[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100625946":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":21,"locations":27,"responsibleParty":67,"collaborators":20,"id":69,"slug":70,"hasResults":71,"nctId":72,"briefTitle":73,"officialTitle":74,"acronym":20,"eligibilityCriteria":75,"healthyVolunteers":71,"sex":76,"minAge":77,"maxAge":78,"enrollmentInfo":79,"targetDuration":20,"studyType":82,"phases":83,"briefSummary":86,"conditions":87,"keywords":89,"overallStatus":30,"whyStopped":20,"lastUpdateSubmitDate":99,"lastUpdatePostDateStruct":100,"startDateStruct":103,"completionDateStruct":105,"leadSponsor":107,"locationsCount":108},{"fullName":5,"class":6},"Precision BioSciences, Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Experimental- Part 1 (Initial Safety) & Part 2 (Expansion) cohort","EXPERIMENTAL","The trial is planned to enroll participants into 2 parts as follows:\n\n* Part 1 (Initial Safety) A total of up to 6 participants may be enrolled.\n* Part 2 (Expansion) Up to 12 participants",[13],"Biological: PBGENE-DMD (IV)",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"BIOLOGICAL","PBGENE-DMD (IV)","Participants will receive a single dose of PBGENE-DMD",[9],null,[22],{"name":23,"role":24,"phone":25,"phoneExt":20,"email":26},"Precision BioSciences Clin Ops","CONTACT","(800) 593-0346","function-DMD@precisionbiosciences.com",[28,50],{"facility":29,"status":30,"city":31,"state":32,"zip":33,"country":34,"countryCode":35,"cosmosGeoPoint":36,"geoPoint":41,"contacts":42},"Arkansas Children's Hospital","RECRUITING","Little Rock","Arkansas","72202","United States","US",{"type":37,"coordinates":38},"Point",[39,40],-92.28959,34.74648,{"lat":40,"lon":39},[43,47],{"name":44,"role":24,"phone":45,"phoneExt":20,"email":46},"Kristin MacLean","501-364-2079","MacleanKD@archildrens.org",{"name":48,"role":49,"phone":20,"phoneExt":20,"email":20},"Aravindhan Veerapandiyan, MD","PRINCIPAL_INVESTIGATOR",{"facility":51,"status":30,"city":52,"state":53,"zip":54,"country":34,"countryCode":35,"cosmosGeoPoint":55,"geoPoint":59,"contacts":60},"Washington University School of Medicine","St Louis","Missouri","63110",{"type":37,"coordinates":56},[57,58],-90.19789,38.62727,{"lat":58,"lon":57},[61,65],{"name":62,"role":24,"phone":63,"phoneExt":20,"email":64},"Natalie Goedeker","314-362-4919","NeuromusclePediatricResearch@wustl.edu",{"name":66,"role":49,"phone":20,"phoneExt":20,"email":20},"Craig Zaidman, MD",{"type":68,"investigatorFullName":20,"investigatorTitle":20,"investigatorAffiliation":20,"oldNameTitle":20,"oldOrganization":20},"SPONSOR","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":80,"type":81},18,"ESTIMATED","INTERVENTIONAL",[84,85],"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.",[88],"Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD",[90,91,92,93,94,95,96,97,98],"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","2026-06-22",{"date":101,"type":102},"2026-06-23","ACTUAL",{"date":104,"type":102},"2026-04-24",{"date":106,"type":81},"2029-12",{"name":5,"class":6},2]