Actively Recruiting
Applying pGz in Mitochondrial Disease
Led by Children's Hospital of Philadelphia · Updated on 2025-11-10
90
Participants Needed
1
Research Sites
284 weeks
Total Duration
On this page
Sponsors
C
Children's Hospital of Philadelphia
Lead Sponsor
U
United States Department of Defense
Collaborating Sponsor
AI-Summary
What this Trial Is About
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).
CONDITIONS
Official Title
Applying pGz in Mitochondrial Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males or females aged 10 to 60 years with a minimum height of 135 cm
- Able to walk and complete routine clinical exercise testing
- Willing and able to complete all study procedures
- For those under 18, parental or guardian permission and child assent as appropriate
- For those 18 and older, able to provide informed consent
- Genetically confirmed primary mitochondrial disease with muscle symptoms for PMD patients
- For PICU patients: males or females aged 10 to 23 years, non-ambulatory, cooperative, cognitively able to follow procedures, admitted with anticipated stay over 24 hours
- For PICU non-PMD neuromuscular diagnosis: genetically confirmed diagnosis
- For all other PICU participants: no known genetic diagnosis and healthy before admission
You will not qualify if you...
- Presence of tracheostomy or non-ambulatory status (for Aim 1)
- Unable to complete routine exercise testing
- Diagnosis or symptoms of vertigo
- Recent hospital admission for acute illness within 1 month
- Severe heart or lung disease
- Cognitive impairment preventing study compliance
- Pregnant or lactating females
- Active alcohol or substance abuse
- Use of investigational agents within 4 weeks
- Employment with the U.S. Department of Defense or military
- History of biliary atresia with asplenia or polysplenia
- Prior liver transplant
- Cystic fibrosis or chronic lung disease
- Portal vein thrombosis or related conditions
- Significant or severe congenital heart disease
- Allergic reaction history to Lumason or its ingredients
- History of intraocular injury or metal fragments that cannot be cleared
- History of bullet, shrapnel, or stabbing wounds not cleared by imaging
- Exposure to metal grinding work
- Claustrophobia or anxiety triggered by enclosed spaces
- Inability to lie flat in MRI for up to 45 minutes
- Unable to perform specific leg exercise during MRI
- Cardiorespiratory instability or contraindications to mobilization (Aim 2)
- Fixed lower limb deformities preventing exercise (Aim 2)
- Patients too sick to cooperate with procedures (Aim 2)
AI-Screening
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Trial Site Locations
Total: 1 location
1
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
K
Katelynn Stanley, BS
CONTACT
D
Daniel McGinn, MSGC
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
DIAGNOSTIC
Number of Arms
3
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