A Phase 1 interventional study of Cardiopulmonary Exercise Testing and pGz Bed in Mitochondrial Myopathies and Mitochondrial Diseases, sponsored by Children's Hospital of Philadelphia. Recruiting at 1 site in United States. Open to participants aged 10 Years to 60 Years, including healthy volunteers. Per ClinicalTrials.gov, last updated 2025-11-10.
Sponsored by Children's Hospital of Philadelphia · Phase 1, Interventional, and Diagnostic
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).
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.
46 studies on the registry are indexed under Mitochondrial Myopathies; 13 are open to participants now.
This study's planned enrollment of 90 is above the median of 26 across 32 interventional studies indexed under Mitochondrial Myopathies.
Browse Mitochondrial Myopathies studies →Children's Hospital of Philadelphia is the lead sponsor of 480 studies on the registry; 85 are open to participants now.
Of its 28 completed or terminated interventional studies of FDA-regulated products, 22 (79%) have results posted.
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Aim 1 Enrollment Criteria Inclusion Criteria for Healthy Controls
Inclusion Criteria for PMD Patients
Exclusion Criteria for All Aim 1 Participants General Exclusion Criteria
Aim 2 Enrollment Criteria Inclusion Criteria for PICU PMD Non-Ambulatory Patients
Inclusion Criteria for PICU non-PMD neuromuscular diagnosis
Inclusion Criteria for all other PICU Participants
Exclusion Criteria for All Aim 2 Participants
Exclusion Criteria Specific to study procedure: CrCEST MRI Scan:
The participant has the interventions/study visits occur in a random order: CPET pGz administration through pGz Bed pGz administration through Gentle Jogger
Diagnostic Test: Cardiopulmonary Exercise Testing · Device: pGz Bed · Device: Gentle Jogger · Drug: 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
Diagnostic Test: Cardiopulmonary Exercise Testing · Device: pGz Bed · Device: Gentle Jogger · Drug: Lumason® contrast agent
All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger
Device: Gentle Jogger · Device: Exercise Pedal · Drug: Lumason® contrast agent
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
Participants will lay down on a passive exercise (pGz) bed for 45 minutes during which the bed will administer passive exercise through periodic acceleration
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)
Participants will exercise while laying down for 20 minutes with an exercise pedal that attaches to the bed
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.
Aim 1: Mean Difference in Maximal Oxygen Consumption between primary mitochondrial disease patients and healthy volunteers
Maximal Oxygen Consumption will be measured only during CPET
Time frame: During Cardiopulmonary Exercise Testing, which will last 1 hour
Aim 2: Arterial-Venous (A-V) O2 difference
This will be measured through blood draws that occur before and after study interventions
Time frame: A total of 4 15 minute blood draws
Aim 1 and 2: Oxygen Consumption
Measured During the study interventions
Time frame: 1 hour per study intervention
Aim 1 and 2: A/B ratio measurement through EKG or Plethsymography
To measure hemodynamic physiologic marker of cardiac output (CO)
Time frame: 1 hour per study intervention
Aim 1 and 2: Heart Rate
To measure hemodynamic physiologic marker of cardiac output (CO)
Time frame: 1 hour per study intervention
Aim 1 and 2: OXPHOS Capacity
Measured through a CrCEST Leg MRI, which measures creative levels and recovery in the leg
Time frame: 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
Measured through venous blood draws
Time frame: 15 minute blood draws that occur pre and and immediately after each study intervention
Aim 1 and 2: Vasodilatation
Measured through a vascular ultrasound with contrast
Time frame: 30 minute ultrasound that occurs pre and immediately after each study intervention
NO release
Measured through Plethsymography
Time frame: 15 minutes, before and immediately after each study intervention
Plasma Nom Levels
Measured through venous blood draws
Time frame: 15 minute blood draws that occur pre and immediately after each study intervention
Post-operative Patient Satisfaction Survey
Participant tolerance to pGz compared to CPET
Time frame: 15 minutes, taken after each study intervention
Borg Scale
Participant tolerance to pGz compared to CPET
Time frame: 15 minutes, taken after each study intervention
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