[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100458331":3},{"organization":4,"armGroups":7,"interventions":22,"overallOfficials":29,"centralContacts":33,"locations":39,"responsibleParty":356,"collaborators":28,"id":358,"slug":359,"hasResults":360,"nctId":361,"briefTitle":362,"officialTitle":363,"acronym":364,"eligibilityCriteria":365,"healthyVolunteers":360,"sex":366,"minAge":367,"maxAge":28,"enrollmentInfo":368,"targetDuration":28,"studyType":371,"phases":372,"briefSummary":374,"conditions":375,"keywords":377,"overallStatus":42,"whyStopped":28,"lastUpdateSubmitDate":380,"lastUpdatePostDateStruct":381,"startDateStruct":384,"completionDateStruct":386,"leadSponsor":388,"locationsCount":389},{"fullName":5,"class":6},"4D Molecular Therapeutics","INDUSTRY",[8,14,18],{"label":9,"type":10,"description":11,"interventionNames":12},"4D-710 Phase 1: Dose Exploration","EXPERIMENTAL","Participants who are ineligible for or intolerant of modulator therapy will receive one of various dose levels of 4D-710 to identify recommended phase 2 dose(s) for further evaluation.",[13],"Biological: 4D-710",{"label":15,"type":10,"description":16,"interventionNames":17},"4D-710 Phase 2: Dose Expansion","Participants will receive a single inhalational administration of 4D-710 at the dose level(s) selected for dose expansion.",[13],{"label":19,"type":10,"description":20,"interventionNames":21},"4D-710 Dose Exploration (Sub-Study)","Participants who are on currently available CFTR modulator therapy will receive a dose of 4D-710 at various dose levels.",[13],[23],{"type":24,"name":25,"description":26,"armGroupLabels":27,"otherNames":28},"BIOLOGICAL","4D-710","4D-710 is an adeno-associated virus (AAV) gene therapy comprised of an AAV capsid variant (4D-A101) carrying a transgene cassette encoding human cystic fibrosis transmembrane conductance regulator with a deletion in the regulatory domain (CFTRΔR).",[19,9,15],null,[30],{"name":31,"affiliation":5,"role":32},"Sam Moskowitz MD","STUDY_DIRECTOR",[34],{"name":35,"role":36,"phone":37,"phoneExt":28,"email":38},"4DMT Patient Advocacy","CONTACT","(888) 748-8881","clinicaltrials@4DMT.com",[40,61,77,93,111,127,143,159,175,191,207,219,230,246,261,277,292,308,324,340],{"facility":41,"status":42,"city":43,"state":44,"zip":45,"country":46,"countryCode":47,"cosmosGeoPoint":48,"geoPoint":53,"contacts":54},"University of Alabama Child Health Research Unit","RECRUITING","Birmingham","Alabama","35233","United States","US",{"type":49,"coordinates":50},"Point",[51,52],-86.80249,33.52066,{"lat":52,"lon":51},[55,58],{"name":56,"role":36,"phone":28,"phoneExt":28,"email":57},"Justin Anderson","justinanderson@uabmc.edu",{"name":59,"role":60,"phone":28,"phoneExt":28,"email":28},"George M. 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Nelson","rachel.nelson@northwestern.edu",{"name":158,"role":60,"phone":28,"phoneExt":28,"email":28},"Manu Jain, MD",{"facility":160,"status":42,"city":161,"state":162,"zip":163,"country":46,"countryCode":47,"cosmosGeoPoint":164,"geoPoint":168,"contacts":169},"University of Kansas Medical Center","Kansas City","Kansas","66160",{"type":49,"coordinates":165},[166,167],-94.62746,39.11417,{"lat":167,"lon":166},[170,173],{"name":171,"role":36,"phone":28,"phoneExt":28,"email":172},"Lawrence Scott","lscott2@kumc.edu",{"name":174,"role":60,"phone":28,"phoneExt":28,"email":28},"Joel