[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"lymphatic-malformation\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:lymphatic-malformation":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,10,0,[8,45,72,117,150,171,200,227,252,278],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100304425","phase-2-efficacy-of-rapamycin-in-the-treatment-of-cervico-facial-lymphatic-malformations-100304425",false,"NCT03243019","Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations","Evaluation of the Efficacy of Rapamycin in the Treatment of Cervico-facial Lymphatic Malformations of Poor Prognosis","RAPAMALYMPH","Inclusion Criteria:\n\n* Patient from 0 to 18 years of age, presenting with poly-cystic suprahyoid or mediastinal lymphatic.\n* with chronic pain or functional respiratory or swallowing impairment with a CDS score \\\u003C 8\n* Curative treatment is not possible or associated with a high risk of morbidity ,mortality and functional and cosmetic impairment\n* Karnofsky Score (\\> 10 years of age) or Lansky score (≤10 years of age) \\> 50%\n* Biology\n\n  * Neutrophils count≥1.0 x 109\u002FL\n  * Platelets count ≥ 100 x 109\u002FL\n  * Hemoglobin ≥ 8 g\u002FdL\n  * Bilirubin ≤ 1,5 ULN\n  * Transaminases \\\u003C 2,5 ULN\n  * Serum albumin ≥ 2 g\u002FdL.\n  * LDL cholesterol \\\u003C160 mg\u002FdL\n  * Triglycerides \\\u003C 150 mg\u002FdL\n* Negative test of pregnancy if relevant\n* Social security affiliation\n* At least 2 months after a previous procedure on the malformation\n\nExclusion Criteria:\n\n* Non-respect of inclusion criteria\n* Other immunosuppressive therapy or long-term general corticosteroid therapy without a 28-day washout period\n* renal failure\n* Liver failure\n* Digestive disease leading to rapamycin malabsorption\n* uncontrolled or severe infectious disease\n* Patients requiring treatment interfering with CYP3A4 isoenzyme (rifampicin, rifabutin, carbamazepine, phenobarbital, phenytoin) or inhibiting CYP3A4 isoenzyme's activity (ketoconazole, voriconazole, itraconazole, telithromycin, clarithromycin, Diltiazem, Verapamil, nicardipine, clotrimazole, fluconazole , troleandomycin, bromocriptine, cimetidine, danazol, protease inhibitors) -patients requiring treatment by cisapride and metoclopramide\n* Concomitant administration of mTOR inhibitor\n* Peanuts or soya allergy\n* Impossibility to receive informed consent\n* Absence of social security affiliation\n* refusal to sign consent\n* Ongoing pregnancy or breastfeeding\n* refusal to participate","ALL","18 Years",{"count":20,"type":21},28,"ESTIMATED","INTERVENTIONAL",[24],"PHASE2","To evaluate the efficacy of Rapamycin in extended cervicofacial lymphatic malformations in pediatric patients. Rapamycin is administered oral for a 6 month period.\n\nThe success rate is determined by volume reduction superior to 1\u002F5e of the initial volume measured by MRI, impact on QOL and reduction of bleeding in case of mucosal involvement.",[27,28],"Lymphatic Malformation","Pediatric",[30,31],"cervico-facial","rapamycin","RECRUITING","2026-05-20",{"date":35,"type":36},"2026-05-22","ACTUAL",{"date":38,"type":36},"2018-06-25",{"date":40,"type":21},"2027-02",{"name":42,"class":43},"University Hospital, Lille","OTHER",2,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":17,"minAge":53,"maxAge":18,"enrollmentInfo":54,"targetDuration":4,"studyType":22,"phases":56,"briefSummary":57,"conditions":58,"keywords":59,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":62,"lastUpdatePostDateStruct":63,"startDateStruct":65,"completionDateStruct":67,"leadSponsor":69,"locationsCount":5},"100506252","phase-2-safety-and-efficacy-study-of-intracystic-tara-002-for-the-treatment-of-lymphatic-malformations-in-participants-6-months-to-less-than-18-years-of-age-100506252","NCT05871970","Safety and Efficacy Study of Intracystic TARA-002 for the Treatment of Lymphatic Malformations in Participants 6 Months to Less Than 18 Years of Age","A Phase 2a\u002Fb Single Arm Open Label Study to Evaluate the Safety and Efficacy