IA Trial Radar | ||
|---|---|---|
El ensayo clínico NCT06819683 para Miopatías mitocondriales, Enfermedad mitocondrial 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. | ||
Un ensayo coincide con los criterios de filtro
Vista de tarjeta
Validation of Nanosensor Oxygen Measurement
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 NCT06819683 está diseñado para estudiar el diagnóstico de Miopatías mitocondriales, Enfermedad mitocondrial. Es un estudio intervencionista de Fase I. Su estado actual es: reclutando. El ensayo se inició el 2 de enero de 2025, con el objetivo de reclutar a 96 participantes. Dirigido por Children's Hospital of Philadelphia, se espera que finalice el 1 de septiembre de 2026. Los datos se actualizaron por última vez en ClinicalTrials.gov el 11 de febrero 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. Mitochondrial Myopathy (MM) cases and healthy volunteers will undergo nanosensor muscle oxygen measurement in exercised (dominant) forearm muscle during handgrip exercise.
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 Validation of Minimally Invasive Oxygen Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
Condiciones médicas
Miopatías mitocondrialesEnfermedad mitocondrialOtros ID del ensayo
- 22-019820
- W81XWH2210590 (Otra financiación) (Department of Defense)
Número del NCT
Inicio del ensayo (real)
2025-01-02
Última actualización
2025-02-11
Fecha de finalización (estimada)
2026-09
Inscripción (prevista)
96
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 |
|---|---|
ExperimentalAffected Mitochondrial Myopathy (MM) Cases 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. | 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. |
Comparador activoHealthy Controls Adult healthy volunteers will be individually matched with corresponding Mitochondrial Myopathy 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 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 after 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 |
| Medida de resultado | Descripción de la medida | Periodo de tiempo |
|---|---|---|
Self-reported pain level assessment | After the exercise procedure, participants will self-report the level of pain/discomfort with the validated Wong-Baker Faces Pain Rating Scale and Numerical Rating Scale. The Wong-Baker Faces Pain Rating Scale is a tool used to assess pain levels in young children by presenting using a combination of faces, numbers, and words to help the participant effectively communicate the severity of their physical pain. The scale includes a happy face indicating 0="no hurt" to a crying face signifying 10="hurts worst." The NRS is a pain screening tool, commonly used to assess pain severity at that moment in time using a 0-10 scale, with zero meaning "no pain" and 10 meaning "the worst pain imaginable." All subjects will respond to both surveys. | up to 40 days after the screening visit |
Patient Satisfaction | Patient satisfaction will be measured by participants completing the Post-operative Patient Satisfaction Survey. The survey consists of one question to gauge self-reported insight on their overall experience. Responses note the experience was "Agreeable", "Neither pleasant nor unpleasant", "Slightly uncomfortable", "Disagreeable", or "Traumatic". | After study procedures are completed (approximately day 40) |
Self-reported fatigue | After the exercise period, participants will self-report the level of fatigue as a measure of disease burden using the Modified Fatigue Impact Scale (MFIS). This scale includes 9 questions regarding subject's fatigue symptoms and how it impacts their life. Answers range from "Strongly Agree" to "Strongly Disagree" with 7 answer choices for each question. | After study procedures are completed (approximately day 40) |
Criterios de elegibilidad
Criterios de edad
Adulto, Adulto mayor
Edad mínima
18 Years
Criterios de sexo
Todos
Admisión de voluntarios sanos
Sí
Inclusion Criteria 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 Mitochondrial Myopathy (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)
- Employed by the U.S. Department of Defense, including U.S. military personnel
Children's Hospital of PhiladelphiaUnited States Department of DefenseContactos centrales del ensayo
Contacto: Zarazuela Zolkipli-Cunningham, MBChB, MRCP, 267-426-1986, [email protected]
Contacto: Daniel McGinn, MS, LCGC, 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, 267-426-1986, [email protected]
Daniel McGinn, MS, LCGC, Contacto, (267) 426-1986, [email protected]
Zarazuela Zolkipli-Cunningham, MBChB, MRCP, Investigador principal
Reclutando