[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100497332":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":12,"centralContacts":16,"locations":27,"responsibleParty":48,"collaborators":50,"id":53,"slug":54,"hasResults":55,"nctId":56,"briefTitle":57,"officialTitle":58,"acronym":10,"eligibilityCriteria":59,"healthyVolunteers":55,"sex":60,"minAge":10,"maxAge":10,"enrollmentInfo":61,"targetDuration":64,"studyType":65,"phases":10,"briefSummary":66,"conditions":67,"keywords":69,"overallStatus":29,"whyStopped":10,"lastUpdateSubmitDate":73,"lastUpdatePostDateStruct":74,"startDateStruct":77,"completionDateStruct":79,"leadSponsor":81,"locationsCount":82},{"fullName":5,"class":6},"Aplastic Anemia and MDS International Foundation","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":10},"PNH Patients",null,"Individuals of any age with a confirmed diagnosis of PNH or diagnosis consistent with PNH are eligible for inclusion.",[13],{"name":14,"affiliation":5,"role":15},"Alice Houk, MS","PRINCIPAL_INVESTIGATOR",[17,22],{"name":14,"role":18,"phone":19,"phoneExt":20,"email":21},"CONTACT","3012797202","101","houk@aamds.org",{"name":23,"role":18,"phone":24,"phoneExt":25,"email":26},"Elizabeth Kottke","475.289.6855","275","ekottke@rarediseases.org",[28],{"facility":5,"status":29,"city":30,"state":31,"zip":32,"country":33,"countryCode":34,"cosmosGeoPoint":35,"geoPoint":40,"contacts":41},"RECRUITING","Bethesda","Maryland","20814","United States","US",{"type":36,"coordinates":37},"Point",[38,39],-77.10026,38.98067,{"lat":39,"lon":38},[42,47],{"name":43,"role":18,"phone":44,"phoneExt":45,"email":46},"Nyaradzo Sirewu, MPH","301-279-7202","126","sirewu@aamds.org",{"name":14,"role":15,"phone":10,"phoneExt":10,"email":10},{"type":49,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR",[51],{"name":52,"class":6},"National Organization for Rare Disorders","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":62,"type":63},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.",[68],"Paroxysmal Nocturnal Hemoglobinuria",[70,71,72,68],"PNH","Registry","Patient Registry","2025-02-18",{"date":75,"type":76},"2025-02-20","ACTUAL",{"date":78,"type":76},"2021-05-06",{"date":80,"type":63},"2031-05-06",{"name":5,"class":6},1]