Evaluation of Home Based Assessments on Participants With DMD

NCT ID: NCT05657938

Last Updated: 2023-08-14

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 17 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2022-10-13
• Study Completion Date: 2023-04-04

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.

Detailed 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.

Conditions

Duchenne Muscular Dystrophy

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

DMD Patients

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

Wearable Device

Intervention Type: 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.

Duchenne Video Assessment (DVA)

Intervention Type: OTHER

The DVA is a standardized tool that documents and assesses compensatory movement strategies as a marker of quality of movement.

Age Matched Controls

Normal male age-matched controls

Wearable Device

Intervention Type: 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.

Duchenne Video Assessment (DVA)

Intervention Type: OTHER

The DVA is a standardized tool that documents and assesses compensatory movement strategies as a marker of quality of movement.

Interventions

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.

Intervention Type: DEVICE

Duchenne Video Assessment (DVA)

The DVA is a standardized tool that documents and assesses compensatory movement strategies as a marker of quality of movement.

Intervention Type: OTHER

Eligibility Criteria

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.

• Minimum Eligible Age: 4 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: Yes

Sponsors

Solid Biosciences Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Carl A Morris, PhD

Role: STUDY_DIRECTOR

Solid Biosciences

Locations

Solid Biosciences

Charlestown, Massachusetts, United States

Countries

United States

Other Identifiers

SB-000-22-001

Identifier Type: -

Identifier Source: org_study_id