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Clinical Trials in Germany / NCT02638389
Recruiting Phase 3

Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care

NCT02638389 · tracked via the Priya Life Science Germany tracker
Phase
Phase 3
Started
2016-01-25
Last updated
2023-02-24

Condition(s) studied

Vascular Malformations

Investigational drug(s) / intervention(s)

Sirolimus →

Sirolimus: evaluate the efficacy and safety of sirolimus in these patients

Study summary

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.

Eligibility

Sex
ALL
Min age
3 Months
Max age
70 Years
Healthy volunteers
No
Inclusion 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 Exclusion Criteria: * 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 within the last 6 months, documented by persistent elevated cardiac enzymes or persistent regional wall abnormalities on assessment of LVEF function, history of documented congestive heart failure (New York Heart Association functional classification III-IV), documented cardiomyopathy, family history of congenital long or short QT, or known history of QT/QTc prolongation of Torsades de Pointes (TdP) * 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, or small bowel resection) * 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 cirrhosis, active chronic hepatitis, severely impaired lung function with a spirometry ≤ 50% of the normal predicted value and/or O2 saturation ≤ 88% at rest, etc.) * Immunocompromised patients, including known seropositivity for HIV * Pregnant or lactating women * Prior treatment with PI3K and/or mTOR inhibitors

Primary outcome measure(s)

Trial sites (3)

FacilityCityRegionStatus
Cliniques Universitaires Saint-Luc, Université Catholique de Louvain Bruxelles Brussels Brussels Capital Recruiting
CHU Caen Caen Brittany Region Recruiting
Universitätsklinikum Freiburg Freiburg im Breisgau Germany Not Yet Recruiting

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT02638389 on ClinicalTrials.gov ↗ ← All trials in Germany