Ignite Creation Date:
2025-12-24 @ 4:52 PM
Ignite Modification Date:
2025-12-28 @ 4:16 AM
Study NCT ID:
NCT04509050
Status:
RECRUITING
Last Update Posted:
2024-08-12
First Post:
2020-08-06
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D003550', 'term': 'Cystic Fibrosis'}], 'ancestors': [{'id': 'D010182', 'term': 'Pancreatic Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D008171', 'term': 'Lung Diseases'}, {'id': 'D012140', 'term': 'Respiratory Tract Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007232', 'term': 'Infant, Newborn, Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000706587', 'term': 'elexacaftor, ivacaftor, tezacaftor drug combination'}, {'id': 'C545203', 'term': 'ivacaftor'}]}}, 'protocolSection': {'designModule': {'bioSpec': {'retention': 'SAMPLES_WITH_DNA', 'description': 'Serum, Plasma, Buffy Coat, Urine, Stool, oropharyngeal (OP) swab, Bronchoalveolar lavage (BAL)'}, 'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'COHORT'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 210}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2020-11-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-08', 'completionDateStruct': {'date': '2029-12-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-08-08', 'studyFirstSubmitDate': '2020-08-06', 'studyFirstSubmitQcDate': '2020-08-10', 'lastUpdatePostDateStruct': {'date': '2024-08-12', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-08-11', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2029-12-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Part A Primary Outcome Measure: Change in weight-for-age z-scores', 'timeFrame': 'Baseline to 12 months', 'description': 'Weight-for-age z-scores over time'}, {'measure': 'Part A Primary Outcome Measure: Change in height-for-age z-scores', 'timeFrame': 'Baseline to 12 months', 'description': 'Height-for-age z-scores over time'}, {'measure': 'Part B Primary Outcome Measure: Change in weight-for-age z-scores', 'timeFrame': 'Baseline to 12 months', 'description': 'Change in weight-for-age z-scores from baseline'}, {'measure': 'Part B Primary Outcome Measure: Change in height-for-age z-scores', 'timeFrame': 'Baseline to 12 months', 'description': 'Change in height-for-age z-scores from baseline'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Cystic Fibrosis', 'CF', 'CFTR Modulator', 'triple combination therapy', 'elexacaftor', 'tezacaftor', 'ivacaftor'], 'conditions': ['Cystic Fibrosis']}, 'descriptionModule': {'briefSummary': '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).', 'detailedDescription': '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/tezacaftor/ivacaftor (elex/tez/iva) on growth. In Part B, subjects will have 1 "before ivacaftor or elex/tez/iva" visit within 30 days before initiation of the therapy and 8 "after ivacaftor or elex/tez/iva" visits over a 60-month follow-up period.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '10 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Part A - children with confirmed diagnosis of cystic fibrosis who are less than 10 years of age and not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy\n\nPart B - children with confirmed diagnosis of cystic fibrosis who are less than 7 years of age (or participated in Part A of the study) with an intention to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy', 'healthyVolunteers': False, 'eligibilityCriteria': '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/tezacaftor/ivacaftor).\n* Physician intent to prescribe ivacaftor or elexacaftor/tezacaftor/ivacaftor.\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/tezacaftor/ivacaftor 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.'}, 'identificationModule': {'nctId': 'NCT04509050', 'acronym': 'BEGIN', 'briefTitle': 'Study to Evaluate Biological & Clinical Effects of Significantly Corrected CFTR Function in Infants & Young Children', 'organization': {'class': 'OTHER', 'fullName': "Seattle Children's Hospital"}, 'officialTitle': 'A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN Study)', 'orgStudyIdInfo': {'id': 'BEGIN-OB-19'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Part A', 'description': 'Children with CF not on ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy.'}, {'label': 'Part B', 'description': 'Children with CF planning to start ivacaftor or elexacaftor/tezacaftor/ivacaftor CFTR modulator therapy. Participants from the Part A cohort of this study may enroll into the Part B cohort if they become eligible for these CFTR modulator therapies and plan to start them.', 'interventionNames': ['Drug: Ivacaftor or elexacaftor/tezacaftor/ivacaftor']}], 'interventions': [{'name': 'Ivacaftor or elexacaftor/tezacaftor/ivacaftor', 'type': 'DRUG', 'otherNames': ['Kalydeco or Trikafta', 'Vertex (VX)-770 or VX-445/VX-661/VX-770'], 'description': 'In Part B, approved CFTR modulator as prescribed at the discretion of the treating physician -not dictated by the BEGIN investigators', 'armGroupLabels': ['Part B']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Heather