Efficacy and Safety of the Mammalian Target of Rapamycin (mTor Rapamycin) Inhibitor in Vascular Malformations (vasca-LM)
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.
|Study Design:||Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Clinical Study on Efficacy and Safety of the mTor Rapamycin Inhibitor Found in the Complex Vascular Malformations|
- Time of duration of the treatment.(Efficacy) [ Time Frame: up to 12 months ]
- The number of adverse events observed [ Time Frame: up to 12 months ]With Common Toxicity Criteria for Adverse Effects version 3
|Study Start Date:||May 2012|
|Study Completion Date:||January 2016|
|Primary Completion Date:||January 2016 (Final data collection date for primary outcome measure)|
Seric level between 10 to 15 ng/ml Pills for the adults and liquid for the children. Twice a day.
Other Name: rapamycin
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01811667
|Cliniques universitaires Saint-Luc|
|Brussels, Belgium, 1200|
|Principal Investigator:||Laurence Boon, MD, PhD||Cliniques universitaires Saint-Luc|