Validation of Nanosensor Oxygen Measurement

NCT ID: NCT06819683

Last Updated: 2025-02-11

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 96 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-01-02
• Study Completion Date: 2026-09-30

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.

Detailed 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.

Conditions

MItochondrial Myopathies; Mitochondrial Disease

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: DIAGNOSTIC
• Blinding Strategy: NONE

Study Groups

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.

Group Type: EXPERIMENTAL

Nanosensor

Intervention Type: 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.

Healthy Controls

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

Group Type: ACTIVE_COMPARATOR

Nanosensor

Intervention Type: 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.

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.

Intervention Type: DEVICE

Eligibility Criteria

Inclusion Criteria

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

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 65 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

United States Department of Defense

FED

Sponsor Role: collaborator

Children's Hospital of Philadelphia

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Zarazuela Zolkipli-Cunningham, MBChB, MRCP

Role: PRINCIPAL_INVESTIGATOR

Children's Hospital of Philadelphia

Locations

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Zarazuela Zolkipli-Cunningham, MBChB, MRCP

Role: CONTACT

MitoMyopathyResearch@chop.edu

267-426-1986

Daniel McGinn, MS, LCGC

Role: CONTACT

MitoMyopathyResearch@chop.edu

267-426-1986

Facility Contacts

Zarazuela Zolkipli-Cunningham, MBChB, MRCP

Role: primary

MitoMyopathyResearch@chop.edu

267-426-1986

Daniel McGinn, MS, LCGC

Role: backup

MitoMyopathyResearch@chop.edu

(267) 426-1986

Other Identifiers

W81XWH2210590

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

22-019820

Identifier Type: -

Identifier Source: org_study_id