About 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.
Eligibility criteria
Qualifiers
Males and females, between the ages of 18 and 65 years, inclusive
Provide informed consent for study participation; able to understand and complete the protocol
Able to ambulate independently
Able to perform bicycle ergometry
Disqualifiers
Unable to provide informed consent and complete all study procedures, including ergometry
Non-ambulatory or unable to ambulate independently
Pregnant
Within 1 month of a recent hospital admission due to acute illness
Trial design
Treatments tested in this trial
- Nanosensor
Treatment groups
Sponsors and collaborators
Children's Hospital of Philadelphia
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
Collaborator