Duchenne Muscular DystrophyARISENCT05712447
Duchenne Muscular Dystrophy Video Assessment Registry
An observational study of Duchenne Muscular Dystrophy, run by The Emmes Company, LLC at 1 site in United States.
Trial summary
ARISE is a prospective and longitudinal clinical study of individuals with Duchenne Muscular Dystrophy (DMD) aimed at creating a database of functional motor abilities in this population to support validation efforts of the Duchenne Video Assessment (DVA). Read more about this study
- Who is it for?
- Ages 2 years and older
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 150 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Sep 2022to 11 Jan 2026
- 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 2 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Participants will be recruited for this study through referrals from collaborators and advocacy groups as well as through direct marketing via social media, email, phone, or other appropriate methods. Advocacy groups will assist with the facilitation of recruitment by making recruitment materials available to the Duchenne community. Participants who provided consent to be contacted by Casimir staff for future studies and shared their contact information with Casimir staff may be contacted. Potential participants may also share their contact information directly with Casimir study staff to learn about the study. Additionally, recruitment for the study may also occur through postings with patient advocacy groups and on social media. Casimir will supply the advocacy groups with language for website postings and group emails.
Who may be able to join
- At least 2 years of age at time of consent
- Written consent (English only) by adult participant or parent/legal guardian of minor participant
- Written assent (English only) if minor participant is at least 7 years of age
- Documentation provided for the participant's diagnosis of DMD (i.e., genetic report, clinic note)
Who cannot join
- Participants will be excluded if they are unable to use a hand to hold a pen, pick up pennies or drive a powerchair.
Trial locations and contacts
1 site in 1 country.
About this study
ARISE is a prospective and longitudinal clinical study of individuals with Duchenne Muscular Dystrophy (DMD) aimed at creating a database of functional motor abilities in this population to support validation efforts of the Duchenne Video Assessment (DVA).
Read the detailed description
ARISE is a longitudinal, observational study of individuals with Duchenne muscular dystrophy (DMD). The Duchenne Video Assessment (DVA) is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA directs caregivers to video record patients doing specific movement activities at home using a secure mobile application. In this study, participants will remotely complete the DVA in addition to existing DMD functional assessments (North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL 2.0)) to evaluate the longitudinal measurement properties of the DVA.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne, DMD, Remote video assessment
- Medical subject headings
- Muscular Dystrophy, Duchenne
What is being measured
The primary outcome is the main question the study is designed to answer.
Duchenne Video Assessment (DVA)
The DVA is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA includes 18 standardized movement tasks that are assigned to participants based on functional subgroup. The DVA directs and trains caregivers to video record patients doing specific movement tasks at home using a secure mobile application. DVA videos are scored by DVA-certified physical therapists using validated scorecards with prespecified compensatory movement criteria.
Measured: 24 months
Secondary outcomes (3)
North Star Ambulatory Assessment (NSAA)
The NSAA is a 17-item tool used to measure functional motor abilities in ambulatory individuals with DMD. Physical therapists with experience administering the NSAA will remotely administer and record the live NSAA sessions via a secure video conference platform. Participants will receive the appropriate standardized testing equipment.
Measured: 24 months
Performance of Upper Limb 2.0 (PUL 2.0)
The PUL 2.0 is a 22-item rating scale used to measure upper limb abilities in individuals with DMD. The test includes an entry test item to define starting functional level and 21 test items subdivided into shoulder level, elbow level, and distal level. Physical therapists with experience administering the PUL 2.0 will remotely administer and record the live PUL 2.0 sessions via secure video conference platform. Participants will receive the appropriate standardized testing equipment.
Measured: 24 months
Caregiver Task-Specific Impression of Change
At each follow-up assessment timepoint, parents/legal guardians or recording partners will complete an electronic questionnaire to rate their impression of change in the participant's ability to perform each DVA movement task since the prior timepoint.
