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Motor Parameters in Patients With Limb Girdle Muscular Dystrophy (EIDY)

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ClinicalTrials.gov Identifier: NCT04772027
Recruitment Status : Recruiting
First Posted : February 25, 2021
Last Update Posted : May 3, 2022
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris

Brief Summary:

The primary objective of the study is to perform 2-year follow up with motor parameters evolution using instrumental assessments in patients with limb girdle muscular dystrophie, and to identify which motor parameters are sensitive to change.

The secondary objectives of the study are:

  • to describe the changes of the parameters obtained from instrumental evaluations in comparison with the changes obtained from clinical assessments.
  • to characterize the muscular impairments, the biomechanical gait disorders, the standing postural control disorders, the biomechanical upper limb disorders in spatial exploration, the limitation of upper limb capacities, the fatigue, the endurance, the patients' participation to their activities and their quality of life, in comparison with a healthy paired population.
  • to highlight the relationships between muscular assessment parameters, biomechanical gait parameters, standing posture control and upper limb spatial exploration.
  • to highlight the relationships between data from instrumental assessments and data from clinical assessments.
  • to highlight the relationships between instrumental assessments data and clinical assessments data on one side, and features of patients (age, sex, duration since diagnosis, type of LGMD, rehabilitation in follow-up, sportive practices...) on the other side.

Condition or disease
Limb Girdle Muscular Dystrophies

Detailed Description:
This is a monocentric study in which 2 sub-groups of people will be enrolled: LGMD patients and healthy volunteers. Each enrolled LGMD patient will have 5 visits during 2 years, one baseline visit and four half-yearly visits. Each enrolled healthy volunteer will have one planned visit only. The duration of enrollments will last 20 months.

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Study Type : Observational
Estimated Enrollment : 80 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Characterization and Identification of Motor Parameters Using Instrumental Assessment in Patients With Limb Girdle Muscular Dystrophy
Actual Study Start Date : April 1, 2021
Estimated Primary Completion Date : January 2025
Estimated Study Completion Date : January 2025


Group/Cohort
Muscular dystrophies group
40 patients with limb girdle muscular dystrophie
Comparator group
40 healthy volunteers without neuromuscular or squeletic disorder



Primary Outcome Measures :
  1. Change from baseline on maximal strength [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Maximal strength and muscular activation of knee extensors will be evaluated by isometric test with an isokinetic dynamometer (Biodex Medical Systems Inc., Shirley, New York, USA).

  2. Change from baseline on peak hip flexion [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Hip flexion peak during gait will be evaluated with tridimensional gait analysis with an optoelectronic system ((Optitrack system, Natural Point Inc. Corvallis, OR, USA)

  3. Change from baseline on speed of center of pression during standing postural control [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Speed of center of pression will be measured by force platforms (AMTI, Advanced Mechanical Technology).

  4. Change from baseline on elbow peak extension during upper limb spatial exploration [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Elbow peak extension will be measured biomechanically with an optoelectronic system during upper limb spatial exploration (Optitrack system, Natural Point Inc. Corvallis, OR, USA)


Secondary Outcome Measures :
  1. Change from baseline on muscular parameters [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Maximal strength during isometric tests of hip, knee flexors, ankle and maximal grip strength. (Biodex Medical Systems Inc., Shirley, New York, USA) Muscular fatigue

  2. Joint kinematic parameters: change from baseline on gait speed [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used.

    Spatio-temporal = speed (m/s)


  3. Joint kinematic parameters: change from baseline on step length [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used.

  4. Joint kinematic parameters: change from baseline on cadence [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used.

  5. Joint kinematic parameters: change from baseline on step width [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used.

  6. Joint kinematic parameters: change from baseline on % single support phase of gait cycle [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used.

  7. Joint kinematic parameters: change from baseline on % swing phase of gait cycle [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used.

  8. Joint kinematic parameters: change from baseline on % support phase of gait cycle [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used.

  9. Change from baseline on kinetic gait parameters [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Kinetic parameters will be obtained from biomechanical gait analysis with AMTI force plateforms (Advanced Mechanical Technology, Waterton, MA, USA).

    This includes peak of ground reaction force in antero-posterior and vertical axis.


  10. Change from baseline on standing postural control parameters: COP displacements [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Parameters of stabilization and postural orientation during postural control assessment on force platforms (AMTI, Advanced Mechanical Technology), Waterton, MA, USA). Parameters of stabilization include:

    Mean and maximal speed of center of pression (COP) displacements.


  11. Change from baseline on standing postural control parameters: elliptic surface [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Parameters of stabilization and postural orientation during postural control assessment on force platforms (AMTI, Advanced Mechanical Technology), Waterton, MA, USA). Parameters of stabilization include:

    Elliptic surface covering 90% of COP positions.


  12. Change from baseline on standing postural control parameters: Amplitude of COP [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Parameters of stabilization and postural orientation during postural control assessment on force platforms (AMTI, Advanced Mechanical Technology), Waterton, MA, USA). Parameters of stabilization include:

    Amplitude of COP shift in anteroposterior and mediolateral axis.


  13. Change from baseline on standing postural control parameters: Romberg quotient [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Parameters of stabilization and postural orientation during postural control assessment on force platforms (AMTI, Advanced Mechanical Technology), Waterton, MA, USA). Parameters of stabilization include:

    Romberg quotient.


