NCT01811667

Efficacy and Safety of the Mammalian Target of Rapamycin (mTor Rapamycin) Inhibitor in Vascular Malformations

Clinical Study on Efficacy and Safety of the mTor Rapamycin Inhibitor Found in the Complex Vascular Malformations

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain · Academic / Other
• Sex: All
• Age: 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 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 lymphatic-vascular organisations suggesting an appealing therapeutic target to treat patients with complex vascular malformations. The aim of this clinical study is to prospectively evaluate the efficacy and the safety of the Rapamycin, an mTOR inhibitor, to treat children and adults with microcystic lymphatic malformations, general lymphatics abnormalities (GLA) or complex vascular malformations for which conventional therapies as surgery or sclerotherapy are ineffective or associated with high risk of important complications.

Conditions

• Cardiovascular Abnormalities

Interventions

Type	Name	Description
DRUG	Sirolimus

Timeline

• Start date: 2012-05-01
• Primary completion: 2016-01-01
• Completion: 2016-01-01
• First posted: 2013-03-14
• Last updated: 2016-04-14

Locations

1 site across 1 country: Belgium