Validation of Nanosensor Oxygen Measurement

NCT06819683 | Recruiting Phase 1 FDA Device

• 2 other identifiers: 22-019820W81XWH2210590
• Study Type: interventional
• Target: 96
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (2)

• 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.

  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)

  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.

  After CPET exercise up to 1-hour

Secondary Outcomes (3)

• Self-reported pain level assessment

  up to 40 days after the screening visit

• Patient Satisfaction

  After study procedures are completed (approximately day 40)

• Self-reported fatigue

  After study procedures are completed (approximately day 40)

Study Arms (2)

Affected Mitochondrial Myopathy (MM) Cases

EXPERIMENTAL

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.

Device: Nanosensor

Healthy Controls

ACTIVE COMPARATOR

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

Device: Nanosensor

Interventions

Nanosensor 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.

Affected Mitochondrial Myopathy (MM) Cases; Healthy Controls

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Able to perform bicycle ergometry
• 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
• 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.
• 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)
• Able to ambulate independently
• Able to perform bicycle ergometry

You may not qualify if:

• Subjects will be excluded if any of the following apply:
• 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
• 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
• Have a tracheostomy
• Have a known bleeding disorder and/or family history (first-degree relative) with a known bleeding disorder
• Daily intake of aspirin or any other anti-platelet therapy which cannot be temporarily discontinued for medical reasons
• 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
• 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
• Prone to hypertrophic scars and keloids
• Have any other known inherited myopathy, such as Duchenne muscular dystrophy or congenital myopathy
• Known allergy to lidocaine
• Have a cognitive impairment that may prevent the ability to complete study procedures

Sponsors & Collaborators

• Children's Hospital of Philadelphia: lead
• United States Department of Defense: collaborator

Study Sites (1)

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

MeSH Terms

Conditions

Mitochondrial Myopathies; Mitochondrial Diseases

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Zarazuela Zolkipli-Cunningham, MBChB, MRCP

  Children's Hospital of Philadelphia

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Zarazuela Zolkipli-Cunningham, MBChB, MRCP

CONTACT

267-426-1986; MitoMyopathyResearch@chop.edu

Daniel McGinn, MS, LCGC

CONTACT

267-426-1986; MitoMyopathyResearch@chop.edu

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: DIAGNOSTIC
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 4, 2025
• First Posted: February 11, 2025
• Study Start: January 2, 2025
• Primary Completion (Estimated): September 1, 2026
• Study Completion (Estimated): September 1, 2026
• Last Updated: February 17, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)