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Behavioural Intervention
Exercise Therapy for Mitochondrial Disease
Phase 1
Recruiting
Led By Zuela Zolkipli-Cunningham, MBChB, MRCP
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
For individuals under the age of 18, parental/guardian permission (informed consent) and as appropriate, child assent
Ambulatory and able to complete routine clinical exercise testing
Timeline
Screening 3 weeks
Treatment Varies
Follow Up aim 1 subjects will complete 2 1 hour mris, aim 2 subjects will complete 1 1-hour mri
Awards & highlights
Study Summary
This trial will study the effects of exercise in patients with primary mitochondrial disease and healthy volunteers, as well as in patients admitted to the Pediatric Intensive Care Unit.
Who is the study for?
This trial is for males and females aged 10-60 with genetically confirmed mitochondrial myopathy, able to perform clinical exercise tests, and can follow study procedures. It excludes pregnant women, those allergic to Lumason®, individuals with severe diseases or conditions that prevent safe participation, recent investigational drug users, non-ambulatory persons, and certain government employees.Check my eligibility
What is being tested?
The study examines the effects of conventional (Cardiopulmonary Exercise Testing or pedal exercise) versus passive exercises (pGz Bed) on patients with primary mitochondrial disease compared to healthy volunteers. Part of the research includes critically ill children in a hospital's intensive care unit.See study design
What are the potential side effects?
Potential side effects may include reactions to the Lumason® contrast agent used during testing such as allergies or discomfort at injection site. The physical exercises might cause fatigue or muscle soreness.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
My parents or guardians have agreed to my participation in this study.
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I can walk and complete basic exercise tests.
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I am between 10 and 60 years old and at least 135 cm tall.
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I have a genetic condition causing muscle weakness, especially after exercise.
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I can understand and follow the study's procedures.
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I am expected to stay in the pediatric intensive care unit for more than a day.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ aim 1 subjects will complete 2 1 hour mris, aim 2 subjects will complete 1 1-hour mri
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~aim 1 subjects will complete 2 1 hour mris, aim 2 subjects will complete 1 1-hour mri
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Aim 1 and 2: Oxygen Consumption
Mitochondrial Diseases
Aim 2: Arterial-Venous (A-V) O2 difference
Secondary outcome measures
Electrocardiography
Aim 1 and 2: Heart Rate
Aim 1 and 2: OXPHOS Capacity
+6 moreTrial Design
3Treatment groups
Experimental Treatment
Group I: Aim 2: PICU PatientsExperimental Treatment3 Interventions
All participants in Aim 2 will have the interventions/study visits occur in the same order: Exercise Pedal and Gentle Jogger
Group II: Aim 1: Primary Mitochondrial Disease PatientsExperimental Treatment4 Interventions
The participant has the interventions/study visits occur in a random order:
CPET pGz administration through pGz Bed pGz administration through Gentle Jogger
Group III: Aim 1: Healthy ControlsExperimental Treatment4 Interventions
The participant has the interventions/study visits occur in a random order:
pGz administration through Gentle Jogger CPET pGz administration through pGz Bed
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Gentle Jogger
2018
N/A
~50
Cardiopulmonary Exercise Testing
2013
Completed Phase 4
~610
Find a Location
Logistics
Participation is compensated
You will be compensated for participating in this trial.
Who is running the clinical trial?
United States Department of DefenseFED
865 Previous Clinical Trials
327,609 Total Patients Enrolled
Children's Hospital of PhiladelphiaLead Sponsor
708 Previous Clinical Trials
8,582,930 Total Patients Enrolled
Zuela Zolkipli-Cunningham, MBChB, MRCPPrincipal InvestigatorAttending Physician
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My parents or guardians have agreed to my participation in this study.I can walk and complete basic exercise tests.You have worked with a metal grinder in the past or are currently working with one, for example, at a construction site.I have had a liver transplant in the past.I am between 10 and 60 years old and at least 135 cm tall.I do not have claustrophobia or conditions worsened by stress in tight spaces.I am allergic to Lumason or its ingredients.I have a genetic condition causing muscle weakness, especially after exercise.You cannot do the regular exercise tests.I was hospitalized for a sudden illness within the last month.I cannot lie flat for 45 minutes in an MRI machine.I have unstable heart or lung conditions.You are currently using alcohol or drugs excessively.You have been wounded by a bullet, shrapnel, or stabbing and the wound cannot be checked by X-ray or other imaging tests.You cannot do a specific leg exercise during the MRI study.I have cystic fibrosis.I have biliary atresia and either lack a spleen or have multiple spleens.I cannot move or be moved safely due to my health condition.I am too ill to follow the study's procedures.I have a serious heart or lung condition.I have a blood clot or abnormality in the vein going to my liver.I can understand and follow the study's procedures.I have permanent leg issues that prevent me from doing leg exercises.I am over 18 and can give my consent.I am willing and able to follow all the study's requirements.I am willing and able to follow study instructions.I am expected to stay in the pediatric intensive care unit for more than a day.I have been diagnosed with or am experiencing symptoms of vertigo.I have a long-term lung condition.I have a tracheostomy.I have had an eye injury or foreign body in my eye area that can't be checked with imaging.I cannot walk by myself.I have severe heart disease.
Research Study Groups:
This trial has the following groups:- Group 1: Aim 1: Primary Mitochondrial Disease Patients
- Group 2: Aim 1: Healthy Controls
- Group 3: Aim 2: PICU Patients
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What fundamental goals does this study seek to accomplish?
"The primary goals of this Cardiopulmonary Exercise Test, which will take one hour to conduct, are twofold: oxygen consumption. Additionally, plasma nom levels (measured through venous blood draws), plasma lactate levels (also measured via venous blood draws), and the participant's tolerance to pGz compared to CPET as scored on a Borg Scale will be assessed for secondary outcomes."
Answered by AI
Who are the eligible participants for this experiment?
"This clinical study is recruiting 90 people with luft disease, aged between 10 and 60. In addition to this demographic criteria, participants must also demonstrate the ability to complete all necessary tests, provide informed consent (if applicable) and gain parental/guardian permission for minors. Those diagnosed with PMD need a confirmed genetic diagnosis of mitochondrial myopathy as well."
Answered by AI
Is the enrollment of participants limited to those aged 45 or younger?
"To be eligible for this experiment, applicants must fall between ages 10 and 60. There are 9 studies designed for participants younger than 18 years old, and an additional 18 experiments tailored to seniors over the age of 65."
Answered by AI
Has the FDA sanctioned Aim 2: PICU Patients?
"Our assessment of the safety profile for Aim 2: PICU Patients is a 1, as this trial is only in its first phase and there is limited data supporting both efficacy and safe usage."
Answered by AI
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