[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"vascular-malformations\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:vascular-malformations":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,9,0,[8,47,78,103,147,171,197,222,247],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100576793","phase-2-a-phase-2-study-of-mutant-selective-pi3k-inhibitor-rly-2608-in-adults-and-children-with-pik3ca-related-overgrowth-spectrum-and-malformations-driven-by-pik3ca-mutation-the-reinspire-study-100576793",false,"NCT06789913","A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation (The ReInspire Study)","A Phase 2 Study of Mutant-selective PI3Kα Inhibitor, RLY-2608, in Adults and Children With PIK3CA Related Overgrowth Spectrum and Malformations Driven by PIK3CA Mutation","Key Inclusion Criteria:\n\n* The participant must have a clinical diagnosis of PROS or a malformation within the ISSVA classification.\n* One or more documented activating PIK3CA mutation(s) that are targeted by selective PI3Kα inhibitors in lesional tissue and\u002For cell-free DNA from the lesion or blood. Some participants may be eligible without a documented PIK3CA mutation, with the sponsor's approval, as long as no other genetic driver has been documented.\n* Lansky (\\\u003C16 yo) or Karnofsky (≥16 yo) performance status of ≥50.\n* Agree to provide archived lesional fluid and\u002For tissue or be willing to undergo pretreatment lesional biopsy (if considered safe and medically feasible) to assess PIK3CA status.\n\nKey Exclusion Criteria:\n\n* Known hypersensitivity to RLY-2608.\n* Any factors that increase the risk of QTc prolongation or risk of arrhythmic events\n* Clinically significant, uncontrolled cardiovascular disease\n* Received disease-directed therapy prior to the first dose of study drug:\n\n  1. Systemic therapy or antibody within 5 half-lives of the therapy.\n  2. Local therapy including radiation, surgery, or other procedures within 28 days; lesion(s) must have demonstrated progression after the procedure.","ALL","2 Years",{"count":19,"type":20},277,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","This is a 3-part Phase 2 randomized study evaluating the safety and efficacy of the mutant-selective PI3Kα inhibitor, zovegalisib (RLY-2608), in adults and children with PIK3CA Related Overgrowth Spectrum (PROS) and malformations driven by PIK3CA mutation. Part 1 is a dose selection, Part 2 is a basket design with exploratory single-arm cohorts for various subpopulations of participants, and Part 3 is randomized, double-blinded study vs placebo.",[26,27,28,29,30,31,32,33],"PIK3CA-Related Overgrowth Spectrum (PROS)","Lymphatic Malformations","Vascular Malformations","PIK3CA Mutation","CLOVES Syndrome","Klippel Trenaunay Syndrome","Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)","Vascular Anomalies","RECRUITING","2026-06-10",{"date":37,"type":38},"2026-06-12","ACTUAL",{"date":40,"type":38},"2025-06-13",{"date":42,"type":20},"2031-10",{"name":44,"class":45},"Relay Therapeutics, Inc.","INDUSTRY",34,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":51,"acronym":52,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":54,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":21,"phases":58,"briefSummary":59,"conditions":60,"keywords":61,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":69,"startDateStruct":71,"completionDateStruct":73,"leadSponsor":75,"locationsCount":4},"100640742","phase-2-treatment-of-low-flow-vascular-malformations-with-bleomycin-electrosclerotherapy-best-100640742","NCT07579962","Treatment of Low-flow Vascular Malformations With Bleomycin Electrosclerotherapy (BEST)","BEST","Inclusion Criteria:\n\n* age ≥ 18\n* patients with low-flow vascular malformations (venous, lymphatic, capillary or mixed type malformations),\n* patients with a low-flow vascular malformation poorly responding or recurring after previous treatment(s),\n* longer lesion diameter not exceeding 25 cm.\n* more than one lesion can be treated. The limiting factor is the maximal dose per patient per treatment session; 10 000 IU in adults,\n* skin or mucosal, superficial or deep-seated lesions can be treated,\n* technical feasibility of the BEST procedure, i.e.: injection of bleomycin and safe placement of electrodes into the vascular malformation are technically feasible.\n\nExclusion Criteria:\n\n* pregnancy and lactation,\n* women of childbearing potential and men not using reliable contraception,\n* in adults, previous bleomycin exposure with a cumulative dose greater than 100 000 IU. In case of abnormal respiratory results\u002Fchest pathology (including previous severe or long COVID) in consultation with a pulmonologist, special care is required, and bleomycin exposure may be contraindicated,\n* known allergy or hypersensitivity to bleomycin,\n* presence of significant central venous drainage precluding sclerotherapy,\n* acute lung infection or severely reduced lung function,\n* bleomycin-related lung toxicity or reduced lung function which can indicate bleomycin-related lung toxicity,\n* ataxia telangiectasia,\n* chronic renal dysfunction.","18 Years","99 Years",{"count":57,"type":20},140,[23],"In biomedical applications, electroporation is used not only for cancer treatment but also for vaccinations, treatment of cardiac arrhythmias and, more recently, for the treatment of vascular malformations. Bleomycin is a frequently used sclerosing agent in the treatment of various vascular malformations. The use of electrical pulses in addition to bleomycin increases the effectiveness of the treatment, similar to electrochemotherapy. Bleomycin electrosclerotherapy (BEST) is a new treatment modality that is effective in the treatment of low-flow malformations (venous and lymphatic malformations) and potentially also high-flow malformations (arteriovenous malformations). Although a limited number of reports have been published to date, more and more centers are using BEST for the treatment of vascular malformations. As part of the International Network for Sharing Practices on Electrochemotherapy (InspECT) consortium, a dedicated working group has been set up to develop standard operating procedures for BEST. Current Operating Procedures have been prepared and will be used in this clinical trial.",[28],[62,63,64,65,66],"vascular malformations","electroporation","bleomycin","electrosclerotherapy","low flow vascular