Back to resultsEffects of Bisphosphonates on OI-Related Hearing Loss
Primary Purpose
Osteogenesis Imperfecta
Status
Recruiting
Phase
Phase 4
Locations
United States
Study Type
Interventional
Intervention
Risedronate Oral Tablet
Sponsored by
About this trial
This is an interventional prevention trial for Osteogenesis Imperfecta focused on measuring hearing loss, bisphosphonate, skeletal dysplasia, brittle bone disorder, osteogenesis imperfecta, OI
Eligibility 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 (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
Sites / Locations
- Hospital for Special SurgeryRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
No Intervention
No Intervention
No Intervention
Arm Label
Adult Treatment Arm
Child (Bisphosphonate Arm)
Child (Control Arm)
Adult Control Arm
Arm Description
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.
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.
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.
Observational arm. Adults (18+ years) with type 1 OI. Changes in hearing, quality of life, and bone density will be monitored.
Outcomes
Primary Outcome Measures
Pure Tone Averages
Average hearing thresholds at 250, 500, 1000, 2000, 3000, 4000, 8000 Hertz
Secondary Outcome Measures
Speech Recognition Scores
Lowest volume participant can hear and understand speech (decibels)
Word Recognition Scores
Percent of words participants correctly repeat in word recognition test (%)
Hearing Handicap Inventory Raw Score
Score 0-40. Lower score is better. Adults (self-reported)
Tinnitus Handicap Inventory Score
Score 0-100. Lower score is better. Adults (self-reported).
Dizziness Handicap Inventory Score
Score 0-100. Lower score is better. Adults (self-reported). Incidence and impact of vertigo in study population.
SF-36 Scale and Summary Scores
Score 0-100. Higher score is better. Adults (self-reported) quality-of-life survey.
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.
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.
DEXA Z-score
Higher score is better. Relative Bone Density
DEXA Bone Mineral Density
Higher score is better. Bone Mineral Density (grams/centimeter^2)
Full Information
NCT ID
NCT04152551
First Posted
October 18, 2019
Last Updated
April 5, 2023
Sponsor
Hospital for Special Surgery, New York
Collaborators
The New York Community Trust, East River Medical Imaging, Northwell Health
1. Study Identification
Unique Protocol Identification Number
NCT04152551
Brief Title
Effects of Bisphosphonates on OI-Related Hearing Loss
Official Title
Effects of Bisphosphonates on OI-Related Hearing Loss: A Pilot Study
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
November 2, 2019 (Actual)
Primary Completion Date
November 2024 (Anticipated)
Study Completion Date
November 2024 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Hospital for Special Surgery, New York
Collaborators
The New York Community Trust, East River Medical Imaging, Northwell Health
4. Oversight
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
Data Monitoring Committee
No
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Osteogenesis Imperfecta
Keywords
hearing loss, bisphosphonate, skeletal dysplasia, brittle bone disorder, osteogenesis imperfecta, OI
7. Study Design
Primary Purpose
Prevention
Study Phase
Phase 4
Interventional Study Model
Single Group Assignment
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)
Allocation
Non-Randomized
Enrollment
100 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Adult Treatment Arm
Arm Type
Experimental
Arm Description
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.
Arm Title
Child (Bisphosphonate Arm)
Arm Type
No Intervention
Arm Description
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.
Arm Title
Child (Control Arm)
Arm Type
No Intervention
Arm Description
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.
Arm Title
Adult Control Arm
Arm Type
No Intervention
Arm Description
Observational arm. Adults (18+ years) with type 1 OI. Changes in hearing, quality of life, and bone density will be monitored.
Intervention Type
Drug
Intervention Name(s)
Risedronate Oral Tablet
Other Intervention Name(s)
actonel
Intervention Description
Oral bisphosphonate
Primary Outcome Measure Information:
Title
Pure Tone Averages
Description
Average hearing thresholds at 250, 500, 1000, 2000, 3000, 4000, 8000 Hertz
Time Frame
Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months
Secondary Outcome Measure Information:
Title
Speech Recognition Scores
Description
Lowest volume participant can hear and understand speech (decibels)
Time Frame
Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months
Title
Word Recognition Scores
Description
Percent of words participants correctly repeat in word recognition test (%)
Time Frame
Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months
Title
Hearing Handicap Inventory Raw Score
Description
Score 0-40. Lower score is better. Adults (self-reported)
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
Title
Tinnitus Handicap Inventory Score
Description
Score 0-100. Lower score is better. Adults (self-reported).
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
Title
Dizziness Handicap Inventory Score
Description
Score 0-100. Lower score is better. Adults (self-reported). Incidence and impact of vertigo in study population.
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
Title
SF-36 Scale and Summary Scores
Description
Score 0-100. Higher score is better. Adults (self-reported) quality-of-life survey.
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
Title
Pediatric Outcomes Data Collection Instrument (PODCI) Score
Description
Score 0-100. Lower score is better. Children (ages 6-10 years), parent-reported. Assessment of overall health and functioning.
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
Title
Adolescent Outcomes Questionnaire Score
Description
Score 0-100. Lower score is better. Children (ages 11-17 years), parent- or self-reported. Assessment of overall health and functioning.
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
Title
DEXA Z-score
Description
Higher score is better. Relative Bone Density
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
Title
DEXA Bone Mineral Density
Description
Higher score is better. Bone Mineral Density (grams/centimeter^2)
Time Frame
Yearly (Baseline, 12, 24, 36, 48, 60 months)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Years
Maximum Age & Unit of Time
100 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility 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 (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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Su Htwe
Phone
(212)774-2355
Email
htwes@hss.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Erin Carter, MS, CGC
Phone
(212) 774-7332
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Cathleen Raggio, MD
Organizational Affiliation
Hospital for Special Surgery, New York
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital for Special Surgery
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Su Htwe
Phone
212-774-2355
Email
htwes@hss.edu
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
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24715559
Citation
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Results Reference
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Citation
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Effects of Bisphosphonates on OI-Related Hearing Loss
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