[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"indolent-systemic-mastocytosis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:indolent-systemic-mastocytosis":27},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,45,74],{"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":25,"conditions":26,"keywords":29,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100432405","phase-2-harbor-study-to-evaluate-efficacy-and-safety-of-blu-263-versus-placebo-in-patients-with-indolent-systemic-mastocytosis-100432405",false,"NCT04910685","(HARBOR) Study to Evaluate Efficacy and Safety of BLU-263 Versus Placebo in Patients With Indolent Systemic Mastocytosis","A Randomized, Double-Blind, Placebo-Controlled Phase 2\u002F3 Study of BLU-263 in Indolent Systemic Mastocytosis","Key Inclusion Criteria:\n\nAll Participants:\n\n-Participant must have an Eastern Cooperative Oncology Group Performance Status (ECOG PS) of 0 to 2.\n\nPart 1 and PK groups:\n\n* Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review\n* Participant must have failed to achieve adequate symptom control for 1 or more Baseline symptoms, as determined by the Investigator, with at least 2 of the following symptom-directed therapies administered: H1 blockers, H2 blockers, proton-pump inhibitors, leukotriene inhibitors, cromolyn sodium, corticosteroids, or omalizumab.\n* Participants must have SDT for ISM symptom management stabilized for at least 14 days prior to starting screening procedures.\n* For participants receiving corticosteroids, the dose must be ≤ 20 mg\u002Fday prednisone or equivalent, and the dose must be stable for ≥ 14 days.\n\nPart K:\n\n-Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review\n\nPart S:\n\n-Participant has confirmed diagnosis of SSM, confirmed by Central Pathology Review of BM biopsy and central review of B- and C-findings by WHO diagnostic criteria.\n\nPart 2:\n\n-Participant has confirmed diagnosis of ISM, confirmed by Central Pathology Review\n\nKey Exclusion Criteria:\n\n* Participant has been diagnosed with any of the following WHO systemic mastocytosis (SM) sub-classifications: cutaneous mastocytosis only, SM with an associated hematologic neoplasm of non-MC lineage (SM-AHN), aggressive SM, mast cell leukemia, or mast cell sarcoma.\n* Participant has been diagnosed with another myeloproliferative disorder.\n* Participant has organ damage attributable to SM.\n* Participant has clinically significant, uncontrolled, cardiovascular disease\n* Participant has a QT interval corrected using Fridericia's formula (QTcF) \\> \\> 470 milliseconds (msec) (for females) or \\> 450 msec (for males).\n* Participant has a history of a primary malignancy that has been diagnosed or required therapy within 3 years. The following prior malignancies are not exclusionary: completely resected basal cell and squamous cell skin cancer, curatively treated localized prostate cancer, and completely resected carcinoma in situ of any site.\n* Time since any cytoreductive therapy including masitinib and midostaurin should be at least 5 half-lives or 14 days (whichever is longer), and for cladribine, interferon alpha, pegylated interferon, or antibody therapy \\\u003C 28 days or 5 half-lives of the drug (whichever is longer), before beginning the screening period.\n* Participant has received radiotherapy or psoralen and ultraviolet A (PUVA) therapy \\\u003C 14 days before beginning the screening period.\n\nOther protocol-defined criteria apply.","ALL","18 Years",{"count":19,"type":20},534,"ESTIMATED","INTERVENTIONAL",[23,24],"PHASE2","PHASE3","This is a randomized, double-blind, placebo-controlled, Phase 2\u002F3 study comparing the efficacy and safety of elenestinib (BLU-263) + symptom directed therapy (SDT) with placebo + SDT in participants with indolent systemic mastocytosis (ISM) whose symptoms are not adequately controlled by SDT. Parts 1 and 2 will enroll participants with ISM. Participants enrolled in Part 2 will roll over onto Part 3 to receive treatment with elenestinib in an open-label fashion following completion of the earlier Part. Part K will enroll participants with ISM who have previously received an approved selective KIT inhibitor. The study also includes pharmacokinetic (PK) groups that will enroll participants with ISM.",[27,28],"Indolent Systemic Mastocytosis","Smoldering Systemic