Treatment of congenital vascular malformations using Sirolimus: improving quality of Life
- Conditions
- congenital vascular malformationsvascular anomalies10047066
- Registration Number
- NL-OMON45838
- Lead Sponsor
- Radboud Universitair Medisch Centrum
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 75
• Diagnosis of Congenital venous malformation, or lymphatic malformation or combined.
• Age between 1-70 years.
• Patients (or legal guardians for children) have to be able to sign the informed consent
• Patients are either refractory to standard care such as medical treatment (low molecular weight heparines, pain medication etc.), surgical resection and/or sclerotherapy/embolization (ineffective or accompanied by major complications) or there is no possibility for surgical intervention anymore. Only patients that have a normal clinical screening (no signs for infection, normal bone marrow function, normal liver and kidney function, normal glucose metabolism etc.) can be included.
• Patients included have no cardiac impairment
• Patients have no gastrointestinal impairment as Sirolimus is absorbed gastro-intestinal and normal function is needed
• No other underlying medical disorder like Down syndrome or other syndromes
• Women of reproductive age have to be informed that contraceptive methods are
mandatory during the study time, pregnant women are excluded as also breast-feeding women
• Karnofsky score > 50
• No written informed consent
• 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.)
• Recent history of primary malignancy <= 5 years
• Impaired cardiac function or clinically significant cardiac diseases
• Immunocompromised patients, including known seropositivity for HIV
• Patient with any other concurrent severe and /or uncontrolled medical condition that
would,in the investigator*s judgment, contraindicated participation in the clinical
study.
• Pregnant or lactating women
• Karnofsky score < 50
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>To investigate whether Sirolimus results in a significant and clinically<br /><br>relevant reduction of pain and an<br /><br>improved quality of life in patients with untreatable vascular malformations.</p><br>
- Secondary Outcome Measures
Name Time Method <p>What length of therapy is required to have and maintain adequate pain reduction?<br /><br>Will response to Sirolimus prolong after stop treatment or will there be a<br /><br>rebound?<br /><br>Will Sirolimus only have effect on pain reduction or will Sirolimus also<br /><br>inhibit growth/progression of the<br /><br>vascular malformation or even lead to reduction of the size of the vascular<br /><br>malformation?<br /><br>Which long term consequences can be observed after treatment with Sirolimus<br /><br>e.g. in children?<br /><br>Are there genetic factors in the vascular malformation that can predict outcome<br /><br>of treatment with Sirolimus?<br /><br>Will Sirolimus lead to a more cost-effective treatment for this patient group?</p><br>