Feasibility, Validation and Application of Digital Tools for the Follow-up of Neuromuscular Patient Mobility in Daily Living

NCT ID: NCT05798325

Last Updated: 2023-09-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

40 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-06-29

Study Completion Date

2024-06-30

Brief Summary

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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

Detailed Description

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Conditions

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Neuromuscular Diseases

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

OTHER

Blinding Strategy

NONE

Study Groups

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Principal

Group Type OTHER

2MWT

Intervention Type OTHER

Two minutes walk tests.

MFM32

Intervention Type OTHER

Motor Function Measurement (MFM) is a quantitative scale created to measure global motor functional abilities in a person with neuromuscular disease.

MyoGrip

Intervention Type OTHER

The MyoGrip is a dynamometer for measuring grip strength.

QOL-gNMD

Intervention Type OTHER

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

Spirometry

Intervention Type DIAGNOSTIC_TEST

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.

Acceleromerty

Intervention Type 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.

10mWT

Intervention Type OTHER

10 meters walking Test.

PUL

Intervention Type OTHER

The Performance of the Upper Limb (PUL) is a functional scale specifically designed for assessing upper limb function in Duchenne muscular dystrophy.

NSAA

Intervention Type OTHER

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.

NSAD

Intervention Type OTHER

The NSAD was developed to measure motor performance in ambulant and non-ambulant subjects as part of the clinical outcome study of dysferlinopathy.

TANS

Intervention Type OTHER

This scale is derived from the North Star Ambulatory Assessment and several items from the Egan Klassifikation Scale (EK2).

MyoPinch

Intervention Type OTHER

The MyoPinch is a high-precision dynamometer that allows the evaluation of key pinch strength.

MyoQuad

Intervention Type OTHER

The MyoQuad is a high-precision dynamometer that allows the evaluation of knee extension strength.

ACTIVLIM

Intervention Type OTHER

Activity limitations, as measured by ACTIVLIM, is defined as the difficulties a patient may have in executing daily activities, whatever the strategies involved.

PREM

Intervention Type OTHER

This questionnaire is made up of simple questions relating to the assessments and assessment tools that were used during the study.

SF-MPQ

Intervention Type OTHER

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.

FSS

Intervention Type OTHER

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.

IPAQ

Intervention Type OTHER

This questionnaire assesses overall physical activity and the level of sedentary lifestyle during the last seven days.

Rang of motion

Intervention Type OTHER

The EasyAngle® is a digital goniometer improving long established goniometric functionality with rotation, inclination, and scoliosis measurement capabilities.

Goniometry

Intervention Type 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.

Video captured monitoring

Intervention Type OTHER

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.

Activity monitoring

Intervention Type DEVICE

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.

Interventions

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2MWT

Two minutes walk tests.

Intervention Type OTHER

MFM32

Motor Function Measurement (MFM) is a quantitative scale created to measure global motor functional abilities in a person with neuromuscular disease.

Intervention Type OTHER

MyoGrip

The MyoGrip is a dynamometer for measuring grip strength.

Intervention Type OTHER

QOL-gNMD

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

Intervention Type OTHER

Spirometry

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.

Intervention Type DIAGNOSTIC_TEST

Acceleromerty

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.

Intervention Type DEVICE

10mWT

10 meters walking Test.

Intervention Type OTHER

PUL

The Performance of the Upper Limb (PUL) is a functional scale specifically designed for assessing upper limb function in Duchenne muscular dystrophy.

Intervention Type OTHER

NSAA

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.

Intervention Type OTHER

NSAD

The NSAD was developed to measure motor performance in ambulant and non-ambulant subjects as part of the clinical outcome study of dysferlinopathy.

Intervention Type OTHER

TANS

This scale is derived from the North Star Ambulatory Assessment and several items from the Egan Klassifikation Scale (EK2).

Intervention Type OTHER

MyoPinch

The MyoPinch is a high-precision dynamometer that allows the evaluation of key pinch strength.

Intervention Type OTHER

MyoQuad

The MyoQuad is a high-precision dynamometer that allows the evaluation of knee extension strength.

Intervention Type OTHER

ACTIVLIM

Activity limitations, as measured by ACTIVLIM, is defined as the difficulties a patient may have in executing daily activities, whatever the strategies involved.

Intervention Type OTHER

PREM

This questionnaire is made up of simple questions relating to the assessments and assessment tools that were used during the study.

Intervention Type OTHER

SF-MPQ

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.

Intervention Type OTHER

FSS

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.

Intervention Type OTHER

IPAQ

This questionnaire assesses overall physical activity and the level of sedentary lifestyle during the last seven days.

Intervention Type OTHER

Rang of motion

The EasyAngle® is a digital goniometer improving long established goniometric functionality with rotation, inclination, and scoliosis measurement capabilities.

Intervention Type OTHER

Goniometry

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.

Intervention Type DEVICE

Video captured monitoring

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.

Intervention Type OTHER

Activity monitoring

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.

Intervention Type DEVICE

Other Intervention Names

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Two minutes walk tests Muscle Motor Function 32 Spirobank Smart Links sensor system Performance of Upper Limb North Star Ambulatory Assessment North Star Assessment for limb-girdle type muscular dystrophies Transition Assessment North Star Patient Reported Experience Measures Modified Short-form McGill Pain Questionnaire Fatigue Severity Scale International Physical Activity Questionnaire EasyAngle Links Sensor System goniometers Activity assessment and physiological parameters

Eligibility Criteria

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Inclusion Criteria

* 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)

Exclusion Criteria

* 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)
Minimum Eligible Age

12 Years

Maximum Eligible Age

60 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Aparito Ltd.

INDUSTRY

Sponsor Role collaborator

Institut de Myologie, France

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Association Institut de Myologie

Paris, , France

Site Status RECRUITING

John Walton Muscular Dystrophy Research Centre

Newcastle upon Tyne, , United Kingdom

Site Status NOT_YET_RECRUITING

Countries

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France United Kingdom

Facility Contacts

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Vincent MD Varlet

Role: primary

+33 1 42 16 58 58

Volker STRAUB, MD

Role: primary

Other Identifiers

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DT4RD

Identifier Type: -

Identifier Source: org_study_id

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