of Intracystic Administration of TARA-002 in Participants Between 6 Months to Less Than 18 Years of Age for the Treatment of Macrocystic and Mixed Cystic Lymphatic Malformations","STARBORN-1","Inclusion Criteria:\n\n* Male or female participants 6 months to less than 18 years of age at the time of informed consent\u002Fassent form was signed\n* Participants whose parent\u002FLAR(s) have voluntarily given written consent and participants who provided assent (if applicable) after the study has been explained to them\n* Participants with macrocystic LM or mixed cystic LM (≥ 50% macrocystic disease measured by volume) of the Head\u002FNeck\u002FMediastinum according to the ISSVA 2018 criteria (ISSVA 2018) measured via LM imaging at Screening to confirm, upon central review, the diagnosis of macrocystic or mixed cystic LM\n* Participants who may have had surgical or sclerotherapy treatment for their LM, but not within six months of the consent\u002Fassent form being signed\n\nExclusion Criteria:\n\n* Penicillin allergy\n* Vascular tumors or combined vascular malformations\n* Microcystic LM or mixed cystic LM with predominant microcystic features\n* LMs of the orbit (orbital LM) as target cyst\n\nFor more information on eligibility criteria, please contact the sponsor.","6 Months",{"count":55,"type":21},38,[24],"This is a Phase 2a\u002Fb single arm open label study to evaluate the safety, reactogenicity, and efficacy of intracystic injection of TARA-002 in participants 6 months to less than 18 years of age for the treatment of macrocystic and mixed cystic lymphatic malformations. The Phase 2a safety lead-in, age de-escalation study is designed to establish the safety of TARA-002 in older participants 6 years to less than 18 years before proceeding to younger participants 2 years to less than 6 years, then 6 months to less than 2 years. The Phase 2b is an expansion study in which enrollment of participants will be initiated after safety has been established in each cohort during the Phase 2a safety lead-in study. Each participant will receive up to 4 injections of TARA-002 spaced approximately 6 weeks apart.",[27],[60,61],"Macrocystic lymphatic malformations","Mixed-cystic lymphatic malformations","2026-05-14",{"date":64,"type":36},"2026-05-18",{"date":66,"type":36},"2023-10-18",{"date":68,"type":21},"2027-05",{"name":70,"class":71},"Protara Therapeutics","INDUSTRY",{"id":73,"slug":74,"hasResults":11,"nctId":75,"briefTitle":76,"officialTitle":77,"acronym":78,"eligibilityCriteria":79,"healthyVolunteers":11,"sex":17,"minAge":80,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":22,"phases":83,"briefSummary":84,"conditions":85,"keywords":103,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":112,"completionDateStruct":114,"leadSponsor":115,"locationsCount":44},"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","2 Years",{"count":82,"type":21},50,[24],"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).",[86,87,88,89,90,27,91,92,93,94,95,96,97,98,99,100,101,102],"Slow-Flow Vascular Malformation","Fast-Flow Vascular Malformation","Vascular Malformations","Venous Malformation","Lymphatic Malformation, Low Flow","Lymphangioma","Arteriovenous Malformations","Venous Malformation, Low Flow","Cystic Hygroma","Vascular Anomaly","Vascular Anomalies","PI3K Gene Mutation","MAP2K1 Gene Mutation","PIK3CA-related Overgrowth Spectrum","Arteriovenous Malformation (AVM)","KRAS G12C","KRAS G12D",[104,105,106,107,108],"Targeted therapy","vascular malformations","skin diseases, vascular","Phosphatidylinositol 3-Kinases","MEK inhibitor 1","2026-04-29",{"date":111,"type":36},"2026-05-05",{"date":113,"type":36},"2024-09-13",{"date":68,"type":21},{"name":116,"class":43},"Murdoch Childrens Research Institute",{"id":118,"slug":119,"hasResults":11,"nctId":120,"briefTitle":121,"officialTitle":121,"acronym":4,"eligibilityCriteria":122,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":123,"targetDuration":125,"studyType":126,"phases":4,"briefSummary":127,"conditions":128,"keywords":138,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":140,"lastUpdatePostDateStruct":141,"startDateStruct":143,"completionDateStruct":145,"leadSponsor":147,"locationsCount":149},"100239778","lymphatic-anomalies-registry-for-the-assessment-of-outcome-data-100239778","NCT02399527","Lymphatic