malformations","NOT_YET_RECRUITING","2026-05-20",{"date":70,"type":38},"2026-05-22",{"date":72,"type":20},"2026-06-30",{"date":74,"type":20},"2032-06-30",{"name":76,"class":77},"Institute of Oncology Ljubljana","OTHER",{"id":79,"slug":80,"hasResults":11,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":84,"eligibilityCriteria":85,"healthyVolunteers":11,"sex":16,"minAge":86,"maxAge":4,"enrollmentInfo":87,"targetDuration":89,"studyType":90,"phases":4,"briefSummary":91,"conditions":92,"keywords":93,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":68,"lastUpdatePostDateStruct":95,"startDateStruct":96,"completionDateStruct":98,"leadSponsor":100,"locationsCount":102},"100530616","treatment-of-low-flow-venous-malformations-with-electrosclerotherapy-prospective-observational-study-100530616","NCT06189092","Treatment of Low-flow Venous Malformations With Electrosclerotherapy. Prospective Observational Study","Trattamento Delle Malformazioni Venose a Basso Flusso Con l'Elettroscleroterapia. Studio Osservazionale Prospettico","BESVAM","Inclusion Criteria:\n\n* Diagnosis of low-flow venous malformations eligible for electrosclerotherapy\n* Non-indication for embolizing treatment\n* Previous treatments are not an exclusion factor, provided that at least 30 days have elapsed.\n\nExclusion Criteria:\n\n* Previous treatment for \\\u003C 30 days\n* Pregnancy and lactation status\n* Patients of childbearing age without contraceptive use\n* Presence of metal synthetic media\n* COPD with FiO2 \\\u003C 30 mmHg\n* Impaired renal function with eGFR\\\u003C30 ml\u002Fmin\u002F1.73mq\n* Patients with Bleomycin intolerance or previous episodes of toxicity Bleomycin-related\n* Patients who have already received a cumulative dose of Bleomycin ≥100 mg\n* Patients who have undergone prior thoracic radiotherapy\n* Patients with a history of seizures and epilepsy","16 Years",{"count":88,"type":20},65,"1 Year","OBSERVATIONAL","Venous malformations (MVs) are congenital abnormalities of the central or periphery caused by developmental errors at different stages of embryogenesis. Histologically they are characterized by large, venous-like vascular spaces. Scleroembolization constitutes the most widespread method in the treatment of venous malformations allowing good results with low invasiveness. Currently, Bleomycin (and its derivatives) is among the most widely used sclerosing agents for slow-flowing vascular malformations (venous and lymphatic malformations) because of the low rate of local serious adverse events such as swelling, necrosis, and nerve injury compared with others.",[28],[94],"Electrosclerotherapy",{"date":70,"type":38},{"date":97,"type":38},"2023-10-28",{"date":99,"type":20},"2027-11-28",{"name":101,"class":77},"Istituto Ortopedico Rizzoli",1,{"id":104,"slug":105,"hasResults":11,"nctId":106,"briefTitle":107,"officialTitle":108,"acronym":109,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":111,"targetDuration":4,"studyType":21,"phases":113,"briefSummary":114,"conditions":115,"keywords":132,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":137,"lastUpdatePostDateStruct":138,"startDateStruct":140,"completionDateStruct":142,"leadSponsor":144,"locationsCount":146},"100514796","phase-2-a-trial-of-targeted-therapies-for-patients-with-slow-flow-or-fast-flow-vascular-malformations-100514796","NCT05983159","A Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations","A Modular Open Label, Signal Seeking, Phase II Trial of Targeted Therapies for Patients With Slow-Flow or Fast-Flow Vascular Malformations (TARGET-VM)","TARGET-VM","MODULE 1\n\nInclusion Criteria:\n\n1. Adult or paediatric patient, 2 years of age or over\n2. Patient has a clinical diagnosis of a slow-flow vascular malformation\n3. Patient has received standard therapy for the vascular malformation or in which, in the opinion of the investigator, standard therapy is not appropriate\n\n   \\- Note: standard therapy may include treatment with sirolimus. A minimum of 14 days since the last dose of sirolimus is required prior to starting treatment with alpelisib\n4. A documented genetic alteration in the PI3K signalling pathway identified by genetic sequencing prior to enrolment in this study\n5. Adequate performance status (Eastern Cooperative Oncology Group Performance Status Scale (ECOG) 0-2 in patients ≥ 16 years of age; Lansky \\> 50 in patients \\\u003C 16 years of age)\n6. Patient has a life expectancy ≥ 12 weeks\n7. Patient is able to swallow and retain oral medication\n8. Adequate haematologic and end-organ function:\n\n   * Haematology: Haemoglobin ≥ 9.0 g\u002FdL; Absolute neutrophil count ≥ 1.5 x 109\u002FL; Platelets ≥ 90 x 109\u002FL, except where bleeding leading to low haemoglobin level is an indication for treatment, in which case haemoglobin \\\u003C 9.0 g\u002FdL is acceptable.\n   * Hepatic function: alanine aminotransferase (ALT) and aspartate aminotransferase (AT) ≤ 3 x ULN; Total bilirubin \\\u003C 2x ULN except for participants with Gilbert's syndrome who may only be included if the total bilirubin is ≤ 3.0 x ULN or direct bilirubin ≤ 1.5 x ULN\n   * Renal function: Serum creatinine \\\u003C 1.5 x ULN\n   * Biochemistry: Calcium (corrected for serum albumin) and magnesium within normal limits or ≤ Grade 1 according to NCI-CTCAE v5.0 if judged clinically not significant by the investigator; Potassium within normal limits, or corrected with supplements\n   * Fasting blood glucose ≤ 7.0 mmol\u002FL and Glycosylated Haemoglobin (HbA1c) ≤ 6.4% (both criteria must be met)\n9. Patient agrees to abstinence or highly effective contraceptive measures for males and women of childbearing potential (WOCBP)\n\n   * Males who are sexually active must use a condom during intercourse while taking alpelisib and for at least 4 weeks after stopping alpelisib and should not father a child in this period. A condom is required to be used also by vasectomised men in order to prevent delivery of the drug via seminal fluid. In addition, male participants must not donate sperm during the study and for at least 4 weeks after stopping alpelisib\n   * Females who are of child-bearing potential, defined as all women physiologically capable of becoming pregnant, must use a highly effective method of contraception during study treatment and for at least 1 week after the last dose of any study treatment. Highly effective contraceptive methods include:\n\n     * Total abstinence (when this is in line with the preferred and usual lifestyle of the subject) Periodic abstinence (eg., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception;\n     * Female sterilisation (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least 6 weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the women has been confirmed by follow up hormone level assessment;\n     * Male partner sterilisation (at least 6 months prior to screening) of the sole partner of a female participant on the study;\n     * Use of oral (estrogen and progesterone), injected or implanted combined hormonal method of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or forms of hormonal contraception that have comparable efficacy (failure rate \\\u003C 1%), for example hormonal vaginal ring or transdermal hormone contraception. In the case of use of oral contraception, women should have been stable on the same pill for a minimum of 3 months before taking study treatment.