[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"otof-gene-mutation\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:otof-gene-mutation":30},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,2,0,[8,45],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":4,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":4,"briefSummary":23,"conditions":24,"keywords":4,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":33,"lastUpdatePostDateStruct":34,"startDateStruct":37,"completionDateStruct":39,"leadSponsor":41,"locationsCount":44},"100470205","natural-history-in-children-up-to-16-years-with-mild-to-profound-hearing-loss-due-to-mutations-in-gjb2--otof-genes-100470205",false,"NCT05402813","Natural History in Children up to 16 Years With Mild to Profound Hearing Loss Due to Mutations in GJB2 \u002F OTOF Genes","Longitudinal Study of the Natural History of Two Autosomal Recessive Non Syndromic Deafness (DFNB1A and DFNB9) in Children up to 16 Years of Age","Otoconex","Main Inclusion Criteria:\n\nParticipants meeting all the following main inclusion criteria will be eligible to participate in the study:\n\n* Aged ≤ 16 years on the date of signed informed consent for cohort 1 and ≤ 10 years for cohort 2;\n* With a diagnosis of non-syndromic, bilateral, mild to profound, sensorineural hearing loss (according to the American Speech Language-Hearing Association);\n* With documented genotyping results showing mutation(s) in GJB2 or OTOF genes;\n* Written informed consent as required by local regulations.\n* Either without Cochlear Implant, or with unilateral or bilateral Cochlear Implant(s)\n\nExclusion Criteria:\n\nParticipants presenting with any of the following main exclusion criteria will not be included in the study\n\n* Other type of deafness, such as unilateral deafness, persistent conductive deafness, malformation syndrome, syndromic deafness, known familial deafness linked to mutations in other genes than OTOF or GJB2;\n* Documented genotyping results showing pathogenic mutation(s) in other gene(s) than GJB2 or OTOF genes in the tested panel;\n* Unable and\u002For unwilling to comply with all the protocol requirements and\u002For study procedures.","ALL","16 Years",{"count":20,"type":21},180,"ESTIMATED","OBSERVATIONAL","The purpose of this study is to follow the natural history of non-syndromic hearing loss caused by mutations in two genes (GJB2 or OTOF) in children up to 16 years of age.",[25,26,27,28,29,30,31],"Sensorineural Hearing Loss, Bilateral","AUNB1","DFNB1A","Congenital Deafness","DFNB9","OTOF Gene Mutation","GJB2 Gene Mutation","RECRUITING","2026-05-28",{"date":35,"type":36},"2026-06-01","ACTUAL",{"date":38,"type":36},"2022-11-18",{"date":40,"type":21},"2031-06",{"name":42,"class":43},"Sensorion","INDUSTRY",1,{"id":46,"slug":47,"hasResults":11,"nctId":48,"briefTitle":49,"officialTitle":50,"acronym":51,"eligibilityCriteria":52,"healthyVolunteers":11,"sex":17,"minAge":53,"maxAge":54,"enrollmentInfo":55,"targetDuration":4,"studyType":57,"phases":58,"briefSummary":61,"conditions":62,"keywords":68,"overallStatus":32,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":5},"100544551","phase-1-a-phase-iii-clinical-trial-with-sens-501-in-children-suffering-from-severe-to-profound-hearing-loss-due-to-otoferlin-otof-mutations-100544551","NCT06370351","A Phase I\u002FII Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations","A Phase I\u002FII, Open-ended, Adaptative, Open Label Dose Escalation and Expansion Clinical Trial to Evaluate the Efficacy and Safety of Unilateral Intracochlear Injection of SENS-501 Using an Injection System in Children with Severe to Profound Hearing Loss Due to Otoferlin Gene Mutations","AUDIOGENE","Inclusion Criteria:\n\n* Children (male or female) ≥ 6 to ≤ 31 months at the time of inclusion\n* Severe to profound hearing loss assessed by auditory brainstem response (ABR)\n* Biallelic mutation in the Otoferlin gene\n* Presence of Otoacoustic emissions (OAEs)\n* Documented normal cochlea and internal auditory canals\n* Patients with intact vestibular function\n\nExclusion Criteria:\n\n* History of chronic, acute, or major disease, or unspecified reasons, that in the opinion of the Investigator, makes the participant unsuitable for participation in the study or constitutes an unacceptable risk.\n* Have been dosed in a previous gene therapy clinical trial\n* Patients with a prior or current cochlear implant\n* Any contraindication to the surgery determined by the surgeon or anesthesia determined by the anesthesiologist, or designee, or history of therapy known as ototoxic (e.g., cisplatin, high dose and long treatment with aminoglycosides, etc.) for an extended period (more than 2 weeks).\n* Participation in any other interventional clinical trial\n* Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the participant or would preclude the participant from successful completion of the study or might interfere with the evaluation of study treatment\n* Anticipated noncompliance with the protocol requirements","6 Months","31 Months",{"count":56,"type":21},12,"INTERVENTIONAL",[59,60],"PHASE1","PHASE2","This study intends to assess safety, tolerability, and efficacy of SENS-501 in children between the ages of 6-31 months with pre-lingual hearing loss due to a mutation in the Otoferlin gene.",[30,29,28,63,64,65,66,67],"Hearing Disorders","Ear Diseases","Otorhinolaryngologic Diseases","Deafness","Hearing Loss, Sensorineural",[30,69,66,70,51,71,29,72,73,74,75,76],"Hearing impairment","otoferlin","Hearing Loss","Hearing disorder","Cochlear implant","SENS-501","Congenital Hearing loss","Gene therapy","2024-09-23",{"date":79,"type":36},"2024-09-26",{"date":81,"type":36},"2024-06-21",{"date":83,"type":21},"2031-07",{"name":42,"class":43}]