Duchenne Muscular Dystrophy (DMD)Acti-nRollNCT07664124
Digital Monitoring of Upper Limb Function in Non-Ambulant DMD
A study testing Performance of the Upper Limb (PUL) Test, Brook test and Dynamometric measurements of muscle strength in people with Duchenne Muscular Dystrophy (DMD), run by Centre Hospitalier Universitaire de Liege at 1 site in Belgium.
Trial summary
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration. Symptoms typically begin in early childhood and result in loss of ambulation by early adolescence, followed by cardiorespiratory complications. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- What is being tested?
- Performance of the Upper Limb (PUL) Test, Brook test and Dynamometric measurements of muscle strength
- Type of treatment
- Medical device
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteBelgium
- Study dates
- 30 Jul 2026to 30 Apr 2030
- Phase
- Interventional
- Are there results?
- Not yet: the study is under way
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 Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient with genetically confirmed Duchenne Muscular Dystrophy (DMD).
- Non-ambulant at the time of inclusion (not able to walk 10m without external aid).
- A legal guardian willing and able to provide written informed consent for participation in the study if < 18 years old.
Who cannot join
- Any acute or chronic condition that, in the opinion of the investigator, may significantly interfere with the assessments and/or motor function progression.
- Participation in an interventional clinical trial.
- No access to internet connection or alternatively no capacity to come on-site to bring the Syde every 6 months after the recording periods for data retrieval by Liège team
- Scoliosis surgery within the previous 6 months or planned within the next year
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Belgium1 site
Centre de référence des maladies neuromusculaire, Centre Hospitalier Régional de la Citadelle
Liège, 4000
About this study
Official title: Feasibility, Reliability, Clinical Validity and Sensitivity of Digital Outcomes to Monitor Upper Limb Function in Non-ambulant Patients With Genetically Confirmed Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration. Symptoms typically begin in early childhood and result in loss of ambulation by early adolescence, followed by cardiorespiratory complications. Although early treatment, including corticosteroids and emerging therapies, can slow disease progression, sensitive tools to monitor functional decline-particularly in non-ambulant patients-remain limited.
Current assessments rely primarily on clinical scales and hospital-based evaluations, which may not detect subtle changes or reflect real-life function. Digital outcome measures derived from wearable sensors offer a promising approach for continuous, objective monitoring in daily life. This study aims to evaluate the feasibility, reliability, clinical validity, and sensitivity of digital measures to assess upper limb function in non-ambulant patients with genetically confirmed DMD. The Syde device, previously validated in ambulant DMD patients, will be investigated for its applicability in this population.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Being tested
- Performance of the Upper Limb (PUL) Test, Brook test, Dynamometric measurements of muscle strength, Forced Vital Capacity (FVC) test, Clinical Global Impression - Severity (CGI-S)
- Keywords
- Non-ambulant, digital endpoint, upper limb
- 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.
Performance of the Upper Limb (PUL) TestDiagnostic test
This test consists of a set of small manoeuvres (lifting a box, writing, etc.) designed to evaluate the upper-limb functionality of non-ambulatory patients. It was developed specifically for use in cases of Duchenne muscular dystrophy.
Brook testDiagnostic test
The Brooke Upper Extremity Functional Rating Scale will be used to assess the global functional level of upper limb involvement. This 6-point ordinal scale classifies patients according to their highest achievable upper limb function, from full arm abduction to absence of useful hand function.
Dynamometric measurements of muscle strengthDiagnostic test
Dynamometric measurements of the maximum force of the following functions will be taken with the MyoTools: palmar grip (MyoGrip) and thumb-index pinch (MyoPinch). Test will be realized on the dominant side. Patients will be encouraged during the test. They will be given three trials and the best score will be entered.
Forced Vital Capacity (FVC) testDiagnostic test
Forced Vital Capacity (FVC) will be assessed using standardized spirometry procedures. The primary parameter will be FVC expressed as percentage of predicted values. At least three acceptable and reproducible maneuvers will be performed according to standard guidelines.
