Measuring oxygen levels in muscle tissue using a nanosensor

The Validation of Minimally Invasive Oxygen Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle

This study is testing a tiny sensor to see if it can accurately measure oxygen levels in the muscles of people with mitochondrial myopathy and healthy volunteers during exercise.

Quick facts

• Phase: PHASE1
• Study type: Interventional
• Enrollment: 96 (estimated)
• Ages: 18 Years to 65 Years
• Sex: All
• Sponsor: Children's Hospital of Philadelphia (other)
• Locations: 1 site (Philadelphia, Pennsylvania)
• Trial ID: NCT06819683 on ClinicalTrials.gov

What this trial studies

This clinical trial aims to validate the use of an electrochemical nanosensor for measuring oxygen levels in the muscle tissue of patients with mitochondrial myopathy (MM) and healthy volunteers. Participants will undergo a handgrip exercise while the nanosensor is placed in their forearm to assess muscle oxygen levels and pain. The study seeks to establish reliable biomarkers that can reflect the severity and progression of MM, which is crucial for evaluating new treatments. The data collected may enhance future diagnostic and therapeutic strategies for mitochondrial diseases.

Who should consider this trial

Good fit: Ideal candidates include adults aged 18 to 65 with genetically-confirmed mitochondrial myopathy or healthy volunteers meeting specific inclusion criteria.

Not a fit: Patients outside the age range of 18 to 65 or those without a confirmed diagnosis of mitochondrial myopathy may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved diagnostic tools and treatment strategies for patients with mitochondrial diseases.

How similar studies have performed: While there have been challenges in previous studies regarding biomarkers for mitochondrial diseases, this approach using a nanosensor is novel and has not been extensively tested.

Eligibility criteria

Inclusion Criteria:

* Inclusion Criteria for Healthy Controls

  1. Males and females, between the ages of 18 and 65 years, inclusive
  2. Provide informed consent for study participation; able to understand and complete the protocol
  3. Able to ambulate independently
  4. Able to perform bicycle ergometry

Inclusion Criteria for Mitochondrial Myopathy (MM) Cases

1. Males and females, between the ages of 18 and 65 years, inclusive
2. Provide informed consent for study participation; able to understand and complete the protocol
3. Genetically-confirmed MM as defined by a diagnosis of primary mitochondrial disease (PMD) with predominant symptoms of myopathy as expressed by exercise intolerance and muscle weakness and fatigue.
4. 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)
5. Able to ambulate independently
6. Able to perform bicycle ergometry

Exclusion Criteria:

Subjects will be excluded if any of the following apply:

1. Unable to provide informed consent and complete all study procedures, including ergometry
2. Non-ambulatory or unable to ambulate independently
3. Pregnant
4. Within 1 month of a recent hospital admission due to acute illness
5. Have severe cardiac disease as defined by an ejection fraction of less than 35% and New York Heart Association Functional Classification Class III; or severe pulmonary disease as defined by the need for supplemental O2 therapy or daytime ventilatory support
6. Have a tracheostomy
7. Have a known bleeding disorder and/or family history (first-degree relative) with a known bleeding disorder
8. Daily intake of aspirin or any other anti-platelet therapy which cannot be temporarily discontinued for medical reasons
9. a) Have known or suspected congenital or acquired immune deficiency; b) concurrent use of immunosuppressive drugs, including corticosteroids; c) past history of recurrent (more than 6 times per year) severe (required hospitalization) skin or soft tissue infections; d) history of infection or delayed wound healing after surgery or biopsy; e) known history of neutropenia with absolute neutrophil count less than 500/mm3
10. Undergo chronic steroid treatment as defined by daily oral intake (for more than 1 month) or have existing untreated endocrinopathies, such as hypothyroidism that caused acquired myopathy
11. Prone to hypertrophic scars and keloids
12. Have any other known inherited myopathy, such as Duchenne muscular dystrophy or congenital myopathy
13. Known allergy to lidocaine
14. Have a cognitive impairment that may prevent the ability to complete study procedures
15. Unable to comply with the requirements of the study protocol and/or unsuitable for the study for any reason, in the opinion of the principal investigator
16. Individuals from vulnerable populations (e.g., prisoners/detainees)
17. Participants who are unable to speak and/or read English (as participants will be required to be proficient to complete study procedures)
18. Employed by the U.S. Department of Defense, including U.S. military personnel

Where this trial is running

Philadelphia, Pennsylvania

• The Children's Hospital of Philadelphia — Philadelphia, Pennsylvania, United States (RECRUITING)

Study contacts

• Principal investigator: Zarazuela Zolkipli-Cunningham, MBChB, MRCP — Children's Hospital of Philadelphia
• Study coordinator: Zarazuela Zolkipli-Cunningham, MBChB, MRCP

Conditions: MItochondrial Myopathies, Mitochondrial Disease