Not provided
Not provided
Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2020-A02090-39 | Registry Identifier | IDRCB |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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:
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Muscular dystrophies group | 40 patients with limb girdle muscular dystrophie | ||
| Comparator group | 40 healthy volunteers without neuromuscular or squeletic disorder |
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| 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 |
| 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 |
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Joint kinematic parameters: change from baseline on gait speed |
Not provided
Inclusion Criteria:
For limb girdle muscular dystrophie group:
For Volunteer group:
Exclusion Criteria:
§ For all:
Not provided
Not provided
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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Céline BONNYAUD, PhD | Contact | +33 (0)1 71 14 49 21 | celine.bonnyaud@aphp.fr | |
| Samuel POUPLIN | Contact | +33 (0)1 47 10 70 61 | samuel.pouplin@aphp.fr |
| Name | Affiliation | Role |
|---|---|---|
| Céline BONNYAUD, PhD | Laboratoire d'analyse du mouvement, Service d'explorations fonctionnelles, Hôpital Raymond Poincaré, APHP | Principal Investigator |
| Samuel POUPLIN | Plateforme Nouvelles Technologies, Hôpital Raymond Poincaré, APHP |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Laboratoire d'analyse du mouvement, Service d'exploration fonctionnelles, Hôpital Raymond Poincaré, APHP | Recruiting | Garche | 92380 | France |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D049288 | Muscular Dystrophies, Limb-Girdle |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
Not provided
Not provided
Not provided
Not provided
Not provided
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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| Change from baseline on 6 minutes walking test distance | 6 minutes walking test | Assessed at 6 months, 12 months, 18 months and 24 months |
| Change from baseline on the Berg Balance Scale score | The Berg Balance Scale | Assessed at 6 months, 12 months, 18 months and 24 months |
| 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 |
| Change from baseline on locomotor | Questionnaires Abiloco | Assessed at 6 months, 12 months, 18 months and 24 months |
| Change from baseline on upper limb capacities | Questionnaires Abilhand | Assessed at 6 months, 12 months, 18 months and 24 months |
| 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 |
| 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 |
| 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 |
| 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 number of sports practice per week | at baseline, 6 months, 12 months, 18 months and 24 months |
| 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 |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |