Duchenne Muscular DystrophyNCT05657938
Evaluation of Home Based Assessments on Participants With DMD
An observational study of Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 4 to 12 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Wearable Device and Duchenne Video Assessment (DVA)
- Type of treatment
- Medical device
- How many people
- 17 participants
- Where
- 1 siteUnited States
- Study dates
- 13 Oct 2022to 4 Apr 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 4 to 12 years
- Men only
- Healthy volunteers accepted
- Child
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.
Who may be able to join
- 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.
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
1 site in 1 country.
About this study
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)
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.
Read the detailed description
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.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Wearable Device, Duchenne Video Assessment (DVA)
- Keywords
- duchenne, muscular dystrophy, DMD
- Medical subject headings
- Muscular Dystrophy, Duchenne, Muscular Dystrophies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Wearable DeviceDevice
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)Other
The DVA is a standardized tool that documents and assesses compensatory movement strategies as a marker of quality of movement.
| Study group | Role | What they receive |
|---|---|---|
| DMD Patients | Ambulatory males aged 4 to <13 years with Duchenne Muscular Dystrophy Receives: Device: Wearable Device; Other: Duchenne Video Assessment (DVA) | |
| Age Matched Controls | Normal male age-matched controls Receives: Device: Wearable Device; Other: Duchenne Video Assessment (DVA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: Day 24-30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2022
- Study start13 Oct 2022
- Primary completion4 Apr 2023
- Study completion4 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Solid Biosciences Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Carl A Morris, PhD, Study director, Solid Biosciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05657938
- Sponsor's protocol ID
- SB-000-22-001
- Status last verified
- Dec 2022
- First submitted
- 26 Oct 2022
- Last updated
- 14 Aug 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05657938
- Acronym / sponsor ID
- SB-000-22-001
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- duchenne, muscular dystrophy, DMD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Solid Biosciences Inc.Industry (pharma, biotech, devices)
- Study start
- 13 Oct 2022 (actual)
- Primary completion
- 4 Apr 2023 (actual)
- Study completion
- 4 Apr 2023 (actual)
- First posted
- 20 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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:
- 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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| DMD Patients | – | Device: Wearable Device, Other: Duchenne Video Assessment (DVA)Ambulatory males aged 4 to <13 years with Duchenne Muscular Dystrophy |
| Age Matched Controls | – | Device: Wearable Device, Other: Duchenne Video Assessment (DVA)Normal male age-matched controls |
Who may qualify
- Ages
- 4 to 12 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child
- 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.
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (16 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Wearable DeviceThe 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. | Device | Age Matched Controls, DMD Patients |
| Duchenne Video Assessment (DVA)The DVA is a standardized tool that documents and assesses compensatory movement strategies as a marker of quality of movement. | Other | Age Matched Controls, DMD Patients |
Primary outcome
Wearable sensor device compliance
Time frame: 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
Time frame: 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
Time frame: Day 24-30
Participants will be asked to complete an exit interview at the conclusion of the study to assess user preference.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Carl A Morris, PhD study director
Solid Biosciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy, run by Solid Biosciences Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Wearable sensor device compliance (time frame: 30 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2023.