The Development of Minimally Invasive Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 24
- 试验地点
- 2
- 主要终点
- Nanosensor-muscle oxygen (Torr) levels
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Diagnostic
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 是
入选标准
- •for Healthy Controls
- •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
- •Inclusion Criteria for MM Cases
- •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
排除标准
- •for All Participants
- •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
- •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
- •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
- •Individuals from vulnerable populations (e.g., prisoners/detainees)
- •Participants who are unable to speak and/or read English (as participants will be required to be proficient to complete study procedures)
研究组 & 干预措施
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.
干预措施: Nanosensor (Device)
Healthy Controls
Adult healthy volunteers will be individually matched with corresponding MM cases based on age, biological sex, and body mass index.
干预措施: Nanosensor (Device)
结局指标
主要结局
Nanosensor-muscle oxygen (Torr) levels
时间窗: 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
次要结局
- Pain and tolerability(At each 2 study visits, up to 6 months.)
研究者
Zarazuela Zolkipli Cunningham
Principal Investigator
Children's Hospital of Philadelphia