[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100592547":3},{"organization":4,"outcomesModule":7,"designInfo":29,"detailedDescription":35,"studyPopulation":28,"armGroups":36,"interventions":43,"overallOfficials":50,"centralContacts":54,"locations":61,"responsibleParty":292,"collaborators":28,"id":294,"slug":295,"hasResults":296,"nctId":297,"briefTitle":298,"officialTitle":299,"acronym":28,"eligibilityCriteria":300,"healthyVolunteers":296,"sex":301,"minAge":302,"maxAge":303,"enrollmentInfo":304,"targetDuration":28,"studyType":307,"phases":308,"briefSummary":310,"conditions":311,"keywords":313,"overallStatus":64,"whyStopped":28,"lastUpdateSubmitDate":323,"lastUpdatePostDateStruct":324,"startDateStruct":327,"completionDateStruct":329,"leadSponsor":331,"locationsCount":332},{"fullName":5,"class":6},"Novartis","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":28},[9],{"measure":10,"description":11,"timeFrame":12},"Log-transformed ratio to Baseline in UPCR (based on FMV)","UPCR is measured based on the geometric mean of 2 FMVs obtained preceding each scheduled visit.","Baseline, Week 38",[14,18,21,24],{"measure":15,"description":16,"timeFrame":17},"Pharmacokinetic Parameter Cmax in Plasma","Cmax is the maximum (peak) observed plasma drug concentration after dose administration (mass x volume-1)","Week 12 (Pre-dose (0), 2, 4, 6, and 8 hours post-dose)",{"measure":19,"description":20,"timeFrame":17},"Pharmacokinetic Parameter AUClast in Plasma","AUClast is the AUC from time zero to the last measurable concentration sampling time (tlast) (mass x time x volume-1)",{"measure":22,"description":23,"timeFrame":17},"Pharmacokinetic Parameter AUCtau in Plasma","AUCtau is the AUC calculated to the end of a dosing interval (tau) at steady-state (amount x time x volume-1)",{"measure":25,"description":26,"timeFrame":27},"Ctrough concentrations","Pre-dose drug concentration","Week 4, Week 12 and Week 38",null,{"allocation":30,"interventionModel":31,"interventionModelDescription":28,"primaryPurpose":32,"observationalModel":28,"timePerspective":28,"maskingInfo":33},"NA","SINGLE_GROUP","TREATMENT",{"masking":34,"maskingDescription":28,"whoMasked":28},"NONE","The study will enroll approximately 34 pediatric patients with a diagnosis of primary IgAN by kidney biopsy performed within 3 years of Screening with \\\u003C 50% tubulointerstitial fibrosis and \\\u003C25% crescents and not previously treated with immunosuppressive or other immunomodulatory agents within 90 days (or 180 days for rituximab) prior to first study drug administration.\n\nThe study comprises the following periods:\n\n* Screening Period (up to 11 weeks)\n* Run-in Period (2 weeks): This period will be approximately 14 days prior to the Enrollment Visit (Day 1) to obtain Baseline assessments, including two UPCR FMV measurements (required prior to Day 1).\n* Treatment Period ( 52 weeks): The study will enroll approximately 34 pediatric patients in a staggered manner. The approach will be to first enroll Cohort 1 (12 to \\\u003C 18 years of age) to collect safety data (including CV surveillance data), tolerability data, PK data and biomarker data for up to 52 weeks (EOS). Cohort 1 participants will be enrolled in a single group of 22 participants. Cohort 2 (2 to \\\u003C 12 years of age) participants will be enrolled in 2 groups with a total 12 participants.