[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"splenomegaly\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:splenomegaly":26},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,41,74],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":17,"enrollmentInfo":18,"targetDuration":4,"studyType":21,"phases":22,"briefSummary":24,"conditions":25,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100542640","phase-2-high-dose-ruxolitinib-and-allogeneic-stem-cell-transplantation-in-myelofibrosis-patients-with-splenomegaly-100542640",false,"NCT06345495","High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly","Inclusion Criteria:\n\n1. Participants 18 years to less than or equal to 75 years.\n2. Able to provide written consent.\n3. Primary or secondary Myelofibrosis (may have received Jak inhibitors including ruxolitinib)\n4. Enlarged spleen by palpation or imaging. For the purpose of this study, splenomegaly is defined as any clinically palpable spleen or spleen larger than 12 cms on imaging.\n5. Has a fully matched (8\u002F8:HLA A, B, C, DRB1) related or matched unrelated donor.\n6. Adequate renal function, including:\n\n   a. Serum creatinine \\\u003C\u002F= 1.5 mg\u002FdL or estimated Glomerular Filtration Rate (eGFR using the CKI-EPI equation) \\>\u002F= 40 ml\u002Fmin\u002F1.73 m2.\n7. Adequate liver function, including:\n\n   1. ALT\u002FAST \\\u003C\u002F= 3 x ULN\n   2. Direct bilirubin \\\u003C\u002F= 1mg\u002FdL\n   3. No history of liver cirrhosis. No ascites.\n8. Female participants of childbearing potential must have negative results for a serum pregnancy test. Female participants must agree to not breastfeed during the study and for 3 months post-completion of the study therapy.\n9. Subjects who are of childbearing potential, sexually active, and at risk of pregnancy must agree to use a highly effective method of contraception for the duration of the active treatment and at least 3 months post-completion of the study therapy. Highly effective methods of contraception include the following:\n\n   1. Hormonal contraception (i.e., birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject\u002FPartner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.\n   2. Men treated or enrolled on this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of study agent administration. Men who are able to have children must use effective birth control while on the study. If the male participant fathers a child or suspects that he has fathered a child while on the study, he must immediately notify his doctor.\n\nExclusion Criteria:\n\n1. Positive beta HCG in females of child-bearing potential defined as not postmenopausal for 24 months or no previous surgical sterilization or lactating females.\n2. Ejection fraction \\\u003C40%\n3. Corrected DLCO \\\u003C 50%\n4. Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:\n\n   1. Uncontrolled and\u002For active systemic infection (viral, bacterial or fungal)\n   2. Active hepatitis B virus (HBV), hepatitis C (HCV), HIV or TB infection or requiring treatment for the same.\n   3. Thrombosis including MI, Stroke, PE, DVT in the past 6 months\n\nNote: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive and anti-hepatitis B core (HBc) antibody negative) or positive anti-HBc antibody from intravenous immunoglobulins (IVIG) may participate.","ALL","18 Years","75 Years",{"count":19,"type":20},30,"ESTIMATED","INTERVENTIONAL",[23],"PHASE2","To learn if giving ruxolitinib and busulfan before a stem cell transplant can help to reduce spleen size and help the transplant to succeed.",[26,27],"Splenomegaly","Myelofibrosis","RECRUITING","2026-03-09",{"date":31,"type":32},"2026-03-11","ACTUAL",{"date":34,"type":32},"2024-10-01",{"date":36,"type":20},"2029-01-01",{"name":38,"class":39},"M.D. Anderson Cancer Center","OTHER",1,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":46,"acronym":4,"eligibilityCriteria":47,"healthyVolunteers":11,"sex":15,"minAge":16,"maxAge":4,"enrollmentInfo":48,"targetDuration":4,"studyType":21,"phases":50,"briefSummary":51,"conditions":52,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":73},"100347260","phase-2-ruxolitinib-in-treating-patients-with-hypereosinophilic-syndrome-or-primary-eosinophilic-disorders-100347260","NCT03801434","Ruxolitinib in Treating Patients With Hypereosinophilic Syndrome or Primary Eosinophilic Disorders","Phase 2 Study of Ruxolitinib in Idiopathic Hypereosinophilic Syndrome and Primary Eosinophilic Disorders","Inclusion Criteria:\n\n* Subject with idiopathic hypereosinophilic syndrome must meet the following:\n\n  * Has as at least 2 readings with an absolute eosinophil count \\>= 1,500\u002Fmm\\^3 in the preceding 3 months prior to starting ruxolitinib (one reading must be during the screening period).\n  * Dependent, intolerant or refractory to corticosteroids OR has relapsed\u002Frefractory disease to other therapy besides corticosteroids.