Actively Recruiting

Phase 3
Age: 3Months - 70Years
All Genders
NCT02638389

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

Led by Cliniques universitaires Saint-Luc- Université Catholique de Louvain · Updated on 2023-02-24

250

Participants Needed

3

Research Sites

740 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 3Months - 70Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Patients with complex vascular anomalies refractory to standard care such as medical treatment, surgery, or sclerotherapy/embolization
  • Adequate medullary function: hemoglobin > 10.0 g/dl, neutrophils > 1500/mm³, and platelets > 100,000/mm³
  • Laboratory values: total serum bilirubin ≤ 1.5 x ULN (or ≤ 3 x ULN total bilirubin with direct bilirubin ≤ 1.5 x ULN for Gilbert Syndrome)
  • Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN (or < 5.0 x ULN if hepatic metastases present)
  • Serum creatinine ≤ 1.5 x ULN or a 24-hour creatinine clearance ≥ 60 mL/min if serum creatinine is ≥ 1.5 x ULN
  • Karnofsky performance status > 50
  • Ability to sign informed consent
  • Women of childbearing age must agree to use contraceptive methods during the study
Not Eligible

You will not qualify if you...

  • Severe or uncontrolled medical conditions that may affect participation or study results, including significant cardiac diseases or recent myocardial infarction within 6 months
  • Gastrointestinal diseases impairing absorption of sirolimus (e.g., ulcerative diseases, uncontrolled nausea/vomiting, diarrhea ≥ Grade 2, malabsorption, small bowel resection)
  • Known hypersensitivity to drugs similar to study treatment
  • Other severe or uncontrolled medical conditions contraindicating study participation (e.g., pancreatitis, liver cirrhosis, active chronic hepatitis, severely impaired lung function with spirometry ≤ 50% predicted or O2 saturation ≤ 88% at rest)
  • Immunocompromised patients, including known HIV seropositivity
  • Pregnant or lactating women
  • Prior treatment with PI3K and/or mTOR inhibitors

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 3 locations

1

Cliniques Universitaires Saint-Luc, Université Catholique de Louvain Bruxelles

Brussels, Brussels Capital, Belgium, 1200

Actively Recruiting

2

CHU Caen

Caen, Brittany Region, France, 14000

Actively Recruiting

3

Universitätsklinikum Freiburg

Freiburg im Breisgau, Germany, 79106

Not Yet Recruiting

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Research Team

L

Laurence M. Boon, MD, PhD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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