Study of OTO-104 in Subjects at Risk From Cisplatin-Induced Hearing Loss

Study Overview
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Brief Summary
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This is a multicenter, Phase 2 study to assess the feasibility, safety and efficacy of OTO-104 given by intratympanic administration in subjects at risk for ototoxicity from cisplatin chemotherapy regimens in the treatment of cancer.

Official Title
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A Multicenter, Randomized, Phase 2 Study to Assess the Feasibility, Safety and Efficacy of OTO-104 Given by Intratympanic Administration in Subjects at Risk for Ototoxicity From Cisplatin Chemotherapy Regimens in the Treatment of Cancer

Conditions
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Cisplatin Induced Hearing Loss

Intervention / Treatment
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* Drug: OTO-104


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subject is aged 6 months to 21 years inclusive. Subject is diagnosed with neuroblastoma, hepatoblastoma, osteosarcoma or extracranial germ cell tumors and has not been previously treated with cisplatin or carboplatin. Subject is scheduled to receive a chemotherapy regimen that includes a cumulative cisplatin dose of ≥ 200 mg/m2. Subject has normal baseline auditory function, defined as ≤ 20 dB from 2000 to 8000 Hz, in both ears and does not have a history of sensorineural hearing loss. Exclusion Criteria: Subject has middle ear effusion upon clinical examination. Subject has a history of central nervous system radiotherapy that encompasses all or part of the cochlea or will receive such radiation therapy during the course of the study. Subject is receiving sodium-thiosulfate or amifostine therapy with chemotherapy. Subject is currently participating on a separate otoprotection clinical study.

Ages Eligible for Study
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Minimum Age: 6 Months
Maximum Age: 21 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: OTO-104<br>One of the subject's ears will receive up to three administrations of study drug prior to cisplatin-based therapy | Drug: OTO-104<br>* 12 mg dexamethasone administered intratympanically<br>|
| No Intervention: Control<br>The ear not receiving OTO-104 will receive no treatment | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Feasibility assessed via a questionnaire | Feasibility to assess incorporating OTO-104 with a cisplatin-based therapy regimen | Up to 18 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hearing function in each ear according to SIOP-Boston Ototoxicity Scale |  | Up to 6 months | 
| Safety as assessed by adverse events |  | Up to 6 months | 
| Local tolerability as assessed by otoscopic examinations |  | Up to 6 months |