[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"4D Molecular Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":154},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,59,86,111],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":51,"completionDateStruct":53,"leadSponsor":55,"locationsCount":58},"100597920","phase-3-single-intravitreal-ivt-injection-of-4d-150-in-patients-with-macular-neovascularization-secondary-to-age-related-macular-degeneration-100597920",false,"NCT07064759","Single Intravitreal (IVT) Injection of 4D-150 in Patients With Macular Neovascularization Secondary to Age-Related Macular Degeneration","A Phase 3, Randomized, Double-Masked, Active-Controlled Trial of a Single Intravitreal Injection of 4D-150 in Adults With Macular Neovascularization Secondary to Age-Related Macular Degeneration","4FRONT-2","Inclusion Criteria:\n\n* ≥50 years of age at time of consent\n* MNV secondary to nAMD with IVT anti-VEGF treatment history in the study eye, defined as EITHER:\n\n  1. Treatment naïve, i.e. no prior IVT anti-VEGF therapy OR\n  2. Previously treated with no more than 4 IVT anti-VEGF injections due to nAMD and received diagnosis of nAMD no more than 6 months prior to the Screening Visit AND documented evidence of anatomical improvement and visual stability\u002Fimprovement in response to previous IVT anti-VEGF treatment, as determined by the Investigator\n* Active subfoveal MNV or juxtafoveal\u002F extrafoveal MNV with a subfoveal component (where activity is defined as evidence of SRF, IRF, subretinal hyperreflective material, or leakage) identified by fluorescein angiography (FA) or spectral domain optical coherence tomography (SD-OCT) , in the study eye, at the Screening Visit confirmed by the Reading Center\n* Demonstrated clinical response to aflibercept and functional stability in the study eye as confirmed by the Reading Center\n* BCVA between 25 and 78 ETDRS letters, inclusive (20\u002F320-20\u002F32 Snellen equivalent) in the study eye at the Screening Visit\n* CST less than or equal to 500 microns in the study eye at screening visit, confirmed by the central reading center\n\nExclusion Criteria:\n\nOcular Conditions:\n\n* MNV due to causes other than nAMD in either eye (Fibrosis, atrophy, or subretinal hemorrhage in the foveal central subfield (1 mm diameter)\n* History of retinal detachment in the study eye\n* History of or presence of active inflammation in either eye\n* Glaucoma or intraocular hypertension requiring more than 2 topical medications for control\n\nOcular Treatments\u002FInterventions:\n\n* Any prior or concomitant treatment for MNV or vitreomacular-interface abnormalities, other than allowed prior IVT anti-VEGF in the study eye\n\nSystemic Conditions and Considerations:\n\n* Major illness or major surgical procedure in the 28 days prior to the Screening Visit\n* Uncontrolled blood pressure\n* Acute coronary syndrome, myocardial infarction or coronary artery revascularization, cerebrovascular accident, transient ischemic attack within 6 months of the Screening Visit\n* History of autoimmune condition that may predispose to the development of uveitis","ALL","50 Years",{"count":20,"type":21},480,"ESTIMATED","INTERVENTIONAL",[24],"PHASE3","A Phase 3, Randomized, Double-Masked, Active-Controlled Trial in Adults with Macular Neovascularization Secondary to Age-Related Macular Degeneration",[27],"Neovascular Age-Related Macular Degeneration (nAMD)",[29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45,15],"Age-related macular degeneration","AMD","Exudative AMD","Neovascular AMD","Exudative age-related macular degeneration","Neovascular age-related macular degeneration","Wet age-related macular degeneration","Wet macular degeneration","Wet AMD","WAMD","nAMD","Retinal gene therapy","Intravitreal gene therapy","Genetic Medicine","Ocular Gene Therapy","Gene Therapy","4FRONT","RECRUITING","2026-04-14",{"date":49,"type":50},"2026-04-16","ACTUAL",{"date":52,"type":50},"2025-07-22",{"date":54,"type":21},"2029-02",{"name":56,"class":57},"4D Molecular Therapeutics","INDUSTRY",94,{"id":60,"slug":61,"hasResults":11,"nctId":62,"briefTitle":63,"officialTitle":64,"acronym":65,"eligibilityCriteria":66,"healthyVolunteers":11,"sex":17,"minAge":67,"maxAge":4,"enrollmentInfo":68,"targetDuration":4,"studyType":22,"phases":70,"briefSummary":72,"conditions":73,"keywords":75,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":85},"100458331","phase-2-4d-710-in-adult-patients-with-cystic-fibrosis-100458331","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","18 Years",{"count":69,"type":21},30,[71],"PHASE2","This is a Phase 1\u002F2 multicenter, open-label, single