Completed

vasca-LMClinical Study on Efficacy and Safety of the mTor Rapamycin Inhibitor Found in the Complex Vascular Malformations

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What is being tested

Sirolimus

Drug
Who is being recruted

Congenital Abnormalities+1

+ Cardiovascular Diseases

+ Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Until 70 Years
+12 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 3
Interventional
Study Start: May 2012
See protocol details

Summary

Principal SponsorCliniques universitaires Saint-Luc- Université Catholique de Louvain
Last updated: April 14, 2016
Sourced from a government-validated database.Claim as a partner

Study start date: May 1, 2012

Actual date on which the first participant was enrolled.

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.

NCT01811667
Principal SponsorCliniques universitaires Saint-Luc- Université Catholique de Louvain
Last updated: April 14, 2016
Sourced from a government-validated database.Claim as a partner

Protocol

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

19 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Until 70 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Congenital AbnormalitiesCardiovascular DiseasesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCardiovascular Abnormalities

Criteria

5 inclusion criteria required to participate
Karnofsky or Landry > 50
Patients must have adequate liver function (LDL-cholesterol, triglycerides,...)
Patients must have adequate organ function: neutrophils > 1500/mm^3, Hb > 8,0 g et platelets > 50.000/mm^3 (no platelets limits for the Kasabach Merritt syndrome)
Patients must have adequate renal function(normal creatinin depending on the age), clearance > 70 ml/min/1.73m^2 and Urin Protein Creatinine ratio < 0.3 g
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7 exclusion criteria prevent from participating
Concomitant drugs such as inhibitors/inducers of cytochrome P450 3A4 (CYP3A4)
Dental equipments or prosthesis interfering onto a radiological examen
Digestive problems modifying the absorption of Rapamycin (gastric tube feeding accepted)
Immunocompromised patients, including known seropositivity for HIV
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Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Seric level between 10 to 15 ng/ml Pills for the adults and liquid for the children. Twice a day.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Cliniques universitaires Saint-Luc

Brussels, BelgiumOpen Cliniques universitaires Saint-Luc in Google Maps
CompletedOne Study Center