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NCT Number: NCT04152551

Effects of Bisphosphonates on OI-Related Hearing Loss

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.

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Key information

Conditions

Osteogenesis Imperfecta Bone Diseases Bone Diseases, Developmental Brittle Bone Disorder Carbohydrate Metabolism, Inborn Errors Collagen Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Ear Diseases Genetic Diseases, Inborn Hearing Disorders Hearing Loss Lysosomal Storage Diseases Metabolic Diseases Metabolism, Inborn Errors Mucinoses Mucopolysaccharidoses Mucopolysaccharidosis IV Musculoskeletal Diseases Nervous System Diseases Neurologic Manifestations Nutritional and Metabolic Diseases Osteochondrodysplasias Otorhinolaryngologic Diseases Pathological Conditions, Signs and Symptoms Sensation Disorders Signs and Symptoms Skin and Connective Tissue Diseases

Age range

6 year–100 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Risedronate Oral Tablet

Drug

Oral bisphosphonate

Other names: actonel

Primary outcomes

  1. Pure Tone Averages

    Time frame: 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 outcomes

  1. Speech Recognition Scores

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

    Lowest volume participant can hear and understand speech (decibels)

  2. Word Recognition Scores

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

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

  3. Hearing Handicap Inventory Raw Score

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

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

  4. Tinnitus Handicap Inventory Score

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

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

  5. Dizziness Handicap Inventory Score

    Time frame: 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.

  6. SF-36 Scale and Summary Scores

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

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

  7. Pediatric Outcomes Data Collection Instrument (PODCI) Score

    Time frame: 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.

  8. Adolescent Outcomes Questionnaire Score

    Time frame: 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.

  9. DEXA Z-score

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

    Higher score is better. Relative Bone Density

  10. DEXA Bone Mineral Density

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

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

Study contacts

Contact information is provided by the study sponsor or research team.

Erin Carter, MS, CGC

CONTACT

(212) 774-7332

Su Htwe

CONTACT

[email protected]

(212)774-2355

Sponsors and collaborators

Lead sponsor

Hospital for Special Surgery, New York

Other

Collaborators

  • East River Medical Imaging
  • Northwell Health
  • The New York Community Trust

Registry information

Official study title

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

Important dates

Study start
2019
Primary completion
2027
Study completion
2027
First posted
Nov 5, 2019
Registry last updated
Apr 28, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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