[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT04509050":3,"trial-entities:NCT04509050":502,"trial-summary:NCT04509050":506},{"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":17,"conditions":18,"keywords":20,"study_type":27,"primary_purpose":28,"phases":29,"enrollment_info":30,"interventions":33,"primary_outcomes":43,"secondary_outcomes":57,"sex":58,"minimum_age":28,"maximum_age":59,"healthy_volunteers":60,"eligibility_criteria":61,"std_ages":75,"locations":77,"central_contacts":482,"overall_officials":491,"references":500,"see_also_links":501},"NCT04509050","BEGIN-OB-19","Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children","A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)","RECRUITING","2027-12-31","2026-07","2026-07-08","2020-11-18","Sonya Heltshe","OTHER",true,"This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact on children with cystic fibrosis (CF).","This is a two-part, multi-center, prospective longitudinal, exploratory study of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators and their impact in children with cystic fibrosis (CF) on endocrine growth factors and height, gastrointestinal function and gut microbiome, lung function and respiratory microbiome, liver and pancreatic function, sweat chloride, inflammatory markers, and bone health.\n\nTotal duration of the study is expected to be 10 years. Part A will be a prospective cross-sequential study to describe the natural history of hormonal growth factors in early childhood and assess the feasibility of additional measurements. In Part A, subjects will have up to 8 visits over a period of up to 5 years.\n\nPart B will be a prospective longitudinal study to observe the effects of administration of either ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor (elex\u002Ftez\u002Fiva) on growth. In Part B, subjects will have 1 \"before ivacaftor or elex\u002Ftez\u002Fiva\" visit within 30 days before initiation of the therapy and 8 \"after ivacaftor or elex\u002Ftez\u002Fiva\" visits over a 60-month follow-up period.",[19],"Cystic Fibrosis",[19,21,22,23,24,25,26],"CF","CFTR Modulator","triple combination therapy","elexacaftor","tezacaftor","ivacaftor","OBSERVATIONAL",null,[],{"count":31,"type":32},210,"ESTIMATED",[34],{"type":35,"name":36,"description":37,"armGroupLabels":38,"otherNames":40},"DRUG","Ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor","In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators",[39],"Part B",[41,42],"Kalydeco or Trikafta","Vertex (VX)-770 or VX-445\u002FVX-661\u002FVX-770",[44,48,51,54],{"measure":45,"description":46,"timeFrame":47},"Part A Primary Outcome Measure: Change in weight-for-age z-scores","Weight-for-age z-scores over time","Baseline to 12 months",{"measure":49,"description":50,"timeFrame":47},"Part A Primary Outcome Measure: Change in height-for-age z-scores","Height-for-age z-scores over time",{"measure":52,"description":53,"timeFrame":47},"Part B Primary Outcome Measure: Change in weight-for-age z-scores","Change in weight-for-age z-scores from baseline",{"measure":55,"description":56,"timeFrame":47},"Part B Primary Outcome Measure: Change in height-for-age z-scores","Change in height-for-age z-scores from baseline",[],"ALL","10 Years",false,{"inclusion":62,"exclusion":69,"raw_text":74},[63,64,65,66,65,67,68],"Part A:","Less than 10 years of age at the first study visit.","Documentation of a CF diagnosis.","Participated in Part A OR less than 7 years of age at the first study visit.","CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor).","Physician intent to prescribe ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor.",[70,71,72,73],"Part A and Part B:","Use of an investigational drug within 28 days prior to and including the first study visit.","Use of ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor within the 28 days prior to and including the first study visit.","Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.","Inclusion Criteria:\n\n* Part A:\n\n  * Less than 10 years of age at the first study visit.\n  * Documentation of a CF diagnosis.\n\nPart B:\n\n* Participated in Part A OR less than 7 years of age at the first study visit.\n* Documentation of a CF diagnosis.\n* CFTR mutations consistent with FDA labeled indication of highly effective modulator therapy (ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor).