A Phase 0 Study to Evaluate the Clinical Readiness of a Wearable Sensor Device and Duchenne Video Assessment in the Home Environment on Participants With Duchenne Muscular Dystrophy (DMD)
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 17
- 试验地点
- 1
- 主要终点
- Wearable sensor device compliance
研究概览
简要总结
This study is designed to evaluate the feasibility, wearability and participant satisfaction of novel outcome assessment tools in DMD patients which are performed in the home environment.
详细描述
This is a low interventional feasibility study to evaluate the use of:
- An FDA 510(k) cleared Class II wearable medical device designed to collect medical grade, clinical quality biometric, physiological and other electronic clinician outcome assessments data in a clinical trial setting, and;
- The Duchenne Video Assessment (DVA) tool to evaluate the quality of movement in patients with Duchenne Muscular Dystrophy (DMD)
This study will provide data on suitability of these tools in future clinical trials.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 4 Years 至 12 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 是
入选标准
- •For DMD patients:
- •Parent/guardian or participant is able to give informed consent and/or assent as required by local regulations, and all are willing and able to comply with the protocol.
- •Participant is assigned male sex at birth and is age 4 to <13 years at time of consent.
- •Participant has a confirmed diagnosis of DMD based on genetic testing and/or clinical records consistent with the diagnosis.
- •Participant has been on a stable glucocorticoid dose for 3 months prior to participation.
- •Participant is ambulatory as defined by the ability to walk down a hallway at home without assistance or support.
- •For healthy age-matched controls:
- •Parent/guardian or participant is able to give informed consent and/or assent as required by local regulations, and all are willing and able to comply with the protocol.
- •Participant is assigned male sex at birth and age 4 to <13 years at time of consent.
排除标准
- •For DMD Patients:
- •Participant is/was enrolled in any interventional study for DMD in the past 3 months or has ever been enrolled in a gene therapy study.
- •Participant is on any approved therapy for DMD except for glucocorticoids.
- •Participant is currently or was previously treated with exon-skipping antisense oligonucleotides such as eteplirsen, golodirsen, casimersen, and viltolarsen.
- •Participant has any prior or ongoing medical condition, medical history, or physical finding that could affect the participant's ability to perform the study assessments.
- •Participant has had major surgery within 3 months prior to recruitment or planned orthopedic surgery for any time during this study which would interfere with the ability to perform assessments.
- •Participant has a history of allergic response to silicones or adhesives.
- •Participant has an active implanted device (e.g., pacemaker). Implanted devices relying on an electrical power source to function are considered active devices.
- •For healthy age-matched controls:
- •Participant has a known musculoskeletal disease or had a musculoskeletal injury in the past 3 months.
- •Participant has other illness that precludes functional testing.
- •Participant is enrolled in any interventional study.
- •Participant has or has had any prior or ongoing medical condition, medical history, or physical finding that could affect the participant's ability to perform the study assessments.
- •Participant has had major surgery within 3 months prior to recruitment or planned orthopedic surgery for any time during this study which would interfere with the ability to perform study assessments.
- •Participant has a history of allergic response to silicones or adhesives.
- •Participant has an active implanted device (e.g., pacemaker). Implanted devices relying on an electrical power source to function are considered active devices.
结局指标
主要结局
Wearable sensor device compliance
时间窗: 30 days
The wearable sensor device is composed of three small sticker-based sensors that are worn on the chest, thigh and ankle to record activity. Participants will wear the sensors during specified periods over the study duration and compliance will be assessed based on recorded activity.
Accuracy of functional evaluation using the Duchenne Video Assessment (DVA) tool in an at-home setting
时间窗: 30 days
The DVA is a digital tool designed to remotely evaluate functional capacity in a natural environment. Participants will complete a set of movement tasks at specified timepoints during the study that will be captured on video and scored by remote evaluators. DVA scoring will be compared with anticipated levels of functional capacity to assess accuracy.
Preference for use of the wearable sensor device and DVA tool as assessed by interview
时间窗: Day 24-30
Participants will be asked to complete an exit interview at the conclusion of the study to assess user preference.
次要结局
未报告次要终点