Using Sirolimus to treat complex vascular anomalies that don't respond to standard care

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase III clinical trial aims to evaluate the efficacy and safety of Sirolimus, an mTOR inhibitor, in treating patients with complex vascular malformations that have not responded to conventional therapies such as surgery or sclerotherapy. The study will enroll approximately 250 patients, including both adults and children, across multiple European centers. By targeting the PI3Kinase/AKT/mTor pathway, the trial seeks to provide a new therapeutic option for patients suffering from significant morbidity and complications due to these vascular conditions.

Why it matters

Who can join: the full rules

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

Where it's running

• Cliniques Universitaires Saint-Luc, Université Catholique de Louvain Bruxelles
  Brussels, Brussels Capital, Belgium Recruiting
• CHU Caen
  Caen, Brittany Region, France Recruiting
• Universitätsklinikum Freiburg
  Freiburg im Breisgau, Germany Not yet recruiting

Study contacts

• Principal investigator: Laurence M Boon, MD, PhD — Cliniques universitaires Saint-Luc- Université Catholique de Louvain
• Study coordinator: Laurence M. Boon, MD, PhD
• Email: laurence.boon@uclouvain.be
• Phone: + 32-2-764 8020

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Bleomycin Electrosclerotherapy for Low-Flow Vascular Malformations

  This trial tests whether adding electrical pulses to bleomycin injections (BEST) can better treat low-flow vascular malformations in adults.

  Recruiting Phase 2 Institute of Oncology Ljubljana · Ljubljana · NCT07579962

• Everolimus treatment for adults with vascular malformations who cannot have effective surgery

  This study will try daily oral everolimus in adults with vascular malformations who are not good candidates for surgery to see if it shrinks lesions and improves symptoms.

  Recruiting Phase 2 Xuanwu Hospital, Beijing · Beijing · NCT07037238

• Treatment for PIK3CA-related overgrowth and malformations using RLY-2608

  This study is testing a new treatment called RLY-2608 to see if it can help adults and children with PIK3CA-related overgrowth and malformations feel better.

  Recruiting at 40 sites Phase 2 Relay Therapeutics, Inc. · Phoenix, Arizona and 39 other locations · NCT06789913

• Treatment for low-flow venous malformations using electrosclerotherapy

  This study is testing if a new treatment using electrosclerotherapy with Bleomycin can safely help people with low-flow venous malformations feel better.

  Recruiting Istituto Ortopedico Rizzoli · Bologna, Emilia-Romagna · NCT06189092

• Targeted therapies for patients with vascular malformations

  This study is testing new targeted treatments for patients with slow-flow and fast-flow vascular malformations to see if they work better than the usual therapies.

  Recruiting at 2 sites Phase 2 Murdoch Childrens Research Institute · Parkville, Victoria and 1 other locations · NCT05983159

• Assessing patient outcomes for vascular malformations

  This study is trying to create a tool that helps people with venous malformations share how their condition affects their daily lives, so doctors can better understand their needs and improve treatment.

  Recruiting Johns Hopkins University · Baltimore, Maryland · NCT04104464

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.