\n   * Women are considered postmenopausal and not of child bearing potential if they have had 12 months of natural (spontaneous) amenorrhoea with an appropriate clinical profile (i.e., age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or bilateral tubal ligation at least 6 weeks before taking study treatment. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered to be not of child bearing potential.\n10. Patient has signed informed consent and is willing and able to comply with the protocol for the duration of the study, including undergoing treatment and scheduled visits and examinations\n\nExclusion Criteria:\n\n1. History of hypersensitivity to any drugs or metabolites of PI3K inhibitors or any of the excipients of alpelisib\n2. Severe infection requiring intravenous antibiotics within 4 weeks prior to enrolment\n3. Patient has had a major surgical procedure within 4 weeks prior to enrolment\n4. Prior use of an alpha-specific PI3K inhibitor\n5. History of pneumonitis or interstitial lung disease\n6. Patient is pregnant or lactating at the time of study registration. A pregnancy test is mandated for women of child-bearing potential in the screening period\n7. Established diagnosis of type I diabetes mellitus, type II diabetes mellitus requiring anti-hyperglycaemic medication or any participant with HbA1c \\> 6.4%\n8. Patient who is currently receiving medication with a known risk of prolonging the QT interval or inducing Torsades de Pointes\n9. Patient is currently receiving any of the following medications and cannot be discontinued 7 days prior to the start of treatment:\n\n   * Strong inducers of CYP3A4\n   * Strong inhibitors of CYP3A4\n   * Inhibitors of BCRP\n10. History of acute pancreatitis within 1 year of screening or past history of chronic pancreatitis\n11. Patient with Child Pugh score B or C\n12. Unresolved osteonecrosis of the jaw\n13. Impairment of GI function or GI disease that may significantly alter the absorption of the study drug based on investigator discretion\n14. Known history of Human Immunodeficiency Virus (HIV) infection (testing for HIV is not mandatory in screening)\n15. Known history of severe cutaneous reactions like Stevens-Johnson Syndrome (SJS), Toxic Epidermal Necrolysis (TEN), Erythema Multiforme (EM), or Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS)\n16. Known history of clinically significant, uncontrolled heart disease and\u002For recent cardiac events including:\n\n    * History of angina pectoris, coronary artery bypass graft (CABG), symptomatic pericarditis, or myocardial infarction within 6 months prior to the start of study treatment;\n    * History of documented congestive heart failure (New York Heart Association functional classification III-IV);\n    * History of impaired Left Ventricular Ejection Fraction (LVEF) \\\u003C 50% (an assessment of LVEF is not mandatory in screening)\n    * History of clinically significant cardiac arrhythmias, (e.g., ventricular tachycardia), complete left bundle branch block, high grade Atrioventricular (AV) block (e.g. bifascicular block, Mobitz type II and third degree AV block without pacemaker in place);\n    * Uncontrolled hypertension defined by a Systolic Blood Pressure (SBP) ≥ 160 mmHg and\u002For Diastolic Blood Pressure (DBP) ≥ 100 mmHg, with or without anti-hypertensive medication. Initiation or adjustment of antihypertensive medication(s) is allowed prior to screening;\n    * Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or corrected QT interval \\> 470 msec at screening (using Fridericia correction);\n    * Bradycardia (heart rate \\\u003C 50 beats per minute at rest), by electrocardiogram (ECG) or pulse\n17. Patient has other concurrent severe and\u002For uncontrolled medical conditions that would, in the Treating Physician's judgement, contraindicate administration of alpelisib (eg. active or uncontrolled severe infection, chronic active hepatitis, immune-compromised, acute or chronic pancreatitis, uncontrolled high blood pressure)\n18. Patient is unable to understand and comply with treatment instructions and requirements\n\nMODULE 2\n\nInclusion Criteria:\n\n1. Adult or paediatric patient, 2 years of age or over where Body Surface Area (BSA) is greater than or equal to 0.4m2.\n2. Patient has a clinical diagnosis of a fast-flow vascular malformation\n3. Patient has received standard therapy for the vascular malformation or in which, in the opinion of the investigator, standard therapy is not appropriate\n\n   \\- Note: standard therapy may include treatment with sirolimus. A minimum of 14 days since the last dose of sirolimus is required prior to starting treatment with mirdametinib\n4. A documented genetic alteration in the RAS-MEK-ERK signalling pathway identified by genetic sequencing prior to enrolment in this study\n5. Adequate performance status (Eastern Cooperative Oncology Group Performance Status Scale (ECOG) 0-2 in patients ≥ 16 years of age; Lansky \\> 50 in patients \\\u003C 16 years of age)\n6. Patient has a life expectancy ≥ 12 weeks\n7. Participant has the ability to swallow capsules whole if the capsule dosage form is being utilized. This criterion does not apply if participant is utilizing the dispersible tablet form of study treatment\n8. Adequate haematologic and end-organ function:\n\n   * Haematology: Haemoglobin ≥ 9.0 g\u002FdL; Absolute neutrophil count ≥ 1.5 x 109\u002FL; Platelets ≥ 90 x 109\u002FL, except where bleeding leading to low haemoglobin level is an indication for treatment, in which case haemoglobin \\\u003C 9.0 g\u002FdL is acceptable.