\n  * Symptomatic from his\u002Fher disease OR has one or more signs of organ damage (assessed by the investigator as possibly-related to eosinophilia or biopsy-proven). This can include skin, lung, cardiac, central nervous system, liver, or gastrointestinal (GI) involvement, or evidence of symptomatic hepatic or splenic enlargement.\n* Subject with lymphocyte-variant hypereosinophilia must meet the following\n\n  * Has at least 2 readings with an absolute eosinophil count \\>= 1,500\u002Fmm\\^3 in the preceding 3 months prior to starting ruxolitinib (one reading must be during the screening period).\n  * Dependent, intolerant or refractory to corticosteroids\\* OR has relapsed\u002Frefractory disease to other therapy besides corticosteroids.\n  * Symptomatic from his\u002Fher disease OR has one or more signs of organ damage (assessed by the investigator as possibly-related to eosinophilia or biopsy-proven). This can include skin, lung, cardiac, central nervous system, liver, or GI involvement, or evidence of symptomatic hepatic or splenic enlargement\n  * Has abnormal T-lymphocyte immuno-phenotype by flow cytometry.\n* Subject with chronic eosinophilic leukemia, not otherwise specified (CEL,NOS) must meet the following\n\n  * Has at least 2 readings with an absolute eosinophil count \\>= 500\u002Fmm\\^3 in the preceding 3 months prior to starting ruxolitinib (one reading must be during the screening period).\n  * Newly-diagnosed OR receiving corticosteroids OR has relapsed\u002Frefractory disease to any therapy besides corticosteroids.\n  * Has increased blasts in the blood or bone marrow (\\> 5% and \\\u003C 20%), and\u002For a clonal cytogenetic or molecular abnormality\n\n    * Subjects with JAK2 mutations are included within this group.\n* Subject with JAK2-rearranged eosinophilic neoplasm must meet the following\n\n  * Has at least 2 readings with an absolute eosinophil count \\>= 500\u002Fmm\\^3 in the preceding 3 months prior to starting ruxolitinib (one reading must be during the screening period).\n  * Newly-diagnosed OR receiving corticosteroids OR has relapsed\u002Frefractory disease to any therapy besides corticosteroids.\n\n    * This group includes subjects with PCM1-JAK2, BCR-JAK2, ETV6-JAK2 or other JAK2 rearrangements.\n* If receiving corticosteroids, must be a stable dose for \\>= 28 days prior to Day 1 (unstable dosing not eligible).\n* Eastern Cooperative Oncology Group (ECOG) performance status =\\\u003C 3.\n* Willing and able to review and execute informed consent (legally-authorized consent acceptable).\n\nExclusion Criteria:\n\n* Active life-threatening complication(s) from underlying eosinophilic disease (i.e., leukostasis; acute thromboembolic disease including central nervous system (CNS) involvement; severe pulmonary or cardiac dysfunction). Stabilization of acute, life-threatening eosinophil-related co-morbidities will allow enrollment of the patient.\n* World Health Organization (WHO)-defined myeloid neoplasm associated with eosinophilia other than CEL NOS and JAK2 rearranged neoplasms (e.g., myelodysplastic syndrome (MDS); myeloproliferative neoplasms (MPN); MDS\u002FMPN overlap disorders; and systemic mastocytosis (SM).\n* Reactive hypereosinophilia due to connective tissue disease, sarcoidosis or eosinophilic granulomatosis with polyangiitis.\n* Organ-restricted ?tissue? eosinophilia with the absence of peripheral eosinophilia in the blood.\n* Invasive malignancy over the previous 2 years except treated early stage carcinomas of the skin, completely resected intraepithelial carcinoma of the cervix, and completely resected papillary thyroid and follicular thyroid cancers.\n* Myeloid or lymphoid neoplasm with eosinophilia and abnormalities of PDGFRA, PDGFRB or FGFR1.\n* Anticipated to receive a hematopoietic stem cell transplant within the first 6 months of treatment on trial.\n* Major surgery within 4 weeks prior to entering the study.\n* Life expectancy of \\\u003C 6 months.\n* Known diagnosis of human immunodeficiency virus (HIV).\n* Known diagnosis of chronic active hepatitis B or C (viral testing is not required). Subjects with a known history of hepatitis B and\u002For C are allowed on trial if at the time of enrollment, the virus is not active and undetected (testing required if there is a known history), and such patients are not actively receiving antiviral treatment specific for hepatitis B and\u002For C.\n* Clinically serious infections requiring ongoing antibiotic therapy.\n* Parasitic infection diagnosed within 24 weeks prior to enrollment.\n* Platelet count =\\\u003C 25 x 10\\^9\u002FL at baseline.\n* Alanine aminotransferase (ALT)\u002Fserum glutamate pyruvate transaminase (SGPT) \\> 4 x upper limit of normal (ULN) or direct bilirubin \\> 4 x ULN (if considered to be unrelated to the underlying eosinophilic disorder).\n* End-stage renal function (creatinine clearance \\[CrCl\\] \\\u003C 15 mL\u002Fmin or glomerular filtration rate \\[GFR\\] \\\u003C 15 mL\u002Fmin) regardless of whether hemodialysis is required.