Validation of Oxygen Nanosensor in Mitochondrial Myopathy

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
Children's Hospital of Philadelphia
Study ID
NCT04086329
Phase
PHASE1
Status
Recruiting
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Conditions

  • Mitochondrial Diseases
  • Mitochondrial Myopathies

Eligibility Criteria

Sex
ALL
Age
18 Years - 65 Years
Healthy Volunteers
Accepted

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.

Study Details

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.

Key Dates

Start date
Jan 17, 2023
Status verified
Jan 2026
Primary completion
Aug 1, 2027
Completion
Aug 1, 2027

Study Design

Enrollment
24 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
DIAGNOSTIC

Arms

  • Other: Affected MM Cases
    Key eligibility criteria for 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.
  • Other: Healthy Controls
    Adult healthy volunteers will be individually matched with corresponding MM cases based on age, biological sex, and body mass index.

Primary Outcome Measure

Nanosensor-muscle oxygen (Torr) levels [ Time Frame: 60 minutes for data collection at each 2 study visits, up to 6 months. ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
The Children's Hospital of PhiladelphiaPhiladelphiaPennsylvania19104
Zarazuela Zolkipli-Cunningham, MBChB, MRCP
[email protected]

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The Children's Hospital of Philadelphia· Philadelphia, PA

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