Health TrialsbyHealthPanel

Applying pGz in Mitochondrial Disease

ConditionsMitochondrial MyopathiesMitochondrial Diseases
Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age10-60
SponsorChildren's Hospital of Philadelphia

About this trial

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).

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

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

Disqualifiers

Tracheostomy

Non-ambulatory

Unable to complete routine exercise testing

Diagnosed with or have symptoms of vertigo

Trial design

Design model

Parallel

Treatments tested in this trial

  • Cardiopulmonary Exercise Testing

    Diagnostic test

    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

  • pGz Bed

    Device

    Participants will lay down on a passive exercise (pGz) bed for 45 minutes during which the bed will administer passive exercise through periodic acceleration

  • Gentle Jogger

    Device

    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)

  • Exercise Pedal

    Device

    Participants will exercise while laying down for 20 minutes with an exercise pedal that attaches to the bed

  • Lumason® contrast agent

    Drug

    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.

Treatment groups

90 Participants
are divided into 3 treatment groups
Group A: Aim 1: Primary Mitochondrial Disease PatientsExperimental treatment 4 interventions
Group B: Aim 1: Healthy ControlsExperimental treatment 4 interventions
Group C: Aim 2: PICU PatientsExperimental treatment 3 interventions

Trial outcomes

Primary outcomes

1

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
2

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
3

Aim 1 and 2: Oxygen Consumption

Measured During the study interventions

Time frame
1 hour per study intervention

Secondary outcomes

1

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
2

Aim 1 and 2: Heart Rate

To measure hemodynamic physiologic marker of cardiac output (CO)

Time frame
1 hour per study intervention
3

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
4

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Hospital of Philadelphia

Lead sponsor

United States Department of Defense

Collaborator

Source ClinicalTrials.gov
Apply now