\n* Follow-up Period (1 week): Participants who completed the Week 52 (EOS) visit and meet all the eligibility criteria may be enrolled in a rollover extension program (REP) in which they will receive open-label iptacopan. Participants who do not enroll in the REP or do not complete Day 1 of the REP within 7 days of the EOS, should be contacted 7 days after the EOS visit for AE and SAE monitoring and any AEs reported by the participant should be recorded in the study case report form (CRF).",[37],{"label":38,"type":39,"description":40,"interventionNames":41},"iptacopan","EXPERIMENTAL","Participants in Cohort 1 (12 to \\\u003C 18 years old) will receive iptacopan at the dose of 200 mg twice per day. Participants in Cohort 2 (2 to \\\u003C 12 years old) will receive iptacopan at a dose tbd.",[42],"Drug: iptacopan",[44],{"type":45,"name":38,"description":46,"armGroupLabels":47,"otherNames":48},"DRUG","Cohort 1 (12 to \\\u003C 18 years of age): Iptacopan 200 mg b.i.d.(twice daily) Cohort 2 (2 to \\\u003C 12 years old): Dosing tbd",[38],[49],"LNP023",[51],{"name":52,"affiliation":52,"role":53},"Novartis Pharmaceuticals","STUDY_DIRECTOR",[55,59],{"name":52,"role":56,"phone":57,"phoneExt":28,"email":58},"CONTACT","1-888-669-6682","novartis.email@novartis.com",{"name":52,"role":56,"phone":60,"phoneExt":28,"email":28},"+41613241111",[62,84,101,117,134,150,162,173,182,193,201,210,220,228,236,244,255,264,273,282],{"facility":63,"status":64,"city":65,"state":66,"zip":67,"country":68,"countryCode":69,"cosmosGeoPoint":70,"geoPoint":75,"contacts":76},"Valley Childrens Hospital","RECRUITING","Madera","California","93636","United States","US",{"type":71,"coordinates":72},"Point",[73,74],-120.06072,36.96134,{"lat":74,"lon":73},[77,81],{"name":78,"role":56,"phone":79,"phoneExt":28,"email":80},"Tania Jimenez","+1 559 486 2000","tjimenez@valleychildrens.org",{"name":82,"role":83,"phone":28,"phoneExt":28,"email":28},"Nianzhou Xiao","PRINCIPAL_INVESTIGATOR",{"facility":85,"status":64,"city":86,"state":87,"zip":88,"country":68,"countryCode":69,"cosmosGeoPoint":89,"geoPoint":93,"contacts":94},"Childrens Hospital Colorado","Aurora","Colorado","80045",{"type":71,"coordinates":90},[91,92],-104.83192,39.72943,{"lat":92,"lon":91},[95,99],{"name":96,"role":56,"phone":97,"phoneExt":28,"email":98},"Claire Giachino","+1 720 777 1234","claire.giachino@childrenscolorado.org",{"name":100,"role":83,"phone":28,"phoneExt":28,"email":28},"Bradley Dixon",{"facility":102,"status":64,"city":103,"state":104,"zip":105,"country":68,"countryCode":69,"cosmosGeoPoint":106,"geoPoint":110,"contacts":111},"Univ of Florida Shands Division","Gainesville","Florida","32610-0224",{"type":71,"coordinates":107},[108,109],-82.32483,29.65163,{"lat":109,"lon":108},[112,115],{"name":113,"role":56,"phone":28,"phoneExt":28,"email":114},"Andrew Sokolow","asokolow@peds.ufl.edu",{"name":116,"role":83,"phone":28,"phoneExt":28,"email":28},"Lawrence Shoemaker",{"facility":118,"status":64,"city":119,"state":120,"zip":121,"country":68,"countryCode":69,"cosmosGeoPoint":122,"geoPoint":126,"contacts":127},"Childrens Hospital of Philadelphia","Philadelphia","Pennsylvania","19104-4399",{"type":71,"coordinates":123},[124,125],-75.16362,39.95238,{"lat":125,"lon":124},[128,132],{"name":129,"role":56,"phone":130,"phoneExt":28,"email":131},"Catherine Justice","+1 267 426 9338","justicec@chop.edu",{"name":133,"role":83,"phone":28,"phoneExt":28,"email":28},"Rebecca Scobell",{"facility":135,"status":64,"city":136,"state":137,"zip":138,"country":68,"countryCode":69,"cosmosGeoPoint":139,"geoPoint":143,"contacts":144},"Prim Childrens Hosp Inv Pharm","Salt Lake City","Utah","84113",{"type":71,"coordinates":140},[141,142],-111.89105,40.76078,{"lat":142,"lon":141},[145,148],{"name":146,"role":56,"phone":28,"phoneExt":28,"email":147},"Gabriella Gourdin","gabriella.gourdin@hsc.utah.edu",{"name":149,"role":83,"phone":28,"phoneExt":28,"email":28},"Raoul Nelson",{"facility":151,"status":64,"city":152,"state":153,"zip":154,"country":155,"countryCode":156,"cosmosGeoPoint":157,"geoPoint":161,"contacts":28},"Novartis Investigative Site","Córdoba","Córdoba