Prevention and Monitoring of Medication-Related Hearing Disability: A Cross-Sectional Analysis of Ototoxicity Trials Registered on ClinicalTrials.gov

Abstract

Medication-related hearing disability is a significant and potentially preventable consequence of various therapies. Although clinical trials are crucial for prevention, treatment, and screening strategies, it remains unclear how well they evaluate disability-related outcomes.
Objective: This study aimed to characterize ototoxicity studies on ClinicalTrials.gov and examine drug classes, clinical contexts, and outcome measures for medication-related hearing disability. Methods: A registry-based cross-sectional analysis used ClinicalTrials.gov (http://www. clinicaltrials. gov). The search and data export were performed on May 11, 2026, with the search term “ototoxicity.” Eligible records included interventional and observational studies on ototoxicity, hearing loss, tinnitus, vestibular issues, audiological, and rehabilitation outcomes related to hearing disabilities. After screening, 48 records were included. Variables extracted were study status, type, phase, age, region, drug/exposure, purpose, and hearing outcomes. Data were analyzed descriptively with frequencies and percentages.
Results: Of the 48 studies included in the analysis, 21 (43.8%) were completed, and 34 (70.8%) were interventional clinical trials. The most common clinical contexts were platinum-based chemotherapy and mixed cancer treatment exposures, accounting for 33 (68.8%) studies, followed by aminoglycoside and anti-infective therapy, with 10 (20.8%) studies. Most studies assessed the incidence, severity, or grade of hearing loss/ototoxicity (41, 85.4%), and objective audiometric or cochlear measures (33, 68.8%). Fewer studies included patient-reported hearing disabilities or hearing-related quality of life (22, 45.8%), tinnitus-related outcomes (18, 37.5%), vestibular or balance-related outcomes (12, 25.0%), rehabilitation or assistive-device outcomes (10, 20.8%), and speech perception outcomes (9, 18.8%). Trial purpose: 22 (45.8%) studies focused on prevention, eight (16.7%) focused on treatment/rehabilitation, and 18 (37.5%) focused on monitoring/screening.
Conclusion: Ototoxicity trials mainly focus on clinical grading and audiometric changes, with less consistent assessment of functional and patient-centered disability outcomes. Future studies should include hearing, communication, tinnitus, vestibular, quality-of-life, and rehabilitation outcomes to better reflect the real-world impact of medication-related hearing disability.