Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care
Recruiting
Phase 3Interventional Study
Vascular Malformations
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
Ready to participate?
Review the details below, then apply to join this clinical trial.
At a Glance
Age
3 Months – 70
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
250 (estimated)
Sponsor
Cliniques universitaires Saint-Luc- Université Catholique de Louvain · Other
Who this trial is looking for
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
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.
Trial Locations
Loading…
Loading trial locations…
Facility
City
State
Country
Status
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,…
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
Picking one helps us show the most relevant trials first.
Your results are loading in the background — you can
change this any time from the results page.
Set your location to continue
Find My Trials uses your location to surface clinical trials near you.
Add your city or zip code to your profile and try again.