Oxygen Nanosensor for Mitochondrial Myopathy
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?
Is the Oxygen Nanosensor safe for use in humans?
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
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
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Measurement
Nanosensor muscle oxygen measurement in exercised forearm muscle during handgrip exercise
Reproducibility Assessment
Repeat nanosensor measurements to assess reproducibility
Follow-up
Participants are monitored for safety and effectiveness after measurements
Treatment Details
Interventions
- Oxygen Nanosensor
Find a Clinic Near You
Who Is Running the Clinical Trial?
Children's Hospital of Philadelphia
Lead Sponsor
National Institutes of Health (NIH)
Collaborator
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator