[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Aplastic Anemia and MDS International Foundation\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":41},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":20,"studyType":21,"phases":4,"briefSummary":22,"conditions":23,"keywords":25,"overallStatus":29,"whyStopped":4,"lastUpdateSubmitDate":30,"lastUpdatePostDateStruct":31,"startDateStruct":34,"completionDateStruct":36,"leadSponsor":38,"locationsCount":5},"100497332","global-pnh-patient-registry-100497332",false,"NCT05755867","Global PNH Patient Registry","Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry","Inclusion Criteria:\n\n\\- Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion. PNH is defined as a genetic mutation in the PIG-A gene.\n\nIndividuals must be willing to provide informed consent. Participants can be:\n\n* legal adult participants who are able to provide their own consent;\n* children and adults unable to provide their own consent, for whom consent must be provided by a Legally Authorized Representative (LAR) who is a legal adult.\n* Individuals must have at least periodic access to the internet and be able to comply with web-based study procedures and data collections\n\nExclusion Criteria:\n\n* Individuals not able to read and understand English.","ALL",{"count":18,"type":19},500,"ESTIMATED","10 Years","OBSERVATIONAL","The primary aim of the Global Paroxysmal Nocturnal Hemoglobinuria (PNH) Patient Registry is to conduct a prospectively-planned and efficient natural history study that will result in a more comprehensive understanding of the disease and its course and pace over time. Other registry objectives include the following:\n\n* Provide a convenient online platform for participants (or caregivers) to self-report cases of PNH.\n* Develop a communications registry within the Global PNH Patient registry (e.g., to notify patients of research studies and clinical trials).\n* Characterize and describe the Global PNH population as a whole, enhancing the understanding of disease prevalence and phenotype as well as the rate of progression of disease characteristics.\n* Assist the PNH community with the development of recommendations and standards of care.\n* Be a case-finding resource to be used for researchers who seek to study the pathophysiology of PNH, retrospectively collate intervention outcomes, and design prospective trials of novel treatments.",[24],"Paroxysmal Nocturnal Hemoglobinuria",[26,27,28,24],"PNH","Registry","Patient Registry","RECRUITING","2025-02-18",{"date":32,"type":33},"2025-02-20","ACTUAL",{"date":35,"type":33},"2021-05-06",{"date":37,"type":19},"2031-05-06",{"name":39,"class":40},"Aplastic Anemia and MDS International Foundation","OTHER",""]