[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"duchenne-muscular-dystrophy-with-mutations-amenable-to-pbgene-dmd\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:duchenne-muscular-dystrophy-with-mutations-amenable-to-pbgene-dmd":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100625946","phase-1-pbgene-dmd-phase-12a-safety-and-preliminary-efficacy-study-in-duchenne-muscular-dystrophy-function-dmd-100625946",false,"NCT07429240","PBGENE-DMD Phase 1\u002F2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)","A Phase 1\u002F2a, Multi-center, Open-label Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of PBGENE-DMD in Participants With Duchenne Muscular Dystrophy (FUNCTION-DMD)","Inclusion Criteria:\n\n1. Males, 2 to 7 years of age, inclusive, at the time of informed consent\u002Fassent\n2. Molecular confirmed DMD diagnosis (DMD mutation fully contained between exons 45 to 55 \\[inclusive\\])\n3. Clinical phenotype consistent with DMD in the opinion of the Investigator\n4. Ability to complete age-appropriate motor testing assessments requirements.\n\n   Participants aged 2 to \\\u003C 4 years at the time of screening must:\n   1. Be able to walk at least 10 meters independently (without assistive devices).\n   2. Be able to rise from the floor without physical assistance (use of a Gowers' maneuver is acceptable).\n\n      Participants aged 4 to 7 years at the time of screening must:\n   3. Be able to walk at least 100 meters independently (without assistive devices).\n   4. Have an NSAA total score between 16 and 29, inclusive.\n5. Participant has received age-appropriate routine childhood immunizations per the local country's national immunization schedule.\n6. The participant's parent(s)\u002FLAR(s) are willing and able to provide written informed consent prior to the initiation of any trial-specific procedures; where applicable, the participant must provide written or verbal assent in accordance with local regulations.\n7. The participant and their parent(s)\u002FLAR(s) are willing to participate in a LTFU study after the completion of this trial.\n\nExclusion Criteria:\n\n1. Prior treatment with any gene therapy, gene editing therapy, or cell-based therapy at any time.\n2. Receipt of any investigational medication or experimental therapy within 6 months prior to Day 1.\n3. Prior or ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose or inability or unwillingness to refrain from initiating or resuming these therapies for at least 5 years following gene therapy administration.\n4. Prior ongoing use of any product designed to increase dystrophin expression, investigational, or otherwise, including exon-skipping therapies, within 6 months of the scheduled Day 1 dose.\n5. Concurrent enrollment in another clinical trial, unless it is observational (non-interventional).\n6. A positive test for antibodies to AAV9\n7. A participant has any condition that would contraindicate treatment with immunosuppression.\n8. Participants with pathogenic mutations in exons 1-44 and\u002For exons 56-79.\n9. Evidence of cardiomyopathy or clinically significant left ventricular dysfunction, defined as LVEF \\\u003C50% on screening echocardiogram.","MALE","2 Years","7 Years",{"count":20,"type":21},18,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","The purpose of this Phase 1\u002F2a trial is to evaluate the safety, tolerability, and preliminary efficacy of PBGENE-DMD in patients with DMD harboring mutations amenable to excision of exons 45-55. Given the limitations of existing therapeutic strategies, PBGENE-DMD represents a novel, innovative approach with the potential for a one-time, durable correction of the underlying genetic defect in the largest molecular subset of patients with DMD.",[28],"Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD",[30,31,32,33,34,35,36,37,38],"Duchenne muscular dystrophy","DMD","X-linked Muscle wasting disease","Muscular dystrophy","Progressive muscle weakness","Gene editing","AAV serotype 9","AAV-9","Exon 45-55 excision","RECRUITING","2026-06-22",{"date":42,"type":43},"2026-06-23","ACTUAL",{"date":45,"type":43},"2026-04-24",{"date":47,"type":21},"2029-12",{"name":49,"class":50},"Precision BioSciences, Inc.","INDUSTRY",2]