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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.
This is a low interventional feasibility study to evaluate the use of:
This study will provide data on suitability of these tools in future clinical trials.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| DMD Patients | Ambulatory males aged 4 to <13 years with Duchenne Muscular Dystrophy |
| |
| Age Matched Controls | Normal male age-matched controls |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Wearable Device | Device | The wearable device used in this study is an FDA 510(k) cleared Class II medical device designed to collect medical grade, clinical quality biometric, physiological and other electronic clinician outcome assessments data in a clinical trial setting. The system processes raw data into recognizable clinical metrics including vital signs, activity and posture classifications, surface electromyography (sEMG), and sleep metrics. The sensors are multi-modal, multilocation, rechargeable and reusable. |
| Measure | Description | Time Frame |
|---|---|---|
| Wearable sensor device compliance | 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. | 30 days |
| Accuracy of functional evaluation using the Duchenne Video Assessment (DVA) tool in an at-home setting | 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. | 30 days |
| Preference for use of the wearable sensor device and DVA tool as assessed by interview | Participants will be asked to complete an exit interview at the conclusion of the study to assess user preference. | Day 24-30 |
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Inclusion Criteria:
For DMD patients:
For healthy age-matched controls:
Exclusion Criteria:
For DMD Patients:
For healthy age-matched controls:
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Approximately twenty ambulatory males aged 4 to <13 years with DMD will be recruited. Of these, approximately 10 participants will be considered Early Ambulatory and 10 will be considered Late Ambulatory. Early ambulatory is defined as able to get off the floor without assistance or use of furniture.
Approximately twenty healthy, age-matched controls will be recruited.
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| Name | Affiliation | Role |
|---|---|---|
| Carl A Morris, PhD | Solid Biosciences | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Solid Biosciences | Charlestown | Massachusetts | 02129 | United States |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| D009136 | Muscular Dystrophies |
| ID | Term |
|---|---|
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
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| ID | Term |
|---|---|
| D000076251 | Wearable Electronic Devices |
| ID | Term |
|---|---|
| D055615 | Electrical Equipment and Supplies |
| D004864 | Equipment and Supplies |
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| Duchenne Video Assessment (DVA) | Other | The DVA is a standardized tool that documents and assesses compensatory movement strategies as a marker of quality of movement. |
|
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |