The Utility of pGz in Primary Mitochondrial Disorders
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 90
- 试验地点
- 1
- 主要终点
- Aim 1 and 2: Oxygen Consumption
研究概览
简要总结
This is a multi-aim study, studying the effects of conventional exercise (measured through Cardiopulomary Exercises Testing or an in-bed pedal exercise) and passive exercise through periodic acceleration (pGz). Aim 1 will focus on the differences between primary mitochondrial disease (PMD) patients and healthy volunteers. Aim 2 is an exploratory aim, which will be studying the effects in patients admitted to the Children's Hospital of Philadelphia Pediatric Intensive Care Unit (PICU).
详细描述
Aim 1: Primary Mitochondrial Disease Patients and Healthy Controls
Individuals will be screened for eligibility for study entry, and answer questions relating to their ability to perform study procedures and their physical activity levels. Individuals who meet study criteria will have 3 study visits, and each study visit will involve a different intervention.
At each of these study visits, individuals will complete one of the following interventions: Cardiopulmonary Exercise Testing (CPET), pGz administration through a bed or recliner, and pGz through a device called a Gentle Jogger. While participants will complete all three study visits, the order of the study visits will occur in random order.
During the study visits, participants will have blood draws before and after the study intervention, a vascular ultrasound with a Lumason contrast agent before and after the study intervention, and a Creatine Chemical Exchange Saturation Transfer (CrCEST) MRI of the lower leg.
Aim 2: Patients in the Pediatric Intensive Care Unit (PICU)
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 10 Years 至 60 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Aim 1: Primary Mitochondrial Disease Patients
The participant has the interventions/study visits occur in a random order:
CPET pGz administration through pGz Bed pGz administration through Gentle Jogger
干预措施: Cardiopulmonary Exercise Testing (Diagnostic Test)
Aim 1: Primary Mitochondrial Disease Patients
The participant has the interventions/study visits occur in a random order:
CPET pGz administration through pGz Bed pGz administration through Gentle Jogger
干预措施: pGz Bed (Device)
Aim 1: Primary Mitochondrial Disease Patients
The participant has the interventions/study visits occur in a random order:
CPET pGz administration through pGz Bed pGz administration through Gentle Jogger
干预措施: Gentle Jogger (Device)
Aim 1: Primary Mitochondrial Disease Patients
The participant has the interventions/study visits occur in a random order:
CPET pGz administration through pGz Bed pGz administration through Gentle Jogger
干预措施: Lumason® contrast agent (Drug)
Aim 1: Healthy Controls
The participant has the interventions/study visits occur in a random order:
pGz administration through Gentle Jogger CPET pGz administration through pGz Bed
干预措施: Cardiopulmonary Exercise Testing (Diagnostic Test)
Aim 1: Healthy Controls
The participant has the interventions/study visits occur in a random order:
pGz administration through Gentle Jogger CPET pGz administration through pGz Bed
干预措施: pGz Bed (Device)
Aim 1: Healthy Controls
The participant has the interventions/study visits occur in a random order:
pGz administration through Gentle Jogger CPET pGz administration through pGz Bed
干预措施: Gentle Jogger (Device)
Aim 1: Healthy Controls
The participant has the interventions/study visits occur in a random order:
pGz administration through Gentle Jogger CPET pGz administration through pGz Bed
干预措施: Lumason® contrast agent (Drug)
Aim 2: PICU Patients
All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger
干预措施: Gentle Jogger (Device)
Aim 2: PICU Patients
All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger
干预措施: Exercise Pedal (Device)
Aim 2: PICU Patients
All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger
干预措施: Lumason® contrast agent (Drug)
结局指标
主要结局
Aim 1 and 2: Oxygen Consumption
时间窗: 1 hour per study intervention
Measured During the study interventions
Aim 1: Mean Difference in Maximal Oxygen Consumption between primary mitochondrial disease patients and healthy volunteers
时间窗: During Cardiopulmonary Exercise Testing, which will last 1 hour
Maximal Oxygen Consumption will be measured only during CPET
Aim 2: Arterial-Venous (A-V) O2 difference
时间窗: A total of 4 15 minute blood draws
This will be measured through blood draws that occur before and after study interventions
次要结局
- Post-operative Patient Satisfaction Survey(15 minutes, taken after each study intervention)
- Borg Scale(15 minutes, taken after each study intervention)
- Aim 1 and 2: A/B ratio measurement through EKG or Plethsymography(1 hour per study intervention)
- Aim 1 and 2: Heart Rate(1 hour per study intervention)
- Aim 1 and 2: OXPHOS Capacity(Aim 1 subjects will complete 2 1 hour MRIs, Aim 2 Subjects will complete 1 1-hour MRI)
- Aim 1 and 2: Plasma Lactate Levels(15 minute blood draws that occur pre and and immediately after each study intervention)
- Aim 1 and 2: Vasodilatation(30 minute ultrasound that occurs pre and immediately after each study intervention)
- NO release(15 minutes, before and immediately after each study intervention)
- Plasma Nom Levels(15 minute blood draws that occur pre and immediately after each study intervention)