Clinical trial NCT04152551: Effects of Bisphosphonates on OI-Related Hearing Loss
Official title: Effects of Bisphosphonates on OI-Related Hearing Loss: a Pilot Study
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: Osteogenesis Imperfecta
Keywords: hearing loss
bisphosphonate
skeletal dysplasia
brittle bone disorder
osteogenesis imperfecta
OI
Lead sponsor: Hospital for Special Surgery, New York
Sponsor class: OTHER
Collaborators: name: The New York Community Trust
name: East River Medical Imaging
name: Northwell Health
First posted: 2019-11-05
Enrollment: 100
Sex: ALL
Minimum age (years): 6
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: yes
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention 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.
Primary purpose: PREVENTION

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.


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

Primary outcomes:
measure: Pure Tone Averages
timeFrame: Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months
Secondary outcomes:
measure: Speech Recognition Scores
timeFrame: Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months
measure: Word Recognition Scores
timeFrame: Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months
measure: Hearing Handicap Inventory Raw Score
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
measure: Tinnitus Handicap Inventory Score
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
measure: Dizziness Handicap Inventory Score
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
measure: SF-36 Scale and Summary Scores
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
measure: Pediatric Outcomes Data Collection Instrument (PODCI) Score
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
measure: Adolescent Outcomes Questionnaire Score
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
measure: DEXA Z-score
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
measure: DEXA Bone Mineral Density
timeFrame: Yearly (Baseline, 12, 24, 36, 48, 60 months)
Other outcomes:
Locations:
city: New York
country: United States
facility: Hospital for Special Surgery
state: New York
Study officials:
affiliation: Hospital for Special Surgery, New York
name: Cathleen Raggio, MD
role: PRINCIPAL_INVESTIGATOR
References:
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