Limb Girdle Muscular Dystrophies Clinical Trial
— EIDYOfficial title:
Characterization and Identification of Motor Parameters Using Instrumental Assessment in Patients With Limb Girdle Muscular Dystrophy
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.
| Status | Recruiting |
| Enrollment | 80 |
| Est. completion date | January 2025 |
| Est. primary completion date | January 2025 |
| Accepts healthy volunteers | Accepts Healthy Volunteers |
| Gender | All |
| Age group | 18 Years to 70 Years |
| Eligibility | 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: ยง 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. |
| Country | Name | City | State |
|---|---|---|---|
| France | Laboratoire d'analyse du mouvement, Service d'exploration fonctionnelles, Hôpital Raymond Poincaré, APHP | Garche |
| Lead Sponsor | Collaborator |
|---|---|
| Assistance Publique - Hôpitaux de Paris |
France,
| Type | Measure | Description | Time frame | Safety issue |
|---|---|---|---|---|
| Primary | Change from baseline on maximal strength | 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). | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Primary | Change from baseline on peak hip flexion | Hip flexion peak during gait will be evaluated with tridimensional gait analysis with an optoelectronic system ((Optitrack system, Natural Point Inc. Corvallis, OR, USA) | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Primary | Change from baseline on speed of center of pression during standing postural control | Speed of center of pression will be measured by force platforms (AMTI, Advanced Mechanical Technology). | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Primary | Change from baseline on elbow peak extension during upper limb spatial exploration | Elbow peak extension will be measured biomechanically with an optoelectronic system during upper limb spatial exploration (Optitrack system, Natural Point Inc. Corvallis, OR, USA) | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on muscular parameters | Maximal strength during isometric tests of hip, knee flexors, ankle and maximal grip strength. (Biodex Medical Systems Inc., Shirley, New York, USA) Muscular fatigue | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Joint kinematic parameters: change from baseline on gait speed | 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) |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Joint kinematic parameters: change from baseline on step length | Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Joint kinematic parameters: change from baseline on cadence | Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Joint kinematic parameters: change from baseline on step width | Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Joint kinematic parameters: change from baseline on % single support phase of gait cycle | Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Joint kinematic parameters: change from baseline on % swing phase of gait cycle | Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Joint kinematic parameters: change from baseline on % support phase of gait cycle | Spatio-temporal from biomechanical gait analysis: an optoelectronic system (Optitrack system, Natural Point Inc. Corvallis, OR, USA) will be used. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on kinetic gait parameters | 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. |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on standing postural control parameters: COP displacements | 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. |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on standing postural control parameters: elliptic surface | 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. |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on standing postural control parameters: Amplitude of COP | 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. |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on standing postural control parameters: Romberg quotient | 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. |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on standing postural orientation parameters | 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. |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on standing postural orientation parameters: limb loading ratio | 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. |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on drinking task with left and right upper limb: velocity and movement time | 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) |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on drinking task with left and right upper limb: movement strategy | 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) |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on drinking task with left and right upper limb: smoothness and coordination movement | 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) |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on drinking task with left and right upper limb: trunk displacement | 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 |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on drinking task with left and right upper limb: angular joint | 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 |
Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on 6 minutes walking test distance | 6 minutes walking test | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on the Berg Balance Scale score | The Berg Balance Scale | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on Brooke Upper Extremity Scale score | Brooke Upper Extremity Scale score for upper extremity capacities. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on locomotor | Questionnaires Abiloco | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on upper limb capacities | Questionnaires Abilhand | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on patient occupations | 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. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on MRC (Medical Research Council) score on flexors and extensors of lower and upper limb | 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. | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on Fatigue Severity Scale (FSS) score | Questionnaire Fatigue Severity Scale (FSS) for fatigue assessment | Assessed at 6 months, 12 months, 18 months and 24 months | |
| Secondary | Change from baseline on number of fall risks | Assessed at 6 months, 12 months, 18 months and 24 months | ||
| Secondary | Change from baseline on number of physiotherapy sessions per week | at baseline, 6 months, 12 months, 18 months and 24 months | ||
| Secondary | Change from baseline on number of sports practice per week | at baseline, 6 months, 12 months, 18 months and 24 months | ||
| Secondary | Change from baseline on Individualized Neuromuscular Quality of Life Questionnaire (INQoL) | Quality of life questionnaire: INQoL | Assessed at 6 months, 12 months, 18 months and 24 months |