\n   * Hepatic function: alanine aminotransferase (ALT) and aspartate aminotransferase (AT) ≤ 2 x upper limit of normal (ULN); Total bilirubin \\\u003C 1.5x ULN (isolated bilirubin \\> 1.5x ULN is acceptable if bilirubin is fractioned and direct bilirubin \\\u003C 35%), except where impaired hepatic function is a consequence of the fast-flow malformation and hence is an indication for treatment, in which case impaired hepatic function is acceptable.\n   * Renal function: Serum creatinine \\\u003C 1.5 x ULN\n   * Biochemistry: Calcium (corrected for serum albumin) and magnesium within normal limits or ≤ Grade 1 according to NCI-CTCAE v5.0 if judged clinically not significant by the investigator; Potassium within normal limits or corrected with supplements; Phosphate ≤ 1x ULN.\n9. Patient agrees to abstinence or highly effective contraceptive measures if of childbearing potential (WOCBP)\n\n   * Males who are sexually active must use a condom during intercourse while taking mirdametinib and for at least 90 days after stopping mirdametinib and should not father a child in this period. A condom is required to be used also by vasectomised men in order to prevent delivery of the drug via seminal fluid. In addition, male participants must not donate sperm during the study and for at least 90 days after stopping mirdametinib\n   * Females who are of child-bearing potential, defined as all individuals physiologically capable of becoming pregnant, must use a highly effective method of contraception during study treatment and for at least 180 days after the last dose of any study treatment. Highly effective contraceptive methods include:\n\n     * Total abstinence (when this is in line with the preferred and usual lifestyle of the subject) Periodic abstinence (eg., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception;\n     * Female sterilisation (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least 6 weeks before taking study treatment. In case of oophorectomy alone, only when the reproductive status of the women has been confirmed by follow up hormone level assessment;\n     * Male partner sterilisation (at least 6 months prior to screening) of the sole partner of a female participant on the study;\n     * Use of oral (estrogen and progesterone), injected or implanted combined hormonal method of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS), or forms of hormonal contraception that have comparable efficacy (failure rate \\\u003C 1%), for example hormonal vaginal ring or transdermal hormone contraception. In the case of use of oral contraception, women should have been stable on the same pill for a minimum of 3 months before taking study treatment.\n   * Women are considered postmenopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhoea with an appropriate clinical profile (i.e., age appropriate, history of vasomotor symptoms) or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy, or bilateral tubal ligation at least 6 weeks before taking study treatment. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered to be not of childbearing potential.\n10. Patient has signed informed consent and is willing and able to comply with the protocol for the duration of the study, including undergoing treatment and scheduled visits and examinations\n\nExclusion Criteria:\n\n1. History of hypersensitivity to any drugs or metabolites of MEK inhibitors or any of the excipients of mirdametinib\n2. Severe infection requiring intravenous antibiotics within 4 weeks prior to enrolment\n3. Patient has had a major surgical procedure within 4 weeks prior to enrolment\n4. Prior use of a MEK inhibitor\n5. Patient has abnormal QT interval corrected by Fridericia's formula (\\> 450 msec for male participants, \\> 470 msec for female participants, or \\> 480 msec for participants with bundle branch block) (triplicate ECG readings taken approximately 2 to 3 minutes apart and averaged) at Screening;\n6. Patient is pregnant or lactating at the time of study registration. A pregnancy test is mandated for persons of child-bearing potential in the screening period\n7. Impairment of GI function or GI disease that may significantly alter the absorption of the study drug based on investigator discretion\n8. Any clinically significant active or known history of liver disease, or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones)\n9. Lymphoma, leukaemia, or any malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years;\n10. Breast cancer within the past 5 years;\n11. Known history of Human Immunodeficiency Virus (HIV) infection (testing for HIV is not mandatory in screening)\n12. Known history of severe cutaneous reactions like Stevens-Johnson Syndrome (SJS), Toxic Epidermal Necrolysis (TEN), Erythema Multiforme (EM), or Drug Reaction with Eosinophilia and Systemic Symptoms (DRESS)\n13. Patient has a history of, or evidence of, retinal pathology on ophthalmologic examination that is considered a risk factor for central serous retinopathy, retinal vein occlusion (RVO), or neovascular macular degeneration. Patients will be excluded from study participation if they have any of the following risk factors for RVO at Screening:\n\n    * Intraocular pressure \\> 21 mmHg;\n    * Serum cholesterol \\> 7.8 mmol\u002FL;\n    * Serum triglycerides \\> 3.4 mmol\u002FL;\n    * Hyperglycaemia (fasting blood glucose \\> 7.0 mol\u002FL );\n    * Age specific hypertension\n\n      * Patients ≥ 13 years of age with a blood pressure ≥ 140\u002F90 mmHg\n      * Patients ≤ 12 years of age with a blood pressure ≥ 95th percentile for age + 12 mmHg\n14. Known history of glaucoma\n15. Known history of clinically significant, uncontrolled heart disease and\u002For recent (within 6 months \\[24 weeks\\] of signing informed consent\u002Fassent) cardiac events including:\n\n    * History of angina pectoris, coronary artery bypass graft (CABG), symptomatic pericarditis, or myocardial infarction within 6 months prior to the start of study treatment;\n    * History of documented congestive heart failure (New York Heart Association functional classification III-IV);\n    * History of clinically significant cardiac arrhythmias, (e.g., ventricular tachycardia), complete left bundle branch block, high grade Atrioventricular (AV) block (e.g. bifascicular block, Mobitz type II and third degree AV block without pacemaker in place);\n    * Uncontrolled hypertension defined by a Systolic Blood Pressure (SBP) ≥ 140 mmHg and\u002For Diastolic Blood Pressure (DBP) ≥ 90 mmHg, with or without anti-hypertensive medication. Initiation or adjustment of antihypertensive medication(s) is allowed prior to screening;\n    * Long QT syndrome, family history of idiopathic sudden death or congenital long QT syndrome, or corrected QT interval \\> 470 msec at screening (using Fridericia correction);\n    * Bradycardia (heart rate \\\u003C 50 beats per minute at rest), by electrocardiogram (ECG) or pulse\n16. Patient has recorded a left ventricular ejection fraction (LVEF) \\\u003C 55% at Screening\n17. Patient has experienced a cerebrovascular accident, transient ischaemic attach, or symptomatic pulmonary embolism within 6 months (24 weeks) of signing informed consent\u002Fassent.