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Tests treatment safety and results for Vascular Malformations
Official title Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care
ClinicalTrials.gov ID: NCT02638389
What this study is testing
What is Sirolimus?
Sirolimus is an investigational medicine, being studied as a potential treatment for vascular malformations.
Also referred to as Rapamycine.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The phosphatidylinositol 3-kinase (PI3Kinase)/Protein Kinase B (AKT)/mammalian target of rapamycin (mTor) pathway plays a role on the development and the venous/lymphatic vascular organisations. The investigators want to study the efficacy and the safety of Rapamycin, an mTor inhibitor.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 3 to 70
You may be able to join if
- Patients with complex vascular anomalies that are refractory to standard care such as medical treatment, surgical resection and/or...
- Patients must have adequate medullary function: Hemoglobine\> 10,0 g/dl, neutrophils \>1500/mm³ and platelets \> 100.000/mm³
- Patients must have the following laboratory values:
- Total serum bilirubin ≤ 1.5 x ULN (or totally bilirubin ≤ 3 x ULN with direct bilirubin ≤ 1.5 x ULN in patients with well documented Gilbert Syndrome)
- Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN (or \< 5.0 x ULN if hepatic metastases are present)
You likely can't join if
- Any of the following concurrent severe and/or uncontrolled medical conditions, which could compromise participation in the study or interfere with...
- Impaired cardiac function or clinically significant cardiac diseases, including unstable angina pectoris, ventricular arrhythmia, valvular disease...
- Impairment of Gastro-Intestinal (GI) function or GI disease that may significantly alter the absorption of sirolimus (e.g., ulcerative diseases...
- Known hypersensitivity to drugs or metabolites from similar classes as study treatment.
- Patient has other concurrent severe and /or uncontrolled medical condition that would,in the investigator's judgment, contraindicated participation...
- Immunocompromised patients, including known seropositivity for HIV
See the full eligibility criteria
- Patients with complex vascular anomalies that are refractory to standard care such as medical treatment, surgical resection and/or sclerotherapy/embolization (ineffective or accompanied by major complications)
- Patients must have adequate medullary function: Hemoglobine\> 10,0 g/dl, neutrophils \>1500/mm³ and platelets \> 100.000/mm³
- Patients must have the following laboratory values:
- Total serum bilirubin ≤ 1.5 x ULN (or totally bilirubin ≤ 3 x ULN with direct bilirubin ≤ 1.5 x ULN in patients with well documented Gilbert Syndrome)
- Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN (or \< 5.0 x ULN if hepatic metastases are present)
- Serum creatinine 1.5 x ULN. If the serum creatinine is ≥ 1.5 x ULN, then a 24-hour Creatinine Clearance must be conducted and the result must be ≥ 60 mL/min.
- Karnofsky \> 50
- Patients have to be able to sign the informed consent
- Women in age of procreation have to be informed that contraceptive methods are mandatory during the study time
- Any of the following concurrent severe and/or uncontrolled medical conditions, which could compromise participation in the study or interfere with the study results:
- Impaired cardiac function or clinically significant cardiac diseases, including unstable angina pectoris, ventricular arrhythmia, valvular disease with documented compromise in cardiac function, myocardial infarction...
- Impairment of Gastro-Intestinal (GI) function or GI disease that may significantly alter the absorption of sirolimus (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea ≥ Grade 2, malabsorption syndrome...
- Known hypersensitivity to drugs or metabolites from similar classes as study treatment.
- Patient has other concurrent severe and /or uncontrolled medical condition that would,in the investigator's judgment, contraindicated participation in the clinical study (e.g. acute or chronic pancreatitis, liver...
- Immunocompromised patients, including known seropositivity for HIV
- Pregnant or lactating women
- Prior treatment with PI3K and/or mTOR inhibitors
The study team makes the final eligibility decision.
Where it's taking place
- Brussels, Brussels Capital, Belgium
- Caen, Brittany Region, France
- Freiburg im Breisgau, Germany
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 3 months to 70 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Brussels, Brussels Capital, Belgium; Caen, Brittany Region, France; Freiburg im Breisgau, Germany. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.