[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Blueprint Medicines Corporation\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":239},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,7,0,[8,47,74,133,161,180,213],{"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":4,"briefSummary":22,"conditions":23,"keywords":27,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100603988","screening-study-for-kit-d816v-mutated-mast-cell-disease-in-select-populations-100603988",false,"NCT07143669","Screening Study for KIT D816V Mutated Mast Cell Disease in Select Populations","A Multicenter Screening Study to Characterize the Prevalence of the KIT D816V Mutation in Patients With Suspected Clonal Mast Cell Disease","Key Inclusion Criteria:\n\n* Cohort 1 participants must meet inclusion criteria for either SMAC-A or SMAC-B:\n\n  1\\. SMAC-A\n* Documented anaphylaxis due to Hymenoptera venom with cardiovascular symptoms or\n* Documented anaphylaxis without known trigger(s) or allergen(s) warranting hospitalization, emergency room visit, and\u002For epinephrine with cardiovascular symptoms 2. SMAC-B\n* Episodic or recurrent signs and symptoms consistent with mast cell activation without known triggers or allergens in at least 2 of the following organ systems: skin, respiratory\u002Fnaso-ocular, gastrointestinal tract, or cardiovascular.\n* Any clinical response on one or more optimally dosed therapies intended to mitigate mast cell mediators, as determined by the Investigator.\n* Cohort 2 participants must have confirmed, known diagnosis of 1 of the following criteria:\n\n  1. Either hypermobile Ehlers-Danlos syndrome or documented history of hypermobility spectrum disorder.\n  2. Postural orthostatic tachycardia syndrome with one or more systemic symptoms.\n  3. Early onset (≤50 years old) osteoporosis or osteopenia.\n* Cohort 3 participants must have documented diagnosis of 1 of the following, according to World Health Organization 5th edition criteria: chronic myelomonocytic leukemia or myelodysplastic syndrome\u002Fmyeloproliferative neoplasm not otherwise specified.\n\nKey Exclusion Criteria:\n\n* Participants previously diagnosed with any of the following:\n\n  1. Monoclonal mast cell activation syndrome with a known KIT mutation\n  2. Cutaneous mastocytosis only (that is, no documentation of systemic mast cell disease via bone marrow biopsy)\n  3. Any subtype of systemic mastocytosis\n  4. Mast cell sarcoma\n* Cohort 2 only: Osteopenia or osteoporosis attributed to known genetic, endocrine, nutritional, or other medical conditions.\n\nNote: Additional protocol-defined criteria apply.","ALL","18 Years",{"count":19,"type":20},450,"ESTIMATED","OBSERVATIONAL","This is a multicenter screening study to characterize the prevalence of the KIT D816V mutation in participants with suspected clonal mast cell disease.",[24,25,26],"Clonal Mast Cell Disease","KIT D816V Mutation","Suspected KITD816V Mutated Clonal Mast Cell Disease",[28,29,30,31,32,33],"Mast Cell Activation Disorder","MDS\u002FMPN Overlap Syndrome","Chronic Myelomonocytic Leukemia","Hypermobility Syndrome Disorder","Postural Orthostatic Tachycardia Syndrome","Early Onset Osteoporosis","RECRUITING","2026-06-23",{"date":37,"type":38},"2026-06-25","ACTUAL",{"date":40,"type":38},"2025-10-17",{"date":42,"type":20},"2028-10-31",{"name":44,"class":45},"Blueprint Medicines Corporation","INDUSTRY",19,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":56,"phases":57,"briefSummary":60,"conditions":61,"keywords":64,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":67,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":73},"100432405","phase-2-harbor-study-to-evaluate-efficacy-and-safety-of-blu-263-versus-placebo-in-patients-with-indolent-systemic-mastocytosis-100432405","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.",{"count":55,"type":20},534,"INTERVENTIONAL",[58,59],"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.",[62,63],"Indolent Systemic Mastocytosis","Smoldering Systemic Mastocytosis",[65,66],"ISM","SSM",{"date":37,"type":38},{"date":69,"type":38},"2021-11-30",{"date":71,"type":20},"2032-09-30",{"name":44,"class":45},71,{"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":56,"phases":83,"briefSummary":85,"conditions":86,"keywords":91,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":124,"lastUpdatePostDateStruct":125,"startDateStruct":127,"completionDateStruct":129,"leadSponsor":131,"locationsCount":132},"100639442","phase-1-a-first-in-human-trial-of-blu-924-sar449336-in-advanced-solid-tumors-harboring-kras-mutations-100639442","NCT07629960","A First-in-Human Trial of BLU-924 (SAR449336) in Advanced Solid Tumors Harboring KRAS Mutations","A Phase 1\u002F2, Open-Label, Dose-Escalation, Dose-Enrichment, and Dose-Expansion Trial to Evaluate the Safety, Tolerability, Pharmacokinetics, and Antitumor Activity of BLU-924 (SAR449336) as Monotherapy and Combination Therapy in Participants With Advanced Pancreatic Cancer, Non-Small Cell Lung Cancer, or Colorectal Cancer Harboring KRAS Mutations","Inclusion Criteria:\n\n1. Pathologically confirmed diagnosis of metastatic Kirsten rat sarcoma viral oncogene homolog (KRAS)-mutant pancreatic ductal adenocarcinoma (PDAC), non-small cell lung cancer (NSCLC), or colorectal cancer (CRC) with evidence of a single KRAS G12C, G12D, G12V, G12A, G12S, or G13D mutation in tumor tissue or circulating tumor deoxyribonucleic acid (ctDNA).