Anomalies Registry for the Assessment of Outcome Data","Inclusion Criteria:\n\n* Clinical diagnosis of complex vascular tumor, malformation or overgrowth syndrome with significant lymphatic component",{"count":124,"type":21},1000,"15 Years","OBSERVATIONAL","Lymphatic anomalies are a rare subset of vascular anomalies that are poorly understood. the understanding of the natural history, long-term outcomes, risk factors for morbidity and mortality, and the relative benefit of medical therapies and procedures is limited.The goal of this project is to better understand these diseases and improve the care of theses rare patients. To do this, the investigators are conducting an observational study of patients with lymphatic anomalies, including an annual follow-up questionnaire to gather prospective data on mortality, morbidity, treatments, and functionality as well as quality of life.",[27,129,130,131,132,133,134,135,136,137],"Generalized Lymphatic Anomaly (GLA)","Central Conducting Lymphatic Anomaly","CLOVES Syndrome","Gorham-Stout Disease (\"Disappearing Bone Disease\")","Blue Rubber Bleb Nevus Syndrome","Kaposiform Lymphangiomatosis","Kaposiform Hemangioendothelioma\u002FTufted Angioma","Klippel-Trenaunay Syndrome","Lymphangiomatosis",[96,139,27],"Lymphatic Anomalies","2026-04-21",{"date":142,"type":36},"2026-04-24",{"date":144,"type":36},"2013-06",{"date":146,"type":21},"2035-06",{"name":148,"class":43},"Boston Children's Hospital",1,{"id":151,"slug":152,"hasResults":11,"nctId":153,"briefTitle":154,"officialTitle":155,"acronym":4,"eligibilityCriteria":156,"healthyVolunteers":11,"sex":17,"minAge":80,"maxAge":4,"enrollmentInfo":157,"targetDuration":4,"studyType":22,"phases":159,"briefSummary":160,"conditions":161,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":162,"lastUpdatePostDateStruct":163,"startDateStruct":165,"completionDateStruct":167,"leadSponsor":169,"locationsCount":149},"100428592","phase-2-weekly-sirolimus-therapy-100428592","NCT04861064","Weekly Sirolimus Therapy","Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations","Inclusion Criteria:\n\n* Patient 2 years of age and older\n* Venous, lymphatic, or venolymphatic malformations\n\nExclusion Criteria:\n\n* Children with contraindication to use of sirolimus\n* Children with history of transplant\n* Children with a history of natural immunodeficiency\n* Children with a history of artificially induced immunodeficiency\n* Children with a history of a serious or life-threatening infection\n* Children taking CYP3A4 inhibiting medications\n* Children taking strong CYP3A4 inducers to avoid subtherapeutic dosing\u002Fexposure.\n* Inability or unwillingness of subject or legal guardian\u002Frepresentative to give informed consent\n* Women that are or may become pregnant o Sirolimus is a Pregnancy Category C drug. No randomized controlled studies have been done on pregnant women. Women of childbearing potential must be on effective contraception prior to, during, and for 12 weeks following sirolimus therapy.",{"count":158,"type":21},24,[24],"In current practice, options for venous and lymphatic malformations remain limited. Recently an oral medication, sirolimus, has been found to benefit patients when taken once or twice a day for several months. Unfortunately there are many side effects associated with this medication, some of which can be severe including, neutropenia, oral ulcerations, and lab abnormalities. The purpose of this study is to determine if once weekly dosed sirolimus will be effective for the treatment of venous and lymphatic malformations. Additionally, the study will evaluate patient satisfaction and identify adverse effects. Participants will be on the medication for 6 months with an option to continue after