Hathorne', 'role': 'CONTACT', 'email': 'hyhathorne@uabmc.edu'}], 'facility': "The Children's Hospital Alabama, University of Alabama at Birmingham", 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '94304', 'city': 'Palo Alto', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jacquelyn Spano', 'role': 'CONTACT', 'email': 'jmzirbes@stanford.edu'}], 'facility': 'Stanford University Medical Center', 'geoPoint': {'lat': 37.44188, 'lon': -122.14302}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mary Cross', 'role': 'CONTACT', 'email': 'mary.cross@childrenscolorado.org'}], 'facility': "Children's Hospital Colorado", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '32207', 'city': 'Jacksonville', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jennifer (Jenn) Gafford', 'role': 'CONTACT', 'email': 'Jennifer.gafford@nemours.org'}], 'facility': "Nemours Children's Clinic", 'geoPoint': {'lat': 30.33218, 'lon': -81.65565}}, {'zip': '33136', 'city': 'Miami', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Ylber (Ivan) Whitaker', 'role': 'CONTACT', 'email': 'yiw2@miami.edu'}], 'facility': 'University of Miami', 'geoPoint': {'lat': 25.77427, 'lon': -80.19366}}, {'zip': '32827', 'city': 'Orlando', 'state': 'Florida', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sherry Wooldridge', 'role': 'CONTACT', 'email': 'sherry.wooldridge@nemours.org'}], 'facility': "The Nemours Children's Clinic - Orlando", 'geoPoint': {'lat': 28.53834, 'lon': -81.37924}}, {'zip': '46202', 'city': 'Indianapolis', 'state': 'Indiana', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Lisa Bendy', 'role': 'CONTACT', 'email': 'lbendy@iu.edu'}], 'facility': 'Riley Hospital for Children', 'geoPoint': {'lat': 39.76838, 'lon': -86.15804}}, {'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mary Teresi', 'role': 'CONTACT', 'email': 'mary-teresi@uiowa.edu'}], 'facility': 'University of Iowa', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '66160', 'city': 'Kansas City', 'state': 'Kansas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Lawrence Scott', 'role': 'CONTACT', 'email': 'lscott2@kumc.edu'}], 'facility': 'University of Kansas Medical Center', 'geoPoint': {'lat': 39.11417, 'lon': -94.62746}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Robert Fowler', 'role': 'CONTACT', 'email': 'Robert.fowler@childrens.harvard.edu'}], 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Dawn Kruse', 'role': 'CONTACT', 'email': 'dmkruse@med.umich.edu'}], 'facility': 'University of Michigan, Michigan Medicine', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}, {'zip': '49503', 'city': 'Grand Rapids', 'state': 'Michigan', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Andrew James', 'role': 'CONTACT', 'email': 'andrew.james@corewellhealth.org'}], 'facility': "Helen DeVos Children's Hospital", 'geoPoint': {'lat': 42.96336, 'lon': -85.66809}}, {'zip': '55404', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Camerone Bey', 'role': 'CONTACT', 'email': 'Camerone.bey@childrensmn.org'}], 'facility': "Children's Hospitals and Clinics of Minnesota", 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '55455', 'city': 'Minneapolis', 'state': 'Minnesota', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'CF Trials Contact', 'role': 'CONTACT', 'email': 'cftrials@umn.edu'}], 'facility': 'The Minnesota Cystic Fibrosis Center', 'geoPoint': {'lat': 44.97997, 'lon': -93.26384}}, {'zip': '64108', 'city': 'Kansas City', 'state': 'Missouri', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Candy Schmoll', 'role': 'CONTACT', 'email': 'cschmoll@cmh.edu'}], 'facility': "Children's Mercy Kansas City", 'geoPoint': {'lat': 39.09973, 'lon': -94.57857}}, {'zip': '63110', 'city': 'St Louis', 'state': 'Missouri', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Irma Bauer', 'role': 'CONTACT', 'email': 'irmabauer@wustl.edu'}], 'facility': "St. Louis Children's Hospital", 'geoPoint': {'lat': 38.62727, 'lon': -90.19789}}, {'zip': '14203', 'city': 'Buffalo', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Christine Roach', 'role': 'CONTACT', 'email': 'croach@upa.chob.edu'}], 'facility': 'The Cystic Fibrosis Center of Western New York', 'geoPoint': {'lat': 42.88645, 'lon': -78.87837}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Hossein Sadeghi', 'role': 'CONTACT', 'email': 'HS762@cumc.Columbia.edu'}], 'facility': "Children's Hospital of New York", 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}, {'zip': '13210', 'city': 'Syracuse', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Mary Forell', 'role': 'CONTACT', 'email': 'forellm@upstate.edu'}], 'facility': 'SUNY Upstate Medical University', 'geoPoint': {'lat': 43.04812, 'lon': -76.14742}}, {'zip': '10595', 'city': 'Valhalla', 'state': 'New York', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Zachary Messer', 'role': 'CONTACT', 'email': 'Zachary_Messer@nymc.edu'}], 'facility': 'New York Medical College at Westchester Medical Center', 'geoPoint': {'lat': 41.07482, 