RecruitingPhase 1NCT05569122

Applying pGz in Mitochondrial Disease

The Utility of pGz in Primary Mitochondrial Disorders


Sponsor

Children's Hospital of Philadelphia

Enrollment

90 participants

Start Date

Mar 22, 2023

Study Type

INTERVENTIONAL

Conditions

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).


Eligibility

Min Age: 10 YearsMax Age: 60 Years

Plain Language Summary

Simplified for easier understanding

This study examines how a treatment called periodic gravitational stimulation (pGz) — a gentle rocking motion applied using a specialized table — affects exercise tolerance and muscle function in people with primary mitochondrial disease (PMD), a rare genetic condition that impairs the body's ability to produce energy. The study also includes healthy controls and hospitalized children with neuromuscular conditions for comparison. You may be eligible if (Aim 1 — outpatient): - You are between 10 and 60 years old and at least 135 cm tall - You can walk and perform routine exercise testing - You have a genetically confirmed mitochondrial myopathy (for patients) OR are a healthy volunteer You may be eligible if (Aim 2 — hospitalized): - You are between 10 and 23 years old - You are admitted to a PICU and expected to stay more than 24 hours - You have a genetically confirmed mitochondrial or neuromuscular disease, or no known genetic diagnosis You may NOT be eligible if: - You have a tracheostomy, severe heart or lung disease, or recent hospitalization within 1 month - You have vertigo, claustrophobia, or cognitive impairment - You are pregnant or breastfeeding - You have had a prior liver transplant, have portal vein thrombosis, or have cystic fibrosis - You have a known allergy to Lumason (the ultrasound contrast agent) - You have metal implants or history of injuries that cannot be cleared for MRI Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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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.


Locations(1)

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

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NCT05569122


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