EUCTR2016-002157-38-NL
Active, not recruiting
Phase 1
Treatment of congenital vascular malformations usingSirolimus: improving quality of Life
Conditionscongenital vascular malformation : Vascular malformationscan involve lymphatic vessels, capillaries, veins and arteries or even combinations. These vascularmalformations are present at birth and grow with the child.MedDRA version: 20.0Level: HLTClassification code 10047091Term: Vascular malformations and acquired anomaliesSystem Organ Class: 100000005125MedDRA version: 20.0Level: LLTClassification code 10047090Term: Vascular malformation peripheralSystem Organ Class: 100000158692MedDRA version: 20.0Level: PTClassification code 10070181Term: Gastrointestinal vascular malformationSystem Organ Class: 10017947 - Gastrointestinal disordersMedDRA version: 20.0Level: PTClassification code 10074979Term: Vascular malformationSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- congenital vascular malformation : Vascular malformationscan involve lymphatic vessels, capillaries, veins and arteries or even combinations. These vascularmalformations are present at birth and grow with the child.
- Sponsor
- Radboud University Medical Center
- Enrollment
- 75
- Status
- Active, not recruiting
- Last Updated
- 4 years ago
Overview
Brief Summary
No summary available.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Diagnosis of Congenital venous malformation, or lymphatic malformation or combined.
- •Age between 1\-60 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
- •Karnofsky score \> 50
- •Are the trial subjects under 18? yes
Exclusion Criteria
- •No written informed consent
- •Known hypersensitivity to drugs or metabolites from similar classes as study
- •Known hypersensitivity to drugs or metabolites from similar classes as study
- •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
- •Pregnant or lactating women
Outcomes
Primary Outcomes
Not specified
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Registry for Patients with Vascular Malformations and Tumors (VasMuT)Q27.8Q27.9Q28.3D18.1M89.5Q82.5Q27.3Q87.2Q85.8Q28.2I78.0Q87.3Q82.8Q87.8Q78.4D18.0C49.9C22.3Other specified congenital malformations of peripheral vascular systemCongenital malformation of peripheral vascular system, unspecifiedOther malformations of cerebral vesselsLymphangioma, any siteOsteolysisCongenital non-neoplastic naevusPeripheral arteriovenous malformationCongenital malformation syndromes predominantly involving limbsOther phakomatoses, not elsewhere classifiedArteriovenous malformation of cerebral vesselsHereditary haemorrhagic telangiectasiaCongenital malformation syndromes involving early overgrowthOther specified congenital malformations of skinOther specified congenital malformation syndromes, not elsewhere classifiedEnchondromatosisHaemangioma, any siteConnective and soft tissue, unspecifiedAngiosarcoma of liverDRKS00026788niversitätsklinik FreiburgZentrum für Kinder- und JugendmedizinPädiatrische Hämatologie und Onkologie1,000
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