[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT05422222":3,"trial-entities:NCT05422222":387,"trial-summary:NCT05422222":391},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":15,"conditions":17,"keywords":19,"study_type":20,"primary_purpose":21,"phases":22,"enrollment_info":24,"interventions":27,"primary_outcomes":39,"secondary_outcomes":49,"sex":92,"minimum_age":93,"maximum_age":94,"healthy_volunteers":95,"eligibility_criteria":96,"std_ages":104,"locations":106,"central_contacts":379,"overall_officials":380,"references":381,"see_also_links":386},"NCT05422222","VX21-121-105","Evaluation of VX-121\u002FTezacaftor\u002FDeutivacaftor in Cystic Fibrosis (CF) Participants 1 Through 11 Years of Age","A Phase 3 Study Evaluating the Pharmacokinetics, Safety, and Tolerability of VX-121\u002FTezacaftor\u002FDeutivacaftor Triple Combination Therapy in Cystic Fibrosis Subjects 1 Through 11 Years of Age","ACTIVE_NOT_RECRUITING","2030-06-30","2026-05","2026-05-28","2022-06-21","Vertex Pharmaceuticals Incorporated","INDUSTRY",null,"The purpose of this study is to evaluate the pharmacokinetics, safety, tolerability and efficacy of VX-121\u002Ftezacaftor\u002Fdeutivacaftor (VX-121\u002FTEZ\u002FD-IVA) in CF participants with at least 1 triple combination responsive (TCR) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.",[18],"Cystic Fibrosis",[],"INTERVENTIONAL","TREATMENT",[23],"PHASE3",{"count":25,"type":26},210,"ESTIMATED",[28],{"type":29,"name":30,"description":31,"armGroupLabels":32,"otherNames":35},"DRUG","VX-121\u002FTEZ\u002FD-IVA","Fixed-dose combination for oral administration.",[33,34],"Part A: VX-121\u002FTEZ\u002FD-IVA","Part B: VX-121\u002FTEZ\u002FD-IVA",[36,37,38],"VX-121\u002FVX-661\u002FVX-561","VX-121\u002FVX-661\u002FCTP-656","VX-121\u002Ftezacaftor\u002Fdeutivacaftor",[40,43,46],{"measure":41,"timeFrame":42},"Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites","From Day 1 up to Day 22",{"measure":44,"timeFrame":45},"Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)","From Day 1 up to Day 50",{"measure":47,"timeFrame":48},"Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)","From Day 1 up to Week 28",[50,53,56,59,61,63,65,67,70,72,74,76,78,80,82,84,86,88,90],{"measure":51,"timeFrame":52},"Part B: Absolute Change in Sweat Chloride (SwCl)","From Baseline Through Week 24",{"measure":54,"timeFrame":55},"Part B: Observed Pre-dose Plasma Concentration (Ctrough) of VX-121, TEZ, D-IVA, and Relevant Metabolites","From Day 1 up to Week 16",{"measure":57,"timeFrame":58},"Part B: Drug Acceptability Assessment Using Modified Facial Hedonic Scale","At Day 1 and Week 24",{"measure":60,"timeFrame":52},"Part B: Absolute Change in Percent Predicted Forced Expiratory Volume (ppFEV1)",{"measure":62,"timeFrame":52},"Part B: Number of Pulmonary Exacerbation (PEx)",{"measure":64,"timeFrame":52},"Part B: Number of CF-Related Hospitalizations",{"measure":66,"timeFrame":52},"Part B: Absolute Change in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain (RD) Score",{"measure":68,"timeFrame":69},"Part B: Absolute Change in Body Mass Index (BMI)","From Baseline at Week 24",{"measure":71,"timeFrame":69},"Part B: Absolute Change in BMI-for-age Z-score",{"measure":73,"timeFrame":69},"Part B: Absolute Change in Weight",{"measure":75,"timeFrame":69},"Part B: Absolute Change in Weight-for-age Z-score",{"measure":77,"timeFrame":69},"Part B: Absolute Change in Weight-for-length",{"measure":79,"timeFrame":69},"Part B: Absolute Change in Weight-for-length Z-score",{"measure":81,"timeFrame":69},"Part B: Absolute Change in Height",{"measure":83,"timeFrame":69},"Part B: Absolute Change in Height-for-age Z-score",{"measure":85,"timeFrame":69},"Part B: Absolute Change in Length",{"measure":87,"timeFrame":69},"Part B: Absolute Change in Length-for-age Z-score",{"measure":89,"timeFrame":52},"Part B: Proportion of Participants With SwCl \u003C60 millimole per liter (mmol\u002FL)",{"measure":91,"timeFrame":52},"Part B: Proportion of Participants With SwCl \u003C30 mmol\u002FL","ALL","1 Year","11 Years",false,{"inclusion":97,"exclusion":99,"raw_text":103},[98],"Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene",[100,101,102],"History