\n18. Patient has other concurrent severe and\u002For uncontrolled medical conditions that would, in the Treating Physician's judgement, contraindicate administration of mirdametinib (eg. active or uncontrolled severe infection, chronic active hepatitis, immune-compromised, acute or chronic pancreatitis, uncontrolled high blood pressure)\n19. Patient is unable to understand and comply with treatment instructions and requirements",{"count":112,"type":20},50,[23],"Recent 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) 48 weeks of alpelisib therapy for participants with slow-flow vascular malformations and a gene mutation in one of these signalling pathways (module 1) and (ii) 48 weeks of mirdametinib therapy for participants with fast-flow vascular malformations and a gene mutations in the other signalling pathway (module 2).",[116,117,28,118,119,120,121,122,123,124,125,33,126,127,128,129,130,131],"Slow-Flow Vascular Malformation","Fast-Flow Vascular Malformation","Venous Malformation","Lymphatic Malformation, Low Flow","Lymphatic Malformation","Lymphangioma","Arteriovenous Malformations","Venous Malformation, Low Flow","Cystic Hygroma","Vascular Anomaly","PI3K Gene Mutation","MAP2K1 Gene Mutation","PIK3CA-related Overgrowth Spectrum","Arteriovenous Malformation (AVM)","KRAS G12C","KRAS G12D",[133,62,134,135,136],"Targeted therapy","skin diseases, vascular","Phosphatidylinositol 3-Kinases","MEK inhibitor 1","2026-04-29",{"date":139,"type":38},"2026-05-05",{"date":141,"type":38},"2024-09-13",{"date":143,"type":20},"2027-05",{"name":145,"class":77},"Murdoch Childrens Research Institute",2,{"id":148,"slug":149,"hasResults":11,"nctId":150,"briefTitle":151,"officialTitle":152,"acronym":4,"eligibilityCriteria":153,"healthyVolunteers":11,"sex":16,"minAge":89,"maxAge":4,"enrollmentInfo":154,"targetDuration":4,"studyType":21,"phases":156,"briefSummary":157,"conditions":158,"keywords":4,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":162,"lastUpdatePostDateStruct":163,"startDateStruct":165,"completionDateStruct":167,"leadSponsor":169,"locationsCount":4},"100629659","phase-2-a-study-to-evaluate-the-efficacy-and-safety-of-everolimus-in-patients-with-teratment-refractory-vascular-anomalies-100629659","NCT07477548","A Study to Evaluate the Efficacy and Safety of Everolimus in Patients With Teratment-refractory Vascular Anomalies","Non-randomized, Phase II, Open-label Study for Efficacy and Safety of Everolimus in Relapsed or Refractory Hemangioendothelioma and Other ISSVA Group I or II Vascular Malformation and Neoplasms","Inclusion Criteria:\n\n① Diagnosis per the 2014 ISSVA classification: Group 1 (hemangioendothelioma, tufted angioma): histologically confirmed tumor, OR Kasabach-Merritt Syndrome (histologically confirmed or histologic diagnosis not feasible) Group 2 (vascular tumors not in Group 1, or vascular malformations): histologically confirmed, OR radiologically diagnosed when biopsy is not feasible\n\n* Age ≥1 year ③ Failure of at least one prior therapy (e.g., vincristine, corticosteroids, interferon), stratified as: Cohort 1: sirolimus-naïve Cohort 2: prior sirolimus failure\n\n  * At least one measurable target lesion ≥1 cm in longest diameter per RECIST 1.1 on CT or MRI\n\n    * ECOG Performance Score 0, 1, or 2 ⑥ WOCBP must have a negative pregnancy test prior to enrollment; adequate contraception required during the study and for 8 weeks after completion ⑦ Written informed consent obtained\n\nExclusion Criteria:\n\n* Pregnancy or breastfeeding (WOCBP must use adequate contraception)\n\n  * Documented allergy or hypersensitivity to everolimus\n\n    ③ Inadequate organ function: Bone marrow: ANC \\\u003C1,000\u002FµL or platelets \\\u003C75,000\u002FµL Renal: serum creatinine \\>1.5×ULN; if \\>1.5×ULN, 24-hour creatinine clearance \\\u003C60 mL\u002Fmin Hepatic: total bilirubin \\>1.5×ULN or ALT \\>3.0×ULN\n    * KMP associated with vascular tumors or malformations is not an exclusion criterion, including: thrombocytopenia (\\\u003C100,000\u002FµL), hypofibrinogenemia, anemia (Hb \\\u003C8 g\u002FdL), consumptive coagulopathy, or overlying skin changes (edema, warmth, erythema, purplish\u002Fdark discoloration)\n\n      * Uncontrolled hyperlipidemia (fasting cholesterol \\>300 mg\u002FdL or triglycerides \\>2.5×ULN)\n\n        * Uncontrolled diabetes (fasting glucose \\>1.5×ULN)\n\n          * Active uncontrolled infection\n\n            * Hepatitis B (HBsAg positive) or hepatitis C (anti-HCV positive) ⑨ Known HIV infection (positive serology)\n\n              * Clinically significant symptomatic pulmonary dysfunction; PFTs and room air SpO₂ performed if clinically indicated; exclusion if FEV₁ ≤70% or DLCO ≤70% of predicted (assessed in patients ≥8 years)\n\n                ⑪ Prior solid organ or hematopoietic stem cell transplantation (bone marrow, liver, kidney, lung, or heart)\n\n                ⑫ Concomitant investigational agents (e.g., mTOR inhibitors: sirolimus, temsirolimus)\n\n                ⑬ Concurrent chemotherapy (e.g., mTOR inhibitors: sirolimus, temsirolimus)\n\n                ⑭ Concurrent other malignancy not meeting eligibility criteria",{"count":155,"type":20},67,[23],"Background and Objectives Vascular anomalies are a heterogeneous group of disorders classified into vascular tumors and vascular malformations according to the ISSVA classification. Although most follow a benign course, a subset causes serious complications including organ dysfunction, chronic pain, thrombocytopenia, and hemorrhage. Kaposiform hemangioendothelioma (KHE) complicated by Kasabach-Merritt Phenomenon (KMP) carries a mortality rate of 14-24%. Surgical resection is the primary treatment when organ damage is not anticipated; however, when surgery is not feasible, pharmacologic therapy is considered. Agents such as interferon, corticosteroids, vincristine, cyclophosphamide, and propranolol have been used with variable efficacy, and no established therapy exists for patients refractory to these treatments.