An interventional study of 2MWT and MFM32 in Neuromuscular Diseases, sponsored by Institut de Myologie, France. Completed at 2 sites in 2 countries. Open to participants aged 12 Years to 60 Years. Per ClinicalTrials.gov, last updated 2026-06-29.
Sponsored by Institut de Myologie, France · Not applicable, Interventional, and Other
The low prevalence of rare diseases hinders the design of clinical studies with sufficient statistical power to demonstrate the efficacy of new drugs. This can only be achieved by setting up international multicentre studies, which is challenging due to a lack of objective, universal outcome measures that generate high-quality, reproducible data. One of the hurdles in attaining universal outcome measures for clinical trials is the difficulty to capture and distinguish ambulatory from non-ambulatory, autonomous and assistive or involuntary movements. This makes a trial assessing the ambulatory phase very challenging at this moment. Excluding many participants from trials and many patients from access to medication.
Integration and validation of the technology in trials, research and patients' lives is essential in overcoming this hurdle. For example, in dystrophinopathies separate outcome measures exist for ambulant and non-ambulant participants, but the relation between these outcome measures or a transitional outcome measure/end point is largely missing.
Following an exhaustive literature review, several tools have been selected to remotely follow various symptoms of neuromuscular patients including weakness, pain, fatigue, cognitive defects, motor impairments (including loss of dexterity, ataxia...), metabolic, respiratory and cardiac troubles, contractures, tremor, falls, hypo or hypersomnia... The toolbox includes common measures for all patients but may include additional measures specific to the patient's symptoms (hence in turn to the patients' disease).
The measurements are designed to not be invasive, intrusive or burdensome for the patient.
DT4RD is going to leverage state-of-the art technology, clinical rating scales and psychometric/data analysis to deliver fit for purpose remote clinical assessments of mobility to ensure maximum patient benefit, specifically:
361 studies on the registry are indexed under Neuromuscular Diseases; 97 are open to participants now.
This study's enrollment of 40 is above the median of 30 across 204 interventional studies indexed under Neuromuscular Diseases.
Browse Neuromuscular Diseases studies →Institut de Myologie, France is the lead sponsor of 30 studies on the registry; 5 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Other: 2MWT · Other: MFM32 · Other: MyoGrip · Other: QOL-gNMD · Diagnostic Test: Spirometry · Device: Acceleromerty · Other: 10mWT · Other: PUL · Other: NSAA · Other: NSAD · Other: TANS · Other: MyoPinch · Other: MyoQuad · Other: ACTIVLIM · Other: PREM · Other: SF-MPQ · Other: FSS · Other: IPAQ · Other: Rang of motion · Device: Goniometry · Other: Video captured monitoring · Device: Activity monitoring
Two minutes walk tests.
Also known as: Two minutes walk tests
Motor Function Measurement (MFM) is a quantitative scale created to measure global motor functional abilities in a person with neuromuscular disease.
Also known as: Muscle Motor Function 32
The MyoGrip is a dynamometer for measuring grip strength.
The "Quality of Life in genetic Neuromuscular Disease" questionnaire (QoL-gNMD) is a patient reported outcome measure (PROM) tool specifically designed for patients with a lowly progressive neuromuscular disease with genetically predominant muscular damage
Forced vital capacity (FVC), peak expiratory flow (PEF) and forced expiratory volume in 1 second (FEV1) will be measured in the hospital with the local hospital device (e.g. Vitalograph) under the supervision of the clinical evaluator, and at home with a portable device.
Also known as: Spirobank Smart
The Links sensor system is a measuring device and consists of the Links Pods and the Links Hub. The Links pods contain an accelerometer, gyroscope and magnetometer to measure position relative to a global reference frame.
Also known as: Links sensor system
10 meters walking Test.
The Performance of the Upper Limb (PUL) is a functional scale specifically designed for assessing upper limb function in Duchenne muscular dystrophy.
Also known as: Performance of Upper Limb
The North Star Ambulatory Assessment (NSAA) is a 17-item rating scale that is used to measure functional motor abilities in ambulant children with Duchenne Muscular Dystrophy.
