24 Participants Needed

Oxygen Nanosensor for Mitochondrial Myopathy

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Overseen BySara Nguyen
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot participate if you are on daily aspirin or anti-platelet therapy that can't be stopped temporarily, or if you are on chronic steroid treatment.

What data supports the effectiveness of the treatment Oxygen Nanosensor for Mitochondrial Myopathy?

Research shows that oxygen therapy can significantly improve muscle metabolism in patients with mitochondrial myopathy, suggesting that treatments enhancing oxygen delivery, like the Oxygen Nanosensor, could be beneficial.12345

Is the Oxygen Nanosensor safe for use in humans?

The research on oxygen sensors, including those tested in rabbits, shows that they are stable and functional for long periods, suggesting they are generally safe for monitoring oxygen levels in tissues. However, specific safety data for humans is not provided in the available studies.678910

How does the Oxygen Nanosensor treatment for Mitochondrial Myopathy differ from other treatments?

The Oxygen Nanosensor treatment is unique because it uses a genetically encoded sensor to measure oxygen levels inside muscle cells in real-time, helping to understand and manage the oxygen dynamics in mitochondrial myopathy, a condition with no standard treatment. This approach is different from traditional methods as it provides detailed insights into how oxygen is used by cells, which is crucial for addressing the muscle metabolism issues caused by mitochondrial DNA mutations.2471112

What is the purpose of this trial?

Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomarkers in mitochondrial myopathy (MM). There is currently a growing number of potential new treatments to be tested through MM clinical intervention trials, which has created a pressing need for quantitative biomarkers that reliably reflect MM disease severity, progression, and therapeutic response.The purpose of the study is to measure the efficacy of an electrochemical oxygen nanosensor to measure in vivo mitochondrial function in human muscle tissue, and its ability to discriminate MM patients from healthy volunteers. The data and results from this nanosensor study may contribute to current and future research, including improved diagnostic and therapeutic approaches for patients with mitochondrial disease.

Research Team

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Zarazuela Zolkipli-Cunningham

Principal Investigator

Children's Hospital of Philadelphia

Eligibility Criteria

This trial is for adults aged 18-65 with genetically confirmed mitochondrial myopathy, characterized by exercise intolerance and muscle weakness. Healthy volunteers must be able to walk, do bike exercises, and give informed consent. People are excluded if they don't meet these criteria or can't follow the study protocol.

Inclusion Criteria

I understand the study and agree to participate.
Previously enrolled (or will enroll) in Children's Hospital of Philadelphia (CHOP) Institutional Review Board (IRB) study #08-006177 (Falk, PI) or CHOP IRB #16-013364 (Zolkipli, PI)
I have a genetic condition causing muscle weakness, especially after exercise.
See 4 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Measurement

Nanosensor muscle oxygen measurement in exercised forearm muscle during handgrip exercise

1 week
1 visit (in-person)

Reproducibility Assessment

Repeat nanosensor measurements to assess reproducibility

1-4 weeks
1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after measurements

4 weeks

Treatment Details

Interventions

  • Oxygen Nanosensor
Trial Overview The trial is testing an electrochemical oxygen nanosensor designed to measure mitochondrial function in human muscle tissue. It aims to distinguish between MM patients and healthy individuals, potentially aiding future diagnosis and treatment of mitochondrial diseases.
Participant Groups
2Treatment groups
Experimental Treatment
Group I: Healthy ControlsExperimental Treatment1 Intervention
Adult healthy volunteers will be individually matched with corresponding MM cases based on age, biological sex, and body mass index.
Group II: Affected MM CasesExperimental Treatment1 Intervention
Key eligibility criteria for MM cases includes physically-capable adults (male and females, ages 18 to 65 years, inclusive) with genetically-confirmed MM with predominant symptoms of myopathy as expressed by exercise intolerance and muscle weakness and fatigue.

Find a Clinic Near You

Who Is Running the Clinical Trial?

Children's Hospital of Philadelphia

Lead Sponsor

Trials
749
Recruited
11,400,000+

National Institutes of Health (NIH)

Collaborator

Trials
2,896
Recruited
8,053,000+

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator

Trials
1,403
Recruited
655,000+

Findings from Research

Oxygen therapy significantly improved the inferred maximal ATP synthesis rate by 33% in patients with mitochondrial myopathy (MM), indicating enhanced muscle metabolism during exercise.
In contrast, oxygen supplementation had only a minimal effect (5% improvement) on ATP synthesis in healthy controls, suggesting that patients with MM may uniquely benefit from therapies that increase oxygen availability.
Supplemental oxygen and muscle metabolism in mitochondrial myopathy patients.Trenell, MI., Sue, CM., Thompson, CH., et al.[2022]
A noninvasive tissue oxymeter using near infrared light revealed extensive tissue oxygenation in patients with mitochondrial myopathy, linking oxygen levels to the severity of mitochondrial DNA mutations.
The study suggests that the degree of oxygenation correlates with defects in the oxidative phosphorylation system, which may contribute to the progression of mitochondrial DNA mutations, highlighting the potential for this diagnostic method in managing older patients.
Extensive tissue oxygenation associated with mitochondrial DNA mutations.Ozawa, T., Sahashi, K., Nakase, Y., et al.[2004]
In a study involving four normal controls and four patients with mitochondrial disorders, normal individuals maintained stable oxygen levels during exercise, while patients exhibited abnormal oxygenation and slow recovery, indicating impaired oxidative phosphorylation.
The study suggests that noninvasive tissue oximetry can effectively assess the severity of mitochondrial myopathy and exercise intolerance by measuring the imbalance between oxygen delivery and utilization.
Measurement of tissue oxygen consumption in patients with mitochondrial myopathy by noninvasive tissue oximetry.Abe, K., Matsuo, Y., Kadekawa, J., et al.[2019]

References

Supplemental oxygen and muscle metabolism in mitochondrial myopathy patients. [2022]
Extensive tissue oxygenation associated with mitochondrial DNA mutations. [2004]
Measurement of tissue oxygen consumption in patients with mitochondrial myopathy by noninvasive tissue oximetry. [2019]
High resolution spatial investigation of intracellular oxygen in muscle cells. [2023]
Non-invasive assessment of muscle oxygenation may aid in optimising transfusion threshold decisions in ambulatory paediatric patients. [2019]
in vivo Monitoring with micro-implantable hypoxia sensor based on tissue acidosis. [2021]
Oxygen microsensor and its application to single cells and mouse pancreatic islets. [2019]
Linking nanomaterial-induced mitochondrial dysfunction to existing adverse outcome pathways for chemicals. [2023]
Mass transfer and gas-phase calibration of implanted oxygen sensors. [2006]
The oxygen sensitivity of a multipoint antimony electrode for tissue pH measurements. A study of the sensitivity for in vivo PO2 variations below 6 kPa. [2019]
11.United Statespubmed.ncbi.nlm.nih.gov
A Chemiresistor Sensor Based on Azo-Polymer and Graphene for Real-Time Monitoring of Mitochondrial Oxygen Consumption. [2020]
12.United Statespubmed.ncbi.nlm.nih.gov
An electrochemical biosensor based on gold microspheres and nanoporous gold for real-time detection of superoxide anion in skeletal muscle tissue. [2020]
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