Applying pGz in Mitochondrial Disease
- Conditions
- Mitochondrial MyopathiesMitochondrial Diseases
- Interventions
- Diagnostic Test: Cardiopulmonary Exercise TestingDevice: pGz BedDevice: Exercise PedalDevice: Gentle Jogger
- Registration Number
- NCT05569122
- Lead Sponsor
- Children's Hospital of Philadelphia
- Brief Summary
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).
- Detailed Description
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)
Individuals will be screened for eligibility for study entry. Individuals who meet study criteria will have 2 study visits during their admission to the PICU. The first study visit will involve a pedal exercise and the second study visit will involve pGz administration through a device called a Gentle Jogger.
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 if able to safely complete, an CrCEST MRI of the lower leg.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 90
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Aim 2: PICU Patients Lumason® contrast agent All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger Aim 1: Healthy Controls Gentle Jogger The participant has the interventions/study visits occur in a random order: pGz administration through Gentle Jogger CPET pGz administration through pGz Bed Aim 1: Primary Mitochondrial Disease Patients Cardiopulmonary Exercise Testing The participant has the interventions/study visits occur in a random order: CPET pGz administration through pGz Bed pGz administration through Gentle Jogger Aim 1: Primary Mitochondrial Disease Patients pGz Bed The participant has the interventions/study visits occur in a random order: CPET pGz administration through pGz Bed pGz administration through Gentle Jogger Aim 1: Primary Mitochondrial Disease Patients Gentle Jogger The participant has the interventions/study visits occur in a random order: CPET pGz administration through pGz Bed pGz administration through Gentle Jogger Aim 1: Primary Mitochondrial Disease Patients Lumason® contrast agent The participant has the interventions/study visits occur in a random order: CPET pGz administration through pGz Bed pGz administration through Gentle Jogger Aim 1: Healthy Controls Cardiopulmonary Exercise Testing The participant has the interventions/study visits occur in a random order: pGz administration through Gentle Jogger CPET pGz administration through pGz Bed Aim 1: Healthy Controls pGz Bed The participant has the interventions/study visits occur in a random order: pGz administration through Gentle Jogger CPET pGz administration through pGz Bed Aim 1: Healthy Controls Lumason® contrast agent The participant has the interventions/study visits occur in a random order: pGz administration through Gentle Jogger CPET pGz administration through pGz Bed Aim 2: PICU Patients Gentle Jogger All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger Aim 2: PICU Patients Exercise Pedal All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger
- Primary Outcome Measures
Name Time Method Aim 1 and 2: Oxygen Consumption 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 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 A total of 4 15 minute blood draws This will be measured through blood draws that occur before and after study interventions
- Secondary Outcome Measures
Name Time Method Post-operative Patient Satisfaction Survey 15 minutes, taken after each study intervention Participant tolerance to pGz compared to CPET
Borg Scale 15 minutes, taken after each study intervention Participant tolerance to pGz compared to CPET
Aim 1 and 2: A/B ratio measurement through EKG or Plethsymography 1 hour per study intervention To measure hemodynamic physiologic marker of cardiac output (CO)
Aim 1 and 2: Heart Rate 1 hour per study intervention To measure hemodynamic physiologic marker of cardiac output (CO)
Aim 1 and 2: OXPHOS Capacity Aim 1 subjects will complete 2 1 hour MRIs, Aim 2 Subjects will complete 1 1-hour MRI Measured through a CrCEST Leg MRI, which measures creative levels and recovery in the leg
Aim 1 and 2: Plasma Lactate Levels 15 minute blood draws that occur pre and and immediately after each study intervention Measured through venous blood draws
Aim 1 and 2: Vasodilatation 30 minute ultrasound that occurs pre and immediately after each study intervention Measured through a vascular ultrasound with contrast
NO release 15 minutes, before and immediately after each study intervention Measured through Plethsymography
Plasma Nom Levels 15 minute blood draws that occur pre and immediately after each study intervention Measured through venous blood draws
Trial Locations
- Locations (1)
Children's Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States