[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT04861064":3,"trial-entities:NCT04861064":112,"trial-summary:NCT04861064":117},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":15,"conditions":17,"keywords":20,"study_type":21,"primary_purpose":22,"phases":23,"enrollment_info":25,"interventions":28,"primary_outcomes":35,"secondary_outcomes":43,"sex":65,"minimum_age":66,"maximum_age":15,"healthy_volunteers":67,"eligibility_criteria":68,"std_ages":83,"locations":87,"central_contacts":102,"overall_officials":106,"references":110,"see_also_links":111},"NCT04861064","00106369","Weekly Sirolimus Therapy","Weekly Sirolimus Therapy for the Treatment of Venous and Lymphatic Malformations","RECRUITING","2027-12","2026-03","2026-03-05","2022-01-18","Medical University of South Carolina","OTHER",null,"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.",[18,19],"Venous Malformation","Lymphatic Malformation",[],"INTERVENTIONAL","TREATMENT",[24],"PHASE2",{"count":26,"type":27},24,"ESTIMATED",[29],{"type":30,"name":31,"description":32,"armGroupLabels":33},"DRUG","Sirolimus","Participants will get Sirolimus (1.5-2 2mg\u002Fm2) weekly for 6 months.",[34],"Treatment Group",[36,40],{"measure":37,"description":38,"timeFrame":39},"Change in size of lesion","Will be measuring the size of the lesions (mm) at each patient visit","Baseline and 6 months",{"measure":41,"description":42,"timeFrame":39},"Change in size of lesion through photograph","Will be evaluating clinical photographs of lesions at each patient visit",[44,48,50,52,54,56,58,61],{"measure":45,"description":46,"timeFrame":47},"Number of side effects experienced","Patient will complete side effect questionnaires at each visit","Month One",{"measure":45,"description":46,"timeFrame":49},"Month Two",{"measure":45,"description":46,"timeFrame":51},"Month Three",{"measure":45,"description":46,"timeFrame":53},"Month Four",{"measure":45,"description":46,"timeFrame":55},"Month Five",{"measure":45,"description":46,"timeFrame":57},"Month Six",{"measure":59,"description":60,"timeFrame":39},"Change in quality of life as assessed by questionnaire","Patient will complete quality of life questionnaire at each visit",{"measure":62,"description":63,"timeFrame":64},"Number of participants with laboratory abnormalities","Standard of care laboratory results (CBC, CMP, triglycerides) will be monitored","From baseline visit to 2 month visit","ALL","2 Years",false,{"inclusion":69,"exclusion":72,"raw_text":82},[70,71],"Patient 2 years of age and older","Venous, lymphatic, or venolymphatic malformations",[73,74,75,76,77,78,79,80,81],"Children with contraindication to use of sirolimus","Children with history of transplant","Children with a history of natural immunodeficiency","Children with a history of artificially induced immunodeficiency","Children with a history of a serious or life-threatening infection","Children taking CYP3A4 inhibiting medications","Children taking strong CYP3A4 inducers to avoid subtherapeutic dosing\u002Fexposure.","Inability or unwillingness of subject or legal guardian\u002Frepresentative to give informed consent","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.","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.",[84,85,86],"CHILD","ADULT","OLDER_ADULT",[88],{"facility":13,"status":8,"city":89,"state":90,"zip":91,"country":92,"contacts":93,"geoPoint":99},"Charleston","South Carolina","29403","United States",[94],{"name":95,"role":96,"phone":97,"email":98},"Stephanie Munie, BS","CONTACT","843-792-9784","munie@musc.edu",{"lat":100,"lon":101},32.77632,-79.93275,[103],{"name":104,"role":96,"phone":105,"email":98},"Stephnie Munie, BS","843-566-2453",[107],{"name":108,"affiliation":13,"role":109},"Chelsea Shope, MSCR","PRINCIPAL_INVESTIGATOR",[],[],{"nct_id":4,"conditions":113,"biomarkers":116},[114,115,18],"Lymphatic malformation","Venous-lymphatic malformation",[],{"nct_id":4,"found":118,"summary":119,"prompt_version":129},true,{"design":120,"status":121,"heading":122,"summary":123,"follow_up":124,"word_count":125,"commitments":126,"compensation":127,"drugs_mentioned":128},"This is an interventional study, meaning participants will receive a specific treatment. It plans to enroll 24 participants.","completed","Weekly Sirolimus for Venous and Lymphatic Malformations","This study is testing if taking the medication sirolimus once a week can help treat venous malformations (abnormal veins) and lymphatic malformations (abnormal lymph vessels). Sirolimus is currently used for these conditions, but usually taken more often, which can cause side effects. This study aims to see if a weekly dose is effective and has fewer side effects. You may be able to join if you are at least 2 years old and have a venous, lymphatic, or venolymphatic malformation. Researchers will measure changes in the size of your malformation over 6 months to see if the treatment is working. The current status of this study is unclear, but it plans to enroll 24 participants.","The study measures changes in your malformation at 6 months, and you may have the option to continue treatment after this time.",115,"Participants will take sirolimus once a week for 6 months. The size of your malformation will be measured at the beginning and end of this 6-month period.","Not stated in the trial record.",[31],"v2"]