A Phase 4 interventional study of Risedronate Oral Tablet in Osteogenesis Imperfecta, sponsored by Hospital for Special Surgery, New York. Recruiting at 1 site in United States. Open to participants aged 6 Years to 100 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2026-04-28.
Sponsored by Hospital for Special Surgery, New York · Phase 4, Interventional, and Prevention
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.
84 studies on the registry are indexed under Osteogenesis Imperfecta; 20 are open to participants now.
This study's planned enrollment of 100 is above the median of 28 across 60 interventional studies indexed under Osteogenesis Imperfecta.
Browse Osteogenesis Imperfecta studies →Hospital for Special Surgery, New York is the lead sponsor of 226 studies on the registry; 40 are open to participants now.
Of its 30 completed or terminated interventional studies of FDA-regulated products, 22 (73%) have results posted.
Counted across the registry records on this site, refreshed daily.
Inclusion Criteria (Adult Treatment Arm):
Inclusion Criteria (Adult Control Arm):
Inclusion Criteria (Child Observational Bisphosphonate Arm)
Inclusion Criteria (Child Observational No Treatment Arm)
Exclusion Criteria (ALL ARMS):
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
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.
Oral bisphosphonate
Also known as: actonel
Pure Tone Averages
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
Speech Recognition Scores
Lowest volume participant can hear and understand speech (decibels)
Time frame: Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months
Word Recognition Scores
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
Hearing Handicap Inventory Raw Score
Score 0-40. Lower score is better. Adults (self-reported)
Time frame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
Tinnitus Handicap Inventory Score
Score 0-100. Lower score is better. Adults (self-reported).
Time frame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
Dizziness Handicap Inventory Score
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)
SF-36 Scale and Summary Scores
Score 0-100. Higher score is better. Adults (self-reported) quality-of-life survey.
Time frame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
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.
Time frame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
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.
Time frame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
DEXA Z-score
Higher score is better. Relative Bone Density
Time frame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
DEXA Bone Mineral Density
Higher score is better. Bone Mineral Density (grams/centimeter\^2)
Time frame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
Plan to share: No
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