\n2. Measurable disease by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.\n3. Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0-1.\n4. Patients must have received all standard therapies for their cancer type in the metastatic setting, unless they are unable to receive such therapies due to clinical characteristics, comorbidities, or other medically justified reasons.\n\nExclusion Criteria:\n\n1. History of additional malignancy within the last 2 years, with some exceptions as specified in the protocol.\n2. Active brain metastases (participants with asymptomatic brain metastases may be eligible).\n3. Have received prior targeted treatment(s) against KRAS, including pan-KRAS inhibitors, multi-RAS inhibitors, mutant-selective KRAS inhibitors, and RAS or KRAS degraders.\n4. Active or uncontrolled systemic infection, such as tuberculosis, Hepatitis B virus (HBV), Hepatitis C virus (HCV), or Human immunodeficiency virus (HIV).\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.",{"count":82,"type":20},265,[84,58],"PHASE1","A first in human study to evaluate the safety, tolerability, pharmacokinetics, and antitumor activity of BLU-924 \u002F SAR449336, a pan-KRAS inhibitor, in participants with advanced Pancreatic Cancer, Non-Small Cell Lung Cancer, or Colorectal Cancer harboring KRAS mutations.",[87,88,89,90],"Advanced Solid Tumor","Non-Small Cell Lung Cancer","Colorectal Neoplasms","Pancreatic Ductal Adenocarcinoma",[92,93,94,95,96,97,98,99,100,101,102,103,104,105,106,107,108,109,110,111,112,113,114,115,116,117,118,119,120,121,122,123],"Kirsten Rat Sarcoma Viral Oncogene Homolog (KRAS) Mutation","Metastatic Non-Small Lung Cell Cancer","Metastatic Colorectal Cancer (CRC)","Metastatic Pancreatic Ductal Adenocarcinoma","KRAS G12A","KRAS G12C","KRAS G12D","KRAS G12S","KRAS G12V","Solid Tumor, Adult","KRAS-mutant","KRAS-positive","KRAS G13D","Pan-KRAS inhibitor","KRAS inhibitor","First-in-human","Solid tumor","Advanced cancer","Adult solid tumor","Metastatic solid tumor","Colorectal cancer","Colon cancer","Rectal cancer","Metastatic colorectal cancer","Pancreatic cancer","Pancreatic ductal adenocarcinoma","PDAC","Metastatic pancreatic cancer","Lung cancer","NSCLC","Precision oncology","Targeted therapy","2026-06-01",{"date":126,"type":38},"2026-06-05",{"date":128,"type":20},"2026-06-30",{"date":130,"type":20},"2031-07",{"name":44,"class":45},1,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":4,"eligibilityCriteria":139,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":140,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":142,"conditions":143,"keywords":144,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":152,"lastUpdatePostDateStruct":153,"startDateStruct":155,"completionDateStruct":157,"leadSponsor":159,"locationsCount":160},"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":141,"type":20},80,"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.",[62],[65,145,146,147,148,149,150,151],"Indolent systemic mastocytosis","Avapritinib","BLU-285","Selective KIT mutation-targeted tyrosine kinase inhibitor","Tyrosine kinase inhibitor","Mastocytosis","Neoplastic mast cells","2026-04-17",{"date":154,"type":38},"2026-04-20",{"date":156,"type":38},"2025-12-09",{"date":158,"type":20},"2028-12-01",{"name":44,"class":45},8,{"id":162,"slug":163,"hasResults":11,"nctId":164,"briefTitle":165,"officialTitle":166,"acronym":4,"eligibilityCriteria":167,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":168,"targetDuration":4,"studyType":21,"phases":4,"briefSummary":170,"conditions":171,"keywords":172,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":152,"lastUpdatePostDateStruct":173,"startDateStruct":175,"completionDateStruct":177,"leadSponsor":179,"locationsCount":132},"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":169,"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.",[62],[65,146,147,148,149,150,151],{"date":174,"type":38},"2026-04-22",{"date":176,"type":38},"2026-04-15",{"date":178,"type":20},"2032-12-01",{"name":44,"class":45},{"id":181,"slug":182,"hasResults":11,"nctId":183,"briefTitle":184,"officialTitle":185,"acronym":4,"eligibilityCriteria":186,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":187,"targetDuration":4,"studyType":56,"phases":189,"briefSummary":190,"conditions":191,"keywords":194,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":204,"lastUpdatePostDateStruct":205,"startDateStruct":207,"completionDateStruct":209,"leadSponsor":211,"locationsCount":212},"100587670","phase-2-study-of-blu-808-in-chronic-inducible-urticaria-cindu-and-chronic-spontaneous-urticaria-csu-100587670","NCT06931405","Study