[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"thrombotic-microangiopathies\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:thrombotic-microangiopathies":25},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,9,0,[8,40,74,98,145,168,193,222,260],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":13,"acronym":4,"eligibilityCriteria":14,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":16,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":5},"100370061","transpire-lung-injury-in-a-longitudinal-cohort-of-pediatric-hsct-patients-100370061",false,"NCT04098445","TRANSPIRE: Lung Injury in a Longitudinal Cohort of Pediatric HSCT Patients","Inclusion Criteria:\n\n* Subjects ≤ 24 years of age undergoing allogeneic or autologous HSCT.\n\nExclusion Criteria:\n\n* Subjects over 24 years of age.","ALL","24 Years",{"count":18,"type":19},2000,"ESTIMATED","OBSERVATIONAL","Hematopoietic stem cell transplant (HSCT) is an effective but toxic therapy and pulmonary morbidity affects as many as 25% of children receiving transplant. Early pulmonary injury includes diffuse alveolar hemorrhage (DAH), thrombotic microangiopathy (TMA) interstitial pneumonitis (IPS) and infection, while later, bronchiolitis obliterans is a complication of chronic GVHD associated with severe morbidity and mortality. Improved diagnosis and treatment of pulmonary complications are urgently needed as survival after HSCT improves, and as HSCT is increasingly used for non-malignant disorders such as sickle cell disease. Currently, there are large and important gaps in the investigator's knowledge regarding incidence, etiology and optimal treatment of pulmonary complications. Moreover, young children unable to perform spirometry are often diagnosed late, and strategies for monitoring therapeutic response are limited.\n\nThis is a prospective multi-institutional cohort study in pediatric patients undergoing allogeneic hematopoietic stem cell transplantation (alloHSCT). Assembly of a large prospective uniformly screened cohort of children receiving HSCT, together with collection of biological samples, will be an effective strategy to identify mechanisms of lung injury, test novel diagnostic strategies for earlier diagnosis, and novel treatments to reduce morbidity and mortality from lung injury after transplant.",[23,24,25,26,27],"Hematopoietic Stem Cell Transplant (HSCT)","Diffuse Alveolar Hemorrhage","Thrombotic Microangiopathies","Interstitial Pneumonitis","Bronchiolitis Obliterans","RECRUITING","2026-06-17",{"date":31,"type":32},"2026-06-22","ACTUAL",{"date":34,"type":32},"2021-09-08",{"date":36,"type":19},"2033-09",{"name":38,"class":39},"Children's Hospital Medical Center, Cincinnati","OTHER",{"id":41,"slug":42,"hasResults":11,"nctId":43,"briefTitle":44,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":15,"minAge":47,"maxAge":4,"enrollmentInfo":48,"targetDuration":4,"studyType":50,"phases":51,"briefSummary":54,"conditions":55,"keywords":57,"overallStatus":63,"whyStopped":4,"lastUpdateSubmitDate":64,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":4},"100628244","phase-1-tgd001-treatment-in-thrombotic-microangiopathies-100628244","NCT07459114","TGD001 Treatment in Thrombotic Microangiopathies","An Adaptive Dose Escalation and Expansion Basket Trial to Explore the Safety, Pharmacology, and Clinical Activity of TGD001 in Immune-Mediated Thrombotic Thrombocytopenic Purpura (iTTP) and Other Thrombotic Microangiopathies","Key Inclusion Criteria:\n\n* 18 years old or older\n* Willing to sign an informed consent form\n* Willing to refrain from sexual intercourse or must use a contraceptive method that is highly effective for 90 days after the last dose of TGD001\n* Symptomatic acute TMA episode\n* Accessible to follow-up\n\nKey Exclusion Criteria:\n\n* Diagnosis other than TMA, which could account for the findings of thrombocytopenia and hemolytic anemia\n* Diagnosis of Shiga-toxin induced HUS\n* Known bone marrow\u002Fgraft failure\n* Diagnosis of ongoing severe, uncontrolled Graft versus Host Disease\n* Received therapeutic plasma exchange (PEX) within 7 days prior to screening\n* Use of an anticoagulant and\u002For thrombolytics\n* Active internal bleeding\n* Any major bleeding episode within the past 30 days, or diagnosis of chronic bleeding conditions\n* Known gastrointestinal ulcer\n* Severe, uncontrolled hypertension, renal impairment requiring dialysis, or