[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"thrombotic-thrombocytopenic-purpura-ttp\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:thrombotic-thrombocytopenic-purpura-ttp":23},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,5,0,[8,39,60,84,103],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":24,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":27,"lastUpdatePostDateStruct":28,"startDateStruct":31,"completionDateStruct":33,"leadSponsor":35,"locationsCount":38},"100620090","a-study-in-children-and-adults-with-congenital-thrombotic-thrombocytopenic-purpura-cttp-treated-with-adzynma-100620090",false,"NCT07353099","A Study in Children and, Adults With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma","A Post-Authorization Safety Study (PASS) to Further Evaluate Real-World Safety in Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated With Adzynma","Inclusion criteria:\n\n1. Is diagnosed with cTTP.\n2. Had received commercially available Adzynma treatment for cTTP within the eligibility period of which there must be a recorded date and dose of at least one Adzynma administration in their medical chart.\n3. Provides a signed informed consent form (informed consent form \\[ICF\\]; or assent and consent forms, if applicable), in accordance with local ethical and institutional requirements.\n\nExclusion criteria:\n\n1. Has a history or presence of a functional ADAMTS13 inhibitor (that is, neutralizing antibodies to ADAMTS13\u002FrADAMTS13).\n2. Has concurrent use of an investigational drug or is enrolled in another clinical trial at the time of index Adzynma infusion.","ALL",{"count":18,"type":19},50,"ESTIMATED","OBSERVATIONAL","Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body's ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13.\n\nThe main aim of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant.\n\nOnly data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during this study.",[23],"Thrombotic Thrombocytopenic Purpura (TTP)",[25],"Drug Therapy","RECRUITING","2026-06-09",{"date":29,"type":30},"2026-06-11","ACTUAL",{"date":32,"type":30},"2026-05-26",{"date":34,"type":19},"2030-04-01",{"name":36,"class":37},"Takeda","INDUSTRY",7,{"id":40,"slug":41,"hasResults":11,"nctId":42,"briefTitle":43,"officialTitle":44,"acronym":4,"eligibilityCriteria":45,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":46,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":48,"conditions":49,"keywords":50,"overallStatus":51,"whyStopped":4,"lastUpdateSubmitDate":52,"lastUpdatePostDateStruct":53,"startDateStruct":55,"completionDateStruct":57,"leadSponsor":59,"locationsCount":4},"100626000","a-study-to-learn-more-about-the-treatment-of-people-with-congenital-thrombotic-thrombocytopenic-purpura-cttp-who-received-recombinant-adamts13-radamts13-as-part-of-the-early-access-program-100626000","NCT07429942","A Study to Learn More About the Treatment of People With Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Who Received Recombinant ADAMTS13 (rADAMTS13) as Part of the Early Access Program","Treatment and Management of Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP): An International, Multi-center Retrospective Chart Review of Patients in the Early Access Program (EAP) Treated With Recombinant ADAMTS13 (rADAMTS13)","Inclusion criteria for the rADAMTS13 EAP are:\n\n* Participants of any age can participate who have a confirmed diagnosis of severe congenital ADAMTS13 deficiency or cTTP.\n* Participants must be on preventative or prophylactic treatment for cTTP or must have had at least one TTP event in the past.\n* Participants must have no other treatment options available (this includes other clinical studies for cTTP).\n\nThe inclusion criteria for this retrospective chart review are:\n\n* Pediatric and adult participants (no age restrictions) with cTTP, treated with rADAMTS13 via the EAP, who received at least two administrations of rADAMTS13 and who have provided consent (or the legal guardians) to participate in this retrospective chart review.\n* As per local regulations, evidence of a personally signed (or signed by a legally acceptable representative) and dated informed consent form\u002Finformed assent form (ICF\u002FIAF) indicating that the participant (or their legal guardian) has been informed of all pertinent aspects of the retrospective chart review or an approval to process data without informed consent granted by an institutional review board\u002Findependent ethics committee (IRB\u002FIEC)) Participants included in the EAP who were\u002Fare transitioned to the commercially available product will have their data abstracted for the duration of their participation in the EAP as well as when they received the commercially available product until the end of chart abstraction.\n\nThere are no additional exclusion criteria for this chart review.",{"count":47,"type":19},94,"Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by inherited changes in the ADAMTS13 gene that reduce the body's ability to produce the ADAMTS13 enzyme. ADAMTS13 normally cleaves ultra-large multimers of a protein called von Willebrand factor (VWF). In cTTP, low ADAMTS13 activity allows these ultra-large VWF multimers to build up and promote blood clot formation in small blood vessels. These clots can restrict blood flow to vital organs and lead to serious complications.\n\nRecombinant ADAMTS13 (rADAMTS13) is a manufactured form of human ADAMTS13 designed to replace the missing enzyme and restore ADAMTS13 activity.