Applying pGz in Mitochondrial Disease (NCT05569122) | Clinical Trial Compass
RecruitingPhase 1
Applying pGz in Mitochondrial Disease
United States90 participantsStarted 2023-03-22
Plain-language 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).
Who can participate
Age range
10 Years – 60 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Aim 1 Enrollment Criteria Inclusion Criteria for Healthy Controls
* 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
* For individuals over the age of 18 the ability to provide informed consent
Inclusion Criteria for PMD Patients
* 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
* Genetically confirmed mitochondrial myopathy (MM) as defined by a diagnosis of primary mitochondrial disease (PMD) with predominant symptoms of myopathy as expressed by exercise intolerance and muscle weakness and fatigue
* Parental/guardian permission (informed consent) and as appropriate, child assent
Exclusion Criteria for All Aim 1 Participants General Exclusion Criteria
* Tracheostomy
* Non-ambulatory
* Unable to complete routine exercise testing
* Diagnosed with or have symptoms of vertigo
* Within 1 month of a recent hospital admission for acute illness
* Severe co-existing cardiac or pulmonary disease
* Cognitive impairment that may preclude ability to comply with study procedures
* Pregnant or lactating females
* Active alcohol and/or substance abuse
…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Aim 1: Mean Difference in Maximal Oxygen Consumption between primary mitochondrial disease patients and healthy volunteers
Timeframe: During Cardiopulmonary Exercise Testing, which will last 1 hour