- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01811667
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
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
Enrollment (Actual)
Phase
- Phase 3
Contacts and Locations
Study Locations
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Brussels, Belgium, 1200
- Cliniques Universitaires Saint-Luc
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
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
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.
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Other Names:
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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
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up to 12 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The number of adverse events observed
Time Frame: up to 12 months
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With Common Toxicity Criteria for Adverse Effects version 3
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up to 12 months
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Collaborators and Investigators
Investigators
- Principal Investigator: Laurence Boon, MD, PhD, Cliniques Universitaires Saint-Luc
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Estimate)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- vasca-LM
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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