as rapamycin, is an immunosuppressant mainly used to prevent rejection after organ transplantation. In recent years, studies have shown that sirolimus has certain potential in the treatment of lymphatic malformations. However, long-term high-dose sirolimus treatment can cause some common complications, such as oral mucositis, which affects the quality of life of patients. More fine-grained control of rapamycin plasma concentrations may help improve the therapeutic effect and reduce the incidence of complications. Therefore, the investigators conducted this study to understand whether low-dose rapamycin is beneficial to the prognosis of patients.

Conditions

• Lymphatic Malformation

Interventions

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

Timeline

• Start date: 2024-11-30
• Primary completion: 2026-12-30
• Completion: 2026-12-30
• First posted: 2024-11-04
• Last updated: 2024-12-31

Locations

1 site across 1 country: China