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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01811667
First Posted: March 14, 2013
Last Update Posted: April 14, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Cliniques universitaires Saint-Luc- Université Catholique de Louvain
  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: Intervention Model: Single Group Assignment
Masking: None (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 ]

Secondary Outcome Measures:
  • The number of adverse events observed [ Time Frame: up to 12 months ]
    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

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 70 Years   (Child, Adult, Senior)
Sexes Eligible for Study:   All
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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): 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
First Submitted: October 5, 2012
First Posted: March 14, 2013
Last Update Posted: April 14, 2016
Last Verified: April 2016

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