liver impairment, or active infection indicated by sepsis\n* Life expectancy less than 3 months independent of TMA disorder\n* Pregnant or breastfeeding","18 Years",{"count":49,"type":19},70,"INTERVENTIONAL",[52,53],"PHASE1","PHASE2","This is a Phase 1\u002F2, prospective, adaptive design trial of TGD001 in participants with suspicion or clinical diagnosis of acute immune thrombotic thrombocytopenic purpura (iTTP) episodes and participants with suspicion or clinical diagnosis of an acute Thrombotic Microangiopathy (TMA) episode. The trial is an open-label, dose escalation and expansion basket trial.",[25,56],"ITP Immune-Mediated Thrombocytopenia",[58,59,25,60,61,62],"iTTP","TMA","immune-mediated thrombotic thrombocytic purplura","thrombotic microangiopathy","TGD001","NOT_YET_RECRUITING","2026-03-06",{"date":66,"type":32},"2026-03-09",{"date":68,"type":19},"2026-05-31",{"date":70,"type":19},"2028-08-30",{"name":72,"class":73},"TargED Biopharmaceuticals B.V.","INDUSTRY",{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":80,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":15,"minAge":47,"maxAge":4,"enrollmentInfo":82,"targetDuration":4,"studyType":50,"phases":84,"briefSummary":86,"conditions":87,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":97},"100538452","efficacy-and-safety-of-immunosuppression-caplacizumab-and-plasma-infusion-without-therapeutic-plasma-exchange-in-immune-mediated-thrombotic-thrombocytopenic-purpura-100538452","NCT06291025","Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura","Efficacy and Safety of Immunosuppression, Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura: Multicentric Non-inferiority Single-arm Study","PEX-FREE","Inclusion Criteria:\n\n* Adult patient ≥ 18 years;\n* Clinical diagnosis of iTTP based on standard clinical and laboratory criteria (French Score ≥ 2): i.e., thrombotic microangiopathy syndrome with platelet count ≤ 30 G\u002FL and serum creatinine ≤ 200 μmol\u002FL; it is not necessary to have the laboratory result confirming the severe ADAMTS13 deficiency for inclusion of patient \\[32\\] (For patient with previous TTPflare, French score can be \\\u003C 2);\n* Patient having read and understood the information letter and signed the Informed Consent Form. If the patient is unable to express his consent, the consent will be signed by his representative ((1) the trusted person, or failing that, (2) a family member, or (3) a close relative of the person concerned). In this case, consent to continue the study will subsequently be requested from the patient (article L1122-1-1 of the CSP);\n* Patient affiliated with, or beneficiary of a social security (national health insurance) plan;\n* For women:\n\n  * Women of childbearing potential :\n\n    * Effective contraception according to WHO definition (estrogen-progestin or intrauterine device or tubal ligation) since at least 1 month and;\n    * Negative blood pregnancy test;\n  * Women surgically sterile (absence of ovaries and\u002For uterus);\n  * Postmenopausal women (non-medically induced amenorrhea for at least 12 months prior to the inclusion visit).\n\nExclusion Criteria:\n\n* Platelet count \\> 100 G\u002FL before plasma treatment;\n* Patients with a French score \\\u003C 2 (a serum creatinine level \\> 200 μmol\u002FL and\u002For with a platelet count \\> 30 G\u002FL), in order to exclude possible cases of hemolytic uremic syndrome (except for patient with previous TTPflare, French score can be \\\u003C 2);\n* Other known causes of cytopenias and\u002For organ failure including but not limited to: uncontrolled cancer, chemotherapy, transplant, drugs, HIV at AIDS stage;\n* Patients with a severe neurological disorder i.e. seizure, coma, focal deficiency, trouble of consciousness;\n* Pregnant women (positive result from a blood pregnancy test) or patients with an imminent project of pregnancy; breastfeeding women (due to lack of pharmacological data for caplacizumab during pregnancy and breastfeeding);\n* Weight \\> 100KG;\n* Congenital TTP;\n* Clinically significant active bleeding or high risk of bleeding (excluding thrombocytopenia);\n* Chronic treatment with anticoagulant that cannot be interrupted safely, including but not limited to: vitamin K antagonists, direct oral anticoagulant, low molecular weight heparin or heparin;\n* Malignant hypertension;\n* Contra-indication to CABLIVI 10 mg powder and solvent for solution