Efficacy and Safety of Sirolimus to Vascular Anomalies

Vascular Anomaly Clinical Trial

Official title: Efficacy and Safety of Sirolimus in the Treatment of the Complicated Vascular Anomalies

Status Completed

Clinical Trial Summary

To evaluate the safety and efficacy of Sirolimus in complicated vascular anomalies in Chinese children

Clinical Trial Description

Vascular anomalies are composed of vascular tumors and vascular malformations. The prognosis of vascular anomalies is significantly variable. Most of them had a benign course. However, complicated vascular anomalies can lead to disfigurement, organ disfunction and life-threatening with significant morbidity and mortality. Traditional treatments, including steroids, vincristine, cyclophosphamide and surgery, had limited response to complicated vascular anomalies. In the past few years, the inhibitor of the mammalian target of rapamycin (mTOR) signaling pathway-sirolimus has emerged as a treatment for severe vascular anomalies. Besides, preclinical studies also showed that the Phosphoinositide 3-kinase (PI3K)/protein kinase B (Akt)/mTOR pathway play an important role in the development of vascular tumors and vascular malformations. However, the exact efficacious rate and complications of sirolimus are still unknow in china because of the lack of large scale of prospective studies. Therefore, it's important to perform this prospective study to determine the safety and efficacy of sirolimus in the treatment of Chinese children with complicated vascular anomalies, and this study will also make contributions to the diagnoses and treatments of vascular anomalies. ;

Related Conditions & MeSH terms

• Congenital Abnormalities
• Vascular Anomaly
• Vascular Malformations

• NCT number: NCT03583307
• Study type: Interventional
• Source: West China Hospital
• Status: Completed
• Phase: N/A
• Start date: June 1, 2018
• Completion date: February 19, 2021