[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"smoldering-systemic-mastocytosis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:smoldering-systemic-mastocytosis":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"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]