Validation of Nanosensor Oxygen Measurement

Last updated: February 10, 2025
Sponsor: Children's Hospital of Philadelphia
Overall Status: Active - Recruiting

Phase

1

Condition

Mitochondrial Diseases

Treatment

Nanosensor

Clinical Study ID

NCT06819683
22-019820
W81XWH2210590
  • Ages 18-65
  • All Genders
  • Accepts Healthy Volunteers

Study Summary

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

Inclusion

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 andcomplete 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 completethe 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 andmuscle 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

Exclusion Criteria:

Subjects will be excluded if any of the following apply:

  1. Unable to provide informed consent and complete all study procedures, includingergometry

  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% andNew York Heart Association Functional Classification Class III; or severe pulmonarydisease as defined by the need for supplemental O2 therapy or daytime ventilatorysupport

  6. Have a tracheostomy

  7. Have a known bleeding disorder and/or family history (first-degree relative) with aknown bleeding disorder

  8. Daily intake of aspirin or any other anti-platelet therapy which cannot betemporarily discontinued for medical reasons

  9. a) Have known or suspected congenital or acquired immune deficiency; b) concurrentuse of immunosuppressive drugs, including corticosteroids; c) past history ofrecurrent (more than 6 times per year) severe (required hospitalization) skin orsoft tissue infections; d) history of infection or delayed wound healing aftersurgery or biopsy; e) known history of neutropenia with absolute neutrophil countless than 500/mm3

  10. Undergo chronic steroid treatment as defined by daily oral intake (for more than 1month) or have existing untreated endocrinopathies, such as hypothyroidism thatcaused acquired myopathy

  11. Prone to hypertrophic scars and keloids

  12. Have any other known inherited myopathy, such as Duchenne muscular dystrophy orcongenital myopathy

  13. Known allergy to lidocaine

  14. Have a cognitive impairment that may prevent the ability to complete studyprocedures

  15. Unable to comply with the requirements of the study protocol and/or unsuitable forthe 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 berequired to be proficient to complete study procedures)

  18. Employed by the U.S. Department of Defense, including U.S. military personnel

Study Design

Total Participants: 96
Treatment Group(s): 1
Primary Treatment: Nanosensor
Phase: 1
Study Start date:
January 02, 2025
Estimated Completion Date:
September 30, 2026

Study Description

This is an investigational device clinical trial. Mitochondrial Myopathy (MM) cases and healthy volunteers will undergo nanosensor muscle oxygen measurement in exercised (dominant) forearm muscle during handgrip exercise.

After placement of the nanosensor in the forearm under local anesthesia, the primary outcome measure is nanosensor-muscle oxygen levels. The secondary outcome measure is an assessment of pain.

Connect with a study center

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania 19104
    United States

    Active - Recruiting

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