\n\nThe PI3K-Akt-mTOR and RAS-MEK-ERK pathways have been identified as key molecular mechanisms underlying vascular anomalies. Targeted therapies against these pathways are emerging, including anti-VEGF antibodies, PI3K\u002FAkt inhibitors (e.g., alpelisib, miransertib), and mTOR inhibitors. Sirolimus has demonstrated clinical benefit in 50-80% of patients with vascular anomalies, with a 96% symptom response rate in KMP-associated vascular tumors. Everolimus, another mTOR inhibitor, is already approved and established for tuberous sclerosis-associated angiomyolipoma and SEGA in pediatric patients, with a well-characterized safety profile. Given its shared mechanism with sirolimus and emerging case reports supporting efficacy in KHE with KMP, this phase 2 study aims to evaluate the efficacy and safety of everolimus in patients with treatment-refractory vascular anomalies.\n\nStudy Design This is a single-center, open-label, uncontrolled phase 2 clinical trial enrolling 67 patients over 60 months from IRB approval, stratified into two cohorts: Cohort 1 (sirolimus-naïve, n=39) and Cohort 2 (prior sirolimus failure, n=28). Everolimus is administered orally at age- and CYP3A4\u002FP-gp inducer-adjusted doses, with maintenance dosing titrated to a target trough level of 5-15 ng\u002FmL. The primary endpoint is overall response rate (ORR) at 6 months. Secondary endpoints include toxicity per NCI CTCAE v4.0, ORR at 12 months, platelet recovery rate at 4 weeks (KMP patients), 1-year overall survival, and 3-year progression-free survival.",[28,122,118,121,159,160,161],"Nevus, Port-Wine","Hemangioendothelioma","Hemangioma","2026-03-12",{"date":164,"type":38},"2026-03-17",{"date":166,"type":20},"2026-04-01",{"date":168,"type":20},"2030-11-30",{"name":170,"class":77},"Yonsei University",{"id":172,"slug":173,"hasResults":11,"nctId":174,"briefTitle":175,"officialTitle":176,"acronym":4,"eligibilityCriteria":177,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":178,"targetDuration":4,"studyType":21,"phases":180,"briefSummary":182,"conditions":183,"keywords":186,"overallStatus":67,"whyStopped":4,"lastUpdateSubmitDate":188,"lastUpdatePostDateStruct":189,"startDateStruct":191,"completionDateStruct":193,"leadSponsor":195,"locationsCount":4},"100624056","microwave-ablation-for-treatment-of-vascular-malformations-efficacy-and-safety-100624056","NCT07404670","Microwave Ablation for Treatment of Vascular Malformations: Efficacy and Safety","A Multicenter, Prospective Study to Analyze the Efficacy and Safety of Microwave Ablation for the Treatment of Vascular Malformations","Inclusion Criteria:\n\n1. Patients with clinically diagnosed vascular malformations (venous malformations (VM) or arteriovenous malformations (AVM)) confirmed by MRI or ultrasound.\n2. Presence of symptomatic lesions (pain, swelling, functional impairment, or ulceration) that require treatment.\n3. Lesions that are amenable to microwave ablation based on preoperative clinical and imaging assessment.\n4. Ability to provide informed consent or parental consent for minors (if applicable).\n\nExclusion Criteria:\n\n1. Capillary malformations or congenital arteriovenous fistula.\n2. Lesions that are too close to critical structures (e.g., major blood vessels or nerves) and cannot be safely treated.\n3. Pregnancy or breastfeeding.\n4. Severe coagulopathy or significant bleeding disorders.\n5. Active infection at the treatment site.\n6. Patients with severe uncontrolled systemic disease that could interfere with treatment or recovery.\n7. Recent treatments (e.g., surgery, sclerotherapy, embolization) for the same lesion within the last 6 months.",{"count":179,"type":20},150,[181],"NA","This clinical trial aims to assess the safety and efficacy of microwave ablation in treating vascular malformations, including both venous malformations (VM) and arteriovenous malformations (AVM). Vascular malformations are abnormal clusters of blood vessels that can cause pain, swelling, and functional impairment, significantly affecting a patient's quality of life. Microwave ablation is a minimally invasive treatment that uses heat to shrink abnormal vessels, but its effectiveness and safety for these conditions need further investigation.\n\nThe trial will enroll 150 patients (100 with venous malformations and 50 with arteriovenous malformations), all of whom will undergo a single session of microwave ablation. Ultrasound guidance will be used during the procedure to precisely target the lesions, while MRI will be used for both preoperative and postoperative evaluations to assess lesion size and track changes over time.\n\nThe primary goals of the study are to determine whether microwave ablation can reduce lesion size and improve symptoms such as pain and swelling. Additionally, the study will monitor adverse events to evaluate the safety of the procedure, including any potential complications like infection, bleeding, or nerve injury.\n\nPatients will be followed for 12 months, with MRI scans taken at 1 month, 3 months, 6 months, and 12 months after the procedure to evaluate lesion shrinkage and monitor for any recurrence. Clinical symptoms will also be assessed at these time points to track improvement.