Effects of Bisphosphonates on OI-Related Hearing Loss

Phase 4

Recruiting

• Conditions: Osteogenesis Imperfecta
• Interventions: Drug: Risedronate Oral Tablet

• Registration Number: NCT04152551
• Lead Sponsor: Hospital for Special Surgery, New York

Brief Summary

Osteogenesis Imperfecta-related hearing loss usually occurs in individuals with mild (type I) OI and is much earlier in onset than age-related hearing loss, with the majority of individuals experiencing some minor hearing loss in their 20s. Bisphosphonates have been successfully used to treat otosclerosis, a common cause of hearing loss similar to OI-related hearing loss. As many individuals with OI-related hearing loss also present with otosclerosis and because of their mechanistic similarities, the investigators propose studying the effects of bisphosphonate treatment on individuals diagnosed with both OI type I and hearing loss, thereby determining its effectiveness as a potential treatment for hearing loss.

The investigators will enroll 50 individuals diagnosed with type I OI and age 18-100. 25 adults will be enrolled into the treatment arm and receive bisphosphonate treatment (must have at least mild hearing loss), while 25 adults will be enrolled into the control arm. The investigators will enroll 25 children (6-17 years of age) diagnosed with OI who are currently receiving bisphosphonate treatment as part of their care for orthopedic symptoms. The investigators will also observe 25 children (6-17 years of age) diagnosed with OI who are NOT currently receiving bisphosphonate treatment. The study duration is 63 months (approximately 5 years). Enrollment is anticipated to begin in November 2019.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-10-18
• Study First Submitted QC: 2019-11-01
• Study Start: 2019-11-02
• Study First Posted: 2019-11-05
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-23
• Last Update Posted: 2025-01-27
• Primary Completion: 2025-11
• Study Completion: 2025-11

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 100

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Adult Treatment Arm	Risedronate Oral Tablet	Intervention treatment arm. Adults (18+ years) with type 1 OI. Must have at least mild hearing loss. Will receive Risedronate (35mg, 0-2x/week as clinically indicated) for duration of study. Changes in hearing, quality of life, and bone density will be monitored.

Primary Outcome Measures

Name	Time	Method
Pure Tone Averages	Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months	Average hearing thresholds at 250, 500, 1000, 2000, 3000, 4000, 8000 Hertz

Secondary Outcome Measures

Name	Time	Method
Word Recognition Scores	Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months	Percent of words participants correctly repeat in word recognition test (%)
DEXA Z-score	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Higher score is better. Relative Bone Density
Hearing Handicap Inventory Raw Score	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Score 0-40. Lower score is better. Adults (self-reported)
Tinnitus Handicap Inventory Score	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Score 0-100. Lower score is better. Adults (self-reported).
Dizziness Handicap Inventory Score	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Score 0-100. Lower score is better. Adults (self-reported). Incidence and impact of vertigo in study population.
SF-36 Scale and Summary Scores	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Score 0-100. Higher score is better. Adults (self-reported) quality-of-life survey.
Pediatric Outcomes Data Collection Instrument (PODCI) Score	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Score 0-100. Lower score is better. Children (ages 6-10 years), parent-reported. Assessment of overall health and functioning.
DEXA Bone Mineral Density	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Higher score is better. Bone Mineral Density (grams/centimeter\^2)
Speech Recognition Scores	Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months	Lowest volume participant can hear and understand speech (decibels)
Adolescent Outcomes Questionnaire Score	Yearly (Baseline, 12, 24, 36, 48, 60 months)	Score 0-100. Lower score is better. Children (ages 11-17 years), parent- or self-reported. Assessment of overall health and functioning.

Trial Locations

Locations (1)

Hospital for Special Surgery; 🇺🇸 New York, New York, United States