Mitochondrial MyopathiesPhase 1NCT05569122
Applying pGz in Mitochondrial Disease
A Phase 1 study testing Cardiopulmonary Exercise Testing, pGz Bed and Gentle Jogger in people with Mitochondrial Myopathies and Mitochondrial Diseases, run by Children's Hospital of Philadelphia at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 10 to 60 yearsHealthy volunteers can take part
- Conditions the study is for
- Mitochondrial Myopathies, Mitochondrial Diseases
- What is being tested?
- Cardiopulmonary Exercise Testing, pGz Bed and Gentle Jogger
- Type of treatment
- Injection or infusion, Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Mar 2023to Sep 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 to 60 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- 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
Show all 59Show fewer
- 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
- Aim 2 Enrollment Criteria Inclusion Criteria for PICU PMD Non-Ambulatory Patients
- 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
Who cannot join
- 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
Show all 24Show fewer
- 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
- Exclusion Criteria Specific to study procedure: CrCEST MRI Scan:
- 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
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
About this study
Official title: The Utility of pGz in Primary Mitochondrial Disorders
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).
Read the detailed description
Aim 1: Primary Mitochondrial Disease Patients and Healthy Controls
Individuals will be screened for eligibility for study entry, and answer questions relating to their ability to perform study procedures and their physical activity levels. Individuals who meet study criteria will have 3 study visits, and each study visit will involve a different intervention.
At each of these study visits, individuals will complete one of the following interventions: Cardiopulmonary Exercise Testing (CPET), pGz administration through a bed or recliner, and pGz through a device called a Gentle Jogger. While participants will complete all three study visits, the order of the study visits will occur in random order.
During the study visits, participants will have blood draws before and after the study intervention, a vascular ultrasound with a Lumason contrast agent before and after the study intervention, and a Creatine Chemical Exchange Saturation Transfer (CrCEST) MRI of the lower leg.
Aim 2: Patients in the Pediatric Intensive Care Unit (PICU)
Individuals will be screened for eligibility for study entry. Individuals who meet study criteria will have 2 study visits during their admission to the PICU. The first study visit will involve a pedal exercise and the second study visit will involve pGz administration through a device called a Gentle Jogger.
During the study visits, participants will have blood draws before and after the study intervention, a vascular ultrasound with a Lumason contrast agent before and after the study intervention, and if able to safely complete, an CrCEST MRI of the lower leg.
- Conditions
- Mitochondrial Myopathies, Mitochondrial Diseases
- Being tested
- Cardiopulmonary Exercise Testing, pGz Bed, Gentle Jogger, Exercise Pedal, Lumason® contrast agent
- Keywords
- Passive Exercise, periodic acceleration
- Medical subject headings
- Mitochondrial Myopathies, Mitochondrial Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Cardiopulmonary Exercise TestingDiagnostic 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 BedDevice
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 JoggerDevice
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 PedalDevice
Participants will exercise while laying down for 20 minutes with an exercise pedal that attaches to the bed
Lumason® contrast agentDrug
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 group | Role | What they receive |
|---|---|---|
| Aim 1: Primary Mitochondrial Disease Patients | Experimental | The participant has the interventions/study visits occur in a random order:
CPET pGz administration through pGz Bed pGz administration through Gentle Jogger Receives: Diagnostic Test: Cardiopulmonary Exercise Testing; Device: pGz Bed; Device: Gentle Jogger; Drug: Lumason® contrast agent |
| Aim 1: Healthy Controls | Experimental | The participant has the interventions/study visits occur in a random order:
pGz administration through Gentle Jogger CPET pGz administration through pGz Bed Receives: Diagnostic Test: Cardiopulmonary Exercise Testing; Device: pGz Bed; Device: Gentle Jogger; Drug: Lumason® contrast agent |
| Aim 2: PICU Patients | Experimental | All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger Receives: Device: Gentle Jogger; Device: Exercise Pedal; Drug: Lumason® contrast agent |
What is being measured
The primary outcome is the main question the study is designed to answer.
