Validation of Oxygen Nanosensor in Mitochondrial Myopathy

Trial statusRecruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-65
SponsorChildren's Hospital of Philadelphia

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

This trial accepts healthy volunteers

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

Design model

Parallel

Treatments tested in this trial

  • Nanosensor

    Device

    The purpose of the study is to test a device called a "nanosensor", which measures oxygen levels (a proxy of mitochondrial function) in muscle. The nanosensor has not been tested in humans nor has it been approved by the FDA. The study nanosensor measures 1.8 mm width x 6 mm length x 0.3 mm depth. Placement of the sterilized nanosensor involves a small incision for manual placement of the nanosensor in muscle forearm tissue.

Treatment groups

24 Participants
are divided into 2 treatment groups
Group A: Affected MM CasesOther 1 intervention
Group B: Healthy ControlsOther 1 intervention

Trial outcomes

Primary outcomes

1

Nanosensor-muscle oxygen (Torr) levels

Nanosensor measured muscle O2 levels at baseline, during handgrip exercise and after exercise

Time frame
60 minutes for data collection at each 2 study visits, up to 6 months.

Secondary outcomes

1

Pain and tolerability

Time frame
At each 2 study visits, up to 6 months.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Children's Hospital of Philadelphia

Lead sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Collaborator