for injection: hypersensitivity to caplacizumab or to any of the excipients;\n* Contra-indication to Plasma treatment;\n* Contra-indication to corticosteroid (= ((methyl)prednisone or (methyl)prednisolone)) or excipients;\n* Contra-indication to rituximab or excipients and to its premedication;\n* Person deprived of liberty by administrative or judicial decision or placed under judicial protection (guardianship or supervision);\n* Participation in another drug interventional clinical trial within 30 days prior to inclusion and during the study.",{"count":83,"type":19},131,[85],"NA","Immune thrombotic thrombocytopenic purpura (iTTP) is caused by a severe, autoantibody-mediated deficiency of ADAMTS13 leading to an accumulation of ultra-large von Willebrand factor multimers in plasma and finally to microthrombi in blood vessels. The current standard of care of iTTP consists in the triple association of daily plasma exchange (PEX, 60 ml\u002Fkg\u002Fday), immunosuppressive agents and anti-adhesive treatment (Caplacizumab). Our group recently reported the outcome of 90 patients with iTTP treated with this triple association and when compared to historical patients, the triplet regimen prevented death, refractoriness and exacerbations. Likewise, plasma volumes were reduced by 2 to 3-fold and the median number of PEX sessions could be reduced from 13 to 6. PEX is an invasive and time-consuming procedure, associated with catheter and plasma-related complications ranging from 22% to 30%. Consequently, to alleviate the burden of care in iTTP, using a regimen without PEX would represent a major and topical goal. Attempts to treat patients with plasma infusion (PI) without PEX were previously reported and provided evidence that large volumes of PI (20-30 ml\u002Fkg\u002Fday) improved the initial outcome of iTTP. However, fluid overload occurred in most cases after 5-7 days, limiting the feasibility of this strategy. Nevertheless, the recent availability of caplacizumab opens the perspective of treating patients with plasma for a shorter period. Recently, strategies without PEX have been carried out in Jehovah's Witnesses with iTTP \\[5\\]. Impressively, improvement was rapid and comparable to those provided with a standard PEX-based treatment. Additionally, a treatment combining caplacizumab and immunosuppression only was successfully performed in six iTTP patients with severe neurologic and\u002For cardiac involvement. The rapid and durable improvement provides evidence that a regimen without plasma seems feasible. However, it's considered that robust data are still lacking to completely remove plasmatherapy from iTTP management. Based on these statements, the objective is to address the efficacy and safety of a PEX-free regimen, combining PI only (15 ml\u002Fkg\u002Fday), corticosteroids\u002Frituximab, and caplacizumab.",[25],"2026-02-13",{"date":90,"type":32},"2026-02-17",{"date":92,"type":32},"2024-04-10",{"date":94,"type":19},"2026-08-01",{"name":96,"class":39},"University Hospital, Rouen",30,{"id":99,"slug":100,"hasResults":11,"nctId":101,"briefTitle":102,"officialTitle":103,"acronym":104,"eligibilityCriteria":105,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":106,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":108,"conditions":109,"keywords":117,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":135,"lastUpdatePostDateStruct":136,"startDateStruct":138,"completionDateStruct":140,"leadSponsor":142,"locationsCount":144},"100608769","retrospective-epidemiological-study-of-patients-in-the-national-cohort-of-the-french-tma-center-100608769","NCT07205861","Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center","Auto-immune Thrombotic Thrombocytopenic Purpura : Retrospective Epidemiological Study of Patients in the National Cohort of the French TMA Center, TWI-LIGHT","TWILIGHT","Inclusion Criteria:\n\n-Patients with a diagnosis of immune mediated TTP\n\nExclusion Criteria:\n\n* Cancer- associated iTTP and HIV-associated iTTP\n* Severe sepsis\n* Disseminated intravascular coagulation with consumption of coagulation factors;\n* Transplant-associated TTP\n* HIV-associated TTP (AIDS stage)\n* Patient not affiliated with a social security scheme\n* Patient or parent's objection to the reuse of their healthcare data for research",{"count":107,"type":19},1200,"Immune thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening disorder