Applying pGz in Mitochondrial Disease

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Children's Hospital of Philadelphia
Study ID
NCT05569122
Phase
PHASE1
Status
Recruiting
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Conditions

  • Mitochondrial Diseases
  • Mitochondrial Myopathies

Eligibility Criteria

Sex
ALL
Age
10 Years - 60 Years
Healthy Volunteers
Accepted

Interventions

  • 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.

Study Details

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).

Key Dates

Start date
Mar 22, 2023
Status verified
Nov 2025
Primary completion
Sep 30, 2027
Completion
Sep 30, 2028

Study Design

Enrollment
90 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
DIAGNOSTIC

Arms

  • Experimental: Aim 1: Primary Mitochondrial Disease Patients
    The participant has the interventions/study visits occur in a random order: CPET pGz administration through pGz Bed pGz administration through Gentle Jogger
  • Experimental: Aim 1: Healthy Controls
    The participant has the interventions/study visits occur in a random order: pGz administration through Gentle Jogger CPET pGz administration through pGz Bed
  • Experimental: Aim 2: PICU Patients
    All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger

Primary Outcome Measure

Aim 1: Mean Difference in Maximal Oxygen Consumption between primary mitochondrial disease patients and healthy volunteers [ Time Frame: During Cardiopulmonary Exercise Testing, which will last 1 hour ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104-

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By research site
Children's Hospital of Philadelphia· Philadelphia, PA

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