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

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

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 Overview

Status

Completed

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

19

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Brussels, Belgium, 1200
        • Cliniques Universitaires Saint-Luc

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 70 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Sirolimus
Seric level between 10 to 15 ng/ml Pills for the adults and liquid for the children. Twice a day.
Other Names:
  • rapamycin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Time of duration of the treatment.(Efficacy)
Time Frame: up to 12 months
up to 12 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The number of adverse events observed
Time Frame: up to 12 months
With Common Toxicity Criteria for Adverse Effects version 3
up to 12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Laurence Boon, MD, PhD, Cliniques Universitaires Saint-Luc

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2012

Primary Completion (Actual)

January 1, 2016

Study Completion (Actual)

January 1, 2016

Study Registration Dates

First Submitted

October 5, 2012

First Submitted That Met QC Criteria

March 12, 2013

First Posted (Estimate)

March 14, 2013

Study Record Updates

Last Update Posted (Estimate)

April 14, 2016

Last Update Submitted That Met QC Criteria

April 13, 2016

Last Verified

April 1, 2016

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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