Mastocytosis",[30,31],"ISM","SSM","RECRUITING","2026-06-23",{"date":35,"type":36},"2026-06-25","ACTUAL",{"date":38,"type":36},"2021-11-30",{"date":40,"type":20},"2032-09-30",{"name":42,"class":43},"Blueprint Medicines Corporation","INDUSTRY",71,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":4,"eligibilityCriteria":51,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":52,"targetDuration":4,"studyType":54,"phases":4,"briefSummary":55,"conditions":56,"keywords":57,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":73},"100612596","a-non-interventional-study-in-participants-with-indolent-systemic-mastocytosis-ism-in-germany-100612596","NCT07255638","A Non-Interventional Study in Participants With Indolent Systemic Mastocytosis (ISM) in Germany","A Prospective Non-Interventional Study to Describe the Effectiveness of Avapritinib (BLU-285), a Selective KIT Mutation-Targeted Tyrosine Kinase Inhibitor, in Patients With Indolent Systemic Mastocytosis and Symptoms That Are Not Adequately Controlled With Symptomatic Treatments in Real-World Settings","Inclusion Criteria:\n\n* Participants is starting avapritinib treatment at the HCP's discretion as part of their routine care (for ISM with moderate to severe symptoms inadequately controlled with symptomatic treatment) and in accordance with approved SmPC.\n\nExclusion Criteria:\n\n* Participant with a potential increased risk for intracranial hemorrhage including those with a history of vascular aneurysm, intracranial hemorrhage, cerebrovascular accident within the prior year, or severe thrombocytopenia.\n* Participant who has previously taken avapritinib as a commercial drug or as part of a clinical study.",{"count":53,"type":20},80,"OBSERVATIONAL","This is a non-interventional study assessing the effectiveness of avapritinib (BLU-285) in the management of ISM in real-world settings in Germany. The study also seeks to address the existing data gap in the natural history and management of participants with ISM.\n\nThe study is designed to follow each participant up to a maximum of 24 months.",[27],[30,58,59,60,61,62,63,64],"Indolent systemic mastocytosis","Avapritinib","BLU-285","Selective KIT mutation-targeted tyrosine kinase inhibitor","Tyrosine kinase inhibitor","Mastocytosis","Neoplastic mast cells","2026-04-17",{"date":67,"type":36},"2026-04-20",{"date":69,"type":36},"2025-12-09",{"date":71,"type":20},"2028-12-01",{"name":42,"class":43},8,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":81,"targetDuration":4,"studyType":54,"phases":4,"briefSummary":83,"conditions":84,"keywords":85,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":86,"startDateStruct":88,"completionDateStruct":90,"leadSponsor":92,"locationsCount":93},"100613313","an-observational-study-in-participants-with-indolent-systemic-mastocytosis-ism-100613313","NCT07264959","An Observational Study in Participants With Indolent Systemic Mastocytosis (ISM)","An Observational Study in Patients With Indolent Systemic Mastocytosis","Inclusion Criteria:\n\n* Male or female adult participants (≥ 18 years of age) with a diagnosis of ISM according to the World Health Organization (WHO) diagnostic criteria\n* Participant is currently being treated or plans to be treated with symptom-directed therapies and\u002For avapritinib for ISM.\n\nExclusion Criteria:\n\n* Participants with advanced systemic mastocytosis (AdvSM) or another associated hematologic neoplasm\n* Participants with smoldering systemic mastocytosis\n* Ongoing participation in interventional studies in systemic mastocytosis (SM) at the time of enrollment\n* Participants currently receiving treatment with a KIT inhibitor other than avapritinib at the time of enrollment.",{"count":82,"type":20},150,"This is a Phase 4, non-interventional, observational study to collect detailed data on the clinical characteristics, clinical outcomes and medical management of ISM in real-world settings. The study will describe the demographic and clinical characteristics of ISM participants, including anaphylaxis and bone manifestations in ISM. Quality of life and disease control will be assessed through participant questionnaires. The study will also evaluate real world ISM treatment management, including use of avapritinib.",[27],[30,59,60,61,62,63,64],{"date":87,"type":36},"2026-04-22",{"date":89,"type":36},"2026-04-15",{"date":91,"type":20},"2032-12-01",{"name":42,"class":43},1]