of solid organ, hematological transplantation, or cancer","Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)","Lung infection with organisms associated with a more rapid decline in pulmonary status","Key Inclusion Criteria:\n\n* Participants with stable CF and at least 1 TCR mutation (including F508del) in the CFTR gene\n\nKey Exclusion Criteria:\n\n* History of solid organ, hematological transplantation, or cancer\n* Hepatic cirrhosis with portal hypertension, moderate hepatic impairment (Child Pugh Score 7 to 9), or severe hepatic impairment (Child Pugh Score 10 to 15)\n* Lung infection with organisms associated with a more rapid decline in pulmonary status\n\nOther protocol defined Inclusion\u002FExclusion criteria may apply.",[105],"CHILD",[107,116,123,131,139,147,155,163,171,179,186,194,202,209,216,224,232,240,248,256,263,269,275,281,288,294,301,307,314,320,326,333,340,347,353,360,366,373],{"facility":108,"city":109,"state":110,"zip":111,"country":112,"geoPoint":113},"Children's Hospital of Orange County","Orange","California","92868","United States",{"lat":114,"lon":115},33.78779,-117.85311,{"facility":117,"city":118,"state":110,"zip":119,"country":112,"geoPoint":120},"Stanford University Clinical and Translational Research Unit","Palo Alto","94304",{"lat":121,"lon":122},37.44188,-122.14302,{"facility":124,"city":125,"state":126,"zip":127,"country":112,"geoPoint":128},"Children's Hospital of Colorado","Aurora","Colorado","80045",{"lat":129,"lon":130},39.72943,-104.83192,{"facility":132,"city":133,"state":134,"zip":135,"country":112,"geoPoint":136},"The Emory Clinic \u002F Children's Healthcare of Atlanta at Egleston","Atlanta","Georgia","30322",{"lat":137,"lon":138},33.749,-84.38798,{"facility":140,"city":141,"state":142,"zip":143,"country":112,"geoPoint":144},"Ann & Robert H. Lurie Children's Hospital of Chicago","Chicago","Illinois","60611",{"lat":145,"lon":146},41.85003,-87.65005,{"facility":148,"city":149,"state":150,"zip":151,"country":112,"geoPoint":152},"Riley Hospital for Children at Indiana University Health","Indianapolis","Indiana","46202",{"lat":153,"lon":154},39.76838,-86.15804,{"facility":156,"city":157,"state":158,"zip":159,"country":112,"geoPoint":160},"Boston Children's Hospital","Boston","Massachusetts","02115",{"lat":161,"lon":162},42.35843,-71.05977,{"facility":164,"city":165,"state":166,"zip":167,"country":112,"geoPoint":168},"Children's Respiratory and Critical Care Specialists, P.A., Children's Hospitals and Clinics of Minnesota","Minneapolis","Minnesota","55404",{"lat":169,"lon":170},44.97997,-93.26384,{"facility":172,"city":173,"state":174,"zip":175,"country":112,"geoPoint":176},"The Children's Mercy Hospital","Kansas City","Missouri","64108",{"lat":177,"lon":178},39.09973,-94.57857,{"facility":180,"city":181,"state":174,"zip":182,"country":112,"geoPoint":183},"Washington University School of Medicine \u002F St. Louis Children's Hospital","St Louis","63110",{"lat":184,"lon":185},38.62727,-90.19789,{"facility":187,"city":188,"state":189,"zip":190,"country":112,"geoPoint":191},"Cohen Children's Medical Center","Lake Success","New York","11042",{"lat":192,"lon":193},40.77066,-73.71763,{"facility":195,"city":196,"state":197,"zip":198,"country":112,"geoPoint":199},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"lat":200,"lon":201},39.12711,-84.51439,{"facility":203,"city":204,"state":197,"zip":205,"country":112,"geoPoint":206},"Rainbow Babies and Children's Hospital\u002FUniversity Hospitals Cleveland Medical Center","Cleveland","44106",{"lat":207,"lon":208},41.4995,-81.69541,{"facility":210,"city":211,"state":197,"zip":212,"country":112,"geoPoint":213},"Nationwide Children's Hospital","Columbus","43205",{"lat":214,"lon":215},39.96118,-82.99879,{"facility":217,"city":218,"state":219,"zip":220,"country":112,"geoPoint":221},"Oregon Health & Science University","Portland","Oregon","97239",{"lat":222,"lon":223},45.52345,-122.67621,{"facility":225,"city":226,"state":227,"zip":228,"country":112,"geoPoint":229},"UPMC Children's Hospital of