this time period.",[89,27],"2026-03-03",{"date":164,"type":36},"2026-03-05",{"date":166,"type":36},"2022-01-18",{"date":168,"type":21},"2027-12",{"name":170,"class":43},"Medical University of South Carolina",{"id":172,"slug":173,"hasResults":11,"nctId":174,"briefTitle":175,"officialTitle":176,"acronym":4,"eligibilityCriteria":177,"healthyVolunteers":11,"sex":17,"minAge":178,"maxAge":179,"enrollmentInfo":180,"targetDuration":4,"studyType":22,"phases":182,"briefSummary":184,"conditions":185,"keywords":186,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":191,"lastUpdatePostDateStruct":192,"startDateStruct":194,"completionDateStruct":196,"leadSponsor":198,"locationsCount":149},"100537222","a-prospective-study-on-the-treatment-of-clm-based-on-icg-imaging-100537222","NCT06275022","A Prospective Study on the Treatment of cLM Based on ICG Imaging","A Prospective Randomized Controlled Study on the Treatment of Cystic Lymphatic Malformation Based on Indocyanine Green-fluorescence Imaging","Inclusion Criteria:\n\n(1) no previous intervention; (2) cLM diagnosed by pretreatment magnetic resonance imaging (MRI); (3)3 to 6 months post-treatment follow-up; (4) Superfacial cLM\n\nExclusion Criteria:\n\n(1) history of iodine allergy; (2) syndromic cLM ; (3) severe liver and kidney dysfunction; (4) intralesional hemorrhage; (5) intralesional infection","30 Days","16 Years",{"count":181,"type":21},110,[183],"NA","The goal of this prospective randomized controlled study is to explore the role of indocyanine green-fluorescence imaging in management of cystic lymphatic malformation.. To clarify the application value of indocyanine green-fluorescence imaging in both diagnosis and treatment of cystic lymphatic malformation (cLM) in children, is helpful for exploring pathogenesis of cLM, and providing a clearer scientific basis for subsequent surgical intervention. It also provides alternative for the future diagnosis and treatment of cLM.\n\nParticipants will receive indocyanine green-fluorescence imaging before operation, while the patients in control group will receive traditional operation.\n\nResearchers will compare difference in curative effect between two groups.",[27],[187,188,189,190],"indocyanine green","children","inflow","lymphography","2026-02-15",{"date":193,"type":36},"2026-02-18",{"date":195,"type":36},"2023-01-01",{"date":197,"type":21},"2026-03-30",{"name":199,"class":43},"Nanjing Children's Hospital",{"id":201,"slug":202,"hasResults":11,"nctId":203,"briefTitle":204,"officialTitle":205,"acronym":4,"eligibilityCriteria":206,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":207,"targetDuration":4,"studyType":126,"phases":4,"briefSummary":209,"conditions":210,"keywords":214,"overallStatus":217,"whyStopped":4,"lastUpdateSubmitDate":218,"lastUpdatePostDateStruct":219,"startDateStruct":221,"completionDateStruct":223,"leadSponsor":225,"locationsCount":149},"100592502","diagnostic-imaging-of-vascular-malformations-using-msot-and-ulm-100592502","NCT06994260","Diagnostic Imaging of Vascular Malformations Using MSOT and ULM","Multispectral Optoacoustic Tomography (MSOT) and Ultrasound Localization Microscopy (ULM) as Diagnostic Imaging for Lymphatic, Venous and Arteriovenous Malformations","Inclusion Criteria:\n\n* Confirmed vascular malformations (arteriovenous, venous, or lymphatic).\n* ≥18 years old and able to give their consent\n\nExclusion Criteria:\n\n* No imaging for diagnostic confirmation has been performed or is planned.