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

• Status: Completed
• Trial end date: 2016-01-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
• Treatments: Everolimus; Sirolimus

Criteria

Inclusion Criteria:

• Patients with complex vascular abnormalities to be threat by a systemic therapy

• Patients must have adequate liver function (LDL-cholesterol, triglycerides,…)

• Patients must have adequate organ function: neutrophils >1500/mm³, Hb > 8,0 g et platelets> 50.000/mm³ (no platelets limits for the Kasabach Merritt syndrome)

• Patients must have adequate renal function(normal creatinin depending on the age), clearance > 70 ml/min/1.73m² and Urin Protein Creatinine ratio <0.3 g.

• Karnofsky or Landry > 50

Exclusion Criteria:

• Dental equipments or prosthesis interfering onto a radiological examen

• Other uncontrolled medical condition (uncontrolled diabetes, hypertension…)

• Concomitant drugs such as inhibitors/inducers of cytochrome P450 3A4 (CYP3A4)

• Immunocompromised patients, including known seropositivity for HIV

• Digestive problems modifying the absorption of Rapamycin (gastric tube feeding accepted)

• Pregnant or nursing (lactating) women

• Prior treatment with phosphatidylinositol 3-kinase (PI3K) and/or mTOR inhibitors