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

Primary Purpose

Cardiovascular Abnormalities

Status

Completed

Phase

Phase 3

Locations

Belgium

Study Type

Interventional

Intervention

Sirolimus

About this trial

This is an interventional treatment trial for Cardiovascular Abnormalities focused on measuring Vascular Abnormalities

Eligibility Criteria

undefined - 70 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Cliniques universitaires Saint-Luc

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Sirolimus

Arm Description

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

Outcomes

Primary Outcome Measures

Time of duration of the treatment.(Efficacy)

Secondary Outcome Measures

The number of adverse events observed

With Common Toxicity Criteria for Adverse Effects version 3

Full Information

NCT ID

NCT01811667

First Posted

October 5, 2012

Last Updated

April 13, 2016

Sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

1. Study Identification

Unique Protocol Identification Number

NCT01811667

Brief Title

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

Acronym

vasca-LM

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2016

Overall Recruitment Status

Completed

Study Start Date

May 2012 (undefined)

Primary Completion Date

January 2016 (Actual)

Study Completion Date

January 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cardiovascular Abnormalities

Keywords

Vascular Abnormalities

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

19 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Sirolimus

Arm Type

Experimental

Arm Description

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

Intervention Type

Drug

Intervention Name(s)

Sirolimus

Other Intervention Name(s)

rapamycin

Primary Outcome Measure Information:

Title

Time of duration of the treatment.(Efficacy)

Time Frame

up to 12 months

Secondary Outcome Measure Information:

Title

The number of adverse events observed

Description

With Common Toxicity Criteria for Adverse Effects version 3

Time Frame

up to 12 months

10. Eligibility

Sex

All

Maximum Age & Unit of Time

70 Years

Accepts Healthy Volunteers

Eligibility 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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Laurence Boon, MD, PhD

Organizational Affiliation

Cliniques universitaires Saint-Luc

Official's Role

Principal Investigator

Facility Information:

Facility Name

Cliniques universitaires Saint-Luc

City

Brussels

ZIP/Postal Code

1200

Country

Belgium

12. IPD Sharing Statement

Learn more about this trial

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

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