The Utility of pGz in Primary Mitochondrial Disorders
Children's Hospital of Philadelphia
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).
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 p…
Eligibility
- Age range
- 10–60 years
- Sex
- All
- Healthy volunteers
- Yes
Aim 1 Enrollment Criteria Inclusion Criteria for Healthy Controls * Males or females, 10 years to 60 years, with a minimum height for participation of 135 cm * Ambulatory and able to complete routine clinical exercise testing * Willing and able to complete all study procedures * For individuals under the age of 18, parental/guardian permission (informed consent) and as appropriate, child assent * For individuals over the age of 18 the ability to provide informed consent Inclusion Criteria for PMD Patients * Males or females, 10 years to 60 years, with a minimum height for participation of 1…
Interventions
- Diagnostic TestCardiopulmonary Exercise Testing
Testing with an exercise bicycle that is considered "standard of care" for determination of exercise capacity. Participants will complete about 20 minutes of pedaling in a stationary exercise bike
- DevicepGz Bed
Participants will lay down on a passive exercise (pGz) bed for 45 minutes during which the bed will administer passive exercise through periodic acceleration
- DeviceGentle Jogger
Participants will have passive exercise delivered through the gentle jogger device for 45 minutes. This may be sitting down (aim 1 participants) or laying down (aim 2 participants)
- DeviceExercise Pedal
Participants will exercise while laying down for 20 minutes with an exercise pedal that attaches to the bed
- DrugLumason® contrast agent
Contrast agent used during a vascular ultrasound of the upper leg. Will occur at each study visit twice before and after pGz bed, gentle jogger, exercise pedal or CPET. Drug Administration will be through an IV line and take about 5 - 10 minutes.
Location
- Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania