Duchenne Muscular DystrophyNCT06054971
NSAA NON-Interventional Study Protocol
An observational study of Duchenne Muscular Dystrophy, run by Red Nucleus Enterprise Solutions, LLC at 1 site in United States.
Trial summary
The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD). The iTakeControl (iTC) software platform will be utilized to remotely administer and score the NSAAs. Read more about this study
- Who is it for?
- Ages 4 to 12 years
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- Remote Administration of NSAA
- How many people
- 31 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Oct 2023to 13 Mar 2025
- 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
- All sexes
- No healthy volunteers
- Child
Study population: Individuals with confirmed diagnosis of DMD
Who may be able to join
- Evidence of a signed and dated informed consent document indicating that the participant's legally authorized representative (LAR) has been informed of all pertinent aspects of the study, along with evidence of age-appropriate child assent.
- Confirmed diagnosis of DMD
- Participant with DMD aged 4 to12 (inclusive) at t Enrollment date
- Participant with DMD is ambulatory without assistive devices, braces, or aids throughout the study
- Caregiver has access to and/or willingness to learn use of a smart phone and the iTC study mobile application
- Participant and caregiver are based in the US throughout study
- Participant and caregiver are fluent in the English language (verbally and in writing)
Who cannot join
- Non-ambulatory DMD participant at any study timepoint
- Caregiver/participant unwilling or unable to administer/perform the NSAA
- Anticipated deterioration of participant's ambulatory status during the study
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Use of Telemedicine and At-Home Video for Remote Administration of the North Star Ambulatory Assessment (NSAA): a Feasibility Study
The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD). The iTakeControl (iTC) software platform will be utilized to remotely administer and score the NSAAs.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Remote Administration of NSAA
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Remote Administration of NSAAOther
Remote Administration of NSAA
| Study group | Role | What they receive |
|---|---|---|
| Group A | Group A will have Live telemedicine assessments completed prior to caregiver Asynchronous Video Receives: Other: Remote Administration of NSAA | |
| Group B | Group B will have Caregiver Asynchronous Video first, followed by live telemedicine Receives: Other: Remote Administration of NSAA |
What is being measured
The primary outcome is the main question the study is designed to answer.
Comparison of performance and ratings of the NSAA using three methods: 1) Live Telemedicine scores 2) Subsequent Recorded Telemedicine scores 3) Caregiver Asynchronous Video scores
Measured: 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Sep 2023
- Study start2 Oct 2023
- Primary completion3 Feb 2025
- Study completion13 Mar 2025
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
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Red Nucleus Enterprise Solutions, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Christopher Jones, PhD, Red Nucleus Enterprise Solutions, LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06054971
- Sponsor's protocol ID
- NSAA-RWE-001
- Status last verified
- Nov 2024
- First submitted
- 12 Sep 2023
- Last updated
- 21 Mar 2025
- 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 21 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Use of Telemedicine and At-Home Video for Remote Administration of the North Star Ambulatory Assessment (NSAA): a Feasibility Study
- ClinicalTrials.gov ID
- NCT06054971
- Acronym / sponsor ID
- NSAA-RWE-001
- Conditions
- Duchenne Muscular Dystrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Red Nucleus Enterprise Solutions, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Pfizer
- Study start
- 2 Oct 2023 (actual)
- Primary completion
- 3 Feb 2025 (actual)
- Study completion
- 13 Mar 2025 (actual)
- First posted
- 26 Sep 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.
The purpose of this non-interventional study is to evaluate the feasibility of remotely administering the North Star Ambulatory Assessment (NSAA) to participants with Duchenne muscular dystrophy (DMD). The iTakeControl (iTC) software platform will be utilized to remotely administer and score the NSAAs.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Group A | – | Other: Remote Administration of NSAAGroup A will have Live telemedicine assessments completed prior to caregiver Asynchronous Video |
| Group B | – | Other: Remote Administration of NSAAGroup B will have Caregiver Asynchronous Video first, followed by live telemedicine |
Who may qualify
- Ages
- 4 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Individuals with confirmed diagnosis of DMD
Key inclusion criteria (7 listed)
- Evidence of a signed and dated informed consent document indicating that the participant's legally authorized representative (LAR) has been informed of all pertinent aspects of the study, along with evidence of age-appropriate child assent.
- Confirmed diagnosis of DMD
- Participant with DMD aged 4 to12 (inclusive) at t Enrollment date
- Participant with DMD is ambulatory without assistive devices, braces, or aids throughout the study
- Caregiver has access to and/or willingness to learn use of a smart phone and the iTC study mobile application
Key exclusion criteria (3 listed)
- Non-ambulatory DMD participant at any study timepoint
- Caregiver/participant unwilling or unable to administer/perform the NSAA
- Anticipated deterioration of participant's ambulatory status during the study
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 |
|---|---|---|
| Remote Administration of NSAARemote Administration of NSAA | Other | Group A, Group B |
Primary outcome
Comparison of performance and ratings of the NSAA using three methods: 1) Live Telemedicine scores 2) Subsequent Recorded Telemedicine scores 3) Caregiver Asynchronous Video scores
Time frame: 6 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 21 Mar 2025.
- 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.
The record lists no contact. Use the official record to reach the sponsor.
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 Red Nucleus Enterprise Solutions, 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 7 inclusion and 3 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 21 Mar 2025. 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?
Comparison of performance and ratings of the NSAA using three methods: 1) Live Telemedicine scores 2) Subsequent Recorded Telemedicine scores 3) Caregiver Asynchronous Video scores (time frame: 6 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 21 Mar 2025.