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

  • STATUS
    Recruiting
  • End date
    Oct 21, 2025
  • participants needed
    250
  • sponsor
    Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Updated on 21 January 2021

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.

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 venous/lymphatic vascular organisations suggesting an appealing therapeutic target to treat patients with venous, lympathic or complex vascular malformations.

Investigators will realize a multicentric phase III study enrolling a higher number of patients to statistically evaluate the efficacy and the safety of the Rapamycin, an mTOR inhibitor, in the treatment of children and adults with vascular malformations for which conventional therapies such as surgery or sclerotherapy are ineffective or associated with high risk of important complications. Nearly 250 patients (200 adults and 50 children) will be enrolled in several european centers.

Details
Condition Vascular anomaly, Cardiovascular Abnormalities, Cardiovascular Abnormalities, vascular malformation
Treatment Sirolimus
Clinical Study IdentifierNCT02638389
SponsorCliniques universitaires Saint-Luc- Université Catholique de Louvain
Last Modified on21 January 2021

Eligibility

Yes No Not Sure

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/mm and platelets > 100.000/mm
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
Clear my responses

How to participate?

Step 1 Connect with a study center
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar

Primary Contact

site

0/250

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note