[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100587944":3},{"organization":4,"armGroups":7,"interventions":18,"overallOfficials":26,"centralContacts":30,"locations":38,"responsibleParty":187,"collaborators":34,"id":189,"slug":190,"hasResults":191,"nctId":192,"briefTitle":193,"officialTitle":194,"acronym":34,"eligibilityCriteria":195,"healthyVolunteers":191,"sex":196,"minAge":197,"maxAge":198,"enrollmentInfo":199,"targetDuration":34,"studyType":202,"phases":203,"briefSummary":205,"conditions":206,"keywords":208,"overallStatus":41,"whyStopped":34,"lastUpdateSubmitDate":213,"lastUpdatePostDateStruct":214,"startDateStruct":217,"completionDateStruct":219,"leadSponsor":221,"locationsCount":222},{"fullName":5,"class":6},"Novartis","INDUSTRY",[8,14],{"label":9,"type":10,"description":11,"interventionNames":12},"LNP023-Cohort 1 (12 \u003C 18 years old)","EXPERIMENTAL","Participants (12 to \\\u003C 18 years old) will take iptacopan at the dose of 200 mg twice per day (in the morning and in the evening).",[13],"Drug: LNP023",{"label":15,"type":10,"description":16,"interventionNames":17},"LNP023 -Cohort 2 (2 to \u003C 12 years old)","Participants (2 to \\\u003C 12 years old) will be dosed based on weight at the Day 1 visit, initially. The study medication dose will be reassessed and re-adjusted as needed based on their weight at Week 12, 26, and 38.",[13],[19],{"type":20,"name":21,"description":22,"armGroupLabels":23,"otherNames":24},"DRUG","LNP023","Cohort 1-administered orally a dosing scheme of 200 mg twice-daily (two 100 mg capsules). Cohort 2- administered orally a dosing scheme based on weight at the Day 1, Week 12, 26 and 38.",[15,9],[25],"Iptacopan",[27],{"name":28,"affiliation":28,"role":29},"Novartis Pharmaceuticals","STUDY_DIRECTOR",[31,36],{"name":28,"role":32,"phone":33,"phoneExt":34,"email":35},"CONTACT","1-888-669-6682",null,"novartis.email@novartis.com",{"name":28,"role":32,"phone":37,"phoneExt":34,"email":34},"+41613241111",[39,61,78,94,106,115,124,133,140,145,156,166,177],{"facility":40,"status":41,"city":42,"state":43,"zip":44,"country":45,"countryCode":46,"cosmosGeoPoint":47,"geoPoint":52,"contacts":53},"Cancer Institute of New Jersey","RECRUITING","New Brunswick","New Jersey","08901","United States","US",{"type":48,"coordinates":49},"Point",[50,51],-74.45182,40.48622,{"lat":51,"lon":50},[54,58],{"name":55,"role":32,"phone":56,"phoneExt":34,"email":57},"Serban Morojanu","+1 732-235-7577","moroiasa@cinj.rutgers.edu",{"name":59,"role":60,"phone":34,"phoneExt":34,"email":34},"Richard DRACHTMAN","PRINCIPAL_INVESTIGATOR",{"facility":62,"status":41,"city":63,"state":64,"zip":65,"country":45,"countryCode":46,"cosmosGeoPoint":66,"geoPoint":70,"contacts":71},"Childrens Hospital of Philadelphia","Philadelphia","Pennsylvania","19104 4399",{"type":48,"coordinates":67},[68,69],-75.16362,39.95238,{"lat":69,"lon":68},[72,76],{"name":73,"role":32,"phone":74,"phoneExt":34,"email":75},"Ariel Rivera","+1 267 425 2136","riveraa12@chop.edu",{"name":77,"role":60,"phone":34,"phoneExt":34,"email":34},"Timothy Olson",{"facility":79,"status":41,"city":80,"state":81,"zip":82,"country":45,"countryCode":46,"cosmosGeoPoint":83,"geoPoint":87,"contacts":88},"St Jude Childrens Research Hospital","Memphis","Tennessee","38105",{"type":48,"coordinates":84},[85,86],-90.04898,35.14953,{"lat":86,"lon":85},[89,92],{"name":90,"role":32,"phone":34,"phoneExt":34,"email":91},"Kelsey Ray","kelsey.ray@stjude.org",{"name":93,"role":60,"phone":34,"phoneExt":34,"email":34},"Marcin Wlodarski",{"facility":95,"status":41,"city":96,"state":97,"zip":98,"country":99,"countryCode":100,"cosmosGeoPoint":101,"geoPoint":105,"contacts":34},"Novartis Investigative Site","Brasília","Federal District","70684-831","Brazil","BR",{"type":48,"coordinates":102},[103,104],-47.92972,-15.77972,{"lat":104,"lon":103},{"facility":95,"status":41,"city":107,"state":108,"zip":109,"country":99,"countryCode":100,"cosmosGeoPoint":110,"geoPoint":114,"contacts":34},"Natal","Rio Grande do Norte","59012 300",{"type":48,"coordinates":111},[112,113],-35.20944,-5.795,{"lat":113,"lon":112},{"facility":95,"status":41,"city":116,"state":117,"zip":118,"country":99,"countryCode":100,"cosmosGeoPoint":119,"geoPoint":123,"contacts":34},"Porto Alegre","Rio Grande do Sul","90035-003",{"type":48,"coordinates":120},[121,122],-51.23019,-30.03283,{"lat":122,"lon":121},{"facility":95,"status":41,"city":125,"state":126,"zip":127,"country":99,"countryCode":100,"cosmosGeoPoint":128,"geoPoint":132,"contacts":34},"Santo André","São