of BLU-808 in Chronic Inducible Urticaria (CIndU) and Chronic Spontaneous Urticaria (CSU)","A Phase 2 Study to Evaluate the Safety, Tolerability, and Clinical Activity of BLU-808, a Wild Type KIT Inhibitor, in Chronic Inducible Urticaria and Chronic Spontaneous Urticaria","Key Inclusion Criteria:\n\n* Part A: Confirmed diagnosis of CIndU for ≥3 months prior to Day 1 that is inadequately controlled with second generation H1-antihistamines.\n* Part B: Confirmed diagnosis of CSU for ≥3 months prior to Day 1 that is inadequately controlled with second generation H1-antihistamines.\n\nKey Exclusion Criteria:\n\n* Part A: Any active urticaria that may interfere with study assessments.\n* Part B: Participant has a clearly defined predominant cause of chronic urticaria or sole trigger such as symptomatic dermographism and cold-induced urticaria.\n* Part A and Part B: Any other skin disease associated with chronic itching or angioedema that might influence the study evaluations and results, skin diseases associated with only wheals and no itch, or autoinflammatory diseases with urticarial lesions.\n* Part A and Part B: Significant medical, psychiatric, or surgical conditions, or physical findings that may affect participant safety, study drug metabolism, study participation, or assessment of study results.\n* Part A and Part B: Abnormal laboratory values that may pose risks or interfere with study participation.\n* Part A and Part B: Pregnancy or plans for pregnancy; breastfeeding.",{"count":188,"type":20},105,[58],"This is a 2-part, proof-of-concept study to be conducted globally, designed to evaluate the safety, tolerability, clinical activity, pharmacokinetics, and pharmacodynamics of BLU-808, a wild type KIT inhibitor, in participants with CIndU (Part A) or CSU (Part B).",[192,193],"Chronic Inducible Urticaria","Chronic Spontaneous Urticaria",[192,193,195,196,197,198,199,200,201,202,203],"BLU-808","CIndU","CSU","Chronic Urticaria","CU","Cold Urticaria","ColdU","Symptomatic Dermographism","SD","2026-02-16",{"date":206,"type":38},"2026-02-17",{"date":208,"type":38},"2025-05-28",{"date":210,"type":20},"2026-12-31",{"name":44,"class":45},47,{"id":214,"slug":215,"hasResults":11,"nctId":216,"briefTitle":217,"officialTitle":218,"acronym":4,"eligibilityCriteria":219,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":220,"targetDuration":4,"studyType":56,"phases":222,"briefSummary":224,"conditions":225,"keywords":227,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":231,"lastUpdatePostDateStruct":232,"startDateStruct":234,"completionDateStruct":236,"leadSponsor":238,"locationsCount":5},"100573570","phase-4-avapritinib-rollover-study-100573570","NCT06748001","Avapritinib Rollover Study","An Open-label, Multicenter, Rollover Study in Patients Who Participated in an Avapritinib Clinical Study","Inclusion Criteria:\n\n* Participated in a Blueprint Medicines sponsored avapritinib clinical study. The Sponsor reserves the right to not roll over patients who have been on drug hold for an extended time period.\n* Completed End of Trial \u002F End of Study (EOT\u002FEOS) visit in the parent study and demonstrated compliance with the parent study requirements.\n* Continue to clinically benefit from treatment with avapritinib.\n* Able to give written informed consent.\n* Agree to continue to use highly effective contraception as defined in this protocol.\n* Female patients of childbearing potential must have negative highly sensitive serum pregnancy test within 20 days before the first dose of avapritinib. Women of nonchildbearing potential do not require this test.\n\nExclusion Criteria:\n\n* Participant is participating in another interventional study.\n* Participant is unwilling or unable to comply with study procedures and study restrictions.\n* Participant is breastfeeding.\n\nOther protocol-defined criteria apply.",{"count":221,"type":20},60,[223],"PHASE4","The primary objective of the rollover study is to evaluate the long-term safety of avapritinib in participants who have completed a Blueprint Medicines sponsored study (parent study) and continued to benefit from avapritinib.",[226],"Mastocytosis, Systemic",[62,65,228,229,230],"Advanced Systemic Mastocytosis","AdvSM","Rollover study","2026-02-05",{"date":233,"type":38},"2026-02-09",{"date":235,"type":38},"2024-11-28",{"date":237,"type":20},"2027-12-31",{"name":44,"class":45},""]