[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100326140":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":21,"centralContacts":22,"locations":32,"responsibleParty":53,"collaborators":21,"id":57,"slug":58,"hasResults":59,"nctId":60,"briefTitle":61,"officialTitle":62,"acronym":21,"eligibilityCriteria":63,"healthyVolunteers":59,"sex":64,"minAge":21,"maxAge":21,"enrollmentInfo":65,"targetDuration":21,"studyType":68,"phases":69,"briefSummary":72,"conditions":73,"keywords":75,"overallStatus":34,"whyStopped":21,"lastUpdateSubmitDate":80,"lastUpdatePostDateStruct":81,"startDateStruct":84,"completionDateStruct":86,"leadSponsor":88,"locationsCount":89},{"fullName":5,"class":6},"University of Southern California","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Gentamicin Sulfate","EXPERIMENTAL","IV Arm:\n\n7.5 mg\u002Fkg gentamicin once daily for 14 days.\n\nTopical Arm:\n\n0.5% gentamicin ointment applied twice daily for 14 days to selected skin sites.",[13],"Drug: Gentamicin Sulfate",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"DRUG","Gentamicin (formulated as gentamicin sulfate) is a well-known, well-characterized antibiotic that has been used for four decades as a treatment against gram negative bacteria. It, like other aminoglycoside antibiotics, has the well documented added potential to facilitate readthrough of premature termination codons in eukaryotic cells and organisms.",[9],[20],"Gentamicin",null,[23,28],{"name":24,"role":25,"phone":26,"phoneExt":21,"email":27},"Mei Chen, Ph.D.","CONTACT","3238650621","chenm@usc.edu",{"name":29,"role":25,"phone":30,"phoneExt":21,"email":31},"David Woodley, M.D.","3238650956","dwoodley@usc.edu",[33],{"facility":5,"status":34,"city":35,"state":36,"zip":37,"country":38,"countryCode":39,"cosmosGeoPoint":40,"geoPoint":45,"contacts":46},"RECRUITING","Los Angeles","California","90033","United States","US",{"type":41,"coordinates":42},"Point",[43,44],-118.24368,34.05223,{"lat":44,"lon":43},[47,50],{"name":48,"role":25,"phone":49,"phoneExt":21,"email":31},"David Woodley, MD","323-865-0956",{"name":51,"role":25,"phone":52,"phoneExt":21,"email":27},"Mei Chen, Ph.D","323-865-0621",{"type":54,"investigatorFullName":55,"investigatorTitle":56,"investigatorAffiliation":5,"oldNameTitle":21,"oldOrganization":21},"PRINCIPAL_INVESTIGATOR","David Woodley","Professor","100326140","phase-1-gentamicin-for-junctional-epidermolysis-bullosa-100326140",false,"NCT03526159","Gentamicin for Junctional Epidermolysis Bullosa","A Pilot Study of the Restoration of Functional Laminin 332 in JEB Patients With Nonsense Mutations After Topical and Intravenous Gentamicin Treatment","Inclusion Criteria:\n\n1\\. JEB patients with nonsense mutations in the LAMB3 gene in either one or two alleles.\n\nExclusion Criteria:\n\n1. JEB patients who do not have nonsense mutations in the LAMB3 gene in either allele.\n2. Pre-existing known auditory impairment.\n3. Pre-existing known renal impairment.\n4. Pre-existing known allergies to aminoglycosides or sulfate compounds.\n5. Pregnancy.","ALL",{"count":66,"type":67},6,"ESTIMATED","INTERVENTIONAL",[70,71],"PHASE1","PHASE2","Herlitz junctional epidermolysis bullosa (H-JEB), an incurable, fatal, inherited skin disease, is caused by loss-of-function mutations in the LAMA3, LAMB3 or LAMC2 genes, resulting in loss of laminin 332 and poor epidermal-dermal adherence. Eighty percent of H-JEB patients have LAMB3 mutations and about 95% of these are nonsense mutations. The investigators recently demonstrated that gentamicin readily induced nonsense mutation readthrough and produced full-length laminin beta3 in several nonsense mutations tested. Importantly, the gentamicin-induced laminin beta3 restored laminin 332 assembly, secretion, and deposition into the dermal-epidermal junction (DEJ). Newly induced laminin 332 reversed abnormal H-JEB cellular phenotypes. Herein, the investigators propose the first clinical trial of gentamicin (by topical and intravenous administration) in JEB patients with nonsense mutations. The milestones will include restored laminin 332 and hemidesmosomes at the DEJ, improved wound closure, and the absence of significant gentamicin side effects.",[74],"Junctional Epidermolysis Bullosa",[76,77,78,79],"Herlitz","Laminin 332","Laminin beta 3","JEB","2020-04-03",{"date":82,"type":83},"2020-04-07","ACTUAL",{"date":85,"type":83},"2018-06-01",{"date":87,"type":67},"2020-08-31",{"name":5,"class":6},1]