\n\nThis study could provide important data on the safety and efficacy of microwave ablation, potentially offering a less invasive treatment option for patients with vascular malformations.",[184,122,185,28],"Venous Malformations","Microwave Ablation",[185,28,184,122,187],"Minimally Invasive Treatment","2026-02-05",{"date":190,"type":38},"2026-02-11",{"date":192,"type":20},"2026-03-01",{"date":194,"type":20},"2028-03-01",{"name":196,"class":77},"Chengdu University of Traditional Chinese Medicine",{"id":198,"slug":199,"hasResults":11,"nctId":200,"briefTitle":201,"officialTitle":201,"acronym":4,"eligibilityCriteria":202,"healthyVolunteers":11,"sex":16,"minAge":54,"maxAge":203,"enrollmentInfo":204,"targetDuration":4,"studyType":21,"phases":206,"briefSummary":207,"conditions":208,"keywords":209,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":213,"lastUpdatePostDateStruct":214,"startDateStruct":216,"completionDateStruct":218,"leadSponsor":220,"locationsCount":102},"100595806","phase-2-an-open-label-single-arm-exploratory-clinical-study-of-everolimus-for-the-treatment-of-vascular-malformations-100595806","NCT07037238","An Open-Label, Single-Arm Exploratory Clinical Study of Everolimus for the Treatment of Vascular Malformations","Inclusion Criteria\n\n* Patients aged ≥18 and ≤65 years;\n* No gender restrictions;\n* Diagnosed with vascular malformation by MRI;\n* No major surgery within the past 3 months;\n* Able to swallow and retain oral medication, with no significant gastrointestinal abnormalities that may affect drug absorption, such as malabsorption syndrome, intestinal obstruction, or extensive gastrointestinal resection;\n* Able to provide peripheral blood samples for biomarker testing at a central laboratory;\n* Patients must have adequate organ and bone marrow function, and must not have received blood transfusions or any supportive treatments (e.g., cytokines or erythropoietin) to increase white blood cells, platelets, or hemoglobin levels within 7 days before screening tests:Absolute neutrophil count ≥1.0×10⁹\u002FL;Hemoglobin ≥90 g\u002FL; Platelets ≥100×10⁹\u002FL;Total bilirubin ≤1.5× upper limit of normal (ULN), or ≤3.0× ULN for patients with Gilbert's syndrome;AST and ALT ≤2.5× ULN;Albumin ≥3 g\u002FdL; Serum creatinine \\\u003C1.5× ULN or creatinine clearance ≥50 mL\u002Fmin;Urine protein \\\u003C2+; if ≥2+, then 24-hour urine protein must be ≤1 g;Coagulation: International normalized ratio (INR) and activated partial thromboplastin time (APTT) ≤1.5× ULN.\n* Patients must voluntarily sign the written informed consent form and be able to complete follow-up;\n* For patients of childbearing potential: they must agree to use highly effective contraceptive methods, such as combined hormonal contraception, progestogen-only hormonal contraception associated with ovulation inhibition, intrauterine devices (IUDs), intrauterine hormonal systems (IUS), bilateral tubal occlusion, or partner vasectomy, or to practice sexual abstinence during the treatment period and for at least 90 days after the last dose. Male patients must agree to refrain from sperm donation for at least 90 days after the last dose.\n\nExclusion Criteria:\n\n* Diagnosed with Hereditary Hemorrhagic Telangiectasia (HHT), Arteriovenous Malformation (AVM), or PTEN Hamartoma Tumor Syndrome (PHTS);\n* Patients who have previously received any of the following treatments after birth: participation in other interventional clinical trials targeting cerebral cavernous malformations (CM);\n* Presence of malignant tumors currently or within the past three years, except for curatively treated non-melanoma skin basal cell carcinoma, ductal carcinoma in situ of the breast, or cervical carcinoma in situ;\n* Unable to undergo MRI scans and\u002For have contraindications for MRI (e.g., interference from prosthetics, orthodontic devices, etc., affecting target lesion volume analysis on MRI);\n* Modified Rankin Scale (mRS) score of 5, respiratory failure, or currently experiencing severe bleeding requiring life-support treatment;\n* Severe renal failure (e.g., creatinine clearance \\\\\\[CrCl\\] \\\u003C 30 mL\u002Fmin, or significantly elevated serum creatinine not correctable by other means), recent history (within past 3 months) of renal failure or end-stage renal disease without effective treatment, or currently undergoing dialysis;\n* Severe hepatic failure, including but not limited to: Child-Pugh Class C or higher, recent (within 3 months) uncontrolled symptoms related to hepatic failure such as ascites, jaundice, coagulopathy, or hepatic encephalopathy, or patients requiring liver transplantation;\n* Currently using other immunosuppressants or patients with immunodeficiency;\n* Patients requiring use of medications that interfere with or inhibit CYP3A4 enzyme activity, or medications such as cisapride or metoclopramide;\n* Patients with dysphagia, active gastrointestinal disorders, malabsorption syndrome, or other conditions that may affect the absorption of the investigational drug;\n* Interstitial pneumonitis, including clinically significant radiation pneumonitis;\n* Severe asthma;\n* Uncontrolled diabetes mellitus;\n* Active drug or alcohol use or dependence that, in the opinion of the investigator, would interfere with adherence to study requirements;\n* First-degree relatives with a history of sudden cardiac death before the age of 50. First-degree relatives are defined as those with a direct bloodline, such as parents and children, grandparents and grandchildren, or maternal grandparents and maternal grandchildren;\n* Active bacterial, fungal, or viral infections, including active hepatitis B (HBsAg positive with HBV DNA \\> 1000 IU\u002FmL or meeting local diagnostic criteria for active HBV infection), hepatitis C (HCV RNA positive), or HIV infection (HIV positive);\n* Pregnant or breastfeeding women. Any patient who becomes pregnant during the trial must withdraw from the study;\n* Known hypersensitivity to everolimus, other rapamycin derivatives, or any of the excipients in this product. Observed allergic reactions to everolimus or related compounds include but are not limited to: hypersensitivity, dyspnea, flushing, chest pain, or angioedema (e.g., airway or tongue swelling with or without respiratory compromise);\n* Other factors, as determined by the investigator, that may lead to early study termination, such as presence of other severe diseases (including psychiatric disorders) requiring concomitant treatment, significantly abnormal lab values, or social\u002Ffamily issues that may affect patient safety or data collection.","65 Years",{"count":205,"type":20},10,[23],"This study is a single-arm exploratory trial conducted by Xuanwu Hospital, Capital Medical University, aiming to evaluate the efficacy and safety of everolimus monotherapy in adult patients with vascular malformations.",[28],[210,211,212],"Vascular malformations","mTOR Inhibitors","Pharmacological Treatment","2025-09-02",{"date":215,"type":38},"2025-09-05",{"date":217,"type":38},"2025-07-01",{"date":219,"type":20},"2026-12-31",{"name":221,"class":77},"Xuanwu Hospital, Beijing",{"id":223,"slug":224,"hasResults":11,"nctId":225,"briefTitle":226,"officialTitle":226,"acronym":4,"eligibilityCriteria":227,"healthyVolunteers":11,"sex":16,"minAge":228,"maxAge":229,"enrollmentInfo":230,"targetDuration":89,"studyType":90,"phases":4,"briefSummary":232,"conditions":233,"keywords":235,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":238,"lastUpdatePostDateStruct":239,"startDateStruct":241,"completionDateStruct":243,"leadSponsor":245,"locationsCount":102},"100370524","patient-reported-outcomes-for-vascular-malformations-embolization-proven-100370524","NCT04104464","Patient Reported Outcomes for Vascular Malformations EmbolizatioN (PROVEN)","Inclusion Criteria:\n\n* Male and Female pediatric patients, aged between 0-17 with diagnosis of vascular malformations.