Ignite Creation Date:
2025-12-24 @ 2:43 PM
Ignite Modification Date:
2025-12-26 @ 12:29 AM
Study NCT ID:
NCT05821959
Status:
RECRUITING
Last Update Posted:
2025-12-18
First Post:
2023-02-08
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006319', 'term': 'Hearing Loss, Sensorineural'}, {'id': 'C563396', 'term': 'Deafness, Autosomal Recessive 9'}, {'id': 'C538268', 'term': 'Auditory neuropathy'}, {'id': 'D003638', 'term': 'Deafness'}, {'id': 'D034381', 'term': 'Hearing Loss'}, {'id': 'D006312', 'term': 'Hearing Loss, Bilateral'}], 'ancestors': [{'id': 'D006311', 'term': 'Hearing Disorders'}, {'id': 'D004427', 'term': 'Ear Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D012678', 'term': 'Sensation Disorders'}, {'id': 'D009461', 'term': 'Neurologic Manifestations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D012816', 'term': 'Signs and Symptoms'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NON_RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 18}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-09-15', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-12', 'completionDateStruct': {'date': '2028-10', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-16', 'studyFirstSubmitDate': '2023-02-08', 'studyFirstSubmitQcDate': '2023-04-18', 'lastUpdatePostDateStruct': {'date': '2025-12-18', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2023-04-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2028-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Frequency of Adverse Events (AEs)', 'timeFrame': 'through trial completion, approximately 1 year', 'description': 'AEs with relationship to the investigational medicinal product and/or to the administration procedure (including the delivery device)'}], 'secondaryOutcomes': [{'measure': 'Auditory Brainstem Response (ABR) Threshold', 'timeFrame': 'through trial completion, approximately 1 year', 'description': 'Changes from baseline in ABR intensity threshold (decibels normal hearing level \\[dB nHL\\])'}, {'measure': 'Performance of the Akouos Delivery Device', 'timeFrame': 'through trial completion, approximately 1 year', 'description': 'Performance based on the frequency of device deficiencies observed during delivery of AAVAnc80-hOTOF to the intracochlear space'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isUnapprovedDevice': True, 'isFdaRegulatedDevice': True}, 'conditionsModule': {'keywords': ['Otoferlin', 'Auditory neuropathy (AN)', 'Deafness, autosomal recessive 9 (DFNB9)', 'Hearing loss', 'Hearing loss, bilateral', 'Hearing loss, sensorineural'], 'conditions': ['Sensorineural Hearing Loss, Bilateral']}, 'descriptionModule': {'briefSummary': 'Part A of this trial will evaluate the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF and will evaluate the Akouos Delivery Device to safely achieve the intended product performance.', 'detailedDescription': 'The following two dose levels will be tested:\n\n* Up to 4.1E11 total vg/cochlea\n* Up to 8.1E11 total vg/cochlea'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': 'Criteria for Inclusion:\n\n1. Participants may be of any age, based on Cohort Criteria\n2. At least two mutations in the otoferlin gene\n3. Clinical presentation of Profound bilateral sensorineural hearing loss as assessed by ABR\n4. Preserved distortion product otoacoustic emissions (DPOAEs)\n5. Able and willing to comply with all study requirements, including willingness to participate in a separate long term follow-up study after completion of this trial\n\nCriteria for Exclusion:\n\n1. Persistent ear infections, anatomic or other abnormalities of the ear, and/or medical conditions that would contraindicate undergoing surgery, anesthesia, and/or administration of investigational gene therapy\n2. Cochlear Implant(s) in the ear(s) to receive AAVAnc80-hOTOF\n3. Prior participation in a clinical trial with an investigational drug, within six months prior to administration, or any prior participation in a gene therapy clinical trial'}, 'identificationModule': {'nctId': 'NCT05821959', 'briefTitle': 'Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss', 'organization': {'class': 'INDUSTRY', 'fullName': 'Akouos, Inc.'