characterized by microangiopathic hemolytic anemia, severe thrombocytopenia, and ischemic organ damage due to microvascular thrombosis. It results from a severe deficiency in the von Willeband factor (vWF)-cleaving protease ADAMTS13, primarily caused by autoantibodies that inhibit its activity. This deficiency leads to accumulation of ultra-large vWF multimers, triggering pathological platelet aggregation and widespread microthrombi. iTTP typically presents with acute neurological symptoms (e.g., confusion, seizures, coma), cardiac events (e.g., myocardial infarction), and multiorgan dysfunction. Without prompt treatment-plasma exchange, immunosuppression, and the vWF inhibitor caplacizumab-mortality exceeds 90%. Survivors face long-term risks, including cardiovascular complications, cognitive impairment, and reduced life expectancy.\n\nThe TWI-LIGHT protocol is a national retrospective epidemiological study coordinated by the French Reference Center for Thrombotic Microangiopathies (CNR-MAT). It aims to analyze long-term outcome in \\>1,200 iTTP patients diagnosed between October 2000 and June 2024. The study leverages pseudonymized data from the CNR-MAT registry, collected via a secure REDCap database.\n\nKey Objectives:\n\n1. Primary: Assess the impact of cardiovascular risk factors (e.g., hypertension, diabetes) and ADAMTS13 activity on life expectancy in iTTP survivors.\n2. Secondary:\n\n   * Evaluate disease burden in underrepresented groups (pregnant\u002Fpostpartum women, children, elderly patients).\n   * Analyze the influence of new therapies (caplacizumab, rituximab, recombinant ADAMTS13) on care pathways.\n   * Identify prognostic factors and treatment practices.\n   * Characterize neurocognitive outcomes and quality of life post-iTTP.\n\nMethodology:\n\n* Design: Non-interventional, retrospective (MR-004 compliance), using data from standard care.\n* Inclusion: Patients with confirmed iTTP (thrombocytopenia, hemolytic anemia, ADAMTS13 \\\u003C10%), diagnosed within the study period, and ≥1 year of follow-up.\n* Exclusion: Cancer-associated iTTP, severe sepsis, or patient opposition to data reuse.\n* Data Collection: Clinical, biological, and therapeutic variables from hospital\u002Fconsultation records, including cardiovascular events, ADAMTS13 activity, and neurocognitive assessments.\n* Analysis: Kaplan-Meier survival curves and Cox regression models to identify risk factors for non-iTTP-related death.\n\nExpected Outcomes:\n\n* Prevalence of cardiovascular comorbidities and their correlation with ADAMTS13 activity.\n* Insights into iTTP subtypes (e.g., gestational, pediatric) and therapeutic efficacy.\n* Evidence-based strategies for personalized long-term management.\n\nEthical Framework:\n\n* AP-HP-sponsored, with oversight from Sorbonne University's ethics committee.\n* Patients informed of data reuse with opt-out rights; data archived for 15 years.\n\nThis landmark study will inform clinical guidelines, optimize survivor care, and address unmet needs in iTTP management through comprehensive, real-world data analysis.",[110,111,25,112,113,114,115,116],"Thrombotic Thrombocytopenic Purpura (TTP)","Immune Thrombotic Thrombocytopenic Purpura","Microangiopathy, Thromboic","Cardiovascular Diseases","Autoimmune Diseases","Rare Diseases","Neurological Manifestations",[118,111,119,120,121,122,123,124,125,126,127,128,129,130,131,132,133,134],"Thrombotic Thrombocytopenic Purpura","Thrombotic Microangiopathies (MeSH: D057049)","Microangiopathies, Thrombotic (MeSH: D057172)","ADAMTS13 Protein (MeSH: C423082)","Autoimmune Diseases (MeSH: D001327)","Cardiovascular Diseases (MeSH: D002318)","Epidemiologic Studies (MeSH: D004812)","Retrospective Studies (MeSH: D012196)","Observational Study (Publication Type)","Risk Factors (MeSH: D012307)","Treatment Outcome (MeSH: D016647)","Rare Diseases (MeSH: D034381)","Longitudinal Studies (MeSH: D008159)","Patient Registry (MeSH: D010329)","Survival (MeSH: D009505)","Quality of Life (MeSH: D011788)","Neurological Manifestations (MeSH: D009462)","2025-09-25",{"date":137,"type":32},"2025-10-03",{"date":139,"type":32},"2024-12-02",{"date":141,"type":19},"2028-12",{"name":143,"class":39},"Assistance Publique - Hôpitaux de