dose trial of 4D-710 investigational gene therapy in adults with cystic fibrosis.",[74],"Cystic Fibrosis Lung",[65,76,44],"Cystic Fibrosis","2026-03-09",{"date":79,"type":50},"2026-03-11",{"date":81,"type":50},"2022-03-29",{"date":83,"type":21},"2031-06",{"name":56,"class":57},20,{"id":87,"slug":88,"hasResults":11,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":4,"eligibilityCriteria":92,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":4,"enrollmentInfo":93,"targetDuration":4,"studyType":22,"phases":95,"briefSummary":97,"conditions":98,"keywords":100,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":102,"lastUpdatePostDateStruct":103,"startDateStruct":105,"completionDateStruct":107,"leadSponsor":109,"locationsCount":110},"100454417","phase-1-4d-150-in-patients-with-neovascular-wet-age-related-macular-degeneration-100454417","NCT05197270","4D-150 in Patients With Neovascular (Wet) Age-Related Macular Degeneration","A Phase 1\u002F2 Dose-Escalation and Randomized, Controlled, Masked Expansion Trial of Intravitreal 4D-150 Gene Therapy in Adults With Neovascular (Wet) Age-Related Macular Degeneration","* 50 years of age\n\nIndividuals eligible to participate in the trial must meet the following inclusion criteria:\n\n1. ≥50 years of age\n2. Diagnosed with CNV secondary to AMD (confirmed by reading center)\n3. BCVA ≥34 ETDRS letters (\\~20\u002F200) in the contralateral eye, and BCVA in the study eye:\n4. Central subfield thickness (CST) and\u002For presence of subretinal or intraretinal fluid requiring continued anti-VEGF therapy in the study eye, as assessed by SD-OCT; and confirmed by a reading center):\n5. Study eye amenable to IVT injection\n6. Ability to perform tests of visual and retinal function and structure, and ability to comply with other protocol-specified procedures\n7. Currently receiving anti-VEGF treatment (e.g. ranibizumab, aflibercept, faricimab or bevacizumab) in the study eye AND has demonstrated a clinical response consistent with anti-VEGF activity within 12 months prior to screening:\n\n   Note: Day -7 aflibercept will be administered ≥ 4 weeks from last IVT anti-VEGF\n8. Subjects receiving 4D-150 agree to use a barrier method (e.g. condom) during intercourse for 6 months after administration of 4D-150 to prevent fluid transmission; sexually active males should not father a child or donate sperm during this period\n9. Medical records available to document history of anti-VEGF therapy (dates, drugs, and dosage) for a minimum of 12 months prior to Screening\n10. Provide written informed consent.\n\nContralateral Eye Sub-study-Specific Criteria:\n\n1. Received 4D-150 in study eye-1 at least 6 months prior to Contralateral Substudy Screening Visit NOTE: Subjects who received 4D-150 in study eye-1 as part of the Shedding Substudy are also eligible to participate in the Contralateral Eye Substudy only after biological samples from all matrices were at or below the limit of quantitation for vector genomes in at least 3 consecutive measurements AND at least 6 months has passed since 4D-150 administration to study eye-1.\n2. Macular neovascularization (MNV) secondary to AMD in study eye-2 as assessed on historical images at any time by SD-OCT (required) and FA when available or fundus photography when available or at screening based on SD-OCT, FA and fundus photography (confirmed by the reading center)\n3. History of at least 1 anti-VEGF injection in study eye-2 within 6 months prior to Screening (last anti-VEGF injection must be at least 28 days prior to Screening) AND has demonstrated a clinical response consistent with anti-VEGF activity by historical OCT at any time point prior to screening with confirmed response by the reading center (Screening OCT can be confirmatory)\n4. BCVA between 25 and 83 ETDRS letters, inclusive (20\u002F320 20\u002F25 Snellen) in study eye-2\n5. Study eye-2 amenable to IVT injection\n6. BCVA ≥34 ETDRS letters (\\~20\u002F200) in the previously treated study eye-1\n7. Ability to comply with protocol-specified procedures and visits, in the Investigator's judgment\n8. Agree to use a barrier method (e.g. condom) during intercourse for 6 months after administration of 4D-150 to study eye-2 to prevent fluid transmission; sexually active males should not father a child or donate sperm during this period\n9. Provide written informed consent.