\n\nThis study aims to describe the impact of cTTP on participants before and after treatment with rADAMTS13. It will also evaluate participants' health outcomes after treatment and describe treatment patterns before and after rADAMTS13, including whether treatment was used to prevent or treat TTP episodes, how often it was given, the amount received, and others. In addition, the study will describe pregnancies and outcomes for the mother and baby before and during treatment with rADAMTS13.\n\nOnly data already available in the medical records of the people who received rADAMTS13 through Takeda's early access program (EAP) for cTTP will be collected and reviewed in this study.",[23],[25],"NOT_YET_RECRUITING","2026-05-27",{"date":54,"type":30},"2026-05-29",{"date":56,"type":19},"2026-06-30",{"date":58,"type":19},"2026-07-06",{"name":36,"class":37},{"id":61,"slug":62,"hasResults":11,"nctId":63,"briefTitle":64,"officialTitle":65,"acronym":66,"eligibilityCriteria":67,"healthyVolunteers":11,"sex":16,"minAge":68,"maxAge":4,"enrollmentInfo":69,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":71,"conditions":72,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":76,"completionDateStruct":78,"leadSponsor":80,"locationsCount":83},"100588781","immunological-aspect-of-thrombotic-thrombocytopenic-purpura-ttp-100588781","NCT06945861","Immunological Aspect of Thrombotic Thrombocytopenic Purpura (TTP)","Immunological Aspects of Thrombotic Thrombocytopenic Purpura (TTP): Characterisation of B and T Lymphocytes Specific for the ADAMTS13 Autoantigen and Therapeutic Implications","Lympho-PTT","Inclusion Criteria:\n\n* age over 18\n* patients with TTP at any stage of diagnosis (acute phase, lasting remission or not, relapse)\n* patients undergoing internal medicine at Rouen University Hospital\n* people who have read and understood the information letter\n* membership of a social security scheme\n\nExclusion Criteria:\n\n\\- a person deprived of liberty by an administrative or judicial decision or a person placed under court protection\u002Fguardianship or guardianship","18 Years",{"count":70,"type":19},44,"The general objective of the proposed project is to characterise phenotypically and functionally ADAMTS13-specific memory B lymphocytes and autoreactive T lymphocytes, in particular follicular helper T lymphocytes, in the acute phase of the disease, but also during its progression after treatment. The aim is to highlight their contribution to the initial pathogenic process, their evolution under treatment, and also their involvement in patients who are refractory to immunosuppressive therapies and during relapses. The aim of this project is to identify early phenotypic or functional parameters that are predictive of relapse and that can be used for personalised optimisation of treatment to maintain remission.",[23],"2026-04-28",{"date":75,"type":30},"2026-05-04",{"date":77,"type":30},"2023-05-11",{"date":79,"type":19},"2028-05-11",{"name":81,"class":82},"University Hospital, Rouen","OTHER",1,{"id":85,"slug":86,"hasResults":11,"nctId":87,"briefTitle":88,"officialTitle":89,"acronym":4,"eligibilityCriteria":90,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":91,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":93,"conditions":94,"keywords":4,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":95,"lastUpdatePostDateStruct":96,"startDateStruct":98,"completionDateStruct":100,"leadSponsor":102,"locationsCount":83},"100550016","a-survey-of-recombinant-adamts13-in-participants-with-congenital-thrombotic-thrombocytopenic-purpura-100550016","NCT06441578","A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura","Special Drug Use Surveillance of ADZYNMA Intravenous 1500 (All-Case Investigation)","Inclusion Criteria:\n\n\\- All participants with congenital thrombotic thrombocytopenic purpura (cTTP), treated with recombinant ADAMTS13\n\nExclusion Criteria:\n\n\\- None",{"count":92,"type":19},40,"This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study.\n\nThe main aim of the study is to check for side effects related from recombinant ADAMTS13 and to check if recombinant ADAMTS13 improves or prevents cTTP.\n\nDuring the study, participants with cTTP will take recombinant ADAMTS13 intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from recombinant ADAMTS13 for 18 months.",[23],"2025-10-02",{"date":97,"type":30},"2025-10-07",{"date":99,"type":30},"2024-05-30",{"date":101,"type":19},"2032-09-30",{"name":36,"class":37},{"id":104,"slug":105,"hasResults":11,"nctId":106,"briefTitle":107,"officialTitle":108,"acronym":109,"eligibilityCriteria":110,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":111,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":113,"conditions":114,"keywords":122,"overallStatus":26,"whyStopped":4,"lastUpdateSubmitDate":140,"lastUpdatePostDateStruct":141,"startDateStruct":143,"completionDateStruct":145,"leadSponsor":147,"locationsCount":83},"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":112,"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.",[23,115,116,117,118,119,120,121],"Immune Thrombotic Thrombocytopenic Purpura","Thrombotic Microangiopathies","Microangiopathy, Thromboic","Cardiovascular Diseases","Autoimmune Diseases","Rare Diseases","Neurological Manifestations",[123,115,124,125,126,127,128,129,130,131,132,133,134,135,136,137,138,139],"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":142,"type":30},"2025-10-03",{"date":144,"type":30},"2024-12-02",{"date":146,"type":19},"2028-12",{"name":148,"class":82},"Assistance Publique - Hôpitaux de Paris"]