Paris",1,{"id":146,"slug":147,"hasResults":11,"nctId":148,"briefTitle":149,"officialTitle":150,"acronym":151,"eligibilityCriteria":152,"healthyVolunteers":11,"sex":15,"minAge":47,"maxAge":4,"enrollmentInfo":153,"targetDuration":155,"studyType":20,"phases":4,"briefSummary":156,"conditions":157,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":135,"lastUpdatePostDateStruct":160,"startDateStruct":162,"completionDateStruct":164,"leadSponsor":166,"locationsCount":144},"100419705","complement-prospective-evaluation-of-thrombotic-microangiopathy-on-endothelium-100419705","NCT04745195","Complement Prospective Evaluation of Thrombotic Microangiopathy on Endothelium","Diagnostic and Risk Criteria for Complement Defects in Thrombotic Microangiopathy and Amplifying Conditions, Such as Severe Hypertension: The COMPETE Study.","COMPETE","Inclusion Criteria:\n\n* Males or females at least 18 years of age;\n* Have acute kidney injury, defined as estimated GFR \\\u003C45 mL\u002Fmin\u002F1.73m2;\n* Have documented TMA either on peripheral blood, defined as Coombs negative microangiopathic hemolytic anemia (hematocrit \\\u003C30%, hemoglobin \\\u003C6.5 mmol\u002FL \\[\\\u003C10 g\u002FdL\\], lactate dehydrogenase \\>500 U\u002FL, and either schistocytes on peripheral blood smear or undetectable haptoglobin), and platelets \\\u003C150,000 per µL, or kidney biopsy;\n* Have primary atypical HUS or a coexisting condition linked to complement dysregulation:\n\n  * Hypertensive emergency, defined as SBP\u002FDBP of \\>180\u002F120 mmHg and impending organ damage secondary to hypertension (at least one of the following: neurologic disease, hypertensive retinopathy grade III and\u002For IV, left ventricular hypertrophy); OR\n  * Pregnancy, including 12 weeks postpartum; OR\n  * Kidney donor recipient; OR\n  * Systemic auto-immune disease associated with TMA, including systemic sclerosis, systemic lupus erythematosus, anti-phospholipid syndrome;\n* Have the ability to understand the requirements of the study, provide written informed consent, and comply with the study protocol procedures.\n\nExclusion Criteria:\n\n* Have secondary causes of hypertensive emergency, including renovascular hypertension, Cushing syndrome, aldosteronism, pheochromocytoma, thyroid disease;\n* Have a nephropathy not related to thrombosis on kidney biopsy;\n* Have ADAMTS13 deficiency, defined as ADAMTS13 activity \\\u003C10%;\n* Have a positive stool culture for Shiga toxin producing bacteria;\n* Have positive serologic test for viral infections, including HIV and CMV;\n* Have a history of malignant disease, excluding non-melanoma skin cancer;\n* Have a history of bone marrow or solid organ transplantation, excluding kidney transplantation;\n* Received at least one of the following agents: chemotherapeutics, sirolimus, anti-VEGF agents;\n* Have a history of recent past exposure to illicit drug(s).",{"count":154,"type":19},42,"12 Months","Thrombotic microangiopathy (TMA) is a severe and life-threatening condition, often affecting the kidneys and brain. It can occur on the background of various clinical conditions. Dysregulation of the alternative pathway of complement may be the etiological factor and this type of TMA is classified, according to the current nomenclature, as primary atypical hemolytic uremic syndrome (HUS). Half the patients with primary atypical HUS present with rare variants in complement genes, although coexisting conditions are often needed for the TMA to become manifest. In patients with secondary atypical HUS, certain coexisting conditions appear to drive the disease and treatment should target the underlying condition to remit the TMA.\n\nRecently, the investigators demonstrated, by using a novel in-house developed functional endothelial cell-based test, that complement dysregulation and overactivation is the dominant cause of disease and its sequelae in a subset of patients with secondary atypical HUS, having impact on treatment and prognosis. The investigators did first prove this concept in patients presenting with TMA and hypertensive emergency. A prospective study is needed to further corroborate these findings along the spectrum of TMA. The investigators hypothesize that their functional endothelial cell-based test, the so-called \"HMEC\" test, can better categorizes the TMA into different groups with potential therapeutic and prognostic implications. Thus, paving the road to the ultimate goal of precision medicine.",[25,158,159],"Hemolytic Uremic Syndrome, Atypical","Hemolytic-Uremic