Pittsburgh","Pittsburgh","Pennsylvania","15224",{"lat":230,"lon":231},40.44062,-79.99589,{"facility":233,"city":234,"state":235,"zip":236,"country":112,"geoPoint":237},"Texas Children's Hospital - Wallace Tower","Houston","Texas","77030",{"lat":238,"lon":239},29.76328,-95.36327,{"facility":241,"city":242,"state":243,"zip":244,"country":112,"geoPoint":245},"Vermont Lung Center","Colchester","Vermont","05446",{"lat":246,"lon":247},44.54394,-73.14791,{"facility":249,"city":250,"state":251,"zip":252,"country":112,"geoPoint":253},"American Family Childrens Hospital","Madison","Wisconsin","53792",{"lat":254,"lon":255},43.07305,-89.40123,{"facility":257,"city":258,"country":259,"geoPoint":260},"The Kids Research Institute Australia","Nedlands","Australia",{"lat":261,"lon":262},-31.98184,115.8073,{"facility":264,"city":265,"country":259,"geoPoint":266},"Women's and Children's Hospital","North Adelaide",{"lat":267,"lon":268},-34.90733,138.59141,{"facility":270,"city":271,"country":259,"geoPoint":272},"The Royal Children's Hospital","Parkville",{"lat":273,"lon":274},-37.78333,144.95,{"facility":276,"city":277,"country":259,"geoPoint":278},"Queensland Children's Hospital","South Brisbane",{"lat":279,"lon":280},-27.48034,153.02049,{"facility":282,"city":283,"country":284,"geoPoint":285},"The Hospital for Sick Children","Toronto","Canada",{"lat":286,"lon":287},43.70643,-79.39864,{"facility":289,"city":290,"country":284,"geoPoint":291},"British Columbia Children's Hospital","Vancouver",{"lat":292,"lon":293},49.24966,-123.11934,{"facility":295,"city":296,"country":297,"geoPoint":298},"CHU Lyon - Hopital Femme Mere-Enfant","Bron","France",{"lat":299,"lon":300},45.73865,4.91303,{"facility":302,"city":303,"country":297,"geoPoint":304},"Hopital Necker, Enfants Malades","Paris",{"lat":305,"lon":306},48.85341,2.3488,{"facility":308,"city":309,"country":310,"geoPoint":311},"Charité - Paediatric Pulmonology Department","Berlin","Germany",{"lat":312,"lon":313},52.52437,13.41053,{"facility":315,"city":316,"country":310,"geoPoint":317},"Universitatsklinikum Essen","Essen",{"lat":318,"lon":319},51.45657,7.01228,{"facility":321,"city":322,"country":310,"geoPoint":323},"Medizinische Hochschule Hannover","Hanover",{"lat":324,"lon":325},52.37052,9.73322,{"facility":327,"city":328,"country":329,"geoPoint":330},"Erasmus Medical Center \u002F Sophia Children's Hospital","Rotterdam","Netherlands",{"lat":331,"lon":332},51.9225,4.47917,{"facility":334,"city":335,"country":336,"geoPoint":337},"Starship Children's Hospital","Grafton","New Zealand",{"lat":338,"lon":339},-36.86029,174.76566,{"facility":341,"city":342,"country":343,"geoPoint":344},"Sahlgrenska Universitetssjukhuset","Gothenburg","Sweden",{"lat":345,"lon":346},57.70716,11.96679,{"facility":348,"city":349,"country":343,"geoPoint":350},"Karolinska University Hospital - Pulmonology","Stockholm",{"lat":351,"lon":352},59.32938,18.06871,{"facility":354,"city":355,"country":356,"geoPoint":357},"Inselspital - Universitaetsspital Bern","Bern","Switzerland",{"lat":358,"lon":359},46.94809,7.44744,{"facility":361,"city":362,"country":356,"geoPoint":363},"Kinderspital Zurich","Zurich",{"lat":364,"lon":365},47.36667,8.55,{"facility":367,"city":368,"country":369,"geoPoint":370},"Children and Young Adults Research Unit","Cardiff","United Kingdom",{"lat":371,"lon":372},51.48,-3.18,{"facility":374,"city":375,"country":369,"geoPoint":376},"Great Ormond Street Hospital for Children","London",{"lat":377,"lon":378},51.50853,-0.12574,[],[],[382],{"pmid":383,"type":384,"citation":385},"39756425","DERIVED","Hoppe JE, Kasi AS, Pittman JE, Jensen R, Thia LP, Robinson P, Tirakitsoontorn P, Ramsey B, Mall MA, Taylor-Cousar JL, McKone EF, Tullis E, Salinas DB, Zhu J, Chen YC, Rodriguez-Romero V, Sosnay PR, Davies G; VX21-121-105 Study Group. Vanzacaftor-tezacaftor-deutivacaftor for children aged 6-11 years with cystic fibrosis (RIDGELINE Trial VX21-121-105): an analysis from a single-arm, phase 3 trial. Lancet Respir Med. 2025 Mar;13(3):244-255. doi: 10.1016\u002FS2213-2600(24)00407-7. Epub 2025 Jan 2.",[],{"nct_id":4,"conditions":388,"biomarkers":389},[18],[390],"CFTR Gene",{"nct_id":4,"found":95,"summary":15,"prompt_version":15}]