Validation of Nanosensor Oxygen Measurement
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 volunteersQualifiers
Inclusion Criteria for Healthy Controls
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
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
DeviceThe 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
70 Participants
are divided into 2 treatment groupsGroup A: Affected Mitochondrial Myopathy (MM) CasesExperimental treatment 1 intervention
Group B: Healthy ControlsActive comparator 1 intervention
Trial outcomes
Primary outcomes
1
Nanosensor-muscle oxygen (Torr) levels during handgrip exercises
Nanosensor-muscle oxygen (Torr) levels will be measure in the (dominant) forearm muscle during handgrip exercises and correlated to the standard measure of venous oxygenation (VO2) levels using standard clinical indirect calorimetry as a measure of tissue oxygen consumption. This data will be reported as the Torr value at each time point during measurement.
Time frame
before, during and after handgrip exercise up to 4 hours or up to 40 days after the screening visit2
Nanosensor-muscle oxygen (Torr) levels post Cardiopulmonary Exercise Testing (CPET)
Nanosensor-muscle oxygen (Torr) levels before cardiopulmonary exercise testing (CPET) will be measured in the (dominant) forearm muscle by comparing exercised muscle oxygen levels to the venous oxygen (VO2max) levels recorded during the CPET exercise. This data will be reported as the Torr value at each time point during measurement.
Time frame
After CPET exercise up to 1-hourSecondary outcomes
1
Self-reported pain level assessment
After the exercise procedure, participants will self-report the level of pain/discomfort with the validated Wong-Baker Faces Pain Rating Scale and Numerical Rating Scale. The Wong-Baker Faces Pain Rating Scale is a tool used to assess pain levels in young children by presenting using a combination of faces, numbers, and words to help the participant effectively communicate the severity of their physical pain. The scale includes a happy face indicating 0="no hurt" to a crying face signifying 10="hurts worst." The NRS is a pain screening tool, commonly used to assess pain severity at that moment in time using a 0-10 scale, with zero meaning "no pain" and 10 meaning "the worst pain imaginable." All subjects will respond to both surveys.
Time frame
up to 40 days after the screening visit2
Patient Satisfaction
Patient satisfaction will be measured by participants completing the Post-operative Patient Satisfaction Survey. The survey consists of one question to gauge self-reported insight on their overall experience. Responses note the experience was "Agreeable", "Neither pleasant nor unpleasant", "Slightly uncomfortable", "Disagreeable", or "Traumatic".
Time frame
After study procedures are completed (approximately day 40)3
Self-reported fatigue
After the exercise period, participants will self-report the level of fatigue as a measure of disease burden using the Modified Fatigue Impact Scale (MFIS). This scale includes 9 questions regarding subject's fatigue symptoms and how it impacts their life. Answers range from "Strongly Agree" to "Strongly Disagree" with 7 answer choices for each question.
Time frame
After study procedures are completed (approximately day 40)Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Children's Hospital of Philadelphia
Lead sponsor
United States Department of Defense
Collaborator
Source ClinicalTrials.gov