Syndrome",{"date":161,"type":32},"2025-10-01",{"date":163,"type":32},"2021-08-11",{"date":165,"type":19},"2026-12-31",{"name":167,"class":39},"Maastricht University Medical Center",{"id":169,"slug":170,"hasResults":11,"nctId":171,"briefTitle":172,"officialTitle":173,"acronym":4,"eligibilityCriteria":174,"healthyVolunteers":11,"sex":15,"minAge":175,"maxAge":176,"enrollmentInfo":177,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":179,"conditions":180,"keywords":182,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":184,"lastUpdatePostDateStruct":185,"startDateStruct":187,"completionDateStruct":189,"leadSponsor":191,"locationsCount":144},"100523975","identification-of-plasma-biomarkers-for-early-diagnosis-of-transplant-associated-thrombotic-microangiopathy-100523975","NCT06102694","Identification of Plasma Biomarkers for Early Diagnosis of Transplant-associated Thrombotic Microangiopathy","Screening and Identification of Plasma Biomarkers for Early Diagnosis of Transplant-associated Thrombotic Microangiopathy Based on Proteomics and Metabolomics Techniques","Inclusion Criteria:\n\n* 1\\. Diagnosis of a hematologic disease (e.g., leukemia, myelodysplastic syndromes,lymphoma) confirmed by histology or other appropriate diagnostic methods.\n* 2\\. undergoing allo-HSCT\n* 3\\. Age 14 years or older\n* 4.Informed consent must be signed before the start of the study. For participants aged 18 and above, the informed consent should be signed by the patient or their immediate family member. Considering the patient's condition, if it is not favorable for the patient to sign, the informed consent may be signed by a legal guardian or immediate family member of the patient.\n\nExclusion Criteria:\n\n* 1\\. Missing or lost follow-up of key clinical data\n* 2\\. failure to collect plasma samples at specific time points after transplantation\n* 3\\. Plasma sample collection time later than the onset date of TMA\n* 4\\. TMA occurrence time later than 180 days after transplantation","14 Years","70 Years",{"count":178,"type":19},500,"The goal of this clinical trial is to learn about plasma biomarkers of diagnosed transplant-associated thrombotic microangiopathy (TA-TMA) in patients undergoing transplantation. The main questions it aims to answer are: whether there are molecules that can accurately diagnose and predict TA-TMA; whether the current biomarkers related to TA-TMA can well predict the occurrence and survival of TA-TMA in adult patients with malignant hematopoietic diseases, for example, acute leukemia. Participants will receive laboratory tests of peripheral blood and urine specimens related to TA-TMA at regular times after transplantation.",[25,181],"Hematopoietic Stem Cell Transplantation",[183,181],"TA-TMA","2025-08-01",{"date":186,"type":32},"2025-08-06",{"date":188,"type":32},"2023-11-16",{"date":190,"type":19},"2025-12",{"name":192,"class":39},"Institute of Hematology & Blood Diseases Hospital, China",{"id":194,"slug":195,"hasResults":11,"nctId":196,"briefTitle":197,"officialTitle":198,"acronym":4,"eligibilityCriteria":199,"healthyVolunteers":11,"sex":15,"minAge":200,"maxAge":201,"enrollmentInfo":202,"targetDuration":4,"studyType":50,"phases":204,"briefSummary":205,"conditions":206,"keywords":207,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":213,"lastUpdatePostDateStruct":214,"startDateStruct":216,"completionDateStruct":218,"leadSponsor":219,"locationsCount":221},"100504957","phase-2-efficacy-and-safety-study-of-narsoplimab-in-pediatric-patients-with-high-risk-hematopoietic-stem-cell-transplant-tma-100504957","NCT05855083","Efficacy and Safety Study of Narsoplimab in Pediatric Patients With High-Risk Hematopoietic Stem Cell Transplant TMA","A Phase 2 Study Evaluating the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Narsoplimab in Pediatric Patients (28 Days to ≤ 18 Years of Age.) With High-Risk Hematopoietic Stem Cell Transplant Thrombotic Microangiopathy","Inclusion Criteria:\n\n1. Age at least 28 days and less than 18 years prior to informed consent (Visit 0).\n2. Have informed consent from at least one parent or legal guardian as required by local law and regulation. Patient informed consent will be required if the patient has reached the local legal age of majority.\n3. Assent from patients as required by local law and regulation.\n4. Have received an allogeneic hematopoietic stem cell transplant for the treatment of benign or malignant disease.