Effects of Bisphosphonates on OI-Related Hearing Loss

Hospital for Special Surgery (HSS)

Status and phase: Enrolling; Phase 4

Conditions: Osteogenesis Imperfecta

Treatments: Drug: Risedronate Oral Tablet

Study type: Interventional

Funder types: Other

Identifiers

NCT04152551

#2018-0700

Details and patient eligibility

About

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.

Enrollment: 100 estimated patients

Sex: All

Ages: 6 to 100 years old

Volunteers: Accepts Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Prevention

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

---

100 participants in 4 patient groups

Adult Treatment Arm

Experimental group

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.

Treatment:

Drug: Risedronate Oral Tablet

Child (Bisphosphonate Arm)

No Intervention group

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.

Child (Control Arm)

No Intervention group

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.

Adult Control Arm

No Intervention group

Description:

Observational arm. Adults (18+ years) with type 1 OI. Changes in hearing, quality of life, and bone density will be monitored.