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El ensayo clínico NCT04086329 para Miopatías mitocondriales, Enfermedades mitocondriales está reclutando. Consulte la vista de tarjeta del Radar de Ensayos Clínicos y las herramientas de descubrimiento de IA para conocer todos los detalles. O haga cualquier pregunta aquí. | ||
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Validation of Oxygen Nanosensor in Mitochondrial Myopathy
Los detalles del ensayo clínico están disponibles principalmente en inglés. ¡Sin embargo, IA Trial Radar puede ayudar! Simplemente haga clic en 'Explicar el ensayo' para ver y discutir la información del ensayo en el idioma que haya seleccionado.
El ensayo clínico NCT04086329 está diseñado para estudiar el diagnóstico de Miopatías mitocondriales, Enfermedades mitocondriales. Es un estudio intervencionista de Fase I. Su estado actual es: reclutando. El ensayo se inició el 17 de enero de 2023, con el objetivo de reclutar a 24 participantes. Dirigido por Children's Hospital of Philadelphia, se espera que finalice el 1 de agosto de 2027. Los datos se actualizaron por última vez en ClinicalTrials.gov el 10 de septiembre de 2025.
Resumen
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.
Descripción detallada
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.
Título oficial
The Development of Minimally Invasive Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
Condiciones médicas
Miopatías mitocondrialesEnfermedades mitocondrialesOtros ID del ensayo
Número del NCT
Inicio del ensayo (real)
2023-01-17
Última actualización
2025-09-10
Fecha de finalización (estimada)
2027-08-01
Inscripción (prevista)
24
Tipo de estudio
Intervencionista
FASE
Fase I
Estado general
Reclutando
Objetivo principal
Diagnóstico
Método de asignación
No aleatorizado
Modelo de intervención
Paralelo
Enmascaramiento
Ninguno (Etiqueta abierta)
Brazos / Intervenciones
| Grupo de participantes | Intervención/Tratamiento |
|---|---|
OtrosAffected 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. | 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. |
OtrosHealthy Controls Adult healthy volunteers will be individually matched with corresponding MM cases based on age, biological sex, and body mass index. | 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. |
Resultado primario
Resultado secundario
| Medida de resultado | Descripción de la medida | Periodo de tiempo |
|---|---|---|
Nanosensor-muscle oxygen (Torr) levels | Nanosensor measured muscle O2 levels at baseline, during handgrip exercise and after exercise | 60 minutes for data collection at each 2 study visits, up to 6 months. |
| Medida de resultado | Descripción de la medida | Periodo de tiempo |
|---|---|---|
Pain and tolerability | At each 2 study visits, up to 6 months. |
Criterios de elegibilidad
Criterios de edad
Adulto, Adulto mayor
Edad mínima
18 Years
Criterios de sexo
Todos
Admisión de voluntarios sanos
Sí
- 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
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
- 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)
Children's Hospital of PhiladelphiaHospitales Universitarios del Gran París327 ensayos clínicos activos para explorarParte responsable del ensayo
Zarazuela Zolkipli Cunningham, Investigador principal, Principal Investigator, Children's Hospital of Philadelphia
Contactos centrales del ensayo
Contacto: Zarazuela Zolkipli-Cunningham, (267) 426 4961, [email protected]
Contacto: Sara Nguyen, (267) 426-1986, [email protected]
1 Sitios del ensayo en 1 países
Pennsylvania
The Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, 19104, United States
Zarazuela Zolkipli-Cunningham, MBChB, MRCP, Contacto, [email protected]
Reclutando