Motor Parameters in Patients With Limb Girdle Muscular Dystrophy

Recruiting

• Conditions: Limb Girdle Muscular Dystrophies

• Registration Number: NCT04772027
• Lead Sponsor: 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.

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.

Study Timeline

• Study First Submitted: 2021-01-07
• Study First Submitted QC: 2021-02-24
• Study First Posted: 2021-02-25
• Study Start: 2021-04-01
• Status Verified: 2023-09
• Last Update Submitted: 2023-09-04
• Last Update Posted: 2023-09-06
• Primary Completion: 2025-01
• Study Completion: 2025-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 80

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change from baseline on peak hip flexion	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)
Change from baseline on maximal strength	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).
Change from baseline on speed of center of pression during standing postural control	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).
Change from baseline on elbow peak extension during upper limb spatial exploration	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

Name	Time	Method
Change from baseline on muscular parameters	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
Joint kinematic parameters: change from baseline on step length	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.
Change from baseline on standing postural control parameters: Romberg quotient	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.
Joint kinematic parameters: change from baseline on step width	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.
Change from baseline on drinking task with left and right upper limb: velocity and movement time	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)
Change from baseline on drinking task with left and right upper limb: trunk displacement	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
Joint kinematic parameters: change from baseline on gait speed	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)
Joint kinematic parameters: change from baseline on % swing phase of gait cycle	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.
Joint kinematic parameters: change from baseline on % support phase of gait cycle	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.
Change from baseline on kinetic gait parameters	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.
Change from baseline on locomotor	Assessed at 6 months, 12 months, 18 months and 24 months	Questionnaires Abiloco
Change from baseline on patient occupations	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.
Change from baseline on number of fall risks	Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on number of physiotherapy sessions per week	at baseline, 6 months, 12 months, 18 months and 24 months
Change from baseline on Individualized Neuromuscular Quality of Life Questionnaire (INQoL)	Assessed at 6 months, 12 months, 18 months and 24 months	Quality of life questionnaire: INQoL
Joint kinematic parameters: change from baseline on % single support phase of gait cycle	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.
Change from baseline on standing postural control parameters: COP displacements	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.
Change from baseline on standing postural orientation parameters	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.
Change from baseline on drinking task with left and right upper limb: movement strategy	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)
Change from baseline on drinking task with left and right upper limb: angular joint	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
Joint kinematic parameters: change from baseline on cadence	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.
Change from baseline on standing postural control parameters: elliptic surface	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.
Change from baseline on standing postural control parameters: Amplitude of COP	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.
Change from baseline on drinking task with left and right upper limb: smoothness and coordination movement	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)
Change from baseline on number of sports practice per week	at baseline, 6 months, 12 months, 18 months and 24 months
Change from baseline on standing postural orientation parameters: limb loading ratio	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.
Change from baseline on 6 minutes walking test distance	Assessed at 6 months, 12 months, 18 months and 24 months	6 minutes walking test
Change from baseline on Brooke Upper Extremity Scale score	Assessed at 6 months, 12 months, 18 months and 24 months	Brooke Upper Extremity Scale score for upper extremity capacities.
Change from baseline on upper limb capacities	Assessed at 6 months, 12 months, 18 months and 24 months	Questionnaires Abilhand
Change from baseline on Fatigue Severity Scale (FSS) score	Assessed at 6 months, 12 months, 18 months and 24 months	Questionnaire Fatigue Severity Scale (FSS) for fatigue assessment
Change from baseline on the Berg Balance Scale score	Assessed at 6 months, 12 months, 18 months and 24 months	The Berg Balance Scale
Change from baseline on MRC (Medical Research Council) score on flexors and extensors of lower and upper limb	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.

Trial Locations

Locations (1)

Laboratoire d'analyse du mouvement, Service d'exploration fonctionnelles, Hôpital Raymond Poincaré, APHP; 🇫🇷 Garche, France