Aim 1: Mean Difference in Maximal Oxygen Consumption between primary mitochondrial disease patients and healthy volunteers
Maximal Oxygen Consumption will be measured only during CPET
Measured: During Cardiopulmonary Exercise Testing, which will last 1 hour
Aim 2: Arterial-Venous (A-V) O2 difference
This will be measured through blood draws that occur before and after study interventions
Measured: A total of 4 15 minute blood draws
Aim 1 and 2: Oxygen Consumption
Measured During the study interventions
Measured: 1 hour per study intervention
Secondary outcomes (9)
Aim 1 and 2: A/B ratio measurement through EKG or Plethsymography
To measure hemodynamic physiologic marker of cardiac output (CO)
Measured: 1 hour per study intervention
Aim 1 and 2: Heart Rate
To measure hemodynamic physiologic marker of cardiac output (CO)
Measured: 1 hour per study intervention
Aim 1 and 2: OXPHOS Capacity
Measured through a CrCEST Leg MRI, which measures creative levels and recovery in the leg
Measured: Aim 1 subjects will complete 2 1 hour MRIs, Aim 2 Subjects will complete 1 1-hour MRI
Aim 1 and 2: Plasma Lactate Levels
Measured through venous blood draws
Measured: 15 minute blood draws that occur pre and and immediately after each study intervention
Aim 1 and 2: Vasodilatation
Measured through a vascular ultrasound with contrast
Measured: 30 minute ultrasound that occurs pre and immediately after each study intervention
NO release
Measured through Plethsymography
Measured: 15 minutes, before and immediately after each study intervention
Plasma Nom Levels
Measured through venous blood draws
Measured: 15 minute blood draws that occur pre and immediately after each study intervention
Post-operative Patient Satisfaction Survey
Participant tolerance to pGz compared to CPET
Measured: 15 minutes, taken after each study intervention
Borg Scale
Participant tolerance to pGz compared to CPET
Measured: 15 minutes, taken after each study intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Oct 2022
- Study start22 Mar 2023
- Primary completionSep 2027 (estimated)
- Study completionSep 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (20)Papers the sponsor has linked to this study.
- Other trials for Mitochondrial MyopathiesStudies of the same condition, open and under way.
- More trials run by Children's Hospital of PhiladelphiaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Aim 1 will have the 3 study interventions/study visits occur in random order. The interventions will be performed in primary mitochondrial disease patients and healthy controls Aim 2 will have all participants complete the study interventions/visits in the same order. Patients will be from the PICU
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Philadelphia
- Sponsor type
- University, hospital or other
- Collaborators
- United States Department of Defense
- Responsible party
- Sponsor
- Investigators
- Zuela Zolkipli-Cunningham, MBChB, MRCP, Principal investigator, Attending Physician
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05569122
- Sponsor's protocol ID
- 21-018532
- Status last verified
- Nov 2025
- First submitted
- 7 Jul 2022
- Last updated
- 10 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Sackner MA, Lopez JR, Banderas V, Adams JA. Can Physical Activity While Sedentary Produce Health Benefits? A Single-Arm Randomized Trial. Sports Med Open. 2020 Oct 2;6(1):47. doi: 10.1186/s40798-020-00278-3.
PubMed 33006749Sackner MA, Patel S, Adams JA. Changes of blood pressure following initiation of physical inactivity and after external addition of pulses to circulation. Eur J Appl Physiol. 2019 Jan;119(1):201-211. doi: 10.1007/s00421-018-4016-7. Epub 2018 Oct 22.
PubMed 30350153Burstein DS, McBride MG, Min J, Paridon AA, Perelman S, Huffman EM, O'Malley S, Del Grosso J, Groepenhoff H, Paridon SM, Brothers JA. Normative Values for Cardiopulmonary Exercise Stress Testing Using Ramp Cycle Ergometry in Children and Adolescents. J Pediatr. 2021 Feb;229:61-69.e5. doi: 10.1016/j.jpeds.2020.09.018. Epub 2020 Sep 11.
PubMed 32926876Taivassalo T, Jensen TD, Kennaway N, DiMauro S, Vissing J, Haller RG. The spectrum of exercise tolerance in mitochondrial myopathies: a study of 40 patients. Brain. 2003 Feb;126(Pt 2):413-23. doi: 10.1093/brain/awg028.
PubMed 12538407Tarnopolsky M. Exercise testing in metabolic myopathies. Phys Med Rehabil Clin N Am. 2012 Feb;23(1):173-86, xii. doi: 10.1016/j.pmr.2011.11.011. Epub 2011 Dec 11.
PubMed 22239882Tarnopolsky M. Exercise testing as a diagnostic entity in mitochondrial myopathies. Mitochondrion. 2004 Sep;4(5-6):529-42. doi: 10.1016/j.mito.2004.07.011. Epub 2004 Sep 30.