Clinical Global Impression - Severity (CGI-S)Diagnostic test
The CGI-S is a clinician-rated scale used at baseline to assess the overall severity of the participant's condition, based on all available clinical information. The rating reflects the clinician's judgment without standardized scoring rules and must be performed by a clinician experienced in Duchenne Muscular Dystrophy. The CGI-S evaluates three domains: physical motor function, respiratory function, and bulbar function. It is completed after all other study assessments (excluding patient-reported outcomes) to ensure a comprehensive evaluation.
Clinical Global Impression - Improvement (CGI-C)Diagnostic test
The CGI-C is used at each follow-up visit to assess changes in the participant's condition relative to baseline. It provides a clinician-determined measure of improvement or worsening, based on overall clinical judgment rather than fixed criteria. Like the CGI-S, it covers physical motor, respiratory, and bulbar domains and is performed after all other study assessments (excluding patient-reported outcomes). The CGI-C is expected to track consistently with prior CGI-S evaluations, reflecting changes in disease status over time.
Patient Global Impression of severity (PGI-S)Other
The PGI-S (Patient Global Impression of Severity) is a simple, validated, single-item self-administered scale used to assess the current severity of a patient's condition. Widely used in clinical trials, it allows patients to rate their condition, on a 4- to 6-point scale (from 'Normal' to 'Very severe').
SydeDevice
Syde is an innovative device intended to be used in a home-based environment. It is composed of two watch-like sensors, each containing a magneto-inertial sensors that record the linear acceleration, the angular velocity, the magnetic field of the movement in all directions.The two watches can be worn as wristwatch or placed near the ankle.
| Study group | Role | What they receive |
|---|---|---|
| Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) | Experimental | Not described Receives: Diagnostic Test: Performance of the Upper Limb (PUL) Test; Diagnostic Test: Brook test; Diagnostic Test: Dynamometric measurements of muscle strength; Diagnostic Test: Forced Vital Capacity (FVC) test; Diagnostic Test: Clinical Global Impression - Severity (CGI-S); Diagnostic Test: Clinical Global Impression - Improvement (CGI-C); Other: Patient Global Impression of severity (PGI-S); Device: Syde |
What is being measured
The primary outcome is the main question the study is designed to answer.
Device usage (recording time)
Total recording time per recording period (hours)
Measured: Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Patient compliance (Min)
Percentage of participants achieving minimal threshold (expected 50h) of recording time per period
Measured: Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Patient compliance (Max)
Percentage of participants achieving optimal threshold (expected 180h) of recording time per period
Measured: Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Secondary outcomes (4)
Reliability
Intraclass Correlation Coefficient (ICC) for two distinct consecutive half-periods
Measured: Baseline, Month 6, Month 12, Month 18, Month 24
Clinical validity of digital outcome vs Brooke
Correlation between digital outcome and Brooke Upper Extremity Rating Scale (Brooke score)
Measured: Baseline, Month 6, Month 12, Month 18, Month 24
Clinical validity of digital outcome vs PUL
Correlation between digital outcome and Performance of Upper Limb (PUL) score
Measured: Baseline, Month 6, Month 12, Month 18, Month 24
Sensitivity of Digital Upper Limb Outcome Measures
Sensitivity will be evaluated by assessing the ability of digital outcome measures to detect change over time combining mean change from baseline, slope of change over time, and standardized response mean (SRM).
