Effects of Bisphosphonates on OI-Related Hearing Loss: A Pilot Study
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 100
- 试验地点
- 2
- 主要终点
- Pure Tone Averages
研究概览
简要总结
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 100 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •(Adult Treatment Arm):
- •Diagnosis of OI type I
- •Diagnosis of at least mild hearing loss (>20dB pure tone average) by audiogram testing
- •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
排除标准
- •(ALL ARMS):
- •Family history of hearing-loss (not related to OI or occupational hearing loss)
- •Pregnancy
研究组 & 干预措施
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.
干预措施: Risedronate Oral Tablet (Drug)
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.
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.
Adult Control Arm
Observational arm. Adults (18+ years) with type 1 OI. Changes in hearing, quality of life, and bone density will be monitored.
结局指标
主要结局
Pure Tone Averages
时间窗: 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
次要结局
- Tinnitus Handicap Inventory Score(Yearly (Baseline, 12, 24, 36, 48, 60 months))
- Word Recognition Scores(Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months)
- DEXA Z-score(Yearly (Baseline, 12, 24, 36, 48, 60 months))
- Hearing Handicap Inventory Raw Score(Yearly (Baseline, 12, 24, 36, 48, 60 months))
- Dizziness Handicap Inventory Score(Yearly (Baseline, 12, 24, 36, 48, 60 months))
- SF-36 Scale and Summary Scores(Yearly (Baseline, 12, 24, 36, 48, 60 months))
- Pediatric Outcomes Data Collection Instrument (PODCI) Score(Yearly (Baseline, 12, 24, 36, 48, 60 months))
- DEXA Bone Mineral Density(Yearly (Baseline, 12, 24, 36, 48, 60 months))
- Speech Recognition Scores(Baseline, 3, 6, 12, 18, 24, 30, 36, 42, 48, 54, 60 months)
- Adolescent Outcomes Questionnaire Score(Yearly (Baseline, 12, 24, 36, 48, 60 months))