[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"gjb2-mediated-hearing-loss\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:gjb2-mediated-hearing-loss":29},{"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":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":32,"overallStatus":39,"whyStopped":4,"lastUpdateSubmitDate":40,"lastUpdatePostDateStruct":41,"startDateStruct":44,"completionDateStruct":46,"leadSponsor":48,"locationsCount":51},"100642992","phase-1-open-label-study-of-sky-gjb2-in-pediatric-subjects-with-gjb2-mediated-hearing-loss-100642992",false,"NCT07627971","Open-label Study of SKY-GJB2 in Pediatric Subjects With GJB2-mediated Hearing Loss","A Phase 1\u002F2, Open-label, Single Dose Study of SKY-GJB2 Given as a Single Unilateral Intracochlear Administration in Pediatric Subjects With GJB2-mediated Hearing Loss","SONIX","Inclusion Criteria:\n\n* Male or female aged 9 months to 7 years at the time of gene therapy administration\n* Subjects must have at least two (biallelic) pathogenic or likely pathogenic variants in the GJB2 gene\n* Bilateral sensorineural hearing loss as assessed by ABR. Hearing loss at ≥85 dB HL for at least one of the frequencies (500-4000 Hz) in the study treatment ear.\n* Subject's parent(s)\u002Fguardian(s) provide informed consent before the initiation of study-related procedures.\n* Subject is able and willing to comply (or provide assent if old enough) will all study requirements.\n\nExclusion Criteria:\n\n* Subject has non-GJB2 mediated hearing loss including genetic, syndromic, or non-syndromic hearing loss that is not associated with GJB2 mutations.\n* Subject has autosomal dominant nonsyndromic hearing loss due to GJB2 mutation.\n* No response on ABR testing.\n* Bilateral Cochlear Implants.","ALL","9 Months","7 Years",{"count":21,"type":22},10,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","Skylark Bio is conducting a study of an investigational new drug called SKY-GJB2 and its delivery device, the SKY-CAT. SKY-GJB2 is a gene therapy that is being developed to treat children who have hearing loss due to changes in the GJB2 gene (also referred to as Connexin 26). The SKY-CAT is a device that delivers the gene therapy.\n\nThe purpose of this study is to:\n\nLearn about the safety and tolerability (does not cause ongoing discomfort) of SKY-GJB2 Evaluate the efficacy of SKY-GJB2 (how well is works)",[29,30,31],"GJB2-mediated Hearing Loss","GJB2 Gene Mutation","DFNB1A",[33,34,35,36,37,38],"GJB2","Hearing Loss, Bilateral","Hearing Loss","Hearing Loss, Sensorineural","Deafness, Autosomal Recessive 1A (DFNB1A)","Connexin 26","RECRUITING","2026-06-25",{"date":42,"type":43},"2026-06-30","ACTUAL",{"date":45,"type":43},"2026-05-28",{"date":47,"type":22},"2028-06",{"name":49,"class":50},"Skylark Bio Inc.","INDUSTRY",2]