Personalized sirolimus regimen for vascular malformations: a retrospective analysis of VASE cohort.
Emmanuel Seront, An Van Damme, Julien Coulie...
https://pubmed.ncbi.nlm.nih.gov/41366677Actively Recruiting
Phase 3
Age: 3Months - 70Years
All Genders
ID02638389
Phase III Multicentric Study Evaluating the 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
260 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying the effectiveness and safety of Rapamycin (sirolimus), an mTor inhibitor, in people with complex vascular malformations that do not respond well to standard treatments like surgery or sclerotherapy. These malformations can cause chronic pain and serious health issues. This phase III multicenter study aims to provide better treatment guidelines by enrolling about 250 patients, including both children and adults. Participants with venous, lymphatic, or complex vascular malformations will receive sirolimus. The study evaluates sirolimus given as a drug to see if it improves the condition and is safe to use. This study includes ongoing treatment and monitoring over a period of up to two years. During the study, participants will regularly assess their symptoms and quality of life using a visual scale every three months. Researchers will also monitor any side effects monthly at first, then every three months. Imaging tests like MRI will measure changes in the malformations after 12 months. Digital photographs and other evaluations will help track treatment effects over the two-year period.
CONDITIONS
Brief 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
You may qualify if you...
- 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)
- Adequate medullary function: Hemoglobin > 10.0 g/dl, neutrophils >1500/mm³ and platelets > 100,000/mm³
- Total serum bilirubin 1.5 x ULN (or total 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 or if higher, a 24-hour Creatinine Clearance 60 mL/min
- Karnofsky performance status > 50
- Ability to sign informed consent
- Women of childbearing age must use contraceptive methods during the study period
You will not qualify if you...
- Severe or uncontrolled medical conditions that interfere with participation, including serious heart diseases such as unstable angina, recent myocardial infarction, congestive heart failure, cardiomyopathy, or known prolonged QT interval
- Impaired gastrointestinal function or diseases affecting sirolimus absorption, such as ulcers, severe nausea or vomiting, diarrhea of grade 2 or higher, malabsorption syndrome, or small bowel resection
- Known allergy to drugs similar to sirolimus
- Other severe or uncontrolled medical conditions like acute or chronic pancreatitis, liver cirrhosis, active chronic hepatitis, severe lung impairment with spirometry < 50% predicted or oxygen saturation < 88% at rest
- Immunocompromised patients, including those with HIV
- Pregnant or breastfeeding women
- Previous treatment with PI3K or mTOR inhibitors
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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Your Study Journey
Screening
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Treatment
Duration - Up to 2 years
Participants receive sirolimus to evaluate its efficacy and safety in treating vascular malformations that are refractory to standard care.
Monthly visits during the first 3 months, then visits every 3 months up to 2 years
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
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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Published Research Related To This Trial
Preliminary results of the European multicentric phase III trial regarding sirolimus in slow-flow vascular malformations.
Emmanuel Seront, An Van Damme, Catherine Legrand...
https://pubmed.ncbi.nlm.nih.gov/37937645