\n\nShedding Substudy-specific Inclusion Criteria:\n\n1. ≥50 years of age\n2. Diagnosed with MNV secondary to AMD as assessed on historical images at any time by spectral domain optical coherence tomography (SD-OCT) (required) and fluorescein angiography when available or fundus photography when available or at screening based on SD-OCT, FA and fundus photography (confirmed by Reading Center)\n3. Best corrected visual acuity (BCVA) between 10 and 83 ETDRS letters, inclusive (\\~20\u002F640 and 20\u002F25, respectively) in the study eye at Screening\n4. BCVA ≥34 ETDRS letters (\\~20\u002F200) in the contralateral eye\n5. Currently receiving anti-VEGF treatment (e.g. ranibizumab, aflibercept, faricimab or bevacizumab) in the study eye; minimum of 1 injection within the last 6 months (last anti-VEGF injection must be at least 28 days prior to screening) AND has demonstrated a clinical response consistent with anti-VEGF activity by historical OCT at any time prior to screening with confirmed response by the reading center (Screening OCT can be confirmatory)\n6. Study eye amenable to IVT injection\n7. Ability to perform tests of visual and retinal function and structure, and ability to comply with other protocol-specified procedures, in the Investigator's judgment\n8. Agree to use a barrier method (e.g. condom) during intercourse for 6 months after administration of 4D-150 to prevent fluid transmission; sexually active males should not father a child or donate sperm during this period\n9. Provide written informed consent.",{"count":94,"type":21},215,[96,71],"PHASE1","Phase 1\u002F2 dose-escalation and randomized, controlled, masked expansion trial in adults with wet AMD undergoing active anti-VEGF treatment.\n\nSubstudies will evaluate the safety and tolerability of 4D-150 contralateral eye dosing and characterize vector shedding.",[99],"Neovascular (Wet) Age-Related Macular Degeneration",[29,30,31,33,32,34,35,36,37,101,40,41,42,43],"wAMD","2026-02-10",{"date":104,"type":50},"2026-02-12",{"date":106,"type":50},"2021-12-09",{"date":108,"type":21},"2031-01",{"name":56,"class":57},25,{"id":112,"slug":113,"hasResults":11,"nctId":114,"briefTitle":115,"officialTitle":116,"acronym":4,"eligibilityCriteria":117,"healthyVolunteers":11,"sex":17,"minAge":67,"maxAge":118,"enrollmentInfo":119,"targetDuration":4,"studyType":22,"phases":121,"briefSummary":122,"conditions":123,"keywords":125,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":146,"lastUpdatePostDateStruct":147,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":5},"100487628","phase-1-4d-310-in-adults-with-fabry-disease-and-cardiac-involvement-100487628","NCT05629559","4D-310 in Adults With Fabry Disease and Cardiac Involvement","An Open-Label, Phase 1\u002F2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement","Inclusion Criteria:\n\n1. Male or female ≥ 18 to ≤65 years of age\n2. Pathogenic GLA mutation consistent with Fabry Disease\n3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement\n4. Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat\n5. Individuals receiving ERT, must be on a stable dose for at least 6 months\n6. Agree to use highly effective contraception\n\nExclusion Criteria:\n\n1. Presence of pre-existing antibodies to 4D-310 capsid or to AGA\n2. eGFR \\\u003C65 mL\u002Fmin\u002F1.73 m2\n3. Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis\n4. HIV, active or chronic hepatitis B or C,\n5. Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control\n6. History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)\n7. Contraindication to systemic corticosteroid therapy or immunosuppressive therapy\n8. Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.\n9. Moderately severe to severe cardiovascular disease or uncontrolled hypertension\n10. Left ventricular ejection fraction of \\\u003C45% on echocardiogram (ECHO)\n11. Currently receiving investigational drug, device or therapy or having ever received gene therapy\n12. History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation\n13. History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)\n14. Pregnant or breast-feeding\n\nNote: Other inclusion and exclusion criteria apply","65 Years",{"count":120,"type":21},18,[96,71],"This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement",[124],"Fabry Disease",[126,127,128,129,130,131,132,133,134,135,136,137,138,139,140,141,142,143,144,145],"Lysosomal Storage Diseases","Nervous System Brain Diseases","Metabolic","Inborn Brain Diseases","Central Nervous System Diseases","Nervous System Diseases","Cerebral Small Vessel Diseases","Cerebrovascular Disorders","Vascular Diseases","Cardiovascular Diseases","Genetic Diseases","X-Linked","Inborn","Metabolic Diseases","Lipid Metabolism Disorders","Sphingolipidoses","Metabolism","Inborn Errors","Lipidoses","Lipid Metabolism","2025-02-19",{"date":148,"type":50},"2025-02-21",{"date":150,"type":50},"2022-10-16",{"date":152,"type":21},"2030-06",{"name":56,"class":57},""]