RecruitingPhase 4

Effects of Bisphosphonates on OI-Related Hearing Loss

United States100 participantsStarted 2019-11-02

Plain-language 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.

Who can participate

Age range

6 Years – 100 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria (Adult Treatment Arm): * Diagnosis of OI type I * Diagnosis of at least mild hearing loss (\>20dB pure tone average) by audiogram testing * 18+ * Vitamin D level \> 30 Inclusion Criteria (Adult Control Arm): * Diagnosis of OI type I Inclusion Criteria (Child Observational Bisphosphonate Arm) * Diagnosis of OI * Age 6-17 years * Currently receiving bisphosphonate treatment as standard of care Inclusion Criteria (Child Observational No Treatment Arm) * Diagnosis of OI * Age 6-17 years * NOT receiving bisphosphonate treatment and will not receive bisphosphonate treatment for the duration of the study Exclusion Criteria (ALL ARMS): * Family history of hearing-loss (not related to OI or occupational hearing loss) * Pregnancy

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Pure Tone Averages

Timeframe: Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months

Trial details

NCT IDNCT04152551

SponsorHospital for Special Surgery, New York

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2027-01

Contact for this trial

Su Htwe

(212)774-2355 htwes@hss.edu

View on ClinicalTrials.gov More Osteogenesis Imperfecta trials