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Effects of Bisphosphonates on OI-Related Hearing Loss

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ClinicalTrials.gov Identifier: NCT04152551
Recruitment Status : Recruiting
First Posted : November 5, 2019
Last Update Posted : September 17, 2020
Sponsor:
Collaborators:
The New York Community Trust
Weill Cornell Medicine
Northwell Health
Information provided by (Responsible Party):
Hospital for Special Surgery, New York

Study Description
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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, ages 18-100, who have documented hearing loss. 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.


Condition or disease Intervention/treatment Phase
Osteogenesis Imperfecta Drug: Risedronate Oral Tablet Phase 4

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Intervention Model Description: 50 adults with type 1 OI and diagnosed hearing loss will be treated with oral risedronate.
Masking: None (Open Label)
Primary Purpose: Prevention
Official Title: Effects of Bisphosphonates on OI-Related Hearing Loss: A Pilot Study
Actual Study Start Date : November 2, 2019
Estimated Primary Completion Date : November 2024
Estimated Study Completion Date : November 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Adult Treatment Arm
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.
 Drug: Risedronate Oral Tablet
Oral bisphosphonate
Other Name: actonel
No Intervention: Child (Bisphosphonate Arm)
Observational (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.
No Intervention: Child (Control Arm)
Observational 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.


Outcome Measures
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Primary Outcome Measures :
  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 Outcome Measures :
  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)


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 100 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

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 (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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04152551


Contacts
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Contact: Holly LoTurco, BS (212)774-2355 loturcoh@hss.edu
Contact: Erin Carter, MS, CGC (212) 774-7332

Locations
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United States, New York
Hospital for Special Surgery Recruiting
New York, New York, United States, 10021
Contact: Holly LoTurco, BS    212-774-2355    loturcoh@hss.edu   
Sponsors and Collaborators
Hospital for Special Surgery, New York
The New York Community Trust
Weill Cornell Medicine
Northwell Health
Investigators
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Principal Investigator: Cathleen Raggio, MD Hospital for Special Surgery, New York
More Information
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Publications:

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Responsible Party: Hospital for Special Surgery, New York
ClinicalTrials.gov Identifier: NCT04152551    
Other Study ID Numbers: #2018-0700
First Posted: November 5, 2019    Key Record Dates
Last Update Posted: September 17, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Keywords provided by Hospital for Special Surgery, New York:
hearing loss
bisphosphonate
skeletal dysplasia
 brittle bone disorder
osteogenesis imperfecta
OI
Additional relevant MeSH terms:
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Osteogenesis Imperfecta
Hearing Loss
Deafness
Hearing Disorders
Ear Diseases
Otorhinolaryngologic Diseases
Sensation Disorders
Neurologic Manifestations
Nervous System Diseases
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
 Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases
Risedronic Acid
Calcium Channel Blockers
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Bone Density Conservation Agents