Also known as: North Star Ambulatory Assessment
The NSAD was developed to measure motor performance in ambulant and non-ambulant subjects as part of the clinical outcome study of dysferlinopathy.
Also known as: North Star Assessment for limb-girdle type muscular dystrophies
This scale is derived from the North Star Ambulatory Assessment and several items from the Egan Klassifikation Scale (EK2).
Also known as: Transition Assessment North Star
The MyoPinch is a high-precision dynamometer that allows the evaluation of key pinch strength.
The MyoQuad is a high-precision dynamometer that allows the evaluation of knee extension strength.
Activity limitations, as measured by ACTIVLIM, is defined as the difficulties a patient may have in executing daily activities, whatever the strategies involved.
This questionnaire is made up of simple questions relating to the assessments and assessment tools that were used during the study.
Also known as: Patient Reported Experience Measures
The main component of the SF-MPQ consists of 15 descriptors (11 sensories; 4 affective) which are rated on an intensity scale as 0 = none, 1 = mild, 2 = moderate or 3 = severe.
Also known as: Modified Short-form McGill Pain Questionnaire
The Fatigue Severity Scale is used to assess the severity of fatigue and its impact on daily living. The patient responds on a Likert-type scale ranging from 1 to 7.
Also known as: Fatigue Severity Scale
This questionnaire assesses overall physical activity and the level of sedentary lifestyle during the last seven days.
Also known as: International Physical Activity Questionnaire
The EasyAngle® is a digital goniometer improving long established goniometric functionality with rotation, inclination, and scoliosis measurement capabilities.
Also known as: EasyAngle
The Links sensor system is a measuring device and consists of the Links Pods and the Links Hub. The Links pods contain an accelerometer, gyroscope and magnetometer to measure position relative to a global reference frame.
Also known as: Links Sensor System goniometers
Once every four weeks, while wearing the Yumen electronic goniometers (Links Sensor System), the patients will be asked to record on video three tasks related to: 1. Upper limbs: hands to head 2. Lower limbs: Sit to stand with or without the help of a person 3. Self-defined important task: A task of the patient's choice, from a list of tasks, representative of his/her motor difficulties or hindrance to autonomy.
Wearable devices (connected watch) will collect health-related data on a 24/7 basis remotely as patients go through their daily routines at home and work.
Also known as: Activity assessment and physiological parameters
Global home evaluations' attendance
Proportion of home assessments carried out compared to the number of home assessments planned
Time frame: Through study completion, an average of 1 year
Compliance with filling out questionnaires at home
Analysis of the proportion of replies to the questionnaires at home
Time frame: Through study completion, an average of 1 year
Compliance regarding using of the spirometer at home
Analysis of the proportion of valid spirometry data collected at home
Time frame: Through study completion, an average of 1 year
Compliance regarding using of the LINKS sensors device at home
Analysis of the proportion of usable accelerometry data collected at home
Time frame: Through study completion, an average of 1 year
Compliance regarding using of the activity sensor at home
Analysis of the proportion of usable data collected at home using the smartwatch
Time frame: Through study completion, an average of 1 year
Compliance regarding achievement of video tasks at home
Analysis of the usability of the video tasks performed at home
Time frame: Through study completion, an average of 1 year
Correlation of home and hospital spirometer Forced vital capacity measurements
Comparison of Forced vital capacity data collected during home and hospital spirometer measurements
Time frame: Through study completion, an average of 1 year
Correlation of home and hospital spirometer peak expiratory flow measurements
Comparison of Peak expiratory flow data collected during home and hospital spirometer measurements
Time frame: Through study completion, an average of 1 year
Correlation of home and hospital spirometer forced expiratory volume in 1 second measurements
Comparison of forced expiratory volume in 1 second data collected during home and hospital spirometer measurements
Time frame: Through study completion, an average of 1 year
Plan to share: Undecided
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This study is completed, as verified in Jun 2026. You cannot join it, but the record below documents what was studied.
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Institut de Myologie, France