[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100482983":3},{"organization":4,"armGroups":7,"interventions":26,"overallOfficials":51,"centralContacts":56,"locations":66,"responsibleParty":80,"collaborators":82,"id":86,"slug":87,"hasResults":88,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":32,"eligibilityCriteria":92,"healthyVolunteers":93,"sex":94,"minAge":95,"maxAge":96,"enrollmentInfo":97,"targetDuration":32,"studyType":100,"phases":101,"briefSummary":103,"conditions":104,"keywords":107,"overallStatus":68,"whyStopped":32,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":114,"completionDateStruct":116,"leadSponsor":118,"locationsCount":119},{"fullName":5,"class":6},"Children's Hospital of Philadelphia","OTHER",[8,17,21],{"label":9,"type":10,"description":11,"interventionNames":12},"Aim 1: Primary Mitochondrial Disease Patients","EXPERIMENTAL","The participant has the interventions\u002Fstudy visits occur in a random order:\n\nCPET pGz administration through pGz Bed pGz administration through Gentle Jogger",[13,14,15,16],"Diagnostic Test: Cardiopulmonary Exercise Testing","Device: pGz Bed","Device: Gentle Jogger","Drug: Lumason® contrast agent",{"label":18,"type":10,"description":19,"interventionNames":20},"Aim 1: Healthy Controls","The participant has the interventions\u002Fstudy visits occur in a random order:\n\npGz administration through Gentle Jogger CPET pGz administration through pGz Bed",[13,14,15,16],{"label":22,"type":10,"description":23,"interventionNames":24},"Aim 2: PICU Patients","All participants in Aim 2 will have the interventions\u002Fstudy visits occur in the same order: Exercise Pedal and Gentle Jogger",[15,25,16],"Device: Exercise Pedal",[27,33,38,42,46],{"type":28,"name":29,"description":30,"armGroupLabels":31,"otherNames":32},"DIAGNOSTIC_TEST","Cardiopulmonary 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",[18,9],null,{"type":34,"name":35,"description":36,"armGroupLabels":37,"otherNames":32},"DEVICE","pGz 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",[18,9],{"type":34,"name":39,"description":40,"armGroupLabels":41,"otherNames":32},"Gentle 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)",[18,9,22],{"type":34,"name":43,"description":44,"armGroupLabels":45,"otherNames":32},"Exercise Pedal","Participants will exercise while laying down for 20 minutes with an exercise pedal that attaches to the bed",[22],{"type":47,"name":48,"description":49,"armGroupLabels":50,"otherNames":32},"DRUG","Lumason® 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.",[18,9,22],[52],{"name":53,"affiliation":54,"role":55},"Zuela Zolkipli-Cunningham, MBChB, MRCP","Attending Physician","PRINCIPAL_INVESTIGATOR",[57,62],{"name":58,"role":59,"phone":60,"phoneExt":32,"email":61},"Katelynn Stanley, BS","CONTACT","215-426-0225","stanleyk2@chop.edu",{"name":63,"role":59,"phone":64,"phoneExt":32,"email":65},"Daniel McGinn, MSGC","215-590-1000","mcginnde@chop.edu",[67],{"facility":5,"status":68,"city":69,"state":70,"zip":71,"country":72,"countryCode":73,"cosmosGeoPoint":74,"geoPoint":79,"contacts":32},"RECRUITING","Philadelphia","Pennsylvania","19104","United States","US",{"type":75,"coordinates":76},"Point",[77,78],-75.16362,39.95238,{"lat":78,"lon":77},{"type":81,"investigatorFullName":32,"investigatorTitle":32,"investigatorAffiliation":32,"oldNameTitle":32,"oldOrganization":32},"SPONSOR",[83],{"name":84,"class":85},"United States Department of Defense","FED","100482983","phase-1-applying-pgz-in-mitochondrial-disease-100482983",false,"NCT05569122","Applying pGz in Mitochondrial Disease","The Utility of pGz in Primary Mitochondrial Disorders","Aim 1 Enrollment Criteria Inclusion Criteria for Healthy Controls\n\n* Males or females, 10 years to 60 years, with a minimum height for participation of 135 cm\n* Ambulatory and able to complete routine clinical exercise testing\n* Willing and able to complete all study procedures\n* For individuals under the age of 18, parental\u002Fguardian permission (informed consent) and as appropriate, child assent\n* For individuals over the age of 18 the ability to provide informed consent\n\nInclusion Criteria for PMD Patients\n\n* Males or females, 10 years to 60 years, with a minimum height for participation of 135 cm\n* Ambulatory and able to complete routine clinical exercise testing\n* Willing and able to complete all study procedures\n* 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\n* Parental\u002Fguardian permission (informed consent) and as appropriate, child assent\n\nExclusion Criteria for All Aim 1 Participants General Exclusion Criteria\n\n* Tracheostomy\n* Non-ambulatory\n* Unable to complete routine exercise testing\n* Diagnosed with or have symptoms of vertigo\n* Within 1 month of a recent hospital admission for acute illness\n* Severe co-existing cardiac or pulmonary disease\n* Cognitive impairment that may preclude ability to comply with study procedures\n* Pregnant or lactating females\n* Active alcohol and\u002For substance abuse\n* At the discretion of the principal investigator (PI), any medical condition that will interfere with or prevent the safe completion of the study\n* Parents\u002Fguardians or subjects who, in the opinion of the Investigator, may be non-compliant with study schedules or procedures\n* Use of investigational agent(s) within 4 weeks\n* Individuals who are employed by the U.S. Department of Defense, including U.S military personal\n* Patients with biliary atresia with asplenia or polysplenia.