Ignite Creation Date:
2025-12-24 @ 11:30 PM
Ignite Modification Date:
2026-01-01 @ 5:20 PM
Study NCT ID:
NCT06696456
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-05-31
First Post:
2024-11-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C563396', 'term': 'Deafness, Autosomal Recessive 9'}, {'id': 'D003638', 'term': 'Deafness'}, {'id': 'D034381', 'term': 'Hearing Loss'}, {'id': 'D006312', 'term': 'Hearing Loss, Bilateral'}, {'id': 'D006319', 'term': 'Hearing Loss, Sensorineural'}, {'id': 'C538268', 'term': 'Auditory neuropathy'}], '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': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'CASE_ONLY'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 30}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2024-11-11', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'completionDateStruct': {'date': '2033-04', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-05-27', 'studyFirstSubmitDate': '2024-11-18', 'studyFirstSubmitQcDate': '2024-11-19', 'lastUpdatePostDateStruct': {'date': '2025-05-31', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-11-20', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2033-04', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Long term safety of AAVAnc80-hOTOF, including the incidence and frequency of late-occurring adverse events (AEs)', 'timeFrame': 'Through study completion, approximately four years.', 'description': 'The following adverse events will be collected:\n\n1. The development of new or exacerbation of malignancies (oncologic), neurologic, rheumatologic or other autoimmune, hematologic disorders, or new infections (if the infection is potentially related to AAVAnc80-hOTOF\n2. AEs that are related to AAVAnc80-hOTOF and/or the administration procedure\n3. AEs of special interest\n4. All serious adverse events regardless of severity or of relationship to participation in the AK-OTOF-101 Clinical Trial'}], 'secondaryOutcomes': [{'measure': 'Auditory Brainstem Response (ABR) Threshold', 'timeFrame': 'Through study completion, approximately four years.', 'description': 'Changes in ABR intensity threshold (decibels normal hearing level \\[dB nHL\\])'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Otoferlin', 'Deafness, autosomal recessive 9 (DFNB9)', 'Hearing loss', 'Hearing loss, bilateral', 'Hearing loss, sensorineural', 'Auditory neuropathy (AN)'], 'conditions': ['Otoferlin Gene-mediated Hearing Loss']}, 'referencesModule': {'seeAlsoLinks': [{'url': 'https://clinicaltrials.gov/study/NCT05821959?cond=otoferlin&rank=3', 'label': 'AK-OTOF-101: Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss'}]}, 'descriptionModule': {'briefSummary': 'This multi-center, observational, post-intervention long term follow-up (LTFU) study will monitor for safety and efficacy of AAVAnc80-hOTOF in individuals with sensorineural hearing loss due to otoferlin gene mutations who have previously received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.', 'detailedDescription': 'After completing one year of initial safety and efficacy assessments following an intracochlear administration of AAVAnc80-hOTOF in AK-OTOF-101, participants will continue to be observed for an additional approximately four years of safety and efficacy follow-up in this LTFU study, for a total of approximately five years of safety and efficacy observations after vector administration. For each participant, evaluations in the LTFU study will occur at approximately one and a half years and two years after vector administration, followed by annual visits through Year 5; the duration in this LTFU study is approximately four years, with the aim of understanding and mitigating any risks related to delayed adverse events, as well as characterizing the durability of effect of the AAVAnc80-hOTOF.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial\n* Legally authorized representative and/or participant willingness to comply with all study requirements, as evidenced by successful completion of the informed consent process or permission and participant assent process (where appropriate)\n\nExclusion Criteria:\n\n* Any condition that would not allow the potential participant to complete follow-up examinations during the course of the study and/or, in the opinion of the Investigator, makes the potential participant unsuitable for the study'}, 'identificationModule': {'nctId': 'NCT06696456', 'briefTitle': 'Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy', 'organization': {'class': 'INDUSTRY', 'fullName': 'Akouos, Inc.'}, 'officialTitle': 'An Evaluation of the Long Term Safety and Efficacy of AAVAnc80-hOTOF Gene Therapy in Individuals With Sensorineural Hearing Loss Due to Otoferlin Gene Mutations', 'orgStudyIdInfo': {'id': 'AK-OTOF-LTFU'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'Study Participants', 'description': 'Individuals with otoferlin gene-mediated hearing loss who received intracochlear administration of AAVAnc80-hOTOF in the AK-OTOF-101 Clinical Trial'}]}, 'contactsLocationsModule': {'locations': [{'zip': '52242', 'city': 'Iowa City', 'state': 'Iowa', 'country': 'United States', 'facility': 'University of Iowa', 'geoPoint': {'lat': 41.66113, 'lon': -91.53017}}, {'zip': '19146', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'country': 'United States', 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}, {'zip': '08950', 'city': 'Esplugues de Llobregat', 'state': 'Barcelona', 'country': 'Spain', 'facility': 'Hospital Sant Joan de Déu', 'geoPoint': {'lat': 41.37732, 'lon': 2.08809}}, {'zip': '(R.O.C), 100', 'city': 'Taipei', 'state': 'Taiwan', 'country': 'Taiwan', 'facility': 'National Taiwan University Hospital', 'geoPoint': {'lat': 25.05306, 'lon': 121.52639}}], 'overallOfficials': [{'name': 'Kathy Reape, M.D.', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Akouos, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Akouos, Inc.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Eli Lilly and Company', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR'}}}}