Feasibility, Validation and Application of Digital Tools for the Follow-up of Neuromuscular Patient Mobility in Daily Living
DT4RD
1 other identifier
interventional
40
2 countries
2
Brief Summary
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:
- Compare face to face clinical data collected in hospital with Patient Generated Data recorded remotely
- Examine how sensors can enhance measurement potentially at home and during clinical visits
- Promote a clear focus on user centered design and the integration of technology
- Use reliability and validity analyses to equate any common measures (those with the same or a similar construct)
- Demonstrate a proof-of-concept model into which different measures can be interchangeable
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for not_applicable
Started Jun 2023
2 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 8, 2023
CompletedFirst Posted
Study publicly available on registry
April 4, 2023
CompletedStudy Start
First participant enrolled
June 29, 2023
CompletedPrimary Completion
Last participant's last visit for primary outcome
June 30, 2024
CompletedStudy Completion
Last participant's last visit for all outcomes
June 30, 2024
CompletedSeptember 28, 2023
September 1, 2023
1 year
March 8, 2023
September 26, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Global home evaluations' attendance
Proportion of home assessments carried out compared to the number of home assessments planned
Through study completion, an average of 1 year
Secondary Outcomes (8)
Compliance with filling out questionnaires at home
Through study completion, an average of 1 year
Compliance regarding using of the spirometer at home
Through study completion, an average of 1 year
Compliance regarding using of the LINKS sensors device at home
Through study completion, an average of 1 year
Compliance regarding using of the activity sensor at home
Through study completion, an average of 1 year
Compliance regarding achievement of video tasks at home
Through study completion, an average of 1 year
- +3 more secondary outcomes
Study Arms (1)
Principal
OTHERInterventions
Motor Function Measurement (MFM) is a quantitative scale created to measure global motor functional abilities in a person with neuromuscular disease.
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.
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.
The Performance of the Upper Limb (PUL) is a functional scale specifically designed for assessing upper limb function in Duchenne muscular dystrophy.
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.
The NSAD was developed to measure motor performance in ambulant and non-ambulant subjects as part of the clinical outcome study of dysferlinopathy.
This scale is derived from the North Star Ambulatory Assessment and several items from the Egan Klassifikation Scale (EK2).
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.
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.
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.
This questionnaire assesses overall physical activity and the level of sedentary lifestyle during the last seven days.
The EasyAngle® is a digital goniometer improving long established goniometric functionality with rotation, inclination, and scoliosis measurement capabilities.
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.
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.
Eligibility Criteria
You may qualify if:
- Aged between 12 and 60 years
- Patients with a genetically confirmed/molecular-proven neuromuscular or neurometabolic disease\*
- Patients experiencing walking difficulties in the home and at high risk of limiting participation and walking outside of the home.
- Written informed consent
- Able to comply with all protocol requirements, including video recording
- Affiliated to or beneficiary of a social security scheme (for France)
You may not qualify if:
- Patients with undefined diagnosis or any diagnosis other than neuromuscular or neurometabolic disease
- Patient walking 10m in less than 10s
- Guardianship/trusteeship
- Pregnant or nursing women
- Patients having relevant concomitant pathologies that, in the appreciation of the investigator could interfere with protocol compliance
- Patients not being affiliated with local social security (for France)
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Institut de Myologie, Francelead
- Aparito Ltd.collaborator
Study Sites (2)
Association Institut de Myologie
Paris, 75013, France
John Walton Muscular Dystrophy Research Centre
Newcastle upon Tyne, NE1 3BZ, United Kingdom
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- OTHER
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 8, 2023
First Posted
April 4, 2023
Study Start
June 29, 2023
Primary Completion
June 30, 2024
Study Completion
June 30, 2024
Last Updated
September 28, 2023
Record last verified: 2023-09