\n* Use of investigational or commercial therapies with the intent to treat the underlying eosinophilic disorder within 28 days of study start, including interferon; imatinib; alemtuzumab; cyclosporine; methotrexate; mepolizumab; benralizumab; or other antibody therapies.\n* Use of hydroxyurea within 7 days of study start.\n* Prior therapy with ruxolitinib or other JAK inhibitors.\n* Previous allergic reactions to JAK inhibitors or excipients.\n* Unwilling to commit to abstinence from heterosexual contact or agree to use and comply with highly effective contraception, 28 days prior to starting study drug, during the treatment period and for 12 weeks after discontinuation of study treatment.\n* Females of childbearing potential who have a positive pregnancy test (urine or serum) during screening period.",{"count":49,"type":20},10,[23],"This phase II trial studies how well ruxolitinib works in treating patients with hypereosinophilic syndrome or primary eosinophilic disorders.",[53,54,55,56,57,58,59,60,61,62,26,63],"BCR-JAK2 Fusion Protein Expression","Blasts 20 Percent or Less of Peripheral Blood White Cells","Blasts More Than 5 Percent of Bone Marrow Nucleated Cells","Blasts More Than 5 Percent of Peripheral Blood White Cells","Blasts Under 20 Percent of Bone Marrow Nucleated Cells","Chronic Eosinophilic Leukemia, Not Otherwise Specified","Eosinophilia","Hepatomegaly","Hypereosinophilic Syndrome","JAK2 Gene Mutation","TEL-JAK2 Fusion Protein Expression","2025-12-20",{"date":66,"type":32},"2025-12-23",{"date":68,"type":32},"2019-11-15",{"date":70,"type":20},"2028-12-30",{"name":72,"class":39},"William Shomali",4,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":80,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":15,"minAge":82,"maxAge":83,"enrollmentInfo":84,"targetDuration":4,"studyType":21,"phases":86,"briefSummary":88,"conditions":89,"keywords":94,"overallStatus":104,"whyStopped":4,"lastUpdateSubmitDate":105,"lastUpdatePostDateStruct":106,"startDateStruct":108,"completionDateStruct":110,"leadSponsor":112,"locationsCount":40},"100582913","study-of-the-prevalence-of-acid-sphingomyelinase-deficiencyniemann-pick-ab-and-b-disease-in-patients-with-diffuse-interstitial-lung-disease-100582913","NCT06869499","Study of the Prevalence of Acid Sphingomyelinase Deficiency\u002FNiemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease","Niemann-PID: Study of the Prevalence of Acid Sphingomyelinase Deficiency\u002FNiemann Pick AB and B Disease in Patients With Diffuse Interstitial Lung Disease","Niemann-PID","Inclusion Criteria:\n\n1. Interstitial lung disease with ground-glass lesions on a chest CT scan certified by a pneumologist\u002Fradiologist or internist.\n2. At least one of the following criteria :\n\n   * Splenomegaly (palpable spleen or craniocaudal length ≥ 13 cm)\n   * Splenectomy\n   * Thrombocytopenia (platelets \\\u003C 150 G\u002FL)\n   * Low HDL-cholesterol (\\\u003C0.4 g\u002Fl or 1.03 mmol\u002Fl)\n   * Notion of parental consanguinity\n3. Have given their written informed consent, in accordance with regulations.\n4. Affiliated to the social security system or entitled beneficiary (excluding AME).\n\nExclusion Criteria:\n\n1. Inability to understand the information provided.\n2. Under guardianship, curatorship or legal protection.\n3. Under restraint or deprived of liberty by judicial or administrative decision.","15 Years","60 Years",{"count":85,"type":20},200,[87],"NA","The goal of this clinical trial is to optimise and facilitate screening for Acid SphingoMyelinase Deficiency (ASMD) disease, by evaluating acid sphingomyelinase activity and, where appropriate, LysoSM levels in a cohort of 200 participants with diffuse interstitial lund disease (ILD) at risk of developing ASMD disease.\n\nILD is common in the general population, so in order to limit the number of differential diagnoses, the population to be studied will be restricted to participants aged between 15 years and 3 months and 60 years, with ILD plus ground-glass opacities on chest CT scan certified by a pulmonologist\u002Fradiologist or internist, AND splenomegaly or splenectomy, and\u002For thrombocytopenia, and\u002For low HDL cholesterol, and\u002For parental consanguinity which increase the sensitivity of ASMD screening.\n\nIn this clinical trail, two procedures are added, participants will be asked for :\n\n* a blood sample to measure the acid sphingomyelinase enzyme activity and LysoSM, if required.\n* a follow-up visit at 6 months",[26,90,91,92,93],"Splenectomy","Thrombopenia","Interstitial Lung Disease (ILD)","Hypocholesterolemia",[95,96,97,98,99,100,101,102,103],"Acid sphingomyelinase deficiency","Ground glass lesions","Niemann Pick A\u002FB, B","lysoSM","smpd1","splenomegaly","interstitial lung disease","screening","acid sphingomyelinase","NOT_YET_RECRUITING","2025-05-14",{"date":107,"type":32},"2025-05-18",{"date":109,"type":20},"2025-05",{"date":111,"type":20},"2029-03-01",{"name":113,"class":39},"Wladimir MAUHIN, Dr"]