Paulo","09090-401",{"type":48,"coordinates":129},[130,131],-46.53833,-23.66389,{"lat":131,"lon":130},{"facility":95,"status":41,"city":126,"state":126,"zip":134,"country":99,"countryCode":100,"cosmosGeoPoint":135,"geoPoint":139,"contacts":34},"01323001",{"type":48,"coordinates":136},[137,138],-46.63611,-23.5475,{"lat":138,"lon":137},{"facility":95,"status":41,"city":126,"state":126,"zip":141,"country":99,"countryCode":100,"cosmosGeoPoint":142,"geoPoint":144,"contacts":34},"04038-002",{"type":48,"coordinates":143},[137,138],{"lat":138,"lon":137},{"facility":95,"status":41,"city":146,"state":147,"zip":148,"country":149,"countryCode":150,"cosmosGeoPoint":151,"geoPoint":155,"contacts":34},"Cali","Valle del Cauca Department","760012","Colombia","CO",{"type":48,"coordinates":152},[153,154],-76.5199,3.43054,{"lat":154,"lon":153},{"facility":95,"status":41,"city":157,"state":34,"zip":158,"country":159,"countryCode":160,"cosmosGeoPoint":161,"geoPoint":165,"contacts":34},"Berlin","13353","Germany","DE",{"type":48,"coordinates":162},[163,164],13.41053,52.52437,{"lat":164,"lon":163},{"facility":95,"status":41,"city":167,"state":168,"zip":169,"country":170,"countryCode":171,"cosmosGeoPoint":172,"geoPoint":176,"contacts":34},"Genova","GE","16147","Italy","IT",{"type":48,"coordinates":173},[174,175],11.87211,45.21604,{"lat":175,"lon":174},{"facility":95,"status":41,"city":178,"state":34,"zip":179,"country":180,"countryCode":181,"cosmosGeoPoint":182,"geoPoint":186,"contacts":34},"Utrecht","3584 CX","Netherlands","NL",{"type":48,"coordinates":183},[184,185],5.12222,52.09083,{"lat":185,"lon":184},{"type":188,"investigatorFullName":34,"investigatorTitle":34,"investigatorAffiliation":34,"oldNameTitle":34,"oldOrganization":34},"SPONSOR","100587944","phase-3-study-to-assess-the-pharmacokinetics-safety-and-tolerability-of-iptacopan-in-pediatric-pnh-patients-100587944",false,"NCT06934967","Study to Assess the Pharmacokinetics, Safety, and Tolerability of Iptacopan in Pediatric PNH Patients","An Open-label, Single-arm, Multicenter, Phase 3 Study to Assess Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric PNH Patients 2 to \u003C18 Years of Age","Inclusion Criteria:\n\n* Male and female participants 2 to \\\u003C 18 years of age with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with red blood cells (RBCs) and with white blood cells granulocytes\u002Fmonocytes clone size ≥ 10%. The minimum body weight for patients in Cohort 1 is 35 kg.\n* Patients being treated with anti-C5 therapy and who have been on a stable regimen (dose and interval) for at least 6 months prior to enrollment, may be screened and enrolled in the study and switched to iptacopan irrespective of their anemia and hemolysis status, at the discretion of the Principal Investigator.\n* Patients who are anti-C5 treatment naive: mean hemoglobin level \\\u003C 10 g\u002FdL confirmed by central laboratory assessment during screening.\n* Patients who are anti-C5 treatment naive: lactate dehydrogenase (LDH) \\> 1.5 × upper limit of normal (ULN) documented by at least 2 laboratory measurements 2 to 6 weeks apart during the screening period, one of which is to be done by the central lab.\n* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster is required, vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated.\n* Vaccination against Haemophilus influenzae is recommended, according to local guidelines, at least 2 weeks before iptacopan.\n\nExclusion Criteria:\n\n* History of hypersensitivity to the study drug or its excipients or to drugs of similar chemical classes.\n* Known or suspected hereditary complement deficiency at screening.\n* History of hematopoietic stem cell transplantation (HSCT) or scheduled for HSCT within 52 weeks from enrollment into the study (Day 1).\n* Patients with laboratory evidence of bone marrow failure (reticulocytes \\\u003C 100 x 10 to the ninth\u002FL; platelets \\\u003C 30 × 10 to the ninth\u002FL; neutrophils \\\u003C 0.5 × 10 to the ninth\u002FL).\n* Active systemic bacterial, viral (including COVID-19), or fungal infection within 14 days prior to study drug administration.\n* Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration.\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.","ALL","2 Years","18 Years",{"count":200,"type":201},12,"ESTIMATED","INTERVENTIONAL",[204],"PHASE3","The purpose of this open-label, single arm, multicenter, phase 3 study is to assess the pharmacokinetics of iptacopan in pediatric patients and to assess whether iptacopan is safe and well tolerated when used for the treatment of pediatric paroxysmal nocturnal hemoglobinuria (PNH) patients 2 to \\\u003C 18 years of age.",[207],"Paroxysmal Nocturnal Hemoglobinuria (PNH)",[209,210,211,212],"Iptacopan,","PNH,","hemoglobin,","anemia","2026-04-13",{"date":215,"type":216},"2026-04-16","ACTUAL",{"date":218,"type":216},"2025-10-28",{"date":220,"type":201},"2031-12-19",{"name":28,"class":6},13]