\n5. Have a diagnosis of HSCT-TMA defined as meeting both of the following criteria:\n\n   * Platelet count \\\u003C 50,000\u002FmL or a decrease in platelet count \\> 50% from the highest value obtained following transplant.\n   * Evidence of microangiopathic hemolysis (presence of schistocytes, serum lactate dehydrogenase \\[LDH\\] \\> upper limit of normal (\\[ULN\\], or haptoglobin \\\u003C lower limit of normal \\[LLN\\])\n6. Have at least one of the following HSCT-TMA high-risk criteria:\n\n   * HSCT-TMA persistence \\> 2 weeks following modification of calcineurin inhibitors or sirolimus OR\n   * Have evidence of high-risk HSCT-TMA defined as at least one of the following:\n\n     * Spot protein\u002Fcreatinine ratio \\> 2 mg\u002Fmg\n     * Serum creatinine \\> 1.5 x the creatinine level prior to TMA development\n     * Biopsy-proven gastrointestinal TMA\n     * TMA-related neurological abnormality\n     * Pericardial or pleural effusion without alternative explanation\n     * Pulmonary hypertension without alternative explanation\n     * Have Grade III or Grade IV graft-versus-host disease (GVHD) or, in the opinion of the Investigator, risk for development of Grade III or Grade IV GVHD if immunosuppression were to be modified\n     * Have elevated serum C5b-9 (\\> 244 ng\u002FmL)\n7. If sexually active and of childbearing potential (for female pediatric patients, defined as starting at onset of menses), must agree to practice a highly effective method of birth control throughout study drug treatment and for at least 12 weeks after the last dose of study drug, such method of birth control defined as one that results in a low failure rate (i.e., less than 1% per year) when used consistently and correctly, such as implants, injectables, combined oral contraceptives, some intrauterine devices, sexual abstinence (abstinence is acceptable when it is in line with the patient's preferred and usual lifestyle and is defined as complete abstinence of sexual intercourse, not periodic abstinence or withdrawal), or vasectomized partner.\n8. Male patients must be willing to avoid fathering children for at least 12 weeks following the last dose of study medication.\n\nExclusion Criteria:\n\n1. All treatments for HSCT-TMA are allowed except eculizumab, ravulizumab, and defibrotide within 3 months prior to informed consent, unless failure of therapy can be documented.\n\n   a. Patients may not be on eculizumab, ravulizumab, or defibrotide for any indication at screening.\n2. Have Shiga toxin-producing Escherichia coli haemolytic uraemic syndrome (STEC-HUS). Test results obtained within 28 days prior to informed consent may be used.\n3. Have ADAMTS13 activity \\\u003C 10%. Test results obtained within 28 days prior to informed consent may be used.\n4. Have a severe, uncontrolled systemic bacterial or fungal infection requiring antimicrobial therapy, or a severe uncontrolled viral infection (as determined by the investigator); prophylactic antimicrobial therapy administered as standard of care is allowed.\n5. Have malignant hypertension (blood pressure \\[BP\\] \\> 99th percentile plus 5 mmHg with bilateral hemorrhages or \"cotton-wool\" exudates on fundoscopic examination).\n6. Due to conditions other than HSCT-TMA, have a poor prognosis with a life expectancy of less than 3 months in the opinion of the Investigator.\n7. If pregnant or lactating.\n8. Have received treatment with an investigational drug or device within 4 weeks of entering study.\n9. Have abnormal liver function tests defined as alanine aminotransferase (ALT) or aspartate aminotransferase (AST) \\> 5 times ULN within 28 days prior to informed consent.\n10. Have a positive test by antigen or polymerase chain reaction (PCR) for human immunodeficiency virus (HIV), if negative within 28 days prior to informed consent, the test does not need to be repeated.\n11. Patient or one or more of the patient's parents or legal guardians are is an employee or an immediate family member of Omeros, the Clinical Research Organization (CRO), an Investigator, or a study staff member.\n12. Have a known hypersensitivity to any constituent of the product.