NCT03583307

Efficacy and Safety of Sirolimus to Vascular Anomalies

Efficacy and Safety of Sirolimus in the Treatment of the Complicated Vascular Anomalies

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 126 (actual)
• Sponsor: West China Hospital · Academic / Other
• Sex: All
• Age: 0 Years – 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed 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.

Conditions

• Vascular Anomaly

Interventions

Type	Name	Description
DRUG	Sirolimus	Sirolimus was initiated at a dosage of 0.8 mg/m2 administered twice daily. Subsequently, the sirolimus dosage was adjusted monthly to achieve trough levels between 10 and 15 ng/mL.

Timeline

• Start date: 2018-06-01
• Primary completion: 2021-02-19
• Completion: 2021-02-19
• First posted: 2018-07-11
• Last updated: 2022-03-15

Locations

1 site across 1 country: China