\n* Physician intent to prescribe ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor.\n\nExclusion Criteria:\n\n* Part A and Part B:\n* Use of an investigational drug within 28 days prior to and including the first study visit.\n* Use of ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor within the 28 days prior to and including the first study visit.\n* Use of chronic oral corticosteroids within the 28 days prior to and including the first study visit.",[76],"CHILD",[78,92,104,116,128,139,150,162,174,186,198,210,221,233,241,252,263,275,285,296,307,319,330,342,354,366,377,388,400,411,422,434,446,458,470],{"facility":79,"status":8,"city":80,"state":81,"zip":82,"country":83,"contacts":84,"geoPoint":89},"The Children's Hospital Alabama, University of Alabama at Birmingham","Birmingham","Alabama","35233","United States",[85],{"name":86,"role":87,"email":88},"Heather Hathorne","CONTACT","hyhathorne@uabmc.edu",{"lat":90,"lon":91},33.52066,-86.80249,{"facility":93,"status":8,"city":94,"state":95,"zip":96,"country":83,"contacts":97,"geoPoint":101},"Stanford University Medical Center","Palo Alto","California","94304",[98],{"name":99,"role":87,"email":100},"Jacquelyn Spano","jmzirbes@stanford.edu",{"lat":102,"lon":103},37.44188,-122.14302,{"facility":105,"status":8,"city":106,"state":107,"zip":108,"country":83,"contacts":109,"geoPoint":113},"Children's Hospital Colorado","Aurora","Colorado","80045",[110],{"name":111,"role":87,"email":112},"Mary Cross","mary.cross@childrenscolorado.org",{"lat":114,"lon":115},39.72943,-104.83192,{"facility":117,"status":8,"city":118,"state":119,"zip":120,"country":83,"contacts":121,"geoPoint":125},"Nemours Children's Clinic","Jacksonville","Florida","32207",[122],{"name":123,"role":87,"email":124},"Jennifer (Jenn) Gafford","Jennifer.gafford@nemours.org",{"lat":126,"lon":127},30.33218,-81.65565,{"facility":129,"status":8,"city":130,"state":119,"zip":131,"country":83,"contacts":132,"geoPoint":136},"University of Miami","Miami","33136",[133],{"name":134,"role":87,"email":135},"Ylber (Ivan) Whitaker","yiw2@miami.edu",{"lat":137,"lon":138},25.77427,-80.19366,{"facility":140,"status":8,"city":141,"state":119,"zip":142,"country":83,"contacts":143,"geoPoint":147},"The Nemours Children's Clinic - Orlando","Orlando","32827",[144],{"name":145,"role":87,"email":146},"Sherry Wooldridge","sherry.wooldridge@nemours.org",{"lat":148,"lon":149},28.53834,-81.37924,{"facility":151,"status":8,"city":152,"state":153,"zip":154,"country":83,"contacts":155,"geoPoint":159},"Riley Hospital for Children","Indianapolis","Indiana","46202",[156],{"name":157,"role":87,"email":158},"Lisa Bendy","lbendy@iu.edu",{"lat":160,"lon":161},39.76838,-86.15804,{"facility":163,"status":8,"city":164,"state":165,"zip":166,"country":83,"contacts":167,"geoPoint":171},"University of Iowa","Iowa City","Iowa","52242",[168],{"name":169,"role":87,"email":170},"Mary Teresi","mary-teresi@uiowa.edu",{"lat":172,"lon":173},41.66113,-91.53017,{"facility":175,"status":8,"city":176,"state":177,"zip":178,"country":83,"contacts":179,"geoPoint":183},"University of Kansas Medical Center","Kansas City","Kansas","66160",[180],{"name":181,"role":87,"email":182},"Lawrence Scott","lscott2@kumc.edu",{"lat":184,"lon":185},39.11417,-94.62746,{"facility":187,"status":8,"city":188,"state":189,"zip":190,"country":83,"contacts":191,"geoPoint":195},"Boston Children's Hospital","Boston","Massachusetts","02115",[192],{"name":193,"role":87,"email":194},"Robert Fowler","Robert.fowler@childrens.harvard.edu",{"lat":196,"lon":197},42.35843,-71.05977,{"facility":199,"status":8,"city":200,"state":201,"zip":202,"country":83,"contacts":203,"geoPoint":207},"University of Michigan, Michigan Medicine","Ann Arbor","Michigan","48109",[204],{"name":205,"role":87,"email":206},"Dawn Kruse","dmkruse@med.umich.edu",{"lat":208,"lon":209},42.27756,-83.74088,{"facility":211,"status":8,"city":212,"state":201,"zip":213,"country":83,"contacts":214,"geoPoint":218},"Helen DeVos Children's Hospital","Grand Rapids","49503",[215],{"name":216,"role":87,"email":217},"Andrew James","andrew.james@corewellhealth.org",{"lat":219,"lon":220},42.96336,-85.66809,{"facility":222,"status":8,"city":223,"state":224,"zip":225,"country":83,"contacts":226,"geoPoint":230},"Children's Hospitals and