Mermis, MD",{"facility":176,"status":42,"city":177,"state":178,"zip":179,"country":46,"countryCode":47,"cosmosGeoPoint":180,"geoPoint":184,"contacts":185},"Johns Hopkins Hospital","Baltimore","Maryland","21287",{"type":49,"coordinates":181},[182,183],-76.61219,39.29038,{"lat":183,"lon":182},[186,189],{"name":187,"role":36,"phone":28,"phoneExt":28,"email":188},"Kaia 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Howrylak, MD, PhD",{"facility":278,"status":42,"city":279,"state":264,"zip":280,"country":46,"countryCode":47,"cosmosGeoPoint":281,"geoPoint":285,"contacts":286},"The Hospital of the University of Pennsylvania","Philadelphia","19104",{"type":49,"coordinates":282},[283,284],-75.16362,39.95238,{"lat":284,"lon":283},[287,290],{"name":288,"role":36,"phone":28,"phoneExt":28,"email":289},"Melissa Molter","melissa.molter@pennmedicine.upenn.edu",{"name":291,"role":60,"phone":28,"phoneExt":28,"email":28},"Daniel Dorgan, MD",{"facility":293,"status":42,"city":294,"state":295,"zip":296,"country":46,"countryCode":47,"cosmosGeoPoint":297,"geoPoint":301,"contacts":302},"Medical University of South Carolina","Charleston","South Carolina","29425",{"type":49,"coordinates":298},[299,300],-79.93275,32.77632,{"lat":300,"lon":299},[303,306],{"name":304,"role":36,"phone":28,"phoneExt":28,"email":305},"Cameron Mathison","mathisoc@musc.edu",{"name":307,"role":60,"phone":28,"phoneExt":28,"email":28},"Patrick Flume, MD",{"facility":309,"status":42,"city":310,"state":311,"zip":312,"country":46,"countryCode":47,"cosmosGeoPoint":313,"geoPoint":317,"contacts":318},"The University of Texas Southwestern Medical Center","Dallas","Texas","75390",{"type":49,"coordinates":314},[315,316],-96.80667,32.78306,{"lat":316,"lon":315},[319,322],{"name":320,"role":36,"phone":28,"phoneExt":28,"email":321},"Maria McLeod","maria.mcleod@utsouthwestern.edu",{"name":323,"role":60,"phone":28,"phoneExt":28,"email":28},"Raksha Jain, MD",{"facility":325,"status":42,"city":326,"state":327,"zip":328,"country":46,"countryCode":47,"cosmosGeoPoint":329,"geoPoint":333,"contacts":334},"Virginia Commonwealth University Health System","Richmond","Virginia","23298",{"type":49,"coordinates":330},[331,332],-77.46026,37.55376,{"lat":332,"lon":331},[335,338],{"name":336,"role":36,"phone":28,"phoneExt":28,"email":337},"Margaret K. Lessard","margaret.lessard@vcuhealth.org",{"name":339,"role":60,"phone":28,"phoneExt":28,"email":28},"Nauman A. Chaudary, MD",{"facility":341,"status":42,"city":342,"state":343,"zip":344,"country":46,"countryCode":47,"cosmosGeoPoint":345,"geoPoint":349,"contacts":350},"University of Washington Medical Center","Seattle","Washington","98195",{"type":49,"coordinates":346},[347,348],-122.33207,47.60621,{"lat":348,"lon":347},[351,354],{"name":352,"role":36,"phone":28,"phoneExt":28,"email":353},"Roshni Prabhu","rprabhu@medicine.washington.edu",{"name":355,"role":60,"phone":28,"phoneExt":28,"email":28},"Christopher Goss, MD",{"type":357,"investigatorFullName":28,"investigatorTitle":28,"investigatorAffiliation":28,"oldNameTitle":28,"oldOrganization":28},"SPONSOR","100458331","phase-2-4d-710-in-adult-patients-with-cystic-fibrosis-100458331",false,"NCT05248230","4D-710 in Adult Patients With Cystic Fibrosis","An Open-label, Phase 1\u002F2 Trial of Gene Therapy 4D-710 in Adults With Cystic Fibrosis","CF","Key Inclusion Criteria (Primary Study):\n\n1. 