  14. Change from baseline on standing postural orientation parameters [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Parameters of stabilization and postural orientation during postural control assessment on force platforms (AMTI, Advanced Mechanical Technology), Waterton, MA, USA).

    The orientation parameters include: mean position of COP in anteroposterior and mediolateral axis.


  15. Change from baseline on standing postural orientation parameters: limb loading ratio [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    Parameters of stabilization and postural orientation during postural control assessment on force platforms (AMTI, Advanced Mechanical Technology), Waterton, MA, USA).

    The orientation parameters include: limb loading ratio.


  16. Change from baseline on drinking task with left and right upper limb: velocity and movement time [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    The spatio-temporal parameters will be obtained from biomechanical analysis with an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) including:

    Velocity and movement time (Total movement time / Peak velocity/ Mean velocity)


  17. Change from baseline on drinking task with left and right upper limb: movement strategy [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    The spatio-temporal parameters will be obtained from biomechanical analysis with an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) including:

    Movement strategy (Time to peak velocity / time to first peak)


  18. Change from baseline on drinking task with left and right upper limb: smoothness and coordination movement [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    The spatio-temporal parameters will be obtained from biomechanical analysis with an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) including:

    Smoothness and coordination movement (number of movements units / interjoint coordination)


  19. Change from baseline on drinking task with left and right upper limb: trunk displacement [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    The spatio-temporal parameters will be obtained from biomechanical analysis with an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) including:

    Trunk displacement


  20. Change from baseline on drinking task with left and right upper limb: angular joint [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]

    The spatio-temporal parameters will be obtained from biomechanical analysis with an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) including:

    Angular joint


  21. Change from baseline on 6 minutes walking test distance [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    6 minutes walking test

  22. Change from baseline on the Berg Balance Scale score [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    The Berg Balance Scale

  23. Change from baseline on Brooke Upper Extremity Scale score [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Brooke Upper Extremity Scale score for upper extremity capacities.

  24. Change from baseline on locomotor [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Questionnaires Abiloco

  25. Change from baseline on upper limb capacities [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Questionnaires Abilhand

  26. Change from baseline on patient occupations [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Patient occupations assessed by COPM (Canadian Occupational Performance Measure). The measurement will be from 1 (not at all performant) to 10 (very performant) as score.

  27. Change from baseline on MRC (Medical Research Council) score on flexors and extensors of lower and upper limb [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Testing MRC (Medical Research Council), a scale for muscle power. The muscle scale grades muscle power on a scale of 0 to 5 in relation to the maximum expected for that muscle.

  28. Change from baseline on Fatigue Severity Scale (FSS) score [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Questionnaire Fatigue Severity Scale (FSS) for fatigue assessment

  29. Change from baseline on number of fall risks [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
  30. Change from baseline on number of physiotherapy sessions per week [ Time Frame: at baseline, 6 months, 12 months, 18 months and 24 months ]
  31. Change from baseline on number of sports practice per week [ Time Frame: at baseline, 6 months, 12 months, 18 months and 24 months ]
  32. Change from baseline on Individualized Neuromuscular Quality of Life Questionnaire (INQoL) [ Time Frame: Assessed at 6 months, 12 months, 18 months and 24 months ]
    Quality of life questionnaire: INQoL



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population

Population with limb girdle muscular dystrophie for 2-year longitudinal follow-up in Raymond Poincaré hospital. The 4 most common types of diseases: calpainopathy, sarcoglycanopathy, dysferlinopathy and alpha-dystroglycanopathy.

Control group: healthy population without neuromuscular or squeletic disorder.

Criteria

Inclusion Criteria:

  • For limb girdle muscular dystrophie group:

    • Patients diagnosed limb girdle muscular dystrophie;
    • Aged between 18 and 70 years;
    • Covered by the French social security scheme;
    • Patient able to maintain upright position alone;
    • Able to walk at least 10 meters and 6 minutes consecutively without help.
  • For Volunteer group:

    • Adult (18 to 70 years) without neuromuscular or squeletic disorder.

Exclusion Criteria:

§ For all:

  • Without associated neuromuscular disorders such as respiratory or muscular-squeletic diseases, apart from the consequences of dystrophy;
  • Contraindication to the tests;
  • Noncontrol cardiac rhythm disorders;
  • Lying down position intolerance due to severe respiratory troubles;
  • Pregnant or breastfeeding woman.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04772027


Contacts
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Contact: Céline BONNYAUD, PhD +33 (0)1 71 14 49 21 celine.bonnyaud@aphp.fr
Contact: Samuel POUPLIN +33 (0)1 47 10 70 61 samuel.pouplin@aphp.fr

Locations
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France
Laboratoire d'analyse du mouvement, Service d'exploration fonctionnelles, Hôpital Raymond Poincaré, APHP Recruiting
Garche, France, 92380
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
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Principal Investigator: Céline BONNYAUD, PhD Laboratoire d'analyse du mouvement, Service d'explorations fonctionnelles, Hôpital Raymond Poincaré, APHP
Study Director: Samuel POUPLIN Plateforme Nouvelles Technologies, Hôpital Raymond Poincaré, APHP
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Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT04772027    
Other Study ID Numbers: APHP200141
2020-A02090-39 ( Registry Identifier: IDRCB )
First Posted: February 25, 2021    Key Record Dates
Last Update Posted: May 3, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Assistance Publique - Hôpitaux de Paris:
Limb Girdle Muscular Dystrophies (LGMD)
motricity
instrumental assessment
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophies, Limb-Girdle
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn