Ignite Creation Date:
2025-12-24 @ 9:28 PM
Ignite Modification Date:
2025-12-29 @ 1:07 PM
Study NCT ID:
NCT06025032
Status:
WITHDRAWN
Last Update Posted:
2025-04-10
First Post:
2023-08-21
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
a Study in Subjects With Otoferlin Mutation-related Hearing Loss Using RNA Base-eDiting Therapy(SOUND)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 0}}, 'statusModule': {'whyStopped': 'Huidagene stopped this program as lack of patients in China', 'overallStatus': 'WITHDRAWN', 'startDateStruct': {'date': '2023-03-30', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2025-04-08', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2025-04-08', 'studyFirstSubmitDate': '2023-08-21', 'studyFirstSubmitQcDate': '2023-08-29', 'lastUpdatePostDateStruct': {'date': '2025-04-10', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-09-06', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-04-08', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Incidence of otological and systemic adverse events', 'timeFrame': '26 weeks', 'description': 'Number of AE(Adverse events),SAE(Serious Adverse Events),DLT(Dose Limiting Toxicities)'}], 'secondaryOutcomes': [{'measure': 'Change from baseline in ABR(Auditory Brainstem Response) intensity threshold (decibels normal hearing level [dB nHL])', 'timeFrame': '26 weeks', 'description': 'ABR intensity threshold (decibels normal hearing level \\[dB nHL\\]) measurement'}, {'measure': 'Change from baseline in hearing performance by behavioral audiometry with pure-tone audiometry', 'timeFrame': '26 weeks', 'description': 'Behavioral audiometry and pure-tone audiometry measurement'}]}, 'oversightModule': {'isUsExport': False, 'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Congenital hearing loss', 'OTOF', 'Q829X', 'HG205', 'CRISPR'], 'conditions': ['Congenital Hearing Loss']}, 'descriptionModule': {'briefSummary': 'The purpose of the study is to determine whether HG205 as CRISPR/Cas13 RNA base-editing therapy is safe and effective for the treatment of hearing loss caused by p.Q829X mutation in OTOF gene.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '16 Years', 'minimumAge': '1 Year', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Male or females between 1 and 16 years of age at the time the subject/parent/legal guardian signs the informed consent form.\n* Willing to adhere to the protocol as evidenced by written informed consent or parental permission and subject assent.\n* Molecular diagnosis of biallelic mutations in the OTOF gene with at least one mutation being p.Q829X through collected blood samples at screening;\n* Based on auditory brainstem response (ABR), clinically diagnosed sensorineural hearing loss (SNHL) with the following hearing thresholds: severe (65 dB ≤ hearing threshold \\< 80 dB) or profound (80 dB ≤ hearing threshold \\< 95 dB) or complete (hearing threshold ≥ 95 dB) hearing loss in both ears.\n* Acceptable hematology, clinical chemistry, and urine laboratory parameters.\n\nExclusion Criteria:\n\n* Pre-existing other hearing-loss conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints or complications of surgery.\n* Presence of cochlear implants in the study ear.\n* Complicating systemic diseases or clinically significant abnormal baseline laboratory values.\n* Complicating systemic diseases would include those in which the disease itself, or the treatment for the disease, can alter hearing function.\n* Prior participation in clinical study with an investigational drug within the past six months.\n* Prior gene therapy treatments.\n* Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.'}, 'identificationModule': {'nctId': 'NCT06025032', 'briefTitle': 'a Study in Subjects With Otoferlin Mutation-related Hearing Loss Using RNA Base-eDiting Therapy(SOUND)', 'organization': {'class': 'INDUSTRY', 'fullName': 'HuidaGene Therapeutics Co., Ltd.'}, 'officialTitle': 'An Open-label, Multiple-cohort, Dose-finding, Investigator-initiated Trial to Evaluate the Safety, Tolerability, and Efficacy of HG205 RNA Base-Editing Therapy in Subjects With OTOF-p.Q829X Mutation-associated Hearing Loss', 'orgStudyIdInfo': {'id': 'HG20501'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HG205', 'description': 'Method of Administration: Once Unilateral intracochlear injection/subject; The duration of the study for each subject includes a screening period, enrollment visit, treatment visit, a 26-week follow-up period, and a 5-year long-term safety follow-up after the injection', 'interventionNames': ['Genetic: HG205']}], 'interventions': [{'name': 'HG205', 'type': 'GENETIC', 'description': 'The study will enroll up to 2 cohorts, evaluating a starting dose plus a higher or lower dose', 'armGroupLabels': ['HG205']}]}, 'contactsLocationsModule': {'locations': [{'city': 'Shanghai', 'country': 'China', 'facility': 'Eye & ENT Hospital of Fudan University', 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}], 'overallOfficials': [{'name': 'Study Director', 'role': 'STUDY_DIRECTOR', 'affiliation': 'HuidaGene Therapeutics Co., Ltd.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'HuidaGene Therapeutics Co., Ltd.', 'class': 'INDUSTRY'}, 'collaborators': [{'name': 'Eye & ENT Hospital of Fudan University', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}