Studying Exercise in Mitochondrial Disease
This study is looking at different ways to help people with mitochondrial diseases. It compares standard exercise (like riding a stationary bike) with "passive exercise" methods, where a device moves your body for you. These passive exercise methods include the pGz Bed and Gentle Jogger, which use gentle, periodic movements. The study also uses an Exercise Pedal for in-bed exercise. Researchers will use a contrast agent called Lumason® during vascular ultrasounds to see how these exercises affect your blood vessels. The main goals are to understand how much oxygen your body uses during exercise and how oxygen is delivered to your tissues. You might be able to join if you are between 10 and 60 years old. The study is currently unclear on its recruitment status and plans to enroll 90 participants.
- Study design
- This is an interventional study with no specified phase, comparing different exercise methods. It plans to enroll 90 participants, including those with primary mitochondrial disease and healthy volunteers.
- What's involved
- You would have 3 study visits, each involving a different exercise intervention (Cardiopulmonary Exercise Testing, pGz Bed, or Gentle Jogger). Each visit includes blood draws, a vascular ultrasound with Lumason® contrast, and an MRI of your leg.
- Compensation
- Not stated in the trial record.
- Follow-up
- Oxygen consumption is measured for 1 hour per study intervention, and blood draws occur over a total of 4 15-minute periods.
AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.
NCT05569122
Applying pGz in Mitochondrial Disease
Recruiting
PHASE1Ages 10–60InterventionalDiagnosticChildren's Hospital of PhiladelphiaInvestigator-initiated
~90 participants
Updated 2025-11-10 on ClinicalTrials.gov
What's tested:Cardiopulmonary Exercise TestingpGz BedGentle JoggerExercise PedalLumason® contrast agent
At a glance
Recruiting sites
1 of 1 listed site is recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Aim 1: Mean Difference in Maximal Oxygen Consumption between primary mitochondrial disease patients and healthy volunteers
Measured over During Cardiopulmonary Exercise Testing, which will last 1 hour
+2 more outcomes measured
Conditions
Where it's being run
1 sites across 1 statesPennsylvania1
Study leadership
- Zuela Zolkipli-Cunningham, MBChB, MRCP · PRINCIPAL_INVESTIGATOR · Attending Physician
Who to contact
Opens a ready-to-send draft in your own email app — review before sending.
Do you actually qualify for this trial?
Add a private profile and we'll compare every criterion below against your situation — and tell you which ones are met, uncertain, or excluding.
Eligibility criteria
Inclusion
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
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
Males or females ages 10 to 23 years (children and adults)
Non-ambulatory
Genetically confirmed mtDNA-PMD
Cooperative and capable of following research procedures
Have cognitive ability to enable cooperation with study procedures
Admitted to the PICU with an anticipated length of stay for \>24 hours
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
Males or females ages 10 to 23 years (children and adults)
Non-ambulatory
Genetically confirmed non-PMD neuromuscular diagnosis
Cooperative and capable of following research procedures
Have cognitive ability to enable cooperation with study procedures
Admitted to the PICU with an anticipated length of stay for \>24 hours
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
Males or females ages 10 to 23 years (children and adults)
Non-ambulatory
No known genetic diagnosis with healthy pre-morbid status, admitted to PICU
Cooperative and capable of following research procedures
Have cognitive ability to enable cooperation with study procedures
Admitted to the PICU with an anticipated length of stay for \>24 hours
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
Exclusion
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
At the discretion of the principal investigator (PI), any medical condition that will interfere with or prevent the safe completion of the study
Parents/guardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures
Use of investigational agent(s) within 4 weeks
Individuals who are employed by the U.S. Department of Defense, including U.S military personal
Patients with biliary atresia with asplenia or polysplenia.
Patients with prior liver transplant.
Patients with cystic fibrosis.
Patients with chronic lung disease.
Patients with portal vein thrombosis, cavernous transformation of the portal vein or absent portal vein.
Patients with significant heart disease or severe congenital heart disease.
Patients with a history of allergic reaction to Lumason®, sulfur hexafluoride, sulfur hexafluoride lipid microsphere components, or other ingredients in Lumason (polyethylene glycol, distearoylphosphatidlycholine (DSPC), dipalmitoylphosphatidylglycerol sodium (DPPG-Na, palmitic acid) or other components of the ultrasound contrast agent
Any history of intraocular injury or fragment in or around the orbit that cannot be cleared through radiologic evaluation
Any history of bullet, shrapnel, or stabbing wounds that cannot be cleared through radiologic evaluation
Past or current employment involving (or exposure to) a metal grinder (e.g., at a construction worksite)
Claustrophobia or any known medical conditions which can be exacerbated by stress, anxiety, or panic attacks triggered by enclosed spaces
Inability to lie flat in an MRI scanner for up to 45 minutes
Unable to perform sub-maximal ankle dorsiflexion leg exercise during the MRI study
Have cognitive impairment that may preclude ability to comply with study procedures
Have cardiorespiratory instability
Patients in whom are so sick that they will not be able to cooperate with the study procedures
Have clear contraindications to mobilization
Have fixed lower limb deformities/contractures that would prohibit lower extremity exercise
Pregnant or lactating females
Active alcohol and/or substance abuse
At the discretion of the principal investigator (PI), any medical condition that will interfere with or prevent the safe completion of the study
Use of investigational agent(s) within 4 weeks
Individual who are employed by the U.S. Department of Defense, including U.S military personal
Patients with biliary atresia with asplenia or polysplenia.
Patients with prior liver transplant.
Patients with cystic fibrosis.
Patients with chronic lung disease.
Patients with portal vein thrombosis, cavernous transformation of the portal vein or absent portal vein.
Patients with significant heart disease or severe congenital heart disease.
Patients with a history of allergic reaction to Lumason®, sulfur hexafluoride, sulfur hexafluoride lipid microsphere components, or other ingredients in Lumason (polyethylene glycol, distearoylphosphatidlycholine (DSPC), dipalmitoylphosphatidylglycerol sodium (DPPG-Na, palmitic acid) or other components of the ultrasound contrast agent
Any history of intraocular injury or fragment in or around the orbit that cannot be cleared through radiologic evaluation
Any history of bullet, shrapnel, or stabbing wounds that cannot be cleared through radiologic evaluation
Past or current employment involving (or exposure to) a metal grinder (e.g., at a construction worksite)
Claustrophobia or any known medical conditions which can be exacerbated by stress, anxiety, or panic attacks triggered by enclosed spaces
Inability to lie flat in an MRI scanner for up to 45 minutes
Unable to perform sub-maximal ankle dorsiflexion leg exercise during the MRI study
What this trial measures
- Aim 1: Mean Difference in Maximal Oxygen Consumption between primary mitochondrial disease patients and healthy volunteersDuring Cardiopulmonary Exercise Testing, which will last 1 hour
Maximal Oxygen Consumption will be measured only during CPET
- Aim 2: Arterial-Venous (A-V) O2 differenceA total of 4 15 minute blood draws
This will be measured through blood draws that occur before and after study interventions
- Aim 1 and 2: Oxygen Consumption1 hour per study intervention
Measured During the study interventions