\n* Lack of written consent\n* \\\u003C18 years old\n* Safety concerns of the study physician (a patient with physical, psychological, or psychiatric conditions that, in the opinion of the study physician, could compromise the patient's safety or the quality of the data, thereby making the patient an unsuitable candidate for the study).",{"count":208,"type":21},15,"This clinical study evaluates the efficacy and accuracy of Multispectral Optoacoustic Tomography (MSOT) and Ultrasound Localization Microscopy (ULM) for imaging and diagnosing vascular malformations (venous, arteriovenous, lymphatic). The study aims to enhance diagnostic precision and improve treatment planning through advanced non-invasive imaging techniques.",[211,212,27,213],"Vascular Malformation","Arteriovenous Malformation","Venous Malformations",[211,89,212,27,215,216],"MSOT","ULM","NOT_YET_RECRUITING","2025-06-20",{"date":220,"type":36},"2025-06-22",{"date":222,"type":21},"2025-08-01",{"date":224,"type":21},"2026-06-01",{"name":226,"class":43},"University Hospital Erlangen",{"id":228,"slug":229,"hasResults":11,"nctId":230,"briefTitle":231,"officialTitle":232,"acronym":233,"eligibilityCriteria":234,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":235,"enrollmentInfo":236,"targetDuration":4,"studyType":22,"phases":238,"briefSummary":239,"conditions":240,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":243,"lastUpdatePostDateStruct":244,"startDateStruct":246,"completionDateStruct":248,"leadSponsor":250,"locationsCount":149},"100584716","institution-of-an-italian-registry-and-biobank-for-biological-sample-collection-100584716","NCT06892964","Institution of an Italian Registry and Biobank for Biological Sample Collection","Prospective and Retrospective Clinical Study of Lymphatic Malformations: Institution of an Italian REGistry of Lymphatic Malformations and of a Biobank for Biological Sample Collection","IReg-LM","Inclusion Criteria:\n\n1. Age 0-100 years\n2. Signed informed consent.\n3. Individuals with any type of ML (isolated, syndromic, congenital or acquired, with or without molecular confirmation) identified by participating centers\n\nExclusion Criteria:\n\n* Patients whose clinical picture is not compatible with points a) (b) and (c) of the inclusion criteria, as assessed by the Investigator.","100 Years",{"count":237,"type":21},70,[183],"Lymphatic malformations (ML), are benign non-neoplastic, rare, resulting from an embryologic abnormal development of the lymphatic system. Sometimes they may be associated with other vascular malformations (venous or arterial)1,2. ML usually appear at birth, in early childhood or during the first years of life (congenital vs. acquired) and are mainly localized in the region of the head and neck, armpits, groin, retroperitoneal tissues, tongue and mucous membranes of the oral cavity since these areas contain a plethora of lymphatic structures1. The main complications due to their location are airway obstruction, difficulty in eating, and bleeding3-5. Infection and bleeding can promote the sudden, progressive and accelerated growth of these lesions1. The location, speed of growth, and the subsequent complications associated with ML, determine the overall severity of the clinical picture and require generally the referral of the affected patient to a specialized center that can ensure a multidisciplinary care3,5. Recently, the International Society for the Study of Vascular Anomalies (ISSVA), has revised and updated the classification of such malformations, emphasizing the distinction between isolated forms and ML in the context of more complex syndromes with multisystem involvement2. Until a few years ago, the only treatment strategies available for ML were scleroembolization, cryotherapy, transcutaneous laser photocoagulation, and surgical resection of the malformation. The recent identification of genetic alterations in the phosphatidylinositol-3-kinase (PI3K)\u002Fprotein kinase B (AKT)\u002Ftarget mammalian pathway of rapamycin (mTOR), which underlies many isolated and syndromic ML pictures6-9, has opened up important prospects for personalized treatment with repurposed drugs6,7,10-20. Over the years, the rarity and complexity of ML management have resulted in a fragmented nature of available information and poor nationwide sharing of diagnostic-clinical-assistance-therapeutic protocols (PDTAs) with the consequent need for many families to undertake multispecialty consultations in various provinces or regions before identifying a suitable Referral Center. In addition, recent acquisitions in genetics have forced specialists, a further reevaluation of those complex clinical pictures of ML, whether isolated or syndromic, that could be candidates for personalized drug treatments.