Wearable Technology to Evaluate Hyperglycemia and Heart Rate Variability in Duchenne Muscular Dystrophy
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Rate of hyperglycemia
研究概览
简要总结
Duchenne Muscular Dystrophy (DMD) is an X-linked disorder that causes muscle wasting, cardiopulmonary failure, and premature death. Heart failure is a leading cause of death in DMD, but substantial knowledge gaps exist regarding predisposing risk factors. In the general population, hyperglycemia, insulin resistance, and decreased heart rate variability (HRV; reflecting autonomic dysfunction) are associated with cardiomyopathy (CM). It is unclear whether these factors are associated with DMD-CM. Closing this knowledge gap may lead to novel screening and therapeutic strategies to delay progression of DMD related CM. Despite risk factors for hyperglycemia, including the use of glucocorticoids, low muscle mass, obesity, and reduced ambulation, little is known regarding glucose abnormalities in DMD. Some of these same risk factors, along with the distance needed to travel for specialty care, present significant barriers to research participation and clinical care for individuals with DMD. Remote wearable technology may improve research participation in this vulnerable population. Therefore, this study will leverage remote wearable technologies to overcome these barriers and define the relationship between dysglycemia and DMD-CM.
In this Aim of the study, the investigators will assess the utility of remote wearable technology to predict changes in traditional metrics of metabolism and cardiac function. In this pilot study, 10 individuals with DMD will undergo cardiac magnetic resonance imaging (CMR) and oral glucose tolerance tests (OGTTs) at baseline and two years. The investigators will remotely assess glycemia (using continuous glucose monitors), HRV (using extended Holter monitors), and activity (using accelerometers) every 6 months over the 2 years and evaluate if changes in wearable metrics predict changes in CMR and OGTT.
详细描述
Risk for hyperglycemia and insulin resistance in DMD: Individuals with DMD have multiple risk factors for abnormal glucose and insulin metabolism: frequent use of glucocorticoid (GC) medication, decreased ambulation/activity, sarcopenia, and obesity. GC use is known to increase the risk of impaired glucose tolerance (IGT) and insulin resistance (IR) in multiple populations. Decreased skeletal muscle mass and function are associated with impaired skeletal muscle insulin sensitivity and type 2 diabetes (T2D). Despite these risks, there are limited data relating glycemia and IR in this population.
This study is a critical first step in evaluating hyperglycemia in DMD and the relationship to autonomic dysfunction. Our findings will help establish screening guidelines and provide a basis for intervention studies targeting glycemia in DMD. Additionally, this study, along with other ongoing studies (Remote study: Wearable Technology to Evaluate Hyperglycemia and Heart Rate Variability in Duchenne Muscular Dystrophy) will establish wearable technology as investigational tools, for potential use in future clinical trials, in individuals with DMD and neuromuscular diseases.
Study Population: This study will include approximately 10 male participants at Vanderbilt with DMD.
DMD is an X-linked disorder affecting approximately 1/3500-6000 males and 1/50 million females. Therefore, only males will be included in this study.
Study Enrollment Period: Expected duration of the study is 6 years.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Cross Sectional
入排标准
- 年龄范围
- 10 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •Inclusion criteria
- •Male- ≥10 years
- •Clinical phenotype of DMD confirmed with muscle biopsy or genotype.
- •Informed consent for individuals ≥18 years
- •Parent/guardian informed consent and child assent for individuals < 18 years
- •Able to undergo non-sedated CMR
- •Exclusion Criteria
- •Refusal to participate
- •Diagnosis of diabetes prior to the study and/or taking insulin or other anti-diabetic drug therapy in < 4 weeks prior to treatment
- •Inability to fast for 10 hours
- •Use of a pacemaker, implantable cardioverter-defibrillator (ICD), or other implanted device
- •Unable to comply with study procedures, in the opinion of the investigator.
结局指标
主要结局
Rate of hyperglycemia
时间窗: 5 time points, each over 10 days
number of glucose measurements ≥140mg/dL over total number of glucoses
Standard deviation of the mean R-to-R segment (SDANN)
时间窗: 5 time points, each over 7 days
correlation of rate of hyperglycemia and SDANN, which reflects heart rate variability
次要结局
- Activity level(5 time points, each over 7 days)
- Standard deviation of normal R to R intervals (SDNN)(5 time points, each over 7 days)
- Rate of significant hyperglycemia(5 time points, each over 10 days)
- Insulin sensitivity(2 time points: initially and approximately 2 years later)
- Coefficient of variation on CGM(5 time points, each over 10 days)
- Left ventricular ejection fraction (LVEF)(2 time points: initially and approximately 2 years later)
- Late gadolinium enhancement (LGE)(2 time points: initially and approximately 2 years later)
研究者
Jaclyn Tamaroff
Assistant Professor of Pediatrics
Vanderbilt University Medical Center