[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT07627971":3,"trial-entities:NCT07627971":129,"trial-summary:NCT07627971":135},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":22,"study_type":29,"primary_purpose":30,"phases":31,"enrollment_info":34,"interventions":37,"primary_outcomes":45,"secondary_outcomes":50,"sex":58,"minimum_age":59,"maximum_age":60,"healthy_volunteers":61,"eligibility_criteria":62,"std_ages":75,"locations":77,"central_contacts":122,"overall_officials":123,"references":124,"see_also_links":125},"NCT07627971","SKY-GJB2-001","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","RECRUITING","2028-06","2026-07","2026-07-15","2026-05-28","Skylark Bio Inc.","INDUSTRY",true,"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)",null,[19,20,21],"GJB2-mediated Hearing Loss","GJB2 Gene Mutation","DFNB1A",[23,24,25,26,27,28],"GJB2","Hearing Loss, Bilateral","Hearing Loss","Hearing Loss, Sensorineural","Deafness, Autosomal Recessive 1A (DFNB1A)","Connexin 26","INTERVENTIONAL","TREATMENT",[32,33],"PHASE1","PHASE2",{"count":35,"type":36},10,"ESTIMATED",[38],{"type":39,"name":40,"description":41,"armGroupLabels":42},"COMBINATION_PRODUCT","SKY-GJB2","SKY-GJB2 is intended to be administered via a one-time unilateral intracochlear infusion using the SKY-CAT delivery device.",[43,44],"Older","Younger",[46],{"measure":47,"description":48,"timeFrame":49},"Safety\u002FFrequency of Adverse Events (AEs)","AEs as it relates to the the gene therapy (SKY-GJB2) and\u002For to the administration procedure (including the delivery device system (SKY-CAT))","Through trial completion, approximately one year",[51,54],{"measure":52,"description":53,"timeFrame":49},"Auditory Brainstem Response (ABR) Threshold","Changes from baseline in ABR intensity threshold (measured in decibels (dB HL))",{"measure":55,"description":56,"timeFrame":57},"Performance of the SKY-CAT delivery device","Looking at safety and effectiveness of the device in delivering the gene therapy.","One month post-op","ALL","9 Months","7 Years",false,{"inclusion":63,"exclusion":69,"raw_text":74},[64,65,66,67,68],"Male or female aged 9 months to 7 years at the time of gene therapy administration","Subjects must have at least two (biallelic) pathogenic or likely pathogenic variants in the GJB2 gene","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.","Subject's parent(s)\u002Fguardian(s) provide informed consent before the initiation of study-related procedures.","Subject is able and willing to comply (or provide assent if old enough) will all study requirements.",[70,71,72,73],"Subject has non-GJB2 mediated hearing loss including genetic, syndromic, or non-syndromic hearing loss that is not associated with GJB2 mutations.","Subject has autosomal dominant nonsyndromic hearing loss due to GJB2 mutation.","No response on ABR testing.","Bilateral Cochlear Implants.","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.",[76],"CHILD",[78,93,106],{"facility":79,"status":8,"city":80,"state":81,"zip":82,"country":83,"contacts":84,"geoPoint":90},"Massachusetts Eye and Ear Infirmary (MEEI)","Boston","Massachusetts","02114","United States",[85],{"name":86,"role":87,"phone":88,"email":89},"Michael Cheung, MSc, CCRP, Clinical Research Project Manager","CONTACT","617-573-6060","centerforclinicalresearchoperations@meei.harvard.edu",{"lat":91,"lon":92},42.35843,-71.05977,{"facility":94,"status":8,"city":95,"state":96,"zip":97,"country":83,"contacts":98,"geoPoint":103},"Hearts for Hearing-Oklahoma City","Oklahoma City","Oklahoma","73120",[99],{"name":100,"role":87,"phone":101,"email":102},"René Gifford, PhD","4055484300","research@heartsforhearing.org",{"lat":104,"lon":105},35.46756,-97.51643,{"facility":107,"status":8,"city":108,"state":109,"zip":110,"country":83,"contacts":111,"geoPoint":119},"Lehigh Valley Hospital-Cedar Crest","Allentown","Pennsylvania","181103",[112,116],{"name":113,"role":87,"phone":114,"email":115},"Ravi N. Samy, MD, FACS","610-402-9543","Ravi.Samy@Jefferson.edu",{"name":117,"role":87,"email":118},"Danielle Koehler, RN (Study Coordinator)","Danielle.Koehler@Jefferson.edu",{"lat":120,"lon":121},40.60843,-75.49018,[],[],[],[126],{"label":127,"url":128},"Related Info","https:\u002F\u002Fsonixstudy.com\u002F",{"nct_id":4,"conditions":130,"biomarkers":133},[131,19,132],"Deafness, Autosomal Recessive 1A","Sensorineural Hearing Loss",[134],"GJB2 Gene",{"nct_id":4,"found":61,"summary":17,"prompt_version":17}]