\n\nThe institution of a national Registry of pathology promoted by the Association of Patients with Lymphatic Malformations, which supports clinicians and families in filling unmet information and clinical care gaps, is a priority project in order to improve the quality of life of patients and the level of care offered to them. In addition, the establishment of a collection of biological specimens, processed according to high quality standards, within a Research Biobank provides the opportunity for patients and their families to maximize the visibility of the specimens, promoting their use in national and international research projects dedicated to ML, in compliance with ELSI (Ethical, Social, and Legal Issues) criteria.\n\nThe study primary Objective is To create a computerized registry for ML that collects both retrospective and prospective data in order to estimate the incidence and prevalence of ML, in different phenotypes.\n\nAs secondary objectives. (i) Establish a collection of biological specimens (Fresh and fixed biopsy tissue; DNA extracted from whole blood, saliva and where possible from biopsy tissue) within the FPG Research Biobank, intended for future research purposes and available to the entire scientific community; ii) Genetically profile patients who have never undergone molecular diagnostics or who have been tested with restricted panels of genes (PIK3CA, AKT, MTOR, PTEN, KRAS and BRAF) (activity performed on patients per clinical practice); (iii) Define the natural history of ML in different phenotypes and genotypes from prenatal to adult age; (iv) Evaluate the clinical outcomes of different treatments (e.g., experimental drug therapy, maxillofacial surgery, vascular surgery, laser therapy, sclerotherapy, compression therapy, etc.) and different modes of care (type and frequency of visits performed) in the short and long term; (v) Assess the impact of ML on the lives of patients and caregivers; (vi) Support the drafting\u002Fupdating of national recommendations and standards of care; vii) to promote and facilitate the implementation of research projects dedicated to ML, fostering the advancement of scientific knowledge on this specific disease area;",[27,241,91,242],"Lymphatic Abnormality","Lymphatic Abnormalities","2025-03-18",{"date":245,"type":36},"2025-03-25",{"date":247,"type":36},"2024-06-27",{"date":249,"type":21},"2044-06-27",{"name":251,"class":43},"Fondazione Policlinico Universitario Agostino Gemelli IRCCS",{"id":253,"slug":254,"hasResults":11,"nctId":255,"briefTitle":256,"officialTitle":256,"acronym":4,"eligibilityCriteria":257,"healthyVolunteers":11,"sex":17,"minAge":258,"maxAge":18,"enrollmentInfo":259,"targetDuration":4,"studyType":22,"phases":261,"briefSummary":263,"conditions":264,"keywords":265,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":269,"lastUpdatePostDateStruct":270,"startDateStruct":272,"completionDateStruct":274,"leadSponsor":276,"locationsCount":149},"100567830","phase-2-different-doses-of-sirolimus-for-the-treatment-of-cystic-lymphatic-malformations-100567830","NCT06673290","Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations","Inclusion Criteria:\n\n* Presenting a LM with the following characteristics:\n\n  1. Male and female;\n  2. Between 0 and 18 years of age;\n  3. LM diagnosis was confirmed by local investigators and by consensus of our multidisciplinary vascular anomaly group at the West China Hospital of Sichuan University based on:\n\nBiopsy; Compatible MRI findings; History and clinical features.\n\nExclusion Criteria:\n\n1. Patients contraindicated for the administration of sirolimus (e.g., those with an allergy to sirolimus or other rapamycin analog)\n2. Exposure to chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy or any other investigational agents within 1 weeks before enrolment on study;\n3. Patients had a history of a major surgery within 2 weeks before enrollment;\n4. Patients who have a history of treatment with sirolimus or other mTOR inhibitor;\n5. Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of enrollment;\n6. Concurrent severe and\u002For uncontrolled medical diseases that could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).\n7. Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus.\n8. Patients with inadequate liver function:\n\n   Total bilirubin higher than or equal to 1.5 × the upper limit of the normal (ULN) for age and alanine aminotransferase and aspartate aminotransferase higher than or equal to 2.5 × the ULN for age.\n9. Patients with inadequate renal function:\n\n   0-5 years of age maximum serum creatinine (mg\u002FdL) of 0.8; 6-10 years of age maximum serum creatinine (mg\u002FdL) of 1.0; 11-14 years of age maximum serum creatinine (mg\u002FdL) of 1.2;\n10. Adequate bone marrow function:\n\n    Absolute neutrophil count lower than 1 × 109\u002FL;\n11. History of a malignancy within 5 years;\n12. HIV infection or known immunodeficiency;\n13. Indication for treatment with corticosteroids, vincristine, interferon-α, sirolimus, or tacrolimus for an indication other than IH;\n14. Patients with an inability to participate in or follow-up during the study treatment and assessment plan;\n15. Inability to give informed consent.","1 Year",{"count":260,"type":21},150,[24,262],"PHASE3","The purpose of this study is to compare the efficacy and safety of different concentration gradients of sirolimus in the treatment of cystic lymphatic malformation.",[27],[27,266,267,268],"sirolimus","Efficacy","Safety","2024-12-27",{"date":271,"type":36},"2024-12-31",{"date":273,"type":36},"2024-11-30",{"date":275,"type":21},"2026-12-30",{"name":277,"class":43},"West China Hospital",{"id":279,"slug":280,"hasResults":11,"nctId":281,"briefTitle":282,"officialTitle":283,"acronym":4,"eligibilityCriteria":284,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":285,"enrollmentInfo":286,"targetDuration":4,"studyType":22,"phases":288,"briefSummary":289,"conditions":290,"keywords":291,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":297,"lastUpdatePostDateStruct":298,"startDateStruct":300,"completionDateStruct":302,"leadSponsor":304,"locationsCount":149},"100549678","phase-2-use-of-bleomycin-in-the-sclerotherapy-of-lymphatic-malformations-for-pediatric-patients-100549678","NCT06437158","Use of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients","Efficacy and Safety of Different Concentrations of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients","Inclusion Criteria:\n\n* Male or female participants less than 14 years of age at the time of informed consent\u002Fassent form was signed.\n* Participants whose parents have voluntarily given written consent and participants who provided assent (if applicable) after the study has been explained to them.\n* Participants with LMs of all sites measured and confirmed via imaging at screening, with rapid progression, resluting in obvious symptoms or dysfunction, which could not be radically resected and could be treated by sclerotherapy.\n\nExclusion Criteria:\n\n* Penicillin allergy.\n* Vascular tumors or combined vascular malformations.\n* Participants who may have had surgical or sclerotherapy treatment by other hardeners.\n* LMs growing slowly, without obvious symptoms or dysfunction, which does not need to be treated prematurely.","14 Years",{"count":287,"type":21},200,[24],"Bleomycin has nowadays been more and more widely used in the sclerotherapy of LMs, which has been proven to be primarily dose dependent. The investigators aim to compare the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of LMs for pediatric patients.",[27],[27,292,293,294,295,296],"Bleomycin","Sclerotherapy","Intracystic injection","High-dose concentrations","Low-dose concentrations","2024-05-31",{"date":299,"type":36},"2024-06-03",{"date":301,"type":36},"2023-03-08",{"date":303,"type":21},"2026-12-31",{"name":277,"class":43}]