[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100442345":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":22,"centralContacts":31,"locations":37,"responsibleParty":125,"collaborators":127,"id":130,"slug":131,"hasResults":132,"nctId":133,"briefTitle":134,"officialTitle":135,"acronym":10,"eligibilityCriteria":136,"healthyVolunteers":132,"sex":137,"minAge":10,"maxAge":10,"enrollmentInfo":138,"targetDuration":10,"studyType":141,"phases":10,"briefSummary":142,"conditions":143,"keywords":147,"overallStatus":40,"whyStopped":10,"lastUpdateSubmitDate":149,"lastUpdatePostDateStruct":150,"startDateStruct":153,"completionDateStruct":155,"leadSponsor":157,"locationsCount":158},{"fullName":5,"class":6},"RECORDATI GROUP","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Male and Female Adult and Pediatric Participants",null,"Patients treated with Carbaglu for the treatment for hyperammonemia due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA)",[13],"Drug: Carglumic Acid",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"DRUG","Carglumic Acid","Current or previous treatment with Carbaglu, the dose of Carbaglu® prescribed will be determined by the investigator for each individual patient.",[9],[21],"Carbaglu®",[23,27],{"name":24,"affiliation":25,"role":26},"William Ludlum, MD","Recordati Rare Diseases Inc","STUDY_DIRECTOR",{"name":28,"affiliation":29,"role":30},"Nicholas Ah Mew, MD","Children's National Research Institute","PRINCIPAL_INVESTIGATOR",[32],{"name":33,"role":34,"phone":35,"phoneExt":10,"email":36},"Anne Marie Cesario","CONTACT","908-849-4907","cesario.a@recordati.com",[38,58,75,92,109],{"facility":39,"status":40,"city":41,"state":42,"zip":43,"country":44,"countryCode":45,"cosmosGeoPoint":46,"geoPoint":51,"contacts":52},"Children's National Hospital","RECRUITING","Washington D.C.","District of Columbia","20010","United States","US",{"type":47,"coordinates":48},"Point",[49,50],-77.03637,38.89511,{"lat":50,"lon":49},[53,57],{"name":54,"role":34,"phone":55,"phoneExt":10,"email":56},"Kara Simpson","(202) 545-2503","ksimpson@childrensnational.org",{"name":28,"role":30,"phone":10,"phoneExt":10,"email":10},{"facility":59,"status":40,"city":60,"state":61,"zip":62,"country":44,"countryCode":45,"cosmosGeoPoint":63,"geoPoint":67,"contacts":68},"University of South Florida","Tampa","Florida","33606",{"type":47,"coordinates":64},[65,66],-82.45843,27.94752,{"lat":66,"lon":65},[69,73],{"name":70,"role":34,"phone":71,"phoneExt":10,"email":72},"Farideh Oberheu","(813) 250-2292","faljalla@usf.edu",{"name":74,"role":30,"phone":10,"phoneExt":10,"email":10},"Amarilis Sanchez-Valle, MD",{"facility":76,"status":40,"city":77,"state":78,"zip":79,"country":44,"countryCode":45,"cosmosGeoPoint":80,"geoPoint":84,"contacts":85},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"type":47,"coordinates":81},[82,83],-87.65005,41.85003,{"lat":83,"lon":82},[86,90],{"name":87,"role":34,"phone":88,"phoneExt":10,"email":89},"Michael Sawin","(312) 227-2816","Msawin@luriechildrens.org",{"name":91,"role":30,"phone":10,"phoneExt":10,"email":10},"Joshua Baker, MD",{"facility":93,"status":40,"city":94,"state":95,"zip":96,"country":44,"countryCode":45,"cosmosGeoPoint":97,"geoPoint":101,"contacts":102},"Riley Children's Hospital","Indianapolis","Indiana","46202",{"type":47,"coordinates":98},[99,100],-86.15804,39.76838,{"lat":100,"lon":99},[103,107],{"name":104,"role":34,"phone":105,"phoneExt":10,"email":106},"Susan Romie","(317) 278-6650","sromie@iu.edu",{"name":108,"role":30,"phone":10,"phoneExt":10,"email":10},"Melissa Lah, MD",{"facility":110,"status":40,"city":111,"state":111,"zip":112,"country":44,"countryCode":45,"cosmosGeoPoint":113,"geoPoint":117,"contacts":118},"Icahn School of Medicine at Mt. Sinai","New York","10029",{"type":47,"coordinates":114},[115,116],-74.00597,40.71427,{"lat":116,"lon":115},[119,123],{"name":120,"role":34,"phone":121,"phoneExt":10,"email":122},"Colleen Donnelly","(212) 241-5983","Colleen.donnelly@mssm.edu",{"name":124,"role":30,"phone":10,"phoneExt":10,"email":10},"Margo Breilyn, MD",{"type":126,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR",[128],{"name":129,"class":6},"Target PharmaSolutions, Inc.","100442345","an-observational-study-of-carbaglu-for-the-treatment-of-mma-and-pa-in-adults-and-pediatrics-100442345",false,"NCT05040178","An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics","A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations","Inclusion Criteria:\n\n1. Provision of signed and dated informed consent\u002Fassent form\n2. Prescribed and treated with Carbaglu®\n3. Have an established diagnosis of PA or MMA defined as follows:\n\n   * Diagnosed with PA by semi quantitative urine organic acid analysis, defined as presence of elevated methylcitric acid and normal methylmalonic acid levels and no evidence of biotin related disorders in the organic acid analysis; OR\n   * Diagnosed with MMA by semi quantitative urine organic acid analysis, defined as elevation of methylmalonic acid and no evidence of vitamin B12 dependent disorder on plasma amino acid analysis (vitamin B12 dependency is defined by documented vitamin B12 responsiveness).\n\nAND\u002FOR\n\n* Confirmation by molecular genetic testing\n\nExclusion Criteria:\n\n* None","ALL",{"count":139,"type":140},20,"ESTIMATED","OBSERVATIONAL","To obtain short-term and long-term clinical safety information, in pediatric and adult patients with PA and MMA treated with Carbaglu®.",[144,145,146],"Hyperammonemia","Methylmalonic Acidemia","Propionic Acidemia",[148],"PA & MMA","2025-04-10",{"date":151,"type":152},"2025-04-15","ACTUAL",{"date":154,"type":152},"2022-06-30",{"date":156,"type":140},"2032-06-30",{"name":5,"class":6},5]