Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies...
Kaposiform HemangioendotheliomaTufted Angioma2 moreIn this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.
Bleomycin Electrosclerotherapy Treatment of Vascular Malformations: A Feasibility Study
Vascular MalformationsThe aim of the BEST study is to examine the feasibility of assessing patient and clinician reported outcome of a single electrosclerotherapy treatment of vascular malformations. Sclerotherapy = injection of Bleomycin into vascular birthmarks to seal off abnormal vascular channels Electroporation = application of an electric field to vessels treated with a handheld needle electrode Electrosclerotherapy = a combination of Bleomycin sclerotherapy and electroporation
Efficacy and Safety of Rapamycin to Complex Vascular Anomalies in Pediatric Patients
Vascular AnomaliesKHE and TA are rare tumors and some of the cases may lead to life-threatening complications including Kasabach-Merritt Phenomenon. Typically treated with steroids and vincristine, a majority of the cases do not have good prognosis. Complex vascular malformations are always managed by surgery,sclerotherapy and embolization therapy. While many of the cases still lead to complications such as disfigurement, chronic pain, recurrent infections, coagulopathies. Different medical centers are exploring new therapy for these tough problems. This study is plotted to determine the efficacy and safety of rapamycin monotherapy in KHE/TA and complex vascular malformations in pediatric patients.
Topical Rapamycin/Sirolimus for Complicated Vascular Anomalies and Other Susceptible Lesions
Vascular AnomalyProposed Study: Treatment protocol for the use of the topical Rapamycin/Sirolimus for Complicated Vascular Anomalies and other susceptible lesions Aim The aim of this treatment study is to evaluate the benefit and tolerability of topical sirolimus applied to cutaneous vascular anomalies in pediatric patients. The primary end point will be individually determined based on improvement in lesional clinical characteristics over baseline Rationale for topical sirolimus use in VA The rationale for the use of topical sirolimus is to minimize these potential side effects and risks. Data for the use of topical sirolimus for vascular anomalies at this time are anecdotal and case reports only. As such, this prospective protocol seeks to determine the effectiveness and tolerability of topical sirolimus on patients with vascular anomalies that have a cutaneous component. Experimental design This is an open-labeled efficacy trial with the aim to determine if topical sirolimus can be safe and efficacious in treating the cutaneous component of complicated vascular anomalies. Patients who meet eligibility criteria with a diagnosis of vascular anomaly (VA) with cutaneous component will be offered treatment with the investigational topical sirolimus. Patients will receive topical sirolimus therapy for a total of six months and will be monitored regularly at the research site for clinical response. Response will be based on pre-determined clinical criteria. Patients will be removed from study if there is no response at three months after initiation of therapy. Clinical response will be defined as improvement in measurable parameters defined at the time of initiation of therapy. These include Size of lesions, measured in two parallel longest diameters Flattening of lesion Number of vesicles Episodes of superinfection or bleeding Improvement in pain Drug Information The topical sirolimus formulation will be made at a concentration of 1% sirolimus ointment. Bulk sirolimus powder will be compounded in a liposomal base in a GMP level pharmaceutical company. This base will enhance drug penetration into the skin. It ensures adequate adhesion to the application area and a low degree of systemic absorption. Due to limited absorption only mild side effects are expected.
Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care
Vascular MalformationsThe 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.
Topical Sirolimus in Cutaneous Lymphatic Malformations
Vascular MalformationsLymphatic MalformationCutaneous microcystic lymphatic malformations (CMLMs) are rare conditions of children and adults resulting from abnormal embryologic development of lymphatic vessels. They present as clusters of vesicles full of lymph and blood of various extent. They ooze and bleed, inducing maceration, esthetic impairment, scars, pain, bacterial infections and impaired quality of life. Currently, treatments for CMLMs are disappointing, and their management is challenging. Sirolimus is an inhibitor of mammalian target of rapamycin (mTOR), a serine/threonine protein kinase involved in cell growth and proliferation, cellular metabolism, autophagy and angio-lymphangiogenesis. Topical sirolimus, known to be efficient and well tolerated in cutaneous angiofibromas linked to tuberous sclerosis, has recently been reported effective in few reports of patients with CMLMs. The objective of this trial is to compare the efficacy and safety of a 12-week application of 0.1% topical sirolimus versus topical vehicle in CMLMs in children and adults.
Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With...
Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)This study is a two periods multi-center Phase II trial, with a 6 months double-blind, placebo-controlled period followed by open label period. It will involve 20 participants with MCAP syndrome. Assessment will be performed at baseline and after 24 months of treatment and will include MRI, biological and clinical results, attention, language, and Intellectual Quotient (IQ) assessment. Appropriate neuropsychological tests will be used according to the age of patients and their level of cognitive impairment.
Functional Navigation in Surgery of Cerebral Tumors and Vascular Malformations
TumorBrain2 moreThe purpose of the study is to assess accuracy, advantages of functional neuronavigation and calculate safe distance from motor areas to brain tumors and vascular malformations in image-guided surgery.
SelfWrap-Assisted Arteriovenous Fistulas
Chronic Kidney DiseasesChronic Kidney Failure15 moreThis is a single-center, prospective, single-arm clinical study to evaluate the feasibility, safety, and performance of VenoStent's SelfWrap® Bioabsorbable Perivascular Wrap on arteriovenous fistulas (AVFs). All participants are chronic kidney disease (CKD) patients already receiving hemodialysis treatments that are referred for creation of a new arteriovenous fistula (AVF).
A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations
Slow-Flow Vascular MalformationFast-Flow Vascular Malformation18 moreRecent studies have demonstrated that growth of vascular malformations can be driven by genetic variants in one of 2 signalling pathways. Targeted drugs specific to these pathways have been developed and shown to be effective in treating cancer. This study will describe the effectiveness of (i) 12 months of alpelisib therapy for participants with slow-flow vascular malformations and a gene mutation in one of these signalling pathways (module 1) and (ii) 12 months of mirdametinib therapy for participants with fast-flow vascular malformations and a gene mutations in the other signalling pathway (module 2).