\n* Patients with prior liver transplant.\n* Patients with cystic fibrosis.\n* Patients with chronic lung disease.\n* Patients with portal vein thrombosis, cavernous transformation of the portal vein or absent portal vein.\n* Patients with significant heart disease or severe congenital heart disease.\n* 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\n* Any history of intraocular injury or fragment in or around the orbit that cannot be cleared through radiologic evaluation\n* Any history of bullet, shrapnel, or stabbing wounds that cannot be cleared through radiologic evaluation\n* Past or current employment involving (or exposure to) a metal grinder (e.g., at a construction worksite)\n* Claustrophobia or any known medical conditions which can be exacerbated by stress, anxiety, or panic attacks triggered by enclosed spaces\n* Inability to lie flat in an MRI scanner for up to 45 minutes\n* Unable to perform sub-maximal ankle dorsiflexion leg exercise during the MRI study\n\nAim 2 Enrollment Criteria Inclusion Criteria for PICU PMD Non-Ambulatory Patients\n\n* Males or females ages 10 to 23 years (children and adults)\n* Non-ambulatory\n* Genetically confirmed mtDNA-PMD\n* Cooperative and capable of following research procedures\n* Have cognitive ability to enable cooperation with study procedures\n* Admitted to the PICU with an anticipated length of stay for \\>24 hours\n* Willing and able to complete all study procedures\n* For individuals under the age of 18, parental\u002Fguardian permission (informed consent) and as appropriate, child assent\n* For individuals over the age of 18 the ability to provide informed consent\n\nInclusion Criteria for PICU non-PMD neuromuscular diagnosis\n\n* Males or females ages 10 to 23 years (children and adults)\n* Non-ambulatory\n* Genetically confirmed non-PMD neuromuscular diagnosis\n* Cooperative and capable of following research procedures\n* Have cognitive ability to enable cooperation with study procedures\n* Admitted to the PICU with an anticipated length of stay for \\>24 hours\n* For individuals under the age of 18, parental\u002Fguardian permission (informed consent) and as appropriate, child assent\n* For individuals over the age of 18 the ability to provide informed consent\n\nInclusion Criteria for all other PICU Participants\n\n* Males or females ages 10 to 23 years (children and adults)\n* Non-ambulatory\n* No known genetic diagnosis with healthy pre-morbid status, admitted to PICU\n* Cooperative and capable of following research procedures\n* Have cognitive ability to enable cooperation with study procedures\n* Admitted to the PICU with an anticipated length of stay for \\>24 hours\n* Willing and able to complete all study procedures\n* For individuals under the age of 18, parental\u002Fguardian permission (informed consent) and as appropriate, child assent\n* For individuals over the age of 18 the ability to provide informed consent\n\nExclusion Criteria for All Aim 2 Participants\n\n* Have cognitive impairment that may preclude ability to comply with study procedures\n* Have cardiorespiratory instability\n* Patients in whom are so sick that they will not be able to cooperate with the study procedures\n* Have clear contraindications to mobilization\n* Have fixed lower limb deformities\u002Fcontractures that would prohibit lower extremity exercise\n* Pregnant or lactating females\n* Active alcohol and\u002For substance abuse\n* At the discretion of the principal investigator (PI), any medical condition that will interfere with or prevent the safe completion of the study\n* Use of investigational agent(s) within 4 weeks\n* Individual who are employed by the U.S. Department of Defense, including U.S military personal\n* Patients with biliary atresia with asplenia or polysplenia.\n* Patients with prior liver transplant.\n* Patients with cystic fibrosis.\n* Patients with chronic lung disease.\n* Patients with portal vein thrombosis, cavernous transformation of the portal vein or absent portal vein.\n* Patients with significant heart disease or severe congenital heart disease.\n* 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\n\nExclusion Criteria Specific to study procedure: CrCEST MRI Scan:\n\n* Any history of intraocular injury or fragment in or around the orbit that cannot be cleared through radiologic evaluation\n* Any history of bullet, shrapnel, or stabbing wounds that cannot be cleared through radiologic evaluation\n* Past or current employment involving (or exposure to) a metal grinder (e.g., at a construction worksite)\n* Claustrophobia or any known medical conditions which can be exacerbated by stress, anxiety, or panic attacks triggered by enclosed spaces\n* Inability to lie flat in an MRI scanner for up to 45 minutes\n* Unable to perform sub-maximal ankle dorsiflexion leg exercise during the MRI study",true,"ALL","10 Years","60 Years",{"count":98,"type":99},90,"ESTIMATED","INTERVENTIONAL",[102],"PHASE1","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).",[105,106],"Mitochondrial Myopathies","Mitochondrial Diseases",[108,109],"Passive Exercise","periodic acceleration","2025-11-05",{"date":112,"type":113},"2025-11-10","ACTUAL",{"date":115,"type":113},"2023-03-22",{"date":117,"type":99},"2028-09",{"name":5,"class":6},1]