Limb Girdle Muscular DystrophiesEIDYNCT04772027
Motor Parameters in Patients With Limb Girdle Muscular Dystrophy
An observational study of Limb Girdle Muscular Dystrophies, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Limb Girdle Muscular Dystrophies
- How many people
- 80 participants
- Where
- 1 siteFrance
- Study dates
- 1 Apr 2021to Jan 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- § 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.
Trial locations and contacts
1 site in 1 country.
France1 site, 1 recruiting
- Recruiting
Laboratoire d'analyse du mouvement, Service d'exploration fonctionnelles, Hôpital Raymond Poincaré, APHP
Garche, 92380
About this study
Official 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.
Read the 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.
- Conditions
- Limb Girdle Muscular Dystrophies
- Keywords
- Limb Girdle Muscular Dystrophies (LGMD), motricity, instrumental assessment
- Medical subject headings
- Muscular Dystrophies, Limb-Girdle
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Muscular dystrophies group | 40 patients with limb girdle muscular dystrophie | |
| Comparator group | 40 healthy volunteers without neuromuscular or squeletic disorder |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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)
Measured: 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).
Measured: 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)
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Secondary outcomes (32)
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
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
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)
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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)
Measured: 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)
Measured: 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)
Measured: 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
Measured: 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
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on 6 minutes walking test distance
6 minutes walking test
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on the Berg Balance Scale score
The Berg Balance Scale
Measured: 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.
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on locomotor
Questionnaires Abiloco
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on upper limb capacities
Questionnaires Abilhand
Measured: 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.
Measured: 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.
Measured: 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
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on number of fall risks
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on number of physiotherapy sessions per week
Measured: at baseline, 6 months, 12 months, 18 months and 24 months
Change from baseline on number of sports practice per week
Measured: 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
Measured: Assessed at 6 months, 12 months, 18 months and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Feb 2021
- Study start1 Apr 2021
- Primary completionJan 2025 (estimated)
- Study completionJan 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limb Girdle Muscular DystrophiesStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Céline BONNYAUD, PhD, Principal investigator, Laboratoire d'analyse du mouvement, Service d'explorations fonctionnelles, Hôpital Raymond Poincaré, APHPSamuel POUPLIN, Study director, Plateforme Nouvelles Technologies, Hôpital Raymond Poincaré, APHP
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04772027
- Sponsor's protocol ID
- APHP200141
- Other IDs
- 2020-A02090-39
- Acronym
- EIDY
- Status last verified
- Sep 2023
- First submitted
- 7 Jan 2021
- Last updated
- 6 Sep 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Characterization and Identification of Motor Parameters Using Instrumental Assessment in Patients With Limb Girdle Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT04772027
- Acronym / sponsor ID
- EIDY / APHP200141
- Conditions
- Limb Girdle Muscular Dystrophies
- Keywords
- Limb Girdle Muscular Dystrophies (LGMD), motricity, instrumental assessment
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 1 Apr 2021 (actual)
- Primary completion
- Jan 2025 (estimated)
- Study completion
- Jan 2025 (estimated)
- First posted
- 25 Feb 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Muscular dystrophies group | – | –40 patients with limb girdle muscular dystrophie |
| Comparator group | – | –40 healthy volunteers without neuromuscular or squeletic disorder |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (8 listed)
- For limb girdle muscular dystrophie group:
- For Volunteer group:
Key exclusion criteria (6 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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).
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)
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).
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)
32 secondary outcomes
Change from baseline on muscular parameters
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Joint kinematic parameters: change from baseline on gait speed
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Joint kinematic parameters: change from baseline on step length
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Joint kinematic parameters: change from baseline on cadence
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Joint kinematic parameters: change from baseline on step width
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
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
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
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
Change from baseline on kinetic gait parameters
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on standing postural control parameters: COP displacements
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on standing postural control parameters: elliptic surface
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on standing postural control parameters: Amplitude of COP
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on standing postural control parameters: Romberg quotient
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on standing postural orientation parameters
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on standing postural orientation parameters: limb loading ratio
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: 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
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on 6 minutes walking test distance
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on the Berg Balance Scale score
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on Brooke Upper Extremity Scale score
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on locomotor
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on upper limb capacities
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on patient occupations
Time frame: 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
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on Fatigue Severity Scale (FSS) score
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on number of fall risks
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Change from baseline on number of physiotherapy sessions per week
Time frame: at baseline, 6 months, 12 months