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Validation of Oxygen Nanosensor in Mitochondrial Myopathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04086329
Recruitment Status : Not yet recruiting
First Posted : September 11, 2019
Last Update Posted : March 16, 2020
Sponsor:
Collaborators:
National Institutes of Health (NIH)
North American Mitochondrial Disease Consortium
Singh Center for Nanotechnology at the University of Pennsylvania
Information provided by (Responsible Party):
Zarazuela Zolkipli Cunningham, Children's Hospital of Philadelphia

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.


Condition or disease Intervention/treatment Phase
Mitochondrial Myopathies Mitochondrial Diseases Device: Nanosensor Not Applicable

Detailed Description:

This is an investigational device clinical trial. MM cases and healthy volunteers will undergo nanosensor muscle oxygen measurement in exercised (dominant) forearm muscle during handgrip exercise. The same measurements will be repeated between 7 and 30 days later in the same forearm and at the same time of day for each participant to assess reproducibility.

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Diagnostic
Official Title: The Development of Minimally Invasive Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
Estimated Study Start Date : September 2020
Estimated Primary Completion Date : November 2021
Estimated Study Completion Date : November 2021


Arm Intervention/treatment
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.
Device: 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.


Healthy Controls
Adult healthy volunteers will be individually matched with corresponding MM cases based on age, biological sex, and body mass index.
Device: 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 Measures :
  1. Nanosensor-muscle oxygen (Torr) levels [ Time Frame: 60 minutes for data collection at each 2 study visits, up to 6 months. ]
    Nanosensor measured muscle O2 levels at baseline, during handgrip exercise and after exercise


Secondary Outcome Measures :
  1. Pain and tolerability [ Time Frame: At each 2 study visits, up to 6 months. ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
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 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 for All Participants

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)

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04086329


Contacts
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Contact: Zarazuela Zolkipli-Cunningham (267) 331-1365 zolkipliz@email.chop.edu
Contact: Sara Nguyen (267) 426-7165 nguyens2@email.chop.edu

Sponsors and Collaborators
Children's Hospital of Philadelphia
National Institutes of Health (NIH)
North American Mitochondrial Disease Consortium
Singh Center for Nanotechnology at the University of Pennsylvania
Investigators
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Principal Investigator: Zarazuela Zolkipli-Cunningham Children's Hospital of Philadelphia
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Responsible Party: Zarazuela Zolkipli Cunningham, Principal Investigator, Children's Hospital of Philadelphia
ClinicalTrials.gov Identifier: NCT04086329    
Other Study ID Numbers: 17-014130
First Posted: September 11, 2019    Key Record Dates
Last Update Posted: March 16, 2020
Last Verified: March 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscular Diseases
Mitochondrial Myopathies
Mitochondrial Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Metabolic Diseases