Clinics of Minnesota","Minneapolis","Minnesota","55404",[227],{"name":228,"role":87,"email":229},"Camerone Bey","Camerone.bey@childrensmn.org",{"lat":231,"lon":232},44.97997,-93.26384,{"facility":234,"status":8,"city":223,"state":224,"zip":235,"country":83,"contacts":236,"geoPoint":240},"The Minnesota Cystic Fibrosis Center","55455",[237],{"name":238,"role":87,"email":239},"CF Trials Contact","cftrials@umn.edu",{"lat":231,"lon":232},{"facility":242,"status":8,"city":176,"state":243,"zip":244,"country":83,"contacts":245,"geoPoint":249},"Children's Mercy Kansas City","Missouri","64108",[246],{"name":247,"role":87,"email":248},"Candy Schmoll","cschmoll@cmh.edu",{"lat":250,"lon":251},39.09973,-94.57857,{"facility":253,"status":8,"city":254,"state":243,"zip":255,"country":83,"contacts":256,"geoPoint":260},"St. Louis Children's Hospital","St Louis","63110",[257],{"name":258,"role":87,"email":259},"Irma Bauer","irmabauer@wustl.edu",{"lat":261,"lon":262},38.62727,-90.19789,{"facility":264,"status":8,"city":265,"state":266,"zip":267,"country":83,"contacts":268,"geoPoint":272},"The Cystic Fibrosis Center of Western New York","Buffalo","New York","14203",[269],{"name":270,"role":87,"email":271},"Christine Roach","croach@upa.chob.edu",{"lat":273,"lon":274},42.88645,-78.87837,{"facility":276,"status":8,"city":266,"state":266,"zip":277,"country":83,"contacts":278,"geoPoint":282},"Children's Hospital of New York","10032",[279],{"name":280,"role":87,"email":281},"Hossein Sadeghi","HS762@cumc.Columbia.edu",{"lat":283,"lon":284},40.71427,-74.00597,{"facility":286,"status":8,"city":287,"state":266,"zip":288,"country":83,"contacts":289,"geoPoint":293},"SUNY Upstate Medical University","Syracuse","13210",[290],{"name":291,"role":87,"email":292},"Mary Forell","forellm@upstate.edu",{"lat":294,"lon":295},43.04812,-76.14742,{"facility":297,"status":8,"city":298,"state":266,"zip":299,"country":83,"contacts":300,"geoPoint":304},"New York Medical College at Westchester Medical Center","Valhalla","10595",[301],{"name":302,"role":87,"email":303},"Zachary Messer","Zachary_Messer@nymc.edu",{"lat":305,"lon":306},41.07482,-73.77513,{"facility":308,"status":8,"city":309,"state":310,"zip":311,"country":83,"contacts":312,"geoPoint":316},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",[313],{"name":314,"role":87,"email":315},"Kelly Thornton","Kelly.Thornton@cchmc.org",{"lat":317,"lon":318},39.12711,-84.51439,{"facility":320,"status":8,"city":321,"state":310,"zip":322,"country":83,"contacts":323,"geoPoint":327},"Nationwide Children's Hospital","Columbus","43205",[324],{"name":325,"role":87,"email":326},"Diana Gilmore","Diana.Gilmore@nationwidechildrens.org",{"lat":328,"lon":329},39.96118,-82.99879,{"facility":331,"status":8,"city":332,"state":333,"zip":334,"country":83,"contacts":335,"geoPoint":339},"Oklahoma Cystic Fibrosis Center","Oklahoma City","Oklahoma","73104",[336],{"name":337,"role":87,"email":338},"CF Center Participant Contact","cfresearchteam@ouhsc.edu",{"lat":340,"lon":341},35.46756,-97.51643,{"facility":343,"status":8,"city":344,"state":345,"zip":346,"country":83,"contacts":347,"geoPoint":351},"Oregon Health Sciences University","Portland","Oregon","97239",[348],{"name":349,"role":87,"email":350},"Jenna Bucher","bucherj@ohsu.edu",{"lat":352,"lon":353},45.52345,-122.67621,{"facility":355,"status":8,"city":356,"state":357,"zip":358,"country":83,"contacts":359,"geoPoint":363},"Hershey Medical Center Pennsylvania State University","Hershey","Pennsylvania","17033",[360],{"name":361,"role":87,"email":362},"Diane M Kitch","dkitch@pennstatehealth.psu.edu",{"lat":364,"lon":365},40.28592,-76.65025,{"facility":367,"status":8,"city":368,"state":357,"zip":369,"country":83,"contacts":370,"geoPoint":374},"Children's Hospital of Philadelphia","Philadelphia","19104",[371],{"name":372,"role":87,"email":373},"Erin Donnelly","Donnellye4@email.chop.edu",{"lat":375,"lon":376},39.95238,-75.16362,{"facility":378,"status":8,"city":379,"state":357,"zip":380,"country":83,"contacts":381,"geoPoint":385},"Children's Hospital of Pittsburgh of UPMC","Pittsburgh","15224",[382],{"name":383,"role":87,"email":384},"Elizabeth Hartigan","elizabeth.hartigan@chp.edu",{"lat":386,"lon":387},40.44062,-79.99589,{"facility":389,"status":8,"city":390,"state":391,"zip":392,"country":83,"contacts":393,"geoPoint":397},"University