PubMed 16120411Taivassalo T, Haller RG. Exercise and training in mitochondrial myopathies. Med Sci Sports Exerc. 2005 Dec;37(12):2094-101. doi: 10.1249/01.mss.0000177446.97671.2a.
PubMed 16331135Adams JA, Uryash A, Bassuk J, Sackner MA, Kurlansky P. Biological basis of neuroprotection and neurotherapeutic effects of Whole Body Periodic Acceleration (pGz). Med Hypotheses. 2014 Jun;82(6):681-7. doi: 10.1016/j.mehy.2014.02.031. Epub 2014 Mar 12.
PubMed 24661939Adams JA, Mangino MJ, Bassuk J, Kurlansky P, Sackner MA. Regional blood flow during periodic acceleration. Crit Care Med. 2001 Oct;29(10):1983-8. doi: 10.1097/00003246-200110000-00022.
PubMed 11588467M. Fujita et al., "Periodic acceleration enhances release of nitric oxide in healthy adults," Int. J. Angiol., vol. 14, no. 1, pp. 11-14, Feb. 2005, doi: 10.1007/s00547-005-2013-2.
Uryash A, Bassuk J, Kurlansky P, Altamirano F, Lopez JR, Adams JA. Antioxidant Properties of Whole Body Periodic Acceleration (pGz). PLoS One. 2015 Jul 2;10(7):e0131392. doi: 10.1371/journal.pone.0131392. eCollection 2015.
PubMed 26133377Uryash A, Bassuk J, Kurlansky P, Altamirano F, Lopez JR, Adams JA. Non-invasive technology that improves cardiac function after experimental myocardial infarction: Whole Body Periodic Acceleration (pGz). PLoS One. 2015 Mar 25;10(3):e0121069. doi: 10.1371/journal.pone.0121069. eCollection 2015.
PubMed 25807532Adams JA, Patel S, Lopez JR, Sackner MA. The Effects of Passive Simulated Jogging on Short-Term Heart Rate Variability in a Heterogeneous Group of Human Subjects. J Sports Med (Hindawi Publ Corp). 2018 Oct 1;2018:4340925. doi: 10.1155/2018/4340925. eCollection 2018.
PubMed 30402499Sackner MA, Gummels E, Adams JA. Nitric oxide is released into circulation with whole-body, periodic acceleration. Chest. 2005 Jan;127(1):30-9. doi: 10.1378/chest.127.1.30.
PubMed 15653959Adams JA, Bassuk J, Wu D, Grana M, Kurlansky P, Sackner MA. Periodic acceleration: effects on vasoactive, fibrinolytic, and coagulation factors. J Appl Physiol (1985). 2005 Mar;98(3):1083-90. doi: 10.1152/japplphysiol.00662.2004. Epub 2004 Oct 22.
PubMed 15501928
Study at a glance
From the registry record, last updated by the sponsor on 10 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Utility of pGz in Primary Mitochondrial Disorders
- ClinicalTrials.gov ID
- NCT05569122
- Acronym / sponsor ID
- 21-018532
- Conditions
- Mitochondrial Myopathies, Mitochondrial Diseases
- Keywords
- Passive Exercise, periodic acceleration
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Children's Hospital of PhiladelphiaUniversity, hospital or other
- Collaborators
- United States Department of Defense
- Study start
- 22 Mar 2023 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2028 (estimated)
- First posted
- 6 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Detailed description
Aim 1: Primary Mitochondrial Disease Patients and Healthy Controls
Individuals will be screened for eligibility for study entry, and answer questions relating to their ability to perform study procedures and their physical activity levels. Individuals who meet study criteria will have 3 study visits, and each study visit will involve a different intervention.
At each of these study visits, individuals will complete one of the following interventions: Cardiopulmonary Exercise Testing (CPET), pGz administration through a bed or recliner, and pGz through a device called a Gentle Jogger. While participants will complete all three study visits, the order of the study visits will occur in random order.
During the study visits, participants will have blood draws before and after the study intervention, a vascular ultrasound with a Lumason contrast agent before and after the study intervention, and a Creatine Chemical Exchange Saturation Transfer (CrCEST) MRI of the lower leg.
