Ignite Creation Date:
2025-12-24 @ 11:52 PM
Ignite Modification Date:
2025-12-31 @ 8:58 PM
Study NCT ID:
NCT06370351
Status:
RECRUITING
Last Update Posted:
2024-09-26
First Post:
2024-04-09
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D006311', 'term': 'Hearing Disorders'}, {'id': 'D004427', 'term': 'Ear Diseases'}, {'id': 'D010038', 'term': 'Otorhinolaryngologic Diseases'}, {'id': 'D003638', 'term': 'Deafness'}, {'id': 'D006319', 'term': 'Hearing Loss, Sensorineural'}, {'id': 'D034381', 'term': 'Hearing Loss'}, {'id': 'C563396', 'term': 'Deafness, Autosomal Recessive 9'}], 'ancestors': [{'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': 12}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2024-06-21', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-09', 'completionDateStruct': {'date': '2031-07', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-09-23', 'studyFirstSubmitDate': '2024-04-09', 'studyFirstSubmitQcDate': '2024-04-12', 'lastUpdatePostDateStruct': {'date': '2024-09-26', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2024-04-17', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-07', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and tolerability of SENS-501', 'timeFrame': '5 years', 'description': 'Number of study-related adverse events (AEs) and serious adverse events (SAEs).'}, {'measure': 'Efficacy of SENS-501 assessed by ABR', 'timeFrame': '5 years', 'description': 'Improvement of hearing threshold measured by auditory brainstem response (ABR)'}], 'secondaryOutcomes': [{'measure': 'Safety and tolerability of SENS-501', 'timeFrame': '5 years', 'description': 'Number of study-related adverse events (AEs) and serious adverse events (SAEs).'}, {'measure': 'Efficacy of SENS-501 assessed by ABR', 'timeFrame': '5 years', 'description': 'Improvement of hearing threshold measured by auditory brainstem response (ABR)'}, {'measure': 'Efficacy of SENS-501 assessed by PTA', 'timeFrame': '5 years', 'description': 'Improvement of hearing threshold measured by Pure-tone audiometry (PTA)'}, {'measure': 'Clinical performance of the administration system', 'timeFrame': '1 day', 'description': "Administration success ; Users' feedback questionnaire on the administration and use of the devices"}, {'measure': 'Safety of the administration system', 'timeFrame': '5 years', 'description': 'Adverse device effects and Device deficiencies, Procedure complications'}, {'measure': 'Usability of the administration system', 'timeFrame': '1 day', 'description': 'Usability questionnaire'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['OTOF Gene Mutation', 'Hearing impairment', 'Deafness', 'otoferlin', 'AUDIOGENE', 'Hearing Loss', 'DFNB9', 'Hearing disorder', 'Cochlear implant', 'SENS-501', 'Congenital Hearing loss', 'Gene therapy'], 'conditions': ['OTOF Gene Mutation', 'DFNB9', 'Congenital Deafness', 'Hearing Disorders', 'Ear Diseases', 'Otorhinolaryngologic Diseases', 'Deafness', 'Hearing Loss, Sensorineural']}, 'descriptionModule': {'briefSummary': '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.', 'detailedDescription': 'It is a multicenter, adaptive open-label, non-randomized, dose-escalation, and expansion study to assess safety, tolerability, and efficacy following intracochlear administration of SENS-501.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '31 Months', 'minimumAge': '6 Months', 'healthyVolunteers': False, 'eligibilityCriteria': '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'}, 'identificationModule': {'nctId': 'NCT06370351', 'acronym': 'AUDIOGENE', 'briefTitle': 'A Phase I/II Clinical Trial with SENS-501 in Children Suffering from Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations', 'organization': {'class': 'INDUSTRY', 'fullName': 'Sensorion'}, 'officialTitle': 'A Phase I/II, 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', 'orgStudyIdInfo': {'id': 'SENS-501-101'}, 'secondaryIdInfos': [{'id': '2023-504466-28-00', 'type': 'CTIS'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Dose escalation - Low dose group', 'description': 'Intracochlear administration of a Low dose of SENS-501 in one ear, with a dedicated administration system', 'interventionNames': ['Combination Product: SENS-501 administration']}, {'type': 'EXPERIMENTAL', 'label': 'Dose escalation - High dose group', 'description': 'Intracochlear administration of a High dose of SENS-501 in one ear, with a dedicated administration system', 'interventionNames': ['Combination Product: SENS-501 administration']}, {'type': 'EXPERIMENTAL', 'label': 'Dose expansion group', 'description': 'Intracochlear administration of SENS-501 in one ear, with a dedicated administration system, at the dose recommended following the Dose escalation phase', 'interventionNames': ['Combination Product: SENS-501 administration']}], 'interventions': [{'name': 'SENS-501 administration', 'type': 'COMBINATION_PRODUCT', 'description': 'Administration of SENS-501 with a dedicated administration system', 'armGroupLabels': ['Dose escalation - High dose group', 'Dose escalation - Low dose group', 'Dose expansion group']}]}, 'contactsLocationsModule': {'locations': [{'zip': 'NSW 2145', 'city': 'Westmead', 'status': 'RECRUITING', 'country': 'Australia', 'contacts': [{'name': 'Clinical Research Coordinator/Nurse', 'role': 'CONTACT', 'email': 'SCHN-CHW-CRC@health.nsw.gov.au', 'phone': '+61 2 7825 3048'}, {'name': 'Catherine Birman, Pr', 'role': 'CONTACT'}], 'facility': 'Childrens Hospital Westmead', 'geoPoint': {'lat': -33.80383, 'lon': 150.98768}}, {'zip': '75015', 'city': 'Paris', 'status': 'RECRUITING', 'country': 'France', 'contacts': [{'name': 'Natalie LOUNDON, Pr', 'role': 'CONTACT', 'email': 'secretariat.loundon@nck.aphp.fr', 'phone': '+33(0)171396782'}, {'name': 'Natalie LOUNDON, Pr', 'role': 'CONTACT'}], 'facility': 'Hopital Necker Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'centralContacts': [{'name': 'Lionel HOVSEPIAN, MD', 'role': 'CONTACT', 'email': 'lionel.hovsepian@sensorion-pharma.com', 'phone': '+33 7 86 31 13 76'}, {'name': 'Géraldine HONNET, MD', 'role': 'CONTACT', 'email': 'geraldine.honnet@sensorion-pharma.com'}], 'overallOfficials': [{'name': 'Natalie LOUNDON, Pr', 'role': 'STUDY_CHAIR', 'affiliation': 'Hopital Necker Enfants Malades'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Sensorion', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}