[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100625946":3},{"organization":4,"outcomesModule":7,"designInfo":32,"detailedDescription":41,"studyPopulation":37,"armGroups":42,"interventions":49,"overallOfficials":37,"centralContacts":55,"locations":61,"responsibleParty":101,"collaborators":37,"id":103,"slug":37,"hasResults":104,"nctId":105,"briefTitle":106,"officialTitle":107,"acronym":37,"eligibilityCriteria":108,"healthyVolunteers":104,"sex":109,"minAge":110,"maxAge":111,"enrollmentInfo":112,"targetDuration":37,"studyType":115,"phases":116,"briefSummary":119,"conditions":120,"keywords":122,"overallStatus":64,"whyStopped":37,"lastUpdateSubmitDate":132,"lastUpdatePostDateStruct":133,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":141},{"fullName":5,"class":6},"Precision BioSciences, Inc.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":18},[9],{"measure":10,"description":11,"timeFrame":12},"Incidence, severity, and causality of treatment-emergent adverse events and serious adverse events","Adverse events and serious adverse events that occur or worsen after initiation of the investigational treatment","From Dosing through Week 104",[14],{"measure":15,"description":16,"timeFrame":17},"Dystrophin expression in skeletal muscle","Measurement of Biologic activity of PBGENE-DMD through dystrophin expression in skeletal muscle","Week 12, Week 52",[19,23,26,29],{"measure":20,"description":21,"timeFrame":22},"Developmental Motor Function 1","Change from baseline in the digital mobility outcome (DMO) of Stride Velocity 95th Centile (SV95C)","Week 52, Week 104",{"measure":24,"description":25,"timeFrame":22},"Developmental Motor Function 2","Change from baseline in Bayley Scales of Infant and Toddler Development, Fourth Edition (Bayley-4) Motor Gross Motor and Fine Motor scores (\\\u003C3 years of age)",{"measure":27,"description":28,"timeFrame":22},"Developmental Motor Function 3","Change from baseline (defined as the first assessment after the participant has reached 3 years of age) in North Star Ambulatory Assessment (NSAA) total score (≥ 3 years of age)",{"measure":30,"description":31,"timeFrame":22},"Developmental Motor Function 4","Change from baseline in participants ≥ 3 years of age in timed performance in 4-stair climb, time to rise (TTR), 10-meter walk\u002Frun (10MWR), 100-meter walk\u002Frun (only age \\>\u002F= 4 years of age)",{"allocation":33,"interventionModel":34,"interventionModelDescription":35,"primaryPurpose":36,"observationalModel":37,"timePerspective":37,"maskingInfo":38},"NA","SINGLE_GROUP","A single dose of PBGENE-DMD administered Intravenously (IV) following screening and pretreatment. Total duration of trial intervention for each participant: approximately 130 weeks","TREATMENT",null,{"masking":39,"maskingDescription":40,"whoMasked":37},"NONE","Open label","This is a Phase 1\u002F2a, open-label, multicenter trial designed to evaluate the safety, tolerability, and primary efficacy of a single IV dose of PBGENE-DMD in male participants with DMD presenting with mutations that may be amenable to treatment with PBGENE-DMD. A structured, multimodal, short-term immunomodulatory regimen will be administered around the time of dosing to mitigate the risk of potential immune-mediated responses.\n\nThe trial consists of two parts: Part 1 is to confirm a safe and well-tolerated single dose of PBGENE-DMD that may be further evaluated in Part 2 (expansion).\n\nA total of up to 18 participants may be enrolled in this trial. Total duration of trial participation for each participant: approximately 130 weeks.",[43],{"label":44,"type":45,"description":46,"interventionNames":47},"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",[48],"Biological: PBGENE-DMD (IV)",[50],{"type":51,"name":52,"description":53,"armGroupLabels":54,"otherNames":37},"BIOLOGICAL","PBGENE-DMD (IV)","Participants will receive a single dose of PBGENE-DMD",[44],[56],{"name":57,"role":58,"phone":59,"phoneExt":37,"email":60},"Precision BioSciences Clin Ops","CONTACT","(800) 593-0346","function-DMD@precisionbiosciences.com",[62,84],{"facility":63,"status":64,"city":65,"state":66,"zip":67,"country":68,"countryCode":69,"cosmosGeoPoint":70,"geoPoint":75,"contacts":76},"Arkansas Children's Hospital","RECRUITING","Little Rock","Arkansas","72202","United States","US",{"type":71,"coordinates":72},"Point",[73,74],-92.28959,34.74648,{"lat":74,"lon":73},[77,81],{"name":78,"role":58,"phone":79,"phoneExt":37,"email":80},"Kristin MacLean","501-364-2079","MacleanKD@archildrens.org",{"name":82,"role":83,"phone":37,"phoneExt":37,"email":37},"Aravindhan Veerapandiyan, MD","PRINCIPAL_INVESTIGATOR",{"facility":85,"status":64,"city":86,"state":87,"zip":88,"country":68,"countryCode":69,"cosmosGeoPoint":89,"geoPoint":93,"contacts":94},"Washington University School of Medicine","St Louis","Missouri","63110",{"type":71,"coordinates":90},[91,92],-90.19789,38.62727,{"lat":92,"lon":91},[95,99],{"name":96,"role":58,"phone":97,"phoneExt":37,"email":98},"Natalie Goedeker","314-362-4919","NeuromusclePediatricResearch@wustl.edu",{"name":100,"role":83,"phone":37,"phoneExt":37,"email":37},"Craig Zaidman, MD",{"type":102,"investigatorFullName":37,"investigatorTitle":37,"investigatorAffiliation":37,"oldNameTitle":37,"oldOrganization":37},"SPONSOR","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":113,"type":114},18,"ESTIMATED","INTERVENTIONAL",[117,118],"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.",[121],"Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD",[123,124,125,126,127,128,129,130,131],"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":134,"type":135},"2026-06-23","ACTUAL",{"date":137,"type":135},"2026-04-24",{"date":139,"type":114},"2029-12",{"name":5,"class":6},2]