[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"alpha-thalassemia\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:alpha-thalassemia":100},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,41,79],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":18,"targetDuration":21,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":29,"lastUpdatePostDateStruct":30,"startDateStruct":33,"completionDateStruct":35,"leadSponsor":37,"locationsCount":40},"100429446","international-registry-of-patients-with-alpha-thalassemia-100429446",false,"NCT04872179","International Registry of Patients With Alpha Thalassemia","International Prospective Registry of Patients With Alpha Thalassemia","ATM Registry","Inclusion Criteria:\n\n* diagnosis of alpha thalassemia (prenatal or postnatal) with genotype consistent with ATM or BHFS phenotype\n* referred to the University of California, San Francisco Fetal Treatment Center for fetal diagnosis, management and\u002For evaluation for the ongoing in utero stem cell transplantation clinical trial\n\nExclusion Criteria:\n\n\\- none","ALL",{"count":19,"type":20},500,"ESTIMATED","30 Years","OBSERVATIONAL","This is an international prospective registry of patients with Alpha thalassemia to understand the natural history of the disease and the outcomes of fetal therapies, with the overall goal of improving the prenatal management of patients with Alpha thalassemia.",[25,26,27],"Alpha-Thalassemia","Alpha Thalassemia Major","Alpha Thalassemia Minor","RECRUITING","2025-04-02",{"date":31,"type":32},"2025-04-04","ACTUAL",{"date":34,"type":32},"2017-01",{"date":36,"type":20},"2037-01",{"name":38,"class":39},"University of California, San Francisco","OTHER",1,{"id":42,"slug":43,"hasResults":11,"nctId":44,"briefTitle":45,"officialTitle":45,"acronym":4,"eligibilityCriteria":46,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":4,"enrollmentInfo":47,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":49,"conditions":50,"keywords":67,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":69,"lastUpdatePostDateStruct":70,"startDateStruct":72,"completionDateStruct":74,"leadSponsor":76,"locationsCount":40},"100557519","flower-following-longitudinal-outcomes-with-epidemiology-for-rare-diseases-100557519","NCT06539169","FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases","Inclusion Criteria:\n\n* Any person with a known or suspected rare disease, defined by their prevalence of fewer than 200,000 individuals nationwide. Diseases include but are not limited to:\n\nAlpha- or Beta- Thalassemia Amyloidosis Amyotrophic Lateral Sclerosis (ALS) Creutzfeldt-Jakob disease (CJD) Cystic Fibrosis (CF) Duchenne Muscular Dystrophy (DMD) Early-onset Alzheimer's Disease Ehlers-Danlos Syndrome (EDS) Huntington's Disease (HD) Gaucher Disease GM1 Gangliosidosis Myasthenia Gravis Pompe Disease Sickle Cell Disease Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Transthyretin Amyloid Polyneuropathy (ATTR-PN)\n\n\\- Patients or their legally-authorized representative must be willing and able to provide informed consent (and assent, if applicable). Deceased persons may participate via consent of their legally-authorized representative in accordance with applicable Federal and state laws\n\nExclusion Criteria:\n\n* Patient or LAR is unable to provide informed consent.\n* Patient resides in a country other than the United States and is unable to provide access to medical records.",{"count":48,"type":20},1000,"FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in the study by electronic consent (eConsent) and sign a medical records release to permit data collection. Medical records are accessed from institutions directly via eFax or paper fax, online from patient electronic medical record (EMR) portals, direct from DNA\u002FRNA sequencing and molecular profiling vendors, and via electronic health information exchanges. Patients and their treating physicians may also optionally provide medical records. Medical records are received in or converted to electronic\u002Fdigitized formats (CCDA, FHIR, PDF), sorted by medical record type (clinic visit, in-patient hospital, out-patient clinic, infusion and out-patient pharmacies, etc.) and made machine-readable to support data annotation, full text searches, and natural language processing (NLP) algorithms to further facilitate feature identification.",[25,51,52,53,54,55,56,57,58,59,60,61,62,63,64,65,66],"Beta-Thalassemia","Amyloidosis","Amyotrophic Lateral Sclerosis","Creutzfeld-Jakob Disease","Cystic Fibrosis","Duchenne Muscular Dystrophy","Early-Onset Alzheimer Disease","Ehlers-Danlos Syndrome","Huntington Disease","Gaucher Disease","GM1 Gangliosidosis","Myasthenia Gravis","Pompe Disease","Sickle Cell Disease","Transthyretin Amyloid Cardiomyopathy","Rare Diseases",[68],"rare diseases","2024-11-12",{"date":71,"type":32},"2024-11-14",{"date":73,"type":32},"2024-06-10",{"date":75,"type":20},"2026-06-10",{"name":77,"class":78},"xCures","INDUSTRY",{"id":80,"slug":81,"hasResults":11,"nctId":82,"briefTitle":83,"officialTitle":83,"acronym":4,"eligibilityCriteria":84,"healthyVolunteers":11,"sex":17,"minAge":85,"maxAge":86,"enrollmentInfo":87,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":89,"conditions":90,"keywords":4,"overallStatus":28,"whyStopped":4,"lastUpdateSubmitDate":91,"lastUpdatePostDateStruct":92,"startDateStruct":94,"completionDateStruct":96,"leadSponsor":98,"locationsCount":40},"100561574","genetic-profile-of-alpha-thalassemia-children-at-sohag-university-hospital--100561574","NCT06591936","Genetic Profile of Alpha Thalassemia Children at Sohag University Hospital .","Inclusion Criteria:\n\n* Acase suspecteed of alpha thalassemia\n* age between 0-18years\n* micocytic hypochromic anemia not iron deficiency anemia or Beta thalassemia\n\nExclusion Criteria:\n\n* Age more than 18 years\n* healthy children free from any chronic hematological disease\n* micocytic hypochromic anemia either iron deficiency anemia or Beta thalassemia","0 Days","18 Years",{"count":88,"type":20},50,"Genetic profile of alpha thalassemia children at sohag university hospital ,the aim to determine the prevelance , molecular character of the disorder, characterized by decreased synthesis of alpha -globin Recent work to provide mechanisms for phenotypic heterogeneity .",[25],"2024-09-08",{"date":93,"type":32},"2024-09-19",{"date":95,"type":32},"2024-08-01",{"date":97,"type":20},"2025-08",{"name":99,"class":39},"Sohag University","Alpha Thalassemia"]