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Osteogenesis ImperfectaPhase 4NCT04152551

Effects of Bisphosphonates on OI-Related Hearing Loss

A Phase 4 study testing Risedronate Oral Tablet in people with Osteogenesis Imperfecta, run by Hospital for Special Surgery, New York at 1 site in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 6 to 100 yearsHealthy volunteers can take part
Conditions the study is for
Osteogenesis Imperfecta
What is being tested?
Risedronate Oral Tablet
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
100 participants
Where
1 siteUnited States
Study dates
2 Nov 2019to Jan 2027
Phase
Phase 4
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 100 years
  • All sexes
  • Healthy volunteers accepted
  • Child, Adult, Older adult

Who may be able to join

  • Diagnosis of OI type I
  • Diagnosis of at least mild hearing loss (>20dB pure tone average) by audiogram testing
  • 18+
  • Vitamin D level > 30
  • Diagnosis of OI type I
  • Diagnosis of OI
Show all 11Show fewer
  • Age 6-17 years
  • Currently receiving bisphosphonate treatment as standard of care
  • Diagnosis of OI
  • Age 6-17 years
  • NOT receiving bisphosphonate treatment and will not receive bisphosphonate treatment for the duration of the study

Who cannot join

  • Family history of hearing-loss (not related to OI or occupational hearing loss)
  • Pregnancy

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Su Htwe contact

(212)774-2355

htwes@hss.edu

Erin Carter, MS, CGC contact

(212) 774-7332

United States1 site, 1 recruiting

  • Hospital for Special Surgery

    New York, New York, 10021

    Su Htwe, (212) 774-2355, htwes@hss.edu

    Recruiting

Other trials in United States

About this study

Official title: Effects of Bisphosphonates on OI-Related Hearing Loss: A Pilot Study

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.

Being tested
Risedronate Oral Tablet
Keywords
hearing loss, bisphosphonate, skeletal dysplasia, brittle bone disorder, osteogenesis imperfecta, OI
Medical subject headings
Osteogenesis Imperfecta, Hearing Loss, Mucopolysaccharidosis IV, Brittle Bone Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Risedronate Oral TabletDrug

    Also called actonel

    Oral bisphosphonate

Study groups in NCT04152551
Study groupRoleWhat they receive
Adult Treatment ArmExperimentalIntervention 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.
Receives: Drug: Risedronate Oral Tablet
Child (Bisphosphonate Arm)No interventionObservational (no investigational intervention) arm. Children (6-17 years) with any type of OI who are already receiving bisphosphonate treatment as standard of care treatment for orthopedic symptoms. Changes in hearing, quality of life, and bone density will be observed for the duration of the study.
Child (Control Arm)No interventionObservational arm. Children (6-17 years) with any type of OI who are not receiving bisphosphonate treatment. Changes in hearing, quality of life, and bone density will be observed for the duration of the study.
Adult Control ArmNo interventionObservational arm. Adults (18+ years) with type 1 OI. Changes in hearing, quality of life, and bone density will be monitored.

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Pure Tone Averages

    Average hearing thresholds at 250, 500, 1000, 2000, 3000, 4000, 8000 Hertz

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

Secondary outcomes (10)

  1. Speech Recognition Scores

    Lowest volume participant can hear and understand speech (decibels)

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

  2. Word Recognition Scores

    Percent of words participants correctly repeat in word recognition test (%)

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

  3. Hearing Handicap Inventory Raw Score

    Score 0-40. Lower score is better. Adults (self-reported)

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

  4. Tinnitus Handicap Inventory Score

    Score 0-100. Lower score is better. Adults (self-reported).

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

  5. Dizziness Handicap Inventory Score

    Score 0-100. Lower score is better. Adults (self-reported). Incidence and impact of vertigo in study population.

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

  6. SF-36 Scale and Summary Scores

    Score 0-100. Higher score is better. Adults (self-reported) quality-of-life survey.

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

  7. Pediatric Outcomes Data Collection Instrument (PODCI) Score

    Score 0-100. Lower score is better. Children (ages 6-10 years), parent-reported. Assessment of overall health and functioning.

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

  8. Adolescent Outcomes Questionnaire Score

    Score 0-100. Lower score is better. Children (ages 11-17 years), parent- or self-reported. Assessment of overall health and functioning.

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

  9. DEXA Z-score

    Higher score is better. Relative Bone Density

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

  10. DEXA Bone Mineral Density

    Higher score is better. Bone Mineral Density (grams/centimeter^2)

    Measured: Yearly (Baseline, 12, 24, 36, 48, 60 months)

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted5 Nov 2019
  2. Study start2 Nov 2019
  3. Primary completionJan 2027 (estimated)
  4. Study completionJan 2027 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Prevention
Allocation
Non-randomised
Intervention model
Single group
Model description
25 adults with type 1 OI and diagnosed hearing loss will be treated with oral risedronate. 25 adults with type 1 OI will be enrolled into the control arm.
Masking
None (open label)
Enrolment
100 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04152551
Sponsor's protocol ID
#2018-0700
Status last verified
Jan 2026
First submitted
18 Oct 2019
Last updated
28 Apr 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Publications

  • Van Dijk FS, Sillence DO. Osteogenesis imperfecta: clinical diagnosis, nomenclature and severity assessment. Am J Med Genet A. 2014 Jun;164A(6):1470-81. doi: 10.1002/ajmg.a.36545. Epub 2014 Apr 8.

