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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
ClinicalTrials.gov Identifier:
NCT01811667
First received: October 5, 2012
Last updated: April 13, 2016
Last verified: April 2016
  Purpose

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.


Condition Intervention Phase
Cardiovascular Abnormalities
Drug: Sirolimus
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
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

Resource links provided by NLM:


Further study details as provided by Cliniques universitaires Saint-Luc- Université Catholique de Louvain:

Primary Outcome Measures:
  • Time of duration of the treatment.(Efficacy) [ Time Frame: up to 12 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • The number of adverse events observed [ Time Frame: up to 12 months ] [ Designated as safety issue: Yes ]
    With Common Toxicity Criteria for Adverse Effects version 3


Enrollment: 19
Study Start Date: May 2012
Study Completion Date: January 2016
Primary Completion Date: January 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Sirolimus
Seric level between 10 to 15 ng/ml Pills for the adults and liquid for the children. Twice a day.
Drug: Sirolimus
Other Name: rapamycin

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.

  Eligibility

Ages Eligible for Study:   up to 70 Years   (Child, Adult, Senior)
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01811667

Locations
Belgium
Cliniques universitaires Saint-Luc
Brussels, Belgium, 1200
Sponsors and Collaborators
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Investigators
Principal Investigator: Laurence Boon, MD, PhD Cliniques universitaires Saint-Luc
  More Information

Responsible Party: Cliniques universitaires Saint-Luc- Université Catholique de Louvain
ClinicalTrials.gov Identifier: NCT01811667     History of Changes
Other Study ID Numbers: vasca-LM 
Study First Received: October 5, 2012
Last Updated: April 13, 2016
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products

Keywords provided by Cliniques universitaires Saint-Luc- Université Catholique de Louvain:
Vascular Abnormalities

Additional relevant MeSH terms:
Vascular Malformations
Congenital Abnormalities
Cardiovascular Abnormalities
Cardiovascular Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs

ClinicalTrials.gov processed this record on August 29, 2016