of Texas Southwestern \u002F Children's Health","Dallas","Texas","75207",[394],{"name":395,"role":87,"email":396},"Keianna Brown","Keianna.brown@utsouthwestern.edu",{"lat":398,"lon":399},32.78306,-96.80667,{"facility":401,"status":8,"city":402,"state":391,"zip":403,"country":83,"contacts":404,"geoPoint":408},"Cook Children's Medical Center","Fort Worth","76104",[405],{"name":406,"role":87,"email":407},"Anna Reyes","anna.reyes@cookchildrens.org",{"lat":409,"lon":410},32.72541,-97.32085,{"facility":412,"status":8,"city":413,"state":391,"zip":414,"country":83,"contacts":415,"geoPoint":419},"Baylor College of Medicine","Houston","77030",[416],{"name":417,"role":87,"email":418},"Tracy Mosely","tlmosely@texaschildrens.org",{"lat":420,"lon":421},29.76328,-95.36327,{"facility":423,"status":8,"city":424,"state":425,"zip":426,"country":83,"contacts":427,"geoPoint":431},"Primary Children's Cystic Fibrosis Center","Salt Lake City","Utah","84113",[428],{"name":429,"role":87,"email":430},"Jane B Vroom","jane.vroom@hsc.utah.edu",{"lat":432,"lon":433},40.76078,-111.89105,{"facility":435,"status":8,"city":436,"state":437,"zip":438,"country":83,"contacts":439,"geoPoint":443},"Vermont Children's Hospital","Burlington","Vermont","05401",[440],{"name":441,"role":87,"email":442},"Julie Sweet","julie.sweet@uvmhealth.org",{"lat":444,"lon":445},44.47588,-73.21207,{"facility":447,"status":8,"city":448,"state":449,"zip":450,"country":83,"contacts":451,"geoPoint":455},"University of Virginia","Charlottesville","Virginia","22903",[452],{"name":453,"role":87,"email":454},"Erica Tharrington","SFV6AB@hscmail.mcc.virginia.edu",{"lat":456,"lon":457},38.02931,-78.47668,{"facility":459,"status":8,"city":460,"state":461,"zip":462,"country":83,"contacts":463,"geoPoint":467},"Seattle Children's Hospital","Seattle","Washington","98105",[464],{"name":465,"role":87,"email":466},"Sharon McNamara","sharon.mcnamara@seattlechildrens.org",{"lat":468,"lon":469},47.60621,-122.33207,{"facility":471,"status":8,"city":472,"state":473,"zip":474,"country":83,"contacts":475,"geoPoint":479},"University of Wisconsin","Madison","Wisconsin","53792",[476],{"name":477,"role":87,"email":478},"Melanie Nelson","mnelson@pediatrics.wisc.edu",{"lat":480,"lon":481},43.07305,-89.40123,[483,487],{"name":484,"role":87,"phone":485,"email":486},"Rachael Buckingham","206-884-7517","rachael.buckingham@seattlechildrens.org",{"name":488,"role":87,"phone":489,"email":490},"Anna Mead","206-884-7531","anna.mead@seattlechildrens.org",[492,495,498],{"name":493,"affiliation":459,"role":494},"Bonnie Ramsey, MD","PRINCIPAL_INVESTIGATOR",{"name":496,"affiliation":497,"role":494},"Lucas Hoffman, MD PhD","University of Washington\u002FSeattle Children's",{"name":499,"affiliation":163,"role":494},"Katie Larson Ode, MD",[],[],{"nct_id":4,"conditions":503,"biomarkers":504},[19],[505],"CFTR Gene",{"nct_id":4,"found":15,"summary":507,"prompt_version":517},{"design":508,"status":509,"heading":510,"summary":511,"follow_up":512,"word_count":513,"commitments":514,"compensation":515,"drugs_mentioned":516},"This is an observational study, meaning participants will receive their prescribed CFTR modulator, and researchers will observe its effects. It is a multi-center study with a planned enrollment of 210 participants.","completed","Observational Study of CFTR Modulators in Children with Cystic Fibrosis","This study is looking at how well treatments like ivacaftor or elexacaftor\u002Ftezacaftor\u002Fivacaftor (CFTR modulators) work in infants and young children with cystic fibrosis (CF). Researchers want to understand how these medicines affect growth, digestion, lung health, and other body functions over time. Children up to 10 years old with a CF diagnosis can join. For some parts of the study, children need to have specific CFTR mutations that respond to these medicines. The main goal is to see changes in weight and height over 12 months. The study status is unclear, and it aims to enroll 210 participants.","Participants in Part B will be followed for 60 months (5 years) after starting treatment.",98,"In Part A, participants will have up to 8 visits over a period of up to 5 years. In Part B, participants will have 1 visit before starting treatment and 8 visits over a 60-month (5-year) follow-up period.","Not stated in the trial record.",[36],"v2"]