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New treatment option for Mitochondrial Myopathies
Official title Applying pGz in Mitochondrial Disease
ClinicalTrials.gov ID: NCT05569122
What this study is testing
What is Lumason® contrast agent?
Lumason® contrast agent is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for mitochondrial myopathies.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 10 to 60. Healthy volunteers may be eligible.
You may be able to join if
- Aim 1 Enrollment 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
You likely can't join if
- 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
See the full eligibility criteria
Who can join
- Aim 1 Enrollment 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 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...
- Parental/guardian permission (informed consent) and as appropriate, child assent
What rules you out
- 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
- 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 people 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...
- 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 Inclusion Criteria for PICU non-PMD neuromuscular diagnosis
- 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 Inclusion Criteria for all other PICU Participants
- 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 Criteria for All Aim 2 Participants
- 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...
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Philadelphia, Pennsylvania, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 10 years to 60 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Philadelphia, Pennsylvania, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.