18 years and older\n2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:\n\n   1. Sweat chloride ≥ 60 mmol\u002FL\n   2. Mutation Status\n\n      * Bi-allelic mutations in the CFTR gene, or\n      * Single mutation in the CFTR gene and clinical manifestations of CF lung disease\n   3. Ineligible for CFTR modulator therapy, or previously received modulator therapy but discontinued due to adverse effects.\n3. Forced expiratory volume in 1 second (FEV1) ≥ 50% and ≤ 90% of predicted (per Global Lung Function Initiative) at Screening\n4. Resting oxygen saturation ≥ 92% on room air at Screening\n\nKey Inclusion Criteria (Sub-Study):\n\n1. 18 years and older\n2. Confirmed diagnosis of cystic fibrosis (CF) and CF lung disease including:\n\n   1. Sweat chloride ≥ 60 mmol\u002FL\n   2. Mutation Status\n\n      * Bi-allelic mutations in the CFTR gene, or\n      * Single mutation in the CFTR gene and clinical manifestations of CF lung disease\n3. Currently on a stable dose of CFTR modulator therapy for a minimum of 60 days prior to Screening and agree to maintain current regimen through the 24-month Observation Period\n4. FEV1 ≥ 40% and \\\u003C 70% predicted (per Global Lung Function Initiative) at Screening, AND\u002FOR experienced at least 2 pulmonary exacerbations in the last year requiring intravenous antibiotics\n\nKey Exclusion Criteria (Primary and Sub Study):\n\n1. Any prior gene therapy for any indication (Exception: mRNA-based or antisense oligonucleotide therapies are not exclusionary)\n2. Active Mycobacterium abscessus infection requiring ongoing treatment at Screening\n3. Active allergic bronchopulmonary aspergillosis requiring management with systemic corticosteroids or antifungal therapy\n4. Smoking (e.g. cigarettes, cigars, cannabis) or use of a vaping product during the 2 months prior to screening; must agree to not smoke or use vaping products during Screening and initial 24-month observation period.\n5. Contraindication to systemic corticosteroid therapy\n6. Requires chronic use of systemic corticosteroids or immunosuppressants to treat another condition\n7. If no known diagnosis of cystic fibrosis related diabetes (CFRD), Type I, or Type II diabetes: Hemoglobin A1C ≥ 6.5% at Screening\n8. If known diagnosis of CFRD, Type I or Type II diabetes: Hemoglobin A1C \\> 7.5% at Screening\n9. Recent history of symptomatic hyperglycemia or unstable blood glucose levels as per Investigator's assessment\n10. Other conditions that, in the Investigator's opinion, may interfere with management of corticosteroid-related hyperglycemia\n11. Body Mass Index (BMI) \\\u003C 16\n12. Laboratory abnormalities at screening:\n\n    * ALT, AST or GGT ≥ 3 × the upper limit of normal (ULN)\n    * Total bilirubin ≥ 2 × ULN\n    * Hemoglobin \\\u003C 10 g\u002FdL\n13. Requirement for continuous or night-time oxygen supplementation\n14. Known CF liver disease with evidence of multilobular cirrhosis\n15. History of thrombosis (excluding catheter-related thrombosis) or conditions associated with increased risk of thrombosis","ALL","18 Years",{"count":369,"type":370},30,"ESTIMATED","INTERVENTIONAL",[373],"PHASE2","This is a Phase 1\u002F2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.",[376],"Cystic Fibrosis Lung",[364,378,379],"Cystic Fibrosis","Gene Therapy","2026-03-09",{"date":382,"type":383},"2026-03-11","ACTUAL",{"date":385,"type":383},"2022-03-29",{"date":387,"type":370},"2031-06",{"name":5,"class":6},20]