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
- 80
- 试验地点
- 2
- 主要终点
- Standard deviation of the mean R-to-R segment (SDANN)
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-CM, now the leading cause of death in patients with DMD. Despite risk factors for hyperglycemia, including the use of glucocorticoids (GCs), sarcopenia, 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.
The goal of this remote study is to evaluate rates of hyperglycemia in individuals with DMD compared to control participants using continuous glucose monitors, and to determine the relationship between hyperglycemia and heart rate variability. Participants will utilize continuous glucose monitors, cardiac monitors, and activity monitors to evaluate glucose levels, heart rate, activity, and sleep.
详细描述
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 (Wearable Technology to Evaluate Hyperglycemia and Heart Rate Variability in Duchenne Muscular Dystrophy - longitudinal aim) 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 40 participants with DMD and 40 age/gender/BMI category, race and ethnicity matched participants without 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.
Study procedures (remote or in-person):
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 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
- 是
入选标准
- •CASE, DMD inclusion criteria:
- •Age ≥10years
- •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
排除标准
- •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
- •Use of a pacemaker, Implantable cardioverter-defibrillator (ICD), or other implanted device
- •Unable to comply with study procedures, in the opinion of the investigator.
- •CONTROL inclusion criteria:
- •Age ≥10years
- •Informed consent for individuals ≥18 years
- •Parent/guardian informed consent and child assent for individuals < 18 years
- •BMI matched by Centers for Disease Control and Prevention (CDC) category (underweight, normal, overweight, obese) to cases.
- •Self-reported race/ethnicity matched to cases.
- •No known evidence of diabetes, impaired fasting glucose, or impaired glucose tolerance:
- •For individuals (all ≥10 years) of age with obesity, we anticipate that they will have hemoglobin A1c (HbA1c) screening based on American Academy of Pediatrics (AAP) recommendations.
- •Participants will be included if they have a normal HbA1c (< 5.7%) or if they have an elevated HbA1c (5.7-6.4%) with no evidence of impaired fasting glucose or impaired glucose tolerance on clinically obtained oral glucose tolerance tests (OGTT) (e.g., fasting glucose <100mg/dL and 2-hour glucose <140mg/dL).
- •CONTROL, 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
- •Use of a pacemaker, Implantable cardioverter-defibrillator (ICD), or other implanted device
- •Unable to comply with study procedures, in the opinion of the investigator.
- •Diagnosis of DMD or Becker muscular dystrophy
结局指标
主要结局
Standard deviation of the mean R-to-R segment (SDANN)
时间窗: once over 7 days
correlation of rate of hyperglycemia and SDANN, which reflects heart rate variability
Rate of hyperglycemia
时间窗: once over 10 days
number of glucose measurements ≥140mg/dL over total number of glucoses compared between individuals with and without DMD
次要结局
- Rate of significant hyperglycemia(once over 10 days)
- Activity level - measured by ActiGraph(once over 7 days)
- Standard deviation of normal R to R intervals (SDNN)(once over 7 days)
- Coefficient of variation on CGM(once over 10 days)
研究者
Jaclyn Tamaroff
Assistant Professor of Pediatrics
Vanderbilt University Medical Center