Aim 2: Patients in the Pediatric Intensive Care Unit (PICU)
Individuals will be screened for eligibility for study entry. Individuals who meet study criteria will have 2 study visits during their admission to the PICU. The first study visit will involve a pedal exercise and the second study visit will involve pGz administration through a device called a Gentle Jogger.
During the study visits, participants will have blood draws before and after the study intervention, a vascular ultrasound with a Lumason contrast agent before and after the study intervention, and if able to safely complete, an CrCEST MRI of the lower leg.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
- Model description
- Aim 1 will have the 3 study interventions/study visits occur in random order. The interventions will be performed in primary mitochondrial disease patients and healthy controls Aim 2 will have all participants complete the study interventions/visits in the same order. Patients will be from the PICU
| Arm | Type | Receives |
|---|---|---|
| Aim 1: Primary Mitochondrial Disease Patients | Experimental | Diagnostic Test: Cardiopulmonary Exercise Testing, Device: pGz Bed, Device: Gentle Jogger, Drug: Lumason® contrast agentThe participant has the interventions/study visits occur in a random order: CPET pGz administration through pGz Bed pGz administration through Gentle Jogger |
| Aim 1: Healthy Controls | Experimental | Diagnostic Test: Cardiopulmonary Exercise Testing, Device: pGz Bed, Device: Gentle Jogger, Drug: Lumason® contrast agentThe participant has the interventions/study visits occur in a random order: pGz administration through Gentle Jogger CPET pGz administration through pGz Bed |
| Aim 2: PICU Patients | Experimental | Device: Gentle Jogger, Device: Exercise Pedal, Drug: Lumason® contrast agentAll participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger |
Who may qualify
- Ages
- 10 to 60 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (59 listed)
- 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
Key exclusion criteria (24 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cardiopulmonary Exercise TestingTesting 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 | Diagnostic test | Aim 1: Healthy Controls, Aim 1: Primary Mitochondrial Disease Patients |
| pGz BedParticipants will lay down on a passive exercise (pGz) bed for 45 minutes during which the bed will administer passive exercise through periodic acceleration | Device | Aim 1: Healthy Controls, Aim 1: Primary Mitochondrial Disease Patients |
| Gentle JoggerParticipants 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 | Aim 1: Healthy Controls, Aim 1: Primary Mitochondrial Disease Patients, Aim 2: PICU Patients |
| Exercise PedalParticipants will exercise while laying down for 20 minutes with an exercise pedal that attaches to the bed | Device | Aim 2: PICU Patients |
| Lumason® contrast agentContrast 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. | Drug | Aim 1: Healthy Controls, Aim 1: Primary Mitochondrial Disease Patients, Aim 2: PICU Patients |
Primary outcome
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
Maximal Oxygen Consumption will be measured only during CPET
Aim 2: Arterial-Venous (A-V) O2 difference
Time frame: A 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 Consumption
Time frame: 1 hour per study intervention
Measured During the study interventions
9 secondary outcomes
Aim 1 and 2: A/B ratio measurement through EKG or Plethsymography
Time frame: 1 hour per study intervention
Aim 1 and 2: Heart Rate
Time frame: 1 hour per study intervention
Aim 1 and 2: OXPHOS Capacity
Time frame: Aim 1 subjects will complete 2 1 hour MRIs, Aim 2 Subjects will complete 1 1-hour MRI
Aim 1 and 2: Plasma Lactate Levels
Time frame: 15 minute blood draws that occur pre and and immediately after each study intervention
Aim 1 and 2: Vasodilatation
Time frame: 30 minute ultrasound that occurs pre and immediately after each study intervention
NO release
Time frame: 15 minutes, before and immediately after each study intervention
Plasma Nom Levels
Time frame: 15 minute blood draws that occur pre and immediately after each study intervention
Post-operative Patient Satisfaction Survey
Time frame: 15 minutes, taken after each study intervention
Borg Scale
Time frame: 15 minutes, taken after each study intervention
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Nov 2025.
- Background: PubMed 33006749Sackner MA, Lopez JR, Banderas V, Adams JA. Can Physical Activity While Sedentary Produce Health Benefits? A Single-Arm Randomized Trial. Sports Med Open. 2020 Oct 2;6(1):47. doi: 10.1186/s40798-020-00278-3.
