CFTR Modulator Effects on Bone and Muscle in Adults With Cystic Fibrosis
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 63
- 试验地点
- 1
- 主要终点
- Identify the effects of CFTR modulators on bone mineral density
研究概览
简要总结
Study is looking at the effects of cystic fibrosis treatment on bone muscle.
详细描述
Cystic fibrosis (CF) is a complex multisystem genetic disease, with pulmonary and gastrointestinal consequences dominating the clinical picture. The life-expectancy of CF patients has increased through several therapeutic advances. Although respiratory failure remains the major cause of mortality in CF, musculoskeletal impairments contribute to major morbidity. In the general population, musculoskeletal conditions are among the most common reasons for seeking medical care, and the risk of osteoporotic fracture increases with age. As the CF population ages, the morbidity related to musculoskeletal effects may increase.
The etiology of CF related bone disease is multifactorial and includes effects of pancreatic insufficiency, poor nutritional status, vitamin D deficiency, glucocorticoid treatment, inflammation, hypogonadism, and sarcopenia, collectively resulting in attenuated bone mineral accrual and low bone density The effect of cystic fibrosis transmembrane conductance regulator (CFTR) modulating drugs on bone disease in CF has not been evaluated. Effects of CFTR modulators may help counter the bone and muscle consequences of CF either directly by effects on bone or muscle cells, or indirectly by improved lung disease, improved nutritional status, decreased systemic inflammation or glucocorticoid use, or subsequent increases in physical activity.
The rationale that underlies the proposed research is that better understanding of the bone and muscle effects of CFTR modulator therapies will help guide strategies to optimize bone accrual, prevent osteoporosis and fractures, and improve functional outcomes in the aging CF population. Set on the backbone of a longitudinal observational cohort study, the study will systematically and comprehensively evaluate changes in bone and muscle mass and strength from baseline to 12 month and 24 month time points among patients receiving CFTR modulator therapies and also among controls not receiving CFTR modulator therapies.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •documented, confirmed diagnosis of CF
- •Age ≥18 years old
- •>21 days since the start of their last pulmonary exacerbation at the baseline visit
- •Provide signed written informed consent to participate
排除标准
- •• Estimated glomerular filtration rate (eGFR) <30 ml/min/m2 using the CKD-EPI equation,
- •Treatment with any osteoporosis medication within 6 months for oral agents or 1 year for intravenous or injectable agents (Subjects may participate if therapy stopped earlier than these time periods).
- •Current treatment with growth hormone or IGF-1
- •Currently pregnant or lactating or planning plan on becoming pregnant during the duration of the study.
- •Life expectancy less than 12 months
- •History of lung transplantation
- •Conditions that in the opinion of the investigators would interfere with the ability to collect or interpret the data, or put the patient at higher safety risk from study procedures.
研究组 & 干预措施
on CFTR
For patients on or near time of initiation CFTR modulator therapy
干预措施: Cftr Modulators (Drug)
结局指标
主要结局
Identify the effects of CFTR modulators on bone mineral density
时间窗: 12 months
Changes from baseline to 12 month in total volumetric BMD and in estimated failure load as measured by HRpQCT at the distal radius and tibia
establish the effect of CFTR modulators on sarcopenia.
时间窗: 12 months
changes from baseline to 12 months in whole body lean mass using body composition software on DXA
次要结局
- establish the effect of CFTR modulators on sarcopenia.(6, 12 and 24 month)
- Identify the effect of CFTR modulators on bone mineral density(6, 12 and 24 month)
研究者
Erik Imel
Associate Professor
Indiana University