Measured: Baseline, Month 6, Month 12, Month 18, Month 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Jun 2026
- Study start30 Jul 2026 (estimated)
- Primary completion30 Jun 2029 (estimated)
- Study completion30 Apr 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Centre Hospitalier Universitaire de Liege
- Sponsor type
- University, hospital or other
- Collaborators
- Centre Hospitalier Régional de la Citadelle, SYSNAV
- Responsible party
- Principal investigator, Laurent Servais, Centre Hospitalier Universitaire de Liege
- Investigators
- Laurent Servais, MD, Principal investigator, Centre Hospitalier Universitaire de Liege
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07664124
- Sponsor's protocol ID
- Acti-nRoll
- Acronym
- Acti-nRoll
- Status last verified
- Jun 2026
- First submitted
- 15 Jun 2026
- Last updated
- 23 Jun 2026
- 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 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility, Reliability, Clinical Validity and Sensitivity of Digital Outcomes to Monitor Upper Limb Function in Non-ambulant Patients With Genetically Confirmed Duchenne Muscular Dystrophy (DMD)
- ClinicalTrials.gov ID
- NCT07664124
- Acronym / sponsor ID
- Acti-nRoll / Acti-nRoll
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Non-ambulant, digital endpoint, upper limb
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Centre Hospitalier Universitaire de LiegeUniversity, hospital or other
- Collaborators
- Centre Hospitalier Régional de la Citadelle, SYSNAV
- Study start
- 30 Jul 2026 (estimated)
- Primary completion
- 30 Jun 2029 (estimated)
- Study completion
- 30 Apr 2030 (estimated)
- First posted
- 23 Jun 2026 (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.
Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder caused by the absence of dystrophin, leading to progressive muscle degeneration. Symptoms typically begin in early childhood and result in loss of ambulation by early adolescence, followed by cardiorespiratory complications. Although early treatment, including corticosteroids and emerging therapies, can slow disease progression, sensitive tools to monitor functional decline-particularly in non-ambulant patients-remain limited.
Current assessments rely primarily on clinical scales and hospital-based evaluations, which may not detect subtle changes or reflect real-life function. Digital outcome measures derived from wearable sensors offer a promising approach for continuous, objective monitoring in daily life. This study aims to evaluate the feasibility, reliability, clinical validity, and sensitivity of digital measures to assess upper limb function in non-ambulant patients with genetically confirmed DMD. The Syde device, previously validated in ambulant DMD patients, will be investigated for its applicability in this population.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) | Experimental | Diagnostic Test: Performance of the Upper Limb (PUL) Test, Diagnostic Test: Brook test, Diagnostic Test: Dynamometric measurements of muscle strength, Diagnostic Test: Forced Vital Capacity (FVC) test, Diagnostic Test: Clinical Global Impression - Severity (CGI-S), Diagnostic Test: Clinical Global Impression - Improvement (CGI-C), Other: Patient Global Impression of severity (PGI-S), Device: Syde |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- Patient with genetically confirmed Duchenne Muscular Dystrophy (DMD).
- Non-ambulant at the time of inclusion (not able to walk 10m without external aid).
- A legal guardian willing and able to provide written informed consent for participation in the study if < 18 years old.
Key exclusion criteria (4 listed)
- Any acute or chronic condition that, in the opinion of the investigator, may significantly interfere with the assessments and/or motor function progression.
- Participation in an interventional clinical trial.
- No access to internet connection or alternatively no capacity to come on-site to bring the Syde every 6 months after the recording periods for data retrieval by Liège team
- Scoliosis surgery within the previous 6 months or planned within the next year
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 |
|---|---|---|
| Performance of the Upper Limb (PUL) TestThis test consists of a set of small manoeuvres (lifting a box, writing, etc.) designed to evaluate the upper-limb functionality of non-ambulatory patients. It was developed specifically for use in cases of Duchenne muscular dystrophy. | Diagnostic test | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
| Brook testThe Brooke Upper Extremity Functional Rating Scale will be used to assess the global functional level of upper limb involvement. This 6-point ordinal scale classifies patients according to their highest achievable upper limb function, from full arm abduction to absence of useful hand function. | Diagnostic test | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
| Dynamometric measurements of muscle strengthDynamometric measurements of the maximum force of the following functions will be taken with the MyoTools: palmar grip (MyoGrip) and thumb-index pinch (MyoPinch). Test will be realized on the dominant side. Patients will be encouraged during the test. They will be given three trials and the best score will be entered. | Diagnostic test | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