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Feb 2023
- Study start1 Sep 2022
- Primary completion11 Jan 2026
- Study completion11 Jan 2026
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
- 150 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Emmes Company, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- United States Department of Defense
- Responsible party
- Sponsor
- Investigators
- Marielle Contesse, PhD, Principal investigator, Emmes Endpoint Solutions
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05712447
- Sponsor's protocol ID
- CAS-CAS005-01
- Acronym
- ARISE
- Status last verified
- Feb 2026
- First submitted
- 30 Nov 2022
- Last updated
- 10 Mar 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Duchenne Muscular Dystrophy Video Assessment Registry
- ClinicalTrials.gov ID
- NCT05712447
- Acronym / sponsor ID
- ARISE / CAS-CAS005-01
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne, DMD, Remote video assessment
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 150 participants (actual)
- Lead sponsor
- The Emmes Company, LLCIndustry (pharma, biotech, devices)
- Collaborators
- United States Department of Defense
- Study start
- 1 Sep 2022 (actual)
- Primary completion
- 11 Jan 2026 (actual)
- Study completion
- 11 Jan 2026 (actual)
- First posted
- 3 Feb 2023 (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.
ARISE is a prospective and longitudinal clinical study of individuals with Duchenne Muscular Dystrophy (DMD) aimed at creating a database of functional motor abilities in this population to support validation efforts of the Duchenne Video Assessment (DVA).
Detailed description
ARISE is a longitudinal, observational study of individuals with Duchenne muscular dystrophy (DMD). The Duchenne Video Assessment (DVA) is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA directs caregivers to video record patients doing specific movement activities at home using a secure mobile application. In this study, participants will remotely complete the DVA in addition to existing DMD functional assessments (North Star Ambulatory Assessment (NSAA) and Performance of Upper Limb (PUL 2.0)) to evaluate the longitudinal measurement properties of the DVA.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Participants will be recruited for this study through referrals from collaborators and advocacy groups as well as through direct marketing via social media, email, phone, or other appropriate methods. Advocacy groups will assist with the facilitation of recruitment by making recruitment materials available to the Duchenne community. Participants who provided consent to be contacted by Casimir staff for future studies and shared their contact information with Casimir staff may be contacted. Potential participants may also share their contact information directly with Casimir study staff to learn about the study. Additionally, recruitment for the study may also occur through postings with patient advocacy groups and on social media. Casimir will supply the advocacy groups with language for website postings and group emails.
Key inclusion criteria (4 listed)
- At least 2 years of age at time of consent
- Written consent (English only) by adult participant or parent/legal guardian of minor participant
- Written assent (English only) if minor participant is at least 7 years of age
- Documentation provided for the participant's diagnosis of DMD (i.e., genetic report, clinic note)
Key exclusion criteria (1 listed)
- Participants will be excluded if they are unable to use a hand to hold a pen, pick up pennies or drive a powerchair.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Duchenne Video Assessment (DVA)
Time frame: 24 months
The DVA is a home-based clinical outcome assessment that measures ease of movement through identification of compensatory movement patterns. The DVA includes 18 standardized movement tasks that are assigned to participants based on functional subgroup. The DVA directs and trains caregivers to video record patients doing specific movement tasks at home using a secure mobile application. DVA videos are scored by DVA-certified physical therapists using validated scorecards with prespecified compensatory movement criteria.
3 secondary outcomes
North Star Ambulatory Assessment (NSAA)
Time frame: 24 months
Performance of Upper Limb 2.0 (PUL 2.0)
Time frame: 24 months
Caregiver Task-Specific Impression of Change
Time frame: 24 months
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 10 Mar 2026.
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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.
Marielle Contesse, PhD principal investigator
Emmes Endpoint Solutions
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 The Emmes Company, LLC 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 4 inclusion and 1 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 10 Mar 2026. 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?
Duchenne Video Assessment (DVA) (time frame: 24 months). 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 10 Mar 2026.