Treatment of congenital vascular malformations with Sirolimus

Phase 1

Active, not recruiting

• 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.; MedDRA version: 20.0Level: HLTClassification code 10047091Term: Vascular malformations and acquired anomaliesSystem Organ Class: 100000005125; MedDRA version: 20.0Level: LLTClassification code 10047090Term: Vascular malformation peripheralSystem Organ Class: 100000158692; MedDRA version: 20.0Level: PTClassification code 10070181Term: Gastrointestinal vascular malformationSystem Organ Class: 10017947 - Gastrointestinal disorders; MedDRA version: 20.0Level: PTClassification code 10074979Term: Vascular malformationSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2016-002157-38-NL
• Lead Sponsor: Radboud University Medical Center

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2017-07-19
• Last Update Posted: 29 November 2021

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: All
• Target Recruitment: 75

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
Number of subjects for this age range: 25
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 48
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

•No written informed consent
•Known hypersensitivity to drugs or metabolites from similar classes as study
treatment.
•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 clinical trial of medicinal product
• Study Design: Not specified