| Forced Vital Capacity (FVC) testForced Vital Capacity (FVC) will be assessed using standardized spirometry procedures. The primary parameter will be FVC expressed as percentage of predicted values. At least three acceptable and reproducible maneuvers will be performed according to standard guidelines. | Diagnostic test | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
| Clinical Global Impression - Severity (CGI-S)The CGI-S is a clinician-rated scale used at baseline to assess the overall severity of the participant's condition, based on all available clinical information. The rating reflects the clinician's judgment without standardized scoring rules and must be performed by a clinician experienced in Duchenne Muscular Dystrophy. The CGI-S evaluates three domains: physical motor function, respiratory function, and bulbar function. It is completed after all other study assessments (excluding patient-reported outcomes) to ensure a comprehensive evaluation. | Diagnostic test | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
| Clinical Global Impression - Improvement (CGI-C)The CGI-C is used at each follow-up visit to assess changes in the participant's condition relative to baseline. It provides a clinician-determined measure of improvement or worsening, based on overall clinical judgment rather than fixed criteria. Like the CGI-S, it covers physical motor, respiratory, and bulbar domains and is performed after all other study assessments (excluding patient-reported outcomes). The CGI-C is expected to track consistently with prior CGI-S evaluations, reflecting changes in disease status over time. | Diagnostic test | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
| Patient Global Impression of severity (PGI-S)The PGI-S (Patient Global Impression of Severity) is a simple, validated, single-item self-administered scale used to assess the current severity of a patient's condition. Widely used in clinical trials, it allows patients to rate their condition, on a 4- to 6-point scale (from 'Normal' to 'Very severe'). | Other | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
| SydeSyde is an innovative device intended to be used in a home-based environment. It is composed of two watch-like sensors, each containing a magneto-inertial sensors that record the linear acceleration, the angular velocity, the magnetic field of the movement in all directions.The two watches can be worn as wristwatch or placed near the ankle. | Device | Non-ambulant participants with Duchenne Muscular Dystrophy (DMD) |
Primary outcome
Device usage (recording time)
Time frame: Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Total recording time per recording period (hours)
Patient compliance (Min)
Time frame: Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Percentage of participants achieving minimal threshold (expected 50h) of recording time per period
Patient compliance (Max)
Time frame: Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24
Percentage of participants achieving optimal threshold (expected 180h) of recording time per period
4 secondary outcomes
Reliability
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24
Clinical validity of digital outcome vs Brooke
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24
Clinical validity of digital outcome vs PUL
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24
Sensitivity of Digital Upper Limb Outcome Measures
Time frame: Baseline, Month 6, Month 12, Month 18, Month 24
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
- Individual participant dataSharing planned: No
Related trials for Duchenne Muscular Dystrophy (DMD)
RecruitingEarly Phase 1
Safety and Dystrophin Expression of SPOT-03 in Duchenne Muscular Dystrophy (DMD) Patients
Duchenne Muscular Dystrophy (DMD)
RecruitingPhase 2
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy
Not yet recruitingPhase 2
A Phase 2 Trial to Investigate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Tegacorat in Male Participants 4 to <7 Years of Age With Duchenne Muscular Dystrophy
Duchenne Muscular Dystrophy (DMD)
Not yet recruitingObservational study
Multimodal Study About Absence of Dp140 in Becker Muscular Dystrophy: Brain-muscle Imaging, Cognition and Muscle Function
Becker Muscular Dystrophy
Enrolling by invitationObservational study
A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice
Duchenne Muscular Dystrophy
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.
Laurent Servais, MD principal investigator
Centre Hospitalier Universitaire de Liege
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Performance of the Upper Limb (PUL) Test, Brook test and Dynamometric measurements of muscle strength in people with Duchenne Muscular Dystrophy (DMD), run by Centre Hospitalier Universitaire de Liege at 1 site in Belgium.
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 3 inclusion and 4 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: opening soon, as of the sponsor's last update on 23 Jun 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 Belgium. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Dounia Chelha (+3243215498, Khadidja.Chelha@citadelle.be), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Device usage (recording time) (time frame: Recording periods at Baseline, Month 6, Month 12, Month 18, Month 24). 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 23 Jun 2026.