}, 'officialTitle': 'A Trial of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations', 'orgStudyIdInfo': {'id': 'AK-OTOF-101'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Cohort 1a and Cohort 1b', 'description': 'Cohort 1a: Two participants aged 7 to 17 years old (inclusive) to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device\n\nCohort 1b: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 1) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age', 'interventionNames': ['Combination Product: AAVAnc80-hOTOF via Akouos Delivery Device']}, {'type': 'EXPERIMENTAL', 'label': 'Cohort 2', 'description': 'Cohort 2: Up to six eligible participants will be enrolled to receive intracochlear administration of AAVAnc80-hOTOF (dose level 2) in the study ear using a sterile, one-time use investigational medical device; the first three participants will be at least two years of age at the time of AAVAnc80-hOTOF administration and the next three participants may be of any age', 'interventionNames': ['Combination Product: AAVAnc80-hOTOF via Akouos Delivery Device']}, {'type': 'EXPERIMENTAL', 'label': 'Bilateral Expansion', 'description': 'Bilateral Expansion: up to four eligible participants of any age, and no cochlear implants, may be eligible for enrollment into the Bilateral Expansion Cohort (simultaneous bilateral administration of AAVAnc80-hOTOF).', 'interventionNames': ['Combination Product: AAVAnc80-hOTOF via Akouos Delivery Device']}], 'interventions': [{'name': 'AAVAnc80-hOTOF via Akouos Delivery Device', 'type': 'COMBINATION_PRODUCT', 'description': 'AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time unilateral intracochlear administration using the Akouos Delivery Device.', 'armGroupLabels': ['Cohort 1a and Cohort 1b', 'Cohort 2']}, {'name': 'AAVAnc80-hOTOF via Akouos Delivery Device', 'type': 'COMBINATION_PRODUCT', 'description': 'AAVAnc80-hOTOF is a sterile suspension intended to be administered via a one-time bilateral intracochlear administration using the Akouos Delivery Device.', 'armGroupLabels': ['Bilateral Expansion']}]}, 'contactsLocationsModule': {'locations': [{'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Kesten Anderson', 'role': 'CONTACT', 'email': 'Kesten-Anderson@uiowa.edu', 'phone': '+1 319-356-8320'}], 'facility': 'University of Iowa', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '02115', 'city': 'Boston', 'state': 'Massachusetts', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Meghan Fitzgerald', 'role': 'CONTACT', 'email': 'OTOFtrial@childrens.harvard.edu', 'phone': '+1 617-355-4729'}], 'facility': "Boston Children's Hospital", 'geoPoint': {'lat': 42.35843, 'lon': -71.05977}}, {'zip': '45229', 'city': 'Cincinnati', 'state': 'Ohio', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Alexandra Sesito', 'role': 'CONTACT', 'email': 'alexandra.sesito@cchmc.org', 'phone': '+1 513 636-4355'}], 'facility': "Cincinnati Children's Hospital Medical Center", 'geoPoint': {'lat': 39.12711, 'lon': -84.51439}}, {'zip': '19146', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Aminat Adegbuji', 'role': 'CONTACT', 'email': 'adegbujia@chop.edu', 'phone': '+1 267-426-8380'}], 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Taha Jan, MD', 'role': 'CONTACT', 'email': 'taha.a.jan@vumc.org', 'phone': '+1 615-421-1228'}], 'facility': 'Vanderbilt Bill Wilkerson Center', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': 'M5G 1X8', 'city': 'Toronto', 'state': 'Ontario', 'status': 'RECRUITING', 'country': 'Canada', 'contacts': [{'name': 'Jaina Negandhi', 'role': 'CONTACT', 'email': 'Jaina.negandhi@sickkids.ca', 'phone': '+1 416 813 6683'}], 'facility': 'The Hospital for Sick Children Jaina.negandhi@sickkids.ca +1 (416) 813 6683', 'geoPoint': {'lat': 43.70643, 'lon': -79.39864}}, {'zip': '100', 'city': 'Taipei', 'status': 'RECRUITING', 'country': 'Taiwan', 'contacts': [{'name': 'Wan-Cian Liao', 'role': 'CONTACT', 'email': 'mulong0328@gmail.com', 'phone': '+886- 953-728-657'}], 'facility': 'National Taiwan University Hospital', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}, {'zip': 'NW1 2PG', 'city': 'London', 'state': 'United Kingdom', 'status': 'RECRUITING', 'country': 'United Kingdom', 'contacts': [{'role': 'CONTACT', 'email': 'UCLH.OTOF.Study@nhs.net'}], 'facility': 'University College London', 'geoPoint': {'lat': 51.50853, 'lon': -0.12574}}], 'centralContacts': [{'name': 'Akouos Clinical Trials', 'role': 'CONTACT', 'email': 'AkouosClinicalTrials@Lilly.com', 'phone': '+1 857 410-1816'}], 'overallOfficials': [{'name': 'Kathy Reape, MD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Akouos, Inc.'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Akouos, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Eli Lilly and Company', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}