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

Launched by CLINIQUES UNIVERSITAIRES SAINT-LUC- UNIVERSITÉ CATHOLIQUE DE LOUVAIN · Dec 18, 2015

Keywords

ClinConnect Summary

This clinical trial is studying the effectiveness and safety of a medication called Sirolimus in treating complex vascular anomalies, which are unusual growths or malformations in blood vessels that have not responded to standard treatments like surgery or medication. The goal is to see if Sirolimus, which affects certain pathways in the body involved in blood vessel formation, can help patients with these challenging conditions.

To participate in this trial, patients need to have specific types of vascular anomalies that haven't improved with traditional treatments. They must also meet certain health criteria, such as having enough healthy blood counts and liver function. Participants will be closely monitored during the study, and women who can become pregnant will need to use contraception. This trial is currently recruiting participants of all ages, and it's an opportunity for those with difficult-to-treat vascular issues to potentially benefit from a new treatment approach.

Gender

ALL

Eligibility criteria

• 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