\n13. Presence of any condition that the Investigator believes would put the patient at risk.","28 Days","17 Years",{"count":203,"type":19},18,[53],"The purpose of this study is to evaluate the safety and efficacy of narsoplimab in pediatric patients with thrombotic microangiopathies (TMA) following hematopoietic stem cell transplant (HSCT).",[25,181],[59,208,209,210,211,212],"HSCT","Pediatric","BMT","OMS721","Narsoplimab","2025-03-24",{"date":215,"type":32},"2025-03-25",{"date":217,"type":32},"2023-05-01",{"date":190,"type":19},{"name":220,"class":73},"Omeros Corporation",16,{"id":223,"slug":224,"hasResults":11,"nctId":225,"briefTitle":226,"officialTitle":226,"acronym":4,"eligibilityCriteria":227,"healthyVolunteers":228,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":229,"targetDuration":231,"studyType":20,"phases":4,"briefSummary":232,"conditions":233,"keywords":248,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":250,"lastUpdatePostDateStruct":251,"startDateStruct":253,"completionDateStruct":255,"leadSponsor":257,"locationsCount":259},"100572008","longitudinal-cohort-of-thrombosis-and-hemostasis-diseases-100572008","NCT06727669","Longitudinal Cohort of Thrombosis and Hemostasis Diseases","Inclusion Criteria:\n\n* Patients who were diagnosed as thrombosis and hemostasis diseases.\n\nExclusion Criteria:\n\n* Long-term follow-up information for patients is not available for any reason, such as not being available or having a serious concomitant disease.\n* Patients with alcohol and drug addictions or mental illness affect their ability to comply with study requirements.\n* According to the investigator, there are conditions that may endanger the patient's safety or affect his\u002Fher compliance.",true,{"count":230,"type":19},3000,"5 Years","This is a multicenter, prospective, longitudinal, observational cohort study to investigate thrombosis and hemostasis diseases in Chinese patients. This study will collect basic information, diagnostic and treatment information, as well as medical expense information of patients from medical records.The incidence and risk factors of thrombosis and hemostasis diseases, the treatment methods, prognosis and medical expenses of these patients in China will be analyzed. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.",[234,118,235,236,237,238,239,25,240,241,242,243,244,245,246,247],"Immune Thrombocytopenia","Hemophilia A, Acquired","Disseminated Intravascular Coagulation","Thrombophilia","Deep Vein Thrombosis","Pulmonary Embolism","Coagulation Factor Deficiency","Hemophilia A","Hemophilia B","Hemophilia B, Acquired","Platelet Dysfunction","Arterial Thromboembolism","Bleeding Disorder","Thrombosis",[247,249],"Hemostasis","2024-12-05",{"date":252,"type":32},"2024-12-11",{"date":254,"type":32},"2024-11-01",{"date":256,"type":19},"2030-12-31",{"name":258,"class":39},"Peking University People's Hospital",5,{"id":261,"slug":262,"hasResults":11,"nctId":263,"briefTitle":264,"officialTitle":265,"acronym":4,"eligibilityCriteria":266,"healthyVolunteers":11,"sex":15,"minAge":4,"maxAge":4,"enrollmentInfo":267,"targetDuration":231,"studyType":20,"phases":4,"briefSummary":268,"conditions":269,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":270,"lastUpdatePostDateStruct":271,"startDateStruct":273,"completionDateStruct":275,"leadSponsor":277,"locationsCount":144},"100488039","construction-of-a-database-for-tma-100488039","NCT05634928","Construction of a Database for TMA","Construction of a Database for Thrombotic Microangiopathy","Inclusion Criteria:\n\n* The patient has elevated lactate dehydrogenase to more than twice the baseline value, platelets less than 20 or decreased by more than 50% within 24 hours, and more than 2% of schistocytes visible on a peripheral blood smear. Alternatively, pathological sections show extensive micro thrombosis.\n\nExclusion Criteria:\n\n* The investigator considers the subject unable or unwilling to cooperate with the study procedures.",{"count":178,"type":19},"Information about patients was collected by reviewing the Hitech case system and telephone and outpatient follow-up, and the case database was constructed by Epidata software. The sample size is expected to be 200 cases, the participating hospital is the First Affiliated Hospital of Soochow University, and the study time frame is from Dec 1, 2022, to Nov 31, 2027. The observation indexes of the study include the basic information of patients' age and gender and the clinical-related data of thrombotic microangiopathy.",[25],"2022-11-22",{"date":272,"type":32},"2022-12-02",{"date":274,"type":19},"2022-12-01",{"date":276,"type":19},"2028-11-30",{"name":278,"class":39},"The First Affiliated Hospital of Soochow University"]