'lon': -73.77513}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Kelly Thornton', 'role': 'CONTACT', 'email': 'Kelly.Thornton@cchmc.org'}], 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '43205', 'city': 'Columbus', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Diana Gilmore', 'role': 'CONTACT', 'email': 'Diana.Gilmore@nationwidechildrens.org'}], 'facility': "Nationwide Children's Hospital", 'geoPoint': {'lat': 39.96118, 'lon': -82.99879}}, {'zip': '73104', 'city': 'Oklahoma City', 'state': 'Oklahoma', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'CF Center Participant Contact', 'role': 'CONTACT', 'email': 'cfresearchteam@ouhsc.edu'}], 'facility': 'Oklahoma Cystic Fibrosis Center', 'geoPoint': {'lat': 35.46756, 'lon': -97.51643}}, {'zip': '97239', 'city': 'Portland', 'state': 'Oregon', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jenna Bucher', 'role': 'CONTACT', 'email': 'bucherj@ohsu.edu'}], 'facility': 'Oregon Health Sciences University', 'geoPoint': {'lat': 45.52345, 'lon': -122.67621}}, {'zip': '17033', 'city': 'Hershey', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Diane M Kitch', 'role': 'CONTACT', 'email': 'dkitch@pennstatehealth.psu.edu'}], 'facility': 'Hershey Medical Center Pennsylvania State University', 'geoPoint': {'lat': 40.28592, 'lon': -76.65025}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Erin Donnelly', 'role': 'CONTACT', 'email': 'Donnellye4@email.chop.edu'}], 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '15224', 'city': 'Pittsburgh', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Elizabeth Hartigan', 'role': 'CONTACT', 'email': 'elizabeth.hartigan@chp.edu'}], 'facility': "Children's Hospital of Pittsburgh of UPMC", 'geoPoint': {'lat': 40.44062, 'lon': -79.99589}}, {'zip': '75207', 'city': 'Dallas', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Keianna Brown', 'role': 'CONTACT', 'email': 'Keianna.brown@utsouthwestern.edu'}], 'facility': "University of Texas Southwestern / Children's Health", 'geoPoint': {'lat': 32.78306, 'lon': -96.80667}}, {'zip': '76104', 'city': 'Fort Worth', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Anna Reyes', 'role': 'CONTACT', 'email': 'anna.reyes@cookchildrens.org'}], 'facility': "Cook Children's Medical Center", 'geoPoint': {'lat': 32.72541, 'lon': -97.32085}}, {'zip': '77030', 'city': 'Houston', 'state': 'Texas', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Tracy Mosely', 'role': 'CONTACT', 'email': 'tlmosely@texaschildrens.org'}], 'facility': 'Baylor College of Medicine', 'geoPoint': {'lat': 29.76328, 'lon': -95.36327}}, {'zip': '84113', 'city': 'Salt Lake City', 'state': 'Utah', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Jane B Vroom', 'role': 'CONTACT', 'email': 'jane.vroom@hsc.utah.edu'}], 'facility': "Primary Children's Cystic Fibrosis Center", 'geoPoint': {'lat': 40.76078, 'lon': -111.89105}}, {'zip': '05401', 'city': 'Burlington', 'state': 'Vermont', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Julie Sweet', 'role': 'CONTACT', 'email': 'julie.sweet@uvmhealth.org'}], 'facility': "Vermont Children's Hospital", 'geoPoint': {'lat': 44.47588, 'lon': -73.21207}}, {'zip': '22903', 'city': 'Charlottesville', 'state': 'Virginia', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Erica Tharrington', 'role': 'CONTACT', 'email': 'SFV6AB@hscmail.mcc.virginia.edu'}], 'facility': 'University of Virginia', 'geoPoint': {'lat': 38.02931, 'lon': -78.47668}}, {'zip': '98105', 'city': 'Seattle', 'state': 'Washington', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Sharon McNamara', 'role': 'CONTACT', 'email': 'sharon.mcnamara@seattlechildrens.org'}], 'facility': "Seattle Children's Hospital", 'geoPoint': {'lat': 47.60621, 'lon': -122.33207}}, {'zip': '53792', 'city': 'Madison', 'state': 'Wisconsin', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Melanie Nelson', 'role': 'CONTACT', 'email': 'mnelson@pediatrics.wisc.edu'}], 'facility': 'University of Wisconsin', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}], 'centralContacts': [{'name': 'Rachael Buckingham', 'role': 'CONTACT', 'email': 'rachael.buckingham@seattlechildrens.org', 'phone': '206-884-7517'}, {'name': 'Anna Mead', 'role': 'CONTACT', 'email': 'anna.mead@seattlechildrens.org', 'phone': '206-884-7531'}], 'overallOfficials': [{'name': 'Bonnie Ramsey, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "Seattle Children's Hospital"}, {'name': 'Lucas Hoffman, MD PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "University of Washington/Seattle Children's"}, {'name': 'Katie Larson Ode, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Iowa'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sonya Heltshe', 'class': 'OTHER'}, 'collaborators': [{'name': 'Cystic Fibrosis Foundation', 'class': 'OTHER'}, {'name': 'University of Washington', 'class': 'OTHER'}, {'name': 'University of Alabama at Birmingham', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'Associate Professor University of Washington', 'investigatorFullName': 'Sonya Heltshe', 'investigatorAffiliation': "Seattle Children's Hospital"}}}}