    PubMed 24715559

  • Pillion JP, Vernick D, Shapiro J. Hearing loss in osteogenesis imperfecta: characteristics and treatment considerations. Genet Res Int. 2011;2011:983942. doi: 10.4061/2011/983942. Epub 2011 Dec 14.

    PubMed 22567374

  • Sillence D. Osteogenesis imperfecta: an expanding panorama of variants. Clin Orthop Relat Res. 1981 Sep;(159):11-25.

    PubMed 7285446

  • Swinnen FK, De Leenheer EM, Coucke PJ, Cremers CW, Dhooge IJ. Audiometric, surgical, and genetic findings in 15 ears of patients with osteogenesis imperfecta. Laryngoscope. 2009 Jun;119(6):1171-9. doi: 10.1002/lary.20155.

    PubMed 19358256

  • Berger G, Hawke M, Johnson A, Proops D. Histopathology of the temporal bone in osteogenesis imperfecta congenita: a report of 5 cases. Laryngoscope. 1985 Feb;95(2):193-9. doi: 10.1288/00005537-198502000-00014.

    PubMed 3918222

  • Vincent R, Wegner I, Stegeman I, Grolman W. Stapedotomy in osteogenesis imperfecta: a prospective study of 32 consecutive cases. Otol Neurotol. 2014 Dec;35(10):1785-9. doi: 10.1097/MAO.0000000000000372.

    PubMed 24751750

  • Plotkin LI, Weinstein RS, Parfitt AM, Roberson PK, Manolagas SC, Bellido T. Prevention of osteocyte and osteoblast apoptosis by bisphosphonates and calcitonin. J Clin Invest. 1999 Nov;104(10):1363-74. doi: 10.1172/JCI6800.

    PubMed 10562298

  • Rogers MJ, Frith JC, Luckman SP, Coxon FP, Benford HL, Monkkonen J, Auriola S, Chilton KM, Russell RG. Molecular mechanisms of action of bisphosphonates. Bone. 1999 May;24(5 Suppl):73S-79S. doi: 10.1016/s8756-3282(99)00070-8. No abstract available.

    PubMed 10321934

  • Drake MT, Clarke BL, Khosla S. Bisphosphonates: mechanism of action and role in clinical practice. Mayo Clin Proc. 2008 Sep;83(9):1032-45. doi: 10.4065/83.9.1032.

    PubMed 18775204

  • Kanzaki S, Ito M, Takada Y, Ogawa K, Matsuo K. Resorption of auditory ossicles and hearing loss in mice lacking osteoprotegerin. Bone. 2006 Aug;39(2):414-9. doi: 10.1016/j.bone.2006.01.155. Epub 2006 Mar 24.

    PubMed 16564235

  • Quesnel AM, Seton M, Merchant SN, Halpin C, McKenna MJ. Third-generation bisphosphonates for treatment of sensorineural hearing loss in otosclerosis. Otol Neurotol. 2012 Oct;33(8):1308-14. doi: 10.1097/MAO.0b013e318268d1b3.

    PubMed 22935809

  • Kang WS, Nguyen K, McKenna CE, Sewell WF, McKenna MJ, Jung DH. Measurement of Ototoxicity Following Intracochlear Bisphosphonate Delivery. Otol Neurotol. 2016 Jul;37(6):621-6. doi: 10.1097/MAO.0000000000001042.

    PubMed 27153329

  • Ting TH, Zacharin MR. Hearing in bisphosphonate-treated children with osteogenesis imperfecta: our experience in thirty six young patients. Clin Otolaryngol. 2012 Jun;37(3):229-33. doi: 10.1111/j.1749-4486.2012.02476.x. No abstract available.

    PubMed 22708940

  • Patel RM, Nagamani SC, Cuthbertson D, Campeau PM, Krischer JP, Shapiro JR, Steiner RD, Smith PA, Bober MB, Byers PH, Pepin M, Durigova M, Glorieux FH, Rauch F, Lee BH, Hart T, Sutton VR. A cross-sectional multicenter study of osteogenesis imperfecta in North America - results from the linked clinical research centers. Clin Genet. 2015 Feb;87(2):133-40. doi: 10.1111/cge.12409. Epub 2014 May 30.

    PubMed 24754836

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Risedronate Oral Tablet in people with Osteogenesis Imperfecta, run by Hospital for Special Surgery, New York at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 11 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 28 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Su Htwe ((212)774-2355, htwes@hss.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Pure Tone Averages (time frame: Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04152551, which also lists 14 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Apr 2026.

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