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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

Must not have

Patients with prior liver transplant

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

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

No Placebo-Only Group

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.

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.

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 below

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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.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have had a liver transplant in the past.

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I am allergic to Lumason or its ingredients.

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I cannot lie flat for 45 minutes in an MRI machine.

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I have unstable heart or lung conditions.

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I have cystic fibrosis.

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I have biliary atresia and either lack a spleen or have multiple spleens.

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I cannot move or be moved safely due to my health condition.

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I have a serious heart or lung condition.

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I have a blood clot or abnormality in the vein going to my liver.

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I have permanent leg issues that prevent me from doing leg exercises.

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I have been diagnosed with or am experiencing symptoms of vertigo.

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I have a long-term lung condition.

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I have a tracheostomy.

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I have had an eye injury or foreign body in my eye area that can't be checked with imaging.

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I cannot walk by myself.

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I have severe heart disease.

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

This trial's timeline: 3 weeks for screening, Varies for treatment, and aim 1 subjects will complete 2 1 hour mris, aim 2 subjects will complete 1 1-hour mri for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Aim 1 and 2: Oxygen Consumption

Mitochondrial Diseases

Aim 2: Arterial-Venous (A-V) O2 difference

Secondary study objectives

Electrocardiography

Aim 1 and 2: Heart Rate

Aim 1 and 2: OXPHOS Capacity

+6 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial 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