Evaluation of Home Based Assessments on Participants With DMD

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT05657938
• Other study ID #: SB-000-22-001
• Status: Completed
• Start date: October 13, 2022
• Est. completion date: April 4, 2023

Study information

• Verified date: December 2022
• Source: Solid Biosciences Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Description:

This is a low interventional feasibility study to evaluate the use of: 1. 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; 2. 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 17
• Est. completion date: April 4, 2023
• Est. primary completion date: April 4, 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: Male
• Age group: 4 Years to 12 Years

Eligibility

Inclusion Criteria:

For DMD patients:

1. 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.

2. Participant is assigned male sex at birth and is age 4 to <13 years at time of consent.

3. Participant has a confirmed diagnosis of DMD based on genetic testing and/or clinical records consistent with the diagnosis.

4. Participant has been on a stable glucocorticoid dose for 3 months prior to participation.

5. Participant is ambulatory as defined by the ability to walk down a hallway at home without assistance or support.

For healthy age-matched controls:

1. 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.

2. Participant is assigned male sex at birth and age 4 to <13 years at time of consent. Exclusion Criteria:

For DMD Patients:

1. 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.

2. Participant is on any approved therapy for DMD except for glucocorticoids.

3. Participant is currently or was previously treated with exon-skipping antisense oligonucleotides such as eteplirsen, golodirsen, casimersen, and viltolarsen.

4. 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.

5. 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.

6. Participant has a history of allergic response to silicones or adhesives.

7. 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:

1. Participant has a known musculoskeletal disease or had a musculoskeletal injury in the past 3 months.

2. Participant has other illness that precludes functional testing.

3. Participant is enrolled in any interventional study.

4. 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.

5. 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.

6. Participant has a history of allergic response to silicones or adhesives.

7. Participant has an active implanted device (e.g., pacemaker). Implanted devices relying on an electrical power source to function are considered active devices.

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

Device:
• Wearable 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.

Other:
• Duchenne Video Assessment (DVA)
The DVA is a standardized tool that documents and assesses compensatory movement strategies as a marker of quality of movement.

Locations

Country	Name	City	State
United States	Solid Biosciences	Charlestown	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Solid Biosciences Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Primary	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