NCT02638389

Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care

Phase III Multicentric Study Evaluating the Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 250 (estimated)
• Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain · Academic / Other
• Sex: All
• Age: 3 Months – 70 Years
• Healthy volunteers: Not accepted

Summary

The phosphatidylinositol 3-kinase (PI3Kinase)/Protein Kinase B (AKT)/mammalian target of rapamycin (mTor) pathway plays a role on the development and the venous/lymphatic vascular organisations. The investigators want to study the efficacy and the safety of Rapamycin, an mTor inhibitor.

Detailed description

The complex vascular malformations induce chronical pains and organic dysfunctions causing significant morbidity and mortality. Therefore, the investigators need to establish guidelines in order to treat these pathologies. Standard treatments such as surgery or interventional radiology are of limited efficacy and related to a high level of recurrences as well as complications. Recent preclinical studies have shown the important role of the PI3Kinase/AKT/mTor pathway on the development and the venous/lymphatic vascular organisations suggesting an appealing therapeutic target to treat patients with venous, lympathic or complex vascular malformations. Investigators will realize a multicentric phase III study enrolling a higher number of patients to statistically evaluate the efficacy and the safety of the Rapamycin, an mTOR inhibitor, in the treatment of children and adults with vascular malformations for which conventional therapies such as surgery or sclerotherapy are ineffective or associated with high risk of important complications. Nearly 250 patients (200 adults and 50 children) will be enrolled in several european centers.

Conditions

• Vascular Malformations

Interventions

Type	Name	Description
DRUG	Sirolimus	evaluate the efficacy and safety of sirolimus in these patients

Timeline

• Start date: 2016-01-25
• Primary completion: 2025-04-01
• Completion: 2030-04-01
• First posted: 2015-12-23
• Last updated: 2023-02-24

Locations

3 sites across 3 countries: Belgium, France, Germany