- Background: PubMed 30350153Sackner MA, Patel S, Adams JA. Changes of blood pressure following initiation of physical inactivity and after external addition of pulses to circulation. Eur J Appl Physiol. 2019 Jan;119(1):201-211. doi: 10.1007/s00421-018-4016-7. Epub 2018 Oct 22.
- Background: PubMed 32926876Burstein DS, McBride MG, Min J, Paridon AA, Perelman S, Huffman EM, O'Malley S, Del Grosso J, Groepenhoff H, Paridon SM, Brothers JA. Normative Values for Cardiopulmonary Exercise Stress Testing Using Ramp Cycle Ergometry in Children and Adolescents. J Pediatr. 2021 Feb;229:61-69.e5. doi: 10.1016/j.jpeds.2020.09.018. Epub 2020 Sep 11.
- Background: PubMed 12538407Taivassalo T, Jensen TD, Kennaway N, DiMauro S, Vissing J, Haller RG. The spectrum of exercise tolerance in mitochondrial myopathies: a study of 40 patients. Brain. 2003 Feb;126(Pt 2):413-23. doi: 10.1093/brain/awg028.
- Background: PubMed 22239882Tarnopolsky M. Exercise testing in metabolic myopathies. Phys Med Rehabil Clin N Am. 2012 Feb;23(1):173-86, xii. doi: 10.1016/j.pmr.2011.11.011. Epub 2011 Dec 11.
- Background: PubMed 16120411Tarnopolsky M. Exercise testing as a diagnostic entity in mitochondrial myopathies. Mitochondrion. 2004 Sep;4(5-6):529-42. doi: 10.1016/j.mito.2004.07.011. Epub 2004 Sep 30.
- Background: PubMed 16331135Taivassalo T, Haller RG. Exercise and training in mitochondrial myopathies. Med Sci Sports Exerc. 2005 Dec;37(12):2094-101. doi: 10.1249/01.mss.0000177446.97671.2a.
- Background: PubMed 24661939Adams JA, Uryash A, Bassuk J, Sackner MA, Kurlansky P. Biological basis of neuroprotection and neurotherapeutic effects of Whole Body Periodic Acceleration (pGz). Med Hypotheses. 2014 Jun;82(6):681-7. doi: 10.1016/j.mehy.2014.02.031. Epub 2014 Mar 12.
- Background: PubMed 11588467Adams JA, Mangino MJ, Bassuk J, Kurlansky P, Sackner MA. Regional blood flow during periodic acceleration. Crit Care Med. 2001 Oct;29(10):1983-8. doi: 10.1097/00003246-200110000-00022.
- BackgroundM. Fujita et al., "Periodic acceleration enhances release of nitric oxide in healthy adults," Int. J. Angiol., vol. 14, no. 1, pp. 11-14, Feb. 2005, doi: 10.1007/s00547-005-2013-2.
- Background: PubMed 26133377Uryash A, Bassuk J, Kurlansky P, Altamirano F, Lopez JR, Adams JA. Antioxidant Properties of Whole Body Periodic Acceleration (pGz). PLoS One. 2015 Jul 2;10(7):e0131392. doi: 10.1371/journal.pone.0131392. eCollection 2015.
- Background: PubMed 25807532Uryash A, Bassuk J, Kurlansky P, Altamirano F, Lopez JR, Adams JA. Non-invasive technology that improves cardiac function after experimental myocardial infarction: Whole Body Periodic Acceleration (pGz). PLoS One. 2015 Mar 25;10(3):e0121069. doi: 10.1371/journal.pone.0121069. eCollection 2015.
Related trials for Mitochondrial Myopathies
RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
RecruitingPhase 2
Deoxynucleosides Pyrimidines as Treatment for Mitochondrial Depletion Syndrome
Mitochondrial Diseases, Mitochondrial Encephalomyopathy
RecruitingPhase 3
KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases
Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD)
Active, not recruitingPhase 2
An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)
Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)
RecruitingPhase 1
Validation of Nanosensor Oxygen Measurement
MItochondrial Myopathies, Mitochondrial Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Zuela Zolkipli-Cunningham, MBChB, MRCP principal investigator
Attending Physician
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cardiopulmonary Exercise Testing, pGz Bed and Gentle Jogger in people with Mitochondrial Myopathies and Mitochondrial Diseases, run by Children's Hospital of Philadelphia at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 59 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 10 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Katelynn Stanley, BS (215-426-0225, stanleyk2@chop.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05569122, which also lists 20 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Nov 2025.