Clinical trial • Not applicable • Rare Disease

AAVANC80-5'HOTOF, AAVANC80-3'HOTOF for Sensorineural hearing loss due to OTOF (otoferlin) mutations

Not applicable trial of AAVANC80-5'HOTOF, AAVANC80-3'HOTOF for Sensorineural hearing loss due to OTOF (otoferlin) mutations.

Overview

open-label, none/not specified-controlled Not applicable trial across 1 site in Spain.

Recruits 12 paediatric patients.

Vulnerable Population: Vulnerable population is selected. Consent/assent handled via informed consent from legally authorised representative and/or participant; permission and participant assent process where appropriate. Specific assent form for 12-17 years and parent/legal guardian ICF documents are listed (age-specific consent/assent materials available).

{"criterion_text":"- Individuals with OTOF-mediated hearing loss who received an intracochlear administration of AAVAnc80- hOTOF in the AK-OTOF-101 clinical trial (either Part A or Part B).\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)."}

{"criterion_text":"- 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. Potential participants will not be excluded based on their sex, gender, race, or ethnicity."}

{"endpoint_text":"- Safety: Incidence and frequency of AEs","definition_or_measurement_approach":""}
{"endpoint_text":"- Safety: Concomitant Medications","definition_or_measurement_approach":""}
{"endpoint_text":"- Safety: Clinical labs: chemistries, hematology (CBC with differential), urinalysis","definition_or_measurement_approach":"Clinical laboratory assessments including chemistries, hematology (CBC with differential), and urinalysis"}
{"endpoint_text":"- Safety: Physical examination and vital signs","definition_or_measurement_approach":""}
{"endpoint_text":"- Safety: Otoscopic examination","definition_or_measurement_approach":""}
{"endpoint_text":"- Safety: Vestibular screening","definition_or_measurement_approach":""}
{"endpoint_text":"- Safety: Tympanometry","definition_or_measurement_approach":""}

{"endpoint_text":"- Efficacy: Diagnostic ABR testing threshold","definition_or_measurement_approach":""}
{"endpoint_text":"- Efficacy: Age-appropriate behavioral audiometry","definition_or_measurement_approach":""}
{"endpoint_text":"- Efficacy: Standard word or speech recognition testing","definition_or_measurement_approach":""}
{"endpoint_text":"- Efficacy: Standard auditory questionnaires","definition_or_measurement_approach":""}

Planned Sample Size: 12
Recruitment Window Months: 102
Consent Approach: Informed consent to be obtained via an informed consent process from the participant and/or legally authorised representative. Permission and participant assent process used where appropriate (assent form specified for 12-17 years). Age-specific documents include parent ICF and assent for 12-17; subject information and informed consent forms available in multiple languages (English and Spanish translations are present in the submitted documents).

Name: Medpace Inc.
Responsibilities: Duties/roles codes: 1,10,12,13,15 (includes 'Laboratory sample management'),2,5,6,7,8,9; contact s.dupre@medpace.com

{"country":"United States","full_name":"Medpace Inc.","duties_or_roles":"Duties/roles codes: 1,10,12,13,15 (includes 'Laboratory sample management'),2,5,6,7,8,9","organisation_type":"Pharmaceutical company"}
{"country":"Ireland","full_name":"Boyd Consultants Limited","duties_or_roles":"Duties/roles codes: 12","organisation_type":"Pharmaceutical company"}

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