Evaluation of Home Based Assessments on Participants With DMD

NCT05657938 | Completed

• 1 other identifier: SB-000-22-001
• Study Type: observational
• Target: 17
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Duchenne Muscular Dystrophy

Keywords

duchenne; muscular dystrophy; DMD

Outcome Measures

Primary Outcomes (3)

• 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

Study Arms (2)

DMD Patients

Ambulatory males aged 4 to \<13 years with Duchenne Muscular Dystrophy

Device: Wearable Device; Other: Duchenne Video Assessment (DVA)

Age Matched Controls

Normal male age-matched controls

Device: Wearable Device; Other: Duchenne Video Assessment (DVA)

Interventions

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.

Age Matched Controls; DMD Patients

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.

Age Matched Controls; DMD Patients

Eligibility Criteria

• Age: 4 Years - 12 Years
• Healthy Volunteers: Yes
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

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.

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Solid Biosciences Inc.: lead

Study Sites (1)

Solid Biosciences

Charlestown, Massachusetts, 02129, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne; Muscular Dystrophies

Interventions

Wearable Electronic Devices

Condition Hierarchy (Ancestors)

Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Electrical Equipment and Supplies; Equipment and Supplies

Study Officials

• Carl A Morris, PhD

  Solid Biosciences

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 26, 2022
• First Posted: December 20, 2022
• Study Start: October 13, 2022
• Primary Completion: April 4, 2023
• Study Completion: April 4, 2023
• Last Updated: August 14, 2023

Record last verified: 2022-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)