Applying pGz in Mitochondrial Disease

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).

Conditions

• Mitochondrial Myopathies
• Mitochondrial Diseases

Interventions

DIAGNOSTIC_TEST

Cardiopulmonary 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

DEVICE

pGz 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

DEVICE

Gentle 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)

DEVICE

Exercise Pedal

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

DRUG

Lumason® 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.

Sponsors & Collaborators

• United States Department of Defense

  collaborator FED

• Children's Hospital of Philadelphia

  lead OTHER

Principal Investigators

• Zuela Zolkipli-Cunningham, MBChB, MRCP · Attending Physician

Study Design

Allocation

NON_RANDOMIZED

Purpose

DIAGNOSTIC

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

10 Years

Max Age

60 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2023-03-22

Primary Completion

2027-09-30

Completion

2028-09-30

FDA Drug

Yes

Countries

• United States

Study Locations