Recruiting
Phase 1

Electrochemical Nanosensor

Sponsor:

Children's Hospital of Philadelphia

Code:

NCT06819683

Conditions

MItochondrial Myopathies

Mitochondrial Disease

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Accepted

Interventions

Nanosensor

Study Details

Brief 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.

Conditions

MItochondrial Myopathies

Mitochondrial Disease

Study ID

NCT06819683

Start date

Jan 2, 2025

Status verified date

Feb, 2026

Completion date

Sep, 2026

Anticipated

Primary completion date

Sep, 2026

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 18 - 65

Healthy Volunteers: Accepted

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

Study Design

Enrollment

96 participants

Anticipated

Allocation

Non randomized

Intervention Model

Parallel Assignment

Primary purpose

Diagnostic

Interventions and Outcome Measures

Arms

experimental: Affected Mitochondrial Myopathy (MM) Cases

Key eligibility criteria for Mitochondrial Myopathy (MM) cases includes physically-capable adults (male and females, ages 18 to 65 years, inclusive) with genetically-confirmed MM with predominant symptoms of myopathy as expressed by exercise intolerance and muscle weakness and fatigue.

active comparator: Healthy Controls

Adult healthy volunteers will be individually matched with corresponding Mitochondrial Myopathy cases based on age, biological sex, and body mass index.

Interventions

Nanosensor

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.

Primary outcome measure

  • Nanosensor-muscle oxygen (Torr) levels during handgrip exercises [ Time Frame: before, during and after handgrip exercise up to 4 hours or up to 40 days after the screening visit ]
  • Nanosensor-muscle oxygen (Torr) levels post Cardiopulmonary Exercise Testing (CPET) [ Time Frame: After CPET exercise up to 1-hour ]

Central Contacts and Locations

Central contacts

Zarazuela Zolkipli-Cunningham, MBChB, MRCP

267-426-1986MitoMyopathyResearch@chop.edu

Locations

The Children's Hospital of Philadelphia

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Zarazuela Zolkipli-Cunningham, MBChB, MRCP

267-426-1986MitoMyopathyResearch@chop.edu

Principal Investigator:

Zarazuela Zolkipli-Cunningham, MBChB, MRCP

More Information

Sponsor

Children's Hospital of Philadelphia

Last update posted

Feb 17, 2026

Last verified

Feb, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Children's Hospital of Philadelphia on 2026-02-17.