Province","X5016KEH","Argentina","AR",{"type":71,"coordinates":158},[159,160],-64.18853,-31.40648,{"lat":160,"lon":159},{"facility":151,"status":64,"city":163,"state":164,"zip":165,"country":166,"countryCode":167,"cosmosGeoPoint":168,"geoPoint":172,"contacts":28},"Brisbane","Queensland","4101","Australia","AU",{"type":71,"coordinates":169},[170,171],153.02809,-27.46794,{"lat":171,"lon":170},{"facility":151,"status":64,"city":174,"state":175,"zip":176,"country":166,"countryCode":167,"cosmosGeoPoint":177,"geoPoint":181,"contacts":28},"Nedlands","Western Australia","6009",{"type":71,"coordinates":178},[179,180],115.8073,-31.98184,{"lat":180,"lon":179},{"facility":151,"status":64,"city":183,"state":184,"zip":185,"country":186,"countryCode":187,"cosmosGeoPoint":188,"geoPoint":192,"contacts":28},"Hangzhou","Zhejiang","310052","China","CN",{"type":71,"coordinates":189},[190,191],120.16142,30.29365,{"lat":191,"lon":190},{"facility":151,"status":64,"city":194,"state":28,"zip":195,"country":186,"countryCode":187,"cosmosGeoPoint":196,"geoPoint":200,"contacts":28},"Beijing","100034",{"type":71,"coordinates":197},[198,199],116.39723,39.9075,{"lat":199,"lon":198},{"facility":151,"status":64,"city":202,"state":28,"zip":203,"country":202,"countryCode":204,"cosmosGeoPoint":205,"geoPoint":209,"contacts":28},"Hong Kong","999077","HK",{"type":71,"coordinates":206},[207,208],114.17469,22.27832,{"lat":208,"lon":207},{"facility":151,"status":64,"city":211,"state":28,"zip":212,"country":213,"countryCode":214,"cosmosGeoPoint":215,"geoPoint":219,"contacts":28},"Beersheba","8457108","Israel","IL",{"type":71,"coordinates":216},[217,218],34.7913,31.25181,{"lat":218,"lon":217},{"facility":151,"status":64,"city":221,"state":28,"zip":222,"country":213,"countryCode":214,"cosmosGeoPoint":223,"geoPoint":227,"contacts":28},"Haifa","3109601",{"type":71,"coordinates":224},[225,226],34.99928,32.81303,{"lat":226,"lon":225},{"facility":151,"status":64,"city":229,"state":28,"zip":230,"country":213,"countryCode":214,"cosmosGeoPoint":231,"geoPoint":235,"contacts":28},"Jerusalem","9103102",{"type":71,"coordinates":232},[233,234],35.21633,31.76904,{"lat":234,"lon":233},{"facility":151,"status":64,"city":237,"state":28,"zip":238,"country":213,"countryCode":214,"cosmosGeoPoint":239,"geoPoint":243,"contacts":28},"Petah Tikva","4920235",{"type":71,"coordinates":240},[241,242],34.88747,32.08707,{"lat":242,"lon":241},{"facility":151,"status":64,"city":245,"state":246,"zip":247,"country":248,"countryCode":249,"cosmosGeoPoint":250,"geoPoint":254,"contacts":28},"Toyoake","Aichi-ken","4701192","Japan","JP",{"type":71,"coordinates":251},[252,253],136.99931,35.038,{"lat":253,"lon":252},{"facility":151,"status":64,"city":256,"state":257,"zip":258,"country":248,"countryCode":249,"cosmosGeoPoint":259,"geoPoint":263,"contacts":28},"Kurume","Fukuoka","8300011",{"type":71,"coordinates":260},[261,262],130.51667,33.31667,{"lat":262,"lon":261},{"facility":151,"status":64,"city":265,"state":266,"zip":267,"country":248,"countryCode":249,"cosmosGeoPoint":268,"geoPoint":272,"contacts":28},"Ōtsu","Shiga","520-2192",{"type":71,"coordinates":269},[270,271],135.86667,35,{"lat":271,"lon":270},{"facility":151,"status":64,"city":274,"state":275,"zip":276,"country":248,"countryCode":249,"cosmosGeoPoint":277,"geoPoint":281,"contacts":28},"Fuchū","Tokyo","1838561",{"type":71,"coordinates":278},[279,280],139.48216,35.67452,{"lat":280,"lon":279},{"facility":151,"status":64,"city":283,"state":28,"zip":284,"country":285,"countryCode":286,"cosmosGeoPoint":287,"geoPoint":291,"contacts":28},"Riyadh","11211","Saudi Arabia","SA",{"type":71,"coordinates":288},[289,290],46.72185,24.68773,{"lat":290,"lon":289},{"type":293,"investigatorFullName":28,"investigatorTitle":28,"investigatorAffiliation":28,"oldNameTitle":28,"oldOrganization":28},"SPONSOR","100592547","phase-3-study-to-assess-the-efficacy-pharmacokinetics-safety-and-tolerability-of-iptacopan-in-pediatric-patients-with-primary-igan-100592547",false,"NCT06994845","Study to Assess the Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients With Primary IgAN","A Single-arm, Multicenter, Phase III Study to Assess Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients of 2 to \u003C18 Years of Age With Primary Immunoglobulin A Nephropathy (IgAN)","Inclusion Criteria:\n\n* Male and female participants 2 to \\\u003C 18 years of age as of Day 1.