[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Entrada Therapeutics, Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":96},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,49,74],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":48},"100645342","phase-2-long-term-extension-study-in-participants-with-duchenne-muscular-dystrophy-amenable-to-exon-skipping-to-evaluate-the-safety-and-efficacy-of-endosomal-escape-vehicle-phosphorodiamidate-morpholino-oligomer-platform-products-elevate-lte-100645342",false,"NCT07682129","Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Evaluate the Safety and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)","A Phase 2, Open-Label Long-Term Extension Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon Skipping to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Efficacy of Endosomal Escape Vehicle Phosphorodiamidate Morpholino Oligomer Platform Products (ELEVATE-LTE)","ELEVATE-LTE","Inclusion Criteria:\n\n* Willing and able to provide consent (if at age of majority) or assent (if a minor)\n* Participant completed clinical study ENTR-601-44-201 or ENTR-601-45-201\n* Males who are sexually active with a female partner of childbearing potential must agree to use condoms during sexual intercourse\n\nExclusion Criteria:\n\n* Any change from the applicable parent study eligibility criteria, including safety events during the parent study, that in the opinion of the investigator in consultation with the medical monitor and\u002For sponsor designee precludes safe use of study drug\n* Participant has a condition or circumstance that in the view of the investigator places the subject at high risk of poor treatment compliance or for not completing the study","MALE","4 Years","20 Years",{"count":21,"type":22},80,"ESTIMATED","INTERVENTIONAL",[25],"PHASE2","This is a study of investigational medicines ENTR-601-44 and ENTR-601-45 designed to evaluate the long-term safety and tolerability of study drugs in participants with Duchenne muscular dystrophy (DMD).\n\nThe investigational medicines are currently being investigated in multiple ascending dose parent studies. After participants complete their respective parent study, there is a need to understand the effects of long-term administration of ENTR-601-44 and ENTR-601-45.\n\nParticipants enrolling in this study will begin this long-term extension (LTE) study at the dose level they received upon completion of the parent study with possible dose escalation in the LTE study based on emerging safety and efficacy data from the parent studies.\n\nParticipants will:\n\n* Receive study treatment in the form of multiple intravenous (IV) infusions (slow injections) into a vein over the course of several weeks\n* Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and excersice tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a results of the study drug.\n\nParticipants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.",[28],"Duchenne Muscular Dystrophy (DMD)",[30,31,32,33,34,35],"Duchenne Muscular Dystrophy","DMD","Exon 44 skipping","Exon 45 skipping","exon skipping therapy","oligonucleotide therapy","NOT_YET_RECRUITING","2026-06-26",{"date":39,"type":40},"2026-07-02","ACTUAL",{"date":42,"type":22},"2026-08",{"date":44,"type":22},"2032-03",{"name":46,"class":47},"Entrada Therapeutics, Inc.","INDUSTRY",17,{"id":50,"slug":51,"hasResults":11,"nctId":52,"briefTitle":53,"officialTitle":54,"acronym":55,"eligibilityCriteria":56,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":57,"targetDuration":4,"studyType":23,"phases":59,"briefSummary":61,"conditions":62,"keywords":63,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":65,"lastUpdatePostDateStruct":66,"startDateStruct":68,"completionDateStruct":70,"leadSponsor":72,"locationsCount":73},"100595928","phase-1-a-study-in-participants-with-duchenne-muscular-dystrophy-amenable-to-exon-45-skipping-to-evaluate-the-safety-and-efficacy-of-entr-601-45-100595928","NCT07038824","A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45","A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping With an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-45, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-45 (ELEVATE-45)","ELEVATE-45","Inclusion Criteria:\n\n1. Genetic diagnosis of DMD and confirmed pathologic variant in the dystrophin gene amenable to exon 45 skipping as reviewed by a central genetic counselor.\n2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.\n3. Part A: 4-20 years of age, inclusive.\n4. Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening.\n5. Adequate muscle for obtaining tissue biopsy as assessed by the investigator.\n6. Other protocol-defined criteria apply.\n\nExclusion Criteria:\n\n1. Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.\n2. Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety.\n3. Use of the following medications :\n\n   1. Prior or current treatment with any exon skipping therapy within the previous 12 months\n   2. Prior or current treatment with any gene therapy\n   3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents from 30 days prior to screening and until the end of the study\n   4. Use of an immunosuppressant (other than systemic or oral corticosteroid for DMD condition) from 30 days prior to screening until the end of the study.\n   5. Treatment with a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat from 30 days prior to screening until the end of the study\n4. Laboratory abnormalities.\n5. Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.\n6. Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and\u002For a QT interval with Fridericia correction method (QTcF) \\>450 msec at Screening or prior to the first dose of study drug on Day 1.\n7. Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).