\n* Male and Female adult patients aged 18-100 with diagnosis of vascular malformations.\n* Vascular malformation symptoms significant enough to seek treatment.\n\nExclusion Criteria:\n\n* Patients with extensive VM not suitable for sclerotherapy.\n* Prior therapy for treatment of a VM within 3 months.\n* Condition or impairment that may render the patient unable to take part in the study (e.g. cognitive, sight, hearing, etc.).","0 Years","100 Years",{"count":231,"type":20},200,"The purpose of this study is to develop a standardized assessment for patients treated for venous malformations (VM).\n\nVenous malformations result from the abnormal development of veins which may result in pain, swelling, bleeding, functional impairment, disfigurement, and psychological distress. The impact of VM on patient quality of life varies based on the location and size of the malformation.\n\nA patient reported outcome (PRO) is a patient's own account of patient's health condition. PRO measures are valued to clinicians, as many treatment effects are known only to the patient. No studies to date have analyzed the validity of existing PRO measures for VM patients.\n\nCurrent assessment does not include all symptoms or take in to account the relevance of VM location. Past studies show a discrepancy between treatment outcomes reported by patients and physicians. Therefore, there is a need to develop VM-specific PROs to better understand the effectiveness and benefits of treatment for VM.",[28,234],"VM - Vascular Malformation",[236,237],"vascular malformation","VM","2025-08-14",{"date":240,"type":38},"2025-08-19",{"date":242,"type":38},"2019-07-22",{"date":244,"type":20},"2026-07",{"name":246,"class":77},"Johns Hopkins University",{"id":248,"slug":249,"hasResults":11,"nctId":250,"briefTitle":251,"officialTitle":252,"acronym":4,"eligibilityCriteria":253,"healthyVolunteers":11,"sex":16,"minAge":254,"maxAge":255,"enrollmentInfo":256,"targetDuration":4,"studyType":21,"phases":258,"briefSummary":260,"conditions":261,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":263,"startDateStruct":265,"completionDateStruct":267,"leadSponsor":269,"locationsCount":271},"100258062","phase-3-efficacy-and-safety-of-sirolimus-in-vascular-anomalies-that-are-refractory-to-standard-care-100258062","NCT02638389","Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care","Phase III Multicentric Study Evaluating the Efficacy and Safety of Sirolimus in Vascular Anomalies That Are Refractory to Standard Care","Inclusion Criteria:\n\n* Patients with complex vascular anomalies that are refractory to standard care such as medical treatment, surgical resection and\u002For sclerotherapy\u002Fembolization (ineffective or accompanied by major complications)\n* Patients must have adequate medullary function: Hemoglobine\\> 10,0 g\u002Fdl, neutrophils \\>1500\u002Fmm³ and platelets \\> 100.000\u002Fmm³\n* Patients must have the following laboratory values:\n* Total serum bilirubin ≤ 1.5 x ULN (or totally bilirubin ≤ 3 x ULN with direct bilirubin ≤ 1.5 x ULN in patients with well documented Gilbert Syndrome)\n* Serum alanine aminotransferase (ALT) and aspartate aminotransferase (AST) ≤ 3 x ULN (or \\\u003C 5.0 x ULN if hepatic metastases are present)\n* Serum creatinine 1.5 x ULN. If the serum creatinine is ≥ 1.5 x ULN, then a 24-hour Creatinine Clearance must be conducted and the result must be ≥ 60 mL\u002Fmin.\n* Karnofsky \\> 50\n* Patients have to be able to sign the informed consent\n* Women in age of procreation have to be informed that contraceptive methods are mandatory during the study time\n\nExclusion Criteria:\n\n* Any of the following concurrent severe and\u002For uncontrolled medical conditions, which could compromise participation in the study or interfere with the study results:\n* Impaired cardiac function or clinically significant cardiac diseases, including unstable angina pectoris, ventricular arrhythmia, valvular disease with documented compromise in cardiac function, myocardial infarction within the last 6 months, documented by persistent elevated cardiac enzymes or persistent regional wall abnormalities on assessment of LVEF function, history of documented congestive heart failure (New York Heart Association functional classification III-IV), documented cardiomyopathy, family history of congenital long or short QT, or known history of QT\u002FQTc prolongation of Torsades de Pointes (TdP)\n* Impairment of Gastro-Intestinal (GI) function or GI disease that may significantly alter the absorption of sirolimus (e.g., ulcerative diseases, uncontrolled nausea, vomiting, diarrhea ≥ Grade 2, malabsorption syndrome, or small bowel resection)\n* Known hypersensitivity to drugs or metabolites from similar classes as study treatment.\n* Patient has other concurrent severe and \u002For 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\u002For O2 saturation ≤ 88% at rest, etc.)\n* Immunocompromised patients, including known seropositivity for HIV\n* Pregnant or lactating women\n* Prior treatment with PI3K and\u002For mTOR inhibitors","3 Months","70 Years",{"count":257,"type":20},250,[259],"PHASE3","The phosphatidylinositol 3-kinase (PI3Kinase)\u002FProtein Kinase B (AKT)\u002Fmammalian target of rapamycin (mTor) pathway plays a role on the development and the venous\u002Flymphatic vascular organisations.\n\nThe investigators want to study the efficacy and the safety of Rapamycin, an mTor inhibitor.",[28],"2023-02-23",{"date":264,"type":38},"2023-02-24",{"date":266,"type":38},"2016-01-25",{"date":268,"type":20},"2030-04-01",{"name":270,"class":77},"Cliniques universitaires Saint-Luc- Université Catholique de Louvain",3]