\n* eGFR ≥ 30 mL\u002Fmin\u002F1.73m2 where eGFR is calculated using the modified Schwartz formula at Screening and confirmed during the Run-in Period.\n* Kidney biopsy-proven primary IgAN\\*, with biopsy performed within 3 years of Screening with \\\u003C 50% tubulointerstitial fibrosis and \\\u003C 25% crescents. In case a kidney biopsy within 3 years from Screening is not available, a kidney biopsy may be performed if it is part of the planned diagnostic approach and clinical management of the participant.\n\n  \\* Note: Primary IgAN is defined as any IgAN that is proven by biopsy showing IgA deposits prevalent over the other classes of immunoglobulins and deemed not to be associated with causes of secondary IgAN as per clinical judgment of the Investigator.\n* The minimum body weight for participants in Cohort 1 is 35 kg at Screening and confirmed at Baseline (Day 1).\n* Proteinuria due to primary diagnosis of IgAN as assessed by UPCR ≥ 1 g\u002Fg (113 mg\u002FmmoL) sampled from FMV at Screening on Day -90 and Day -60 as well as during the Run-in Period despite treatment with maximum tolerated dose of ACE inhibitor\u002FARB for at least 120 days prior to Day 1. Note: UPCR will be assessed based on one FMV sample at Day -90 and based on the geometric mean of 2 FMV samples for the Day -60 visit and during the Run-in Period.\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* All participants must have been on supportive care including stable dose regimen of ACE inhibitor or ARB at either the locally approved maximal daily dose per body weight, or the maximally tolerated dose (per Investigator's judgment for pediatric use), for at least 120 days before first study drug administration. In addition, if participants are taking diuretics, other antihypertensive medication, or other background medication for IgAN (such as SGLT2 inhibitors), the doses should also be stabilized for at least 120 days prior to the first dosing of study treatment.\n\nExclusion Criteria:\n\n* Any secondary IgAN observed at Screening (and confirmed at Baseline\u002FDay 1) as defined by the Investigator; secondary IgAN can be associated with cirrhosis, celiac disease, human immunodeficiency virus (HIV) infection, herpes simplex virus infection, dermatitis herpetiformis, seronegative arthritis, small-cell carcinoma, lymphoma, disseminated tuberculosis, bronchiolitis obliterans, inflammatory bowel disease, and familial mediterranean fever.\n* A clinical diagnosis of immunoglobulin A vasculitis (IgAV or Henoch-Schonlein purpura) based on typical palpable purpura with or without arthralgia and abdominal pain.\n* Evidence of significant urinary obstruction or difficulty in voiding at Screening (and confirmed at Baseline\u002FDay 1); any urinary tract disorder or any chronic kidney disease other than IgAN at Screening and before first study drug administration.\n* Current acute kidney injury (AKI) defined by Acute Kidney Injury Network (AKIN) criteria within 4 weeks of screening.\n* Presence of rapidly progressive glomerulonephritis (RPGN) as defined by 50% decline in eGFR within 3 months prior to Screening or during the Screening and Run-in periods.\n* Presence of nephrotic syndrome at Screening based on the investigator's judgment.\n* History or current diagnosis of ECG abnormalities indicating significant risk for study participants such clinically significant cardiac arrhythmias, e.g. sustained ventricular tachycardia, or clinically significant second- or third-degree atrioventricular (AV) block without a pacemaker.\n* History or current diagnosis of clinically significant echocardiogram abnormalities indicating significant risk for study participants including but not limited to clinically significant functional, morphological and\u002For structural abnormalities, which may include left ventricular ejection fraction \\\u003C 50% and\u002For left ventricular hypertrophy due to uncontrolled blood pressure at Screening.\n* Participants of 12 to \\\u003C 18 years of age with systolic blood pressure (SBP) \\\u003C 80 mmHg or \\> 150 mmHg, or diastolic blood pressure (DBP) \\\u003C 50 mmHg or \\> 95 mmHg, or pulse rate \\\u003C 50 bpm or \\> 110 bpm; participants of 6 to \\\u003C 12 years of age with SBP \\\u003C 70 mmHg or \\> 140 mmHg, or DBP \\\u003C 40 mmHg or \\> 90 mmHg or pulse rate \\\u003C 52 bpm or \\> 156 bpm; participants of 2 to \\\u003C 6 years of age with SBP \\\u003C 70 mmHg or \\> 120 mmHg, or DBP \\\u003C 40 mmHg or \\> 80 mmHg or pulse rate \\\u003C 52 bpm or \\> 156 bpm at Screening.\n* Participants previously treated with immunosuppressive or other immunomodulatory agents such as but not limited to cyclophosphamide, rituximab, infliximab, canakinumab, mycophenolate mofetil (MMF) or mycophenolate sodium (MPS), calcineurin inhibitors, complement inhibitors (other than iptacopan), oral budesonide, systemic corticosteroids exposure (≥ 0.5 mg\u002Fkg\u002Fday of prednisone\u002Fprednisolone equivalent or \\> 7.5 mg\u002Fd prednisone\u002Fprednisolone equivalent total exposure in a single day) within 120 days (or 180 days for rituximab) prior to first study drug administration. Participants treated with endothelin (receptor) antagonists (including sparsentan) within 120 days prior to first study drug administration.\n* All transplanted participants (any solid organ transplantation, including bone marrow transplantation).\n* History of recurrent invasive infections caused by encapsulated organisms, such as meningococcus and pneumococcus.\n* Major concurrent comorbidities at Screening including but not limited to advanced cardiac disease (e.g. New York Heart Association (NYHA) class III (for 6 to \\\u003C18 years of age), Ross class III (for 2 to \\\u003C 6 years of age), severe pulmonary disease (e.g. World Health Organization (WHO) class III (for 17 years of age); Pulmonary Vascular Research Institute (PVRI) class III (for 2 to \\\u003C 17 years of age), or hepatic disease (e.g. active hepatitis) that in the opinion of the investigator precludes participation in the study.\n* Current use of any homeopathic and\u002For herbal medications for IgAN disease progression, such as but not limited to Lei Gong Teng at Screening (and confirmed at Baseline\u002FDay 1).\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply","ALL","2 Years","18 Years",{"count":305,"type":306},31,"ESTIMATED","INTERVENTIONAL",[309],"PHASE3","The study is an open-label, single arm, multicenter, Phase III study to determine proteinuria reduction, pharmacokinetics (PK), safety and tolerability (including CV surveillance) of iptacopan in primary immunoglobulin A nephropathy (IgAN) pediatric patients aged 2 to \\\u003C18 years.",[312],"Primary Immunoglobulin A Nephropathy (IgAN)",[314,315,316,317,318,319,320,321,322],"Iptacopan,","primary IgA nephropathy,","IgAN,","proteinuria,","UPCR,","eGFR,","kidney function decline,","pediatric,","adolescents","2026-09-03",{"date":325,"type":326},"2026-09-09","ACTUAL",{"date":328,"type":326},"2025-11-27",{"date":330,"type":306},"2030-06-30",{"name":52,"class":6},20]