\n8. Other protocol-defined criteria apply.",{"count":58,"type":22},24,[60,25],"PHASE1","This is a study of the investigational medicine ENTR-601-45 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition. The researchers want to: Test how safe ENTR-601-45 is, learn about any side effects, and look at the potential positive effects of ENTR-601-45, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-45 and placebo are both called study treatments.\n\nThe study has 2 parts: Part A: to evaluate if ENTR-601-45 is safe and to determine the best dose of ENTR-601-45 for Part B. Part B: to further evaluate the effect and safety of ENTR-601-45 at the dose determined in Part A. Participants will be able to roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated.\n\nParticipants will:\n\n* Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B\n* Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, muscle biopsies and exercise tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a result of the study drug.\n\nParticipants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.",[28],[30,31,34,35],"RECRUITING","2026-05-15",{"date":67,"type":40},"2026-05-18",{"date":69,"type":40},"2025-08-30",{"date":71,"type":22},"2029-03-01",{"name":46,"class":47},15,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":80,"eligibilityCriteria":81,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":82,"targetDuration":4,"studyType":23,"phases":83,"briefSummary":84,"conditions":85,"keywords":86,"overallStatus":64,"whyStopped":4,"lastUpdateSubmitDate":87,"lastUpdatePostDateStruct":88,"startDateStruct":90,"completionDateStruct":92,"leadSponsor":94,"locationsCount":95},"100595854","phase-1-a-study-in-participants-with-duchenne-muscular-dystrophy-amenable-to-exon-44-skipping-to-evaluate-the-safety-and-efficacy-of-entr-601-44-100595854","NCT07037862","A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44","A 2-Part, Randomized, Double-Blind, Placebo-Controlled Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping With an Initial Multiple Ascending Dose Part A to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ENTR-601-44, Followed by Part B to Evaluate the Safety and Efficacy of ENTR-601-44 (ELEVATE-44)","ELEVATE-44","Principal inclusion criteria\n\n1. Genetic diagnosis of Duchenne muscular dystrophy (DMD) and confirmed pathologic variant in the dystrophin gene amenable to exon 44 skipping as reviewed by a central genetic counselor.\n2. Assigned male at birth with clinical signs compatible with Duchenne muscular dystrophy as determined by the investigator.\n3. Part A: 4-20 years of age, inclusive.\n4. Ambulatory Status Part A: ambulatory with a Performance of the Upper Limb v2.0 (PUL 2.0) Entry as per protocol at Screening\n5. Adequate muscle for obtaining tissue biopsy as assessed by the investigator.\n6. Other protocol-defined criteria apply.\n\nPrincipal exclusion criteria\n\n1. Any significant concomitant medical condition that might interfere with the ability to comply with protocol requirements.\n2. Has an acute illness within 4 weeks prior to the first dose of study drug which may interfere with study measurements or jeopardize participant's safety.\n3. Use of the following medications:\n\n   1. Prior treatment with any exon skipping therapy at any time\n   2. Prior treatment with any gene therapy at any time\n   3. Use of anti-coagulants, anti-thrombotics, or anti-platelet agents\n   4. Use of an immunosuppressants (other than oral corticosteroids for DMD conditions)\n   5. Has taken or is currently taking a histone deacetylase (HDAC) inhibitor, including (but not limited to) givinostat\n4. Laboratory abnormalities.\n5. Daytime ventilator dependence or any use of invasive mechanical ventilation via tracheostomy.\n6. Has an abnormal electrocardiogram (ECG) reading assessed as clinically significant by the investigator, and\u002For a QT interval with Fridericia correction method (QTcF) \\>450 msec at Screening or prior to the first dose of study drug on Day 1.\n7. Received any experimental or investigational drug, etc. within 3 months prior to first dose or within 5 half-lives (whichever is longer).\n8. Other protocol-defined criteria apply.",{"count":58,"type":22},[60,25],"This is a study of the investigational medicine ENTR-601-44 in participants who have Duchenne muscular dystrophy (DMD), a rare genetic condition.\n\nThe researchers want to: Test how safe ENTR-601-44 is, learn about any side effects, and look at the potential positive effects of ENTR-601-44, compared to placebo. Placebo looks like the investigational medicine but does not contain any active ingredient. In this summary ENTR-601-44 and placebo are both called study treatments.\n\nThe study has 2 parts:\n\n* Part A\n\n  * A Double-Blind Period, to evaluate if ENTR-601-44 is safe and to determine the best dose of ENTR-601-44 for Part B.\n  * Following the Double-Blind period, participants will roll into an open-label treatment period during which the safety and efficacy of extended dosing will be evaluated.\n* Part B\n\n  * To further evaluate the effect and safety of ENTR-601-44 at the dose determined in Part A.\n\nParticipants will:\n\n* Receive study treatment in the form of multiple intravenous (IV) infusions (slow injection) into a vein over the course of several weeks in Part A and in Part B\n* Visit the clinic regularly for checkups and tests such as: blood and urine tests, physical examinations, questionnaires, and exercise tests. Participants will have a muscle biopsy at the beginning of their participation and after their last dose to allow researchers to compare whether there have been changes in the muscle as a result of the study drug.\n\nParticipants are allowed to continue receiving their standard of care therapy for DMD during the study, as long as their health remains stable.",[28],[30,31,34,35],"2026-03-06",{"date":89,"type":40},"2026-03-09",{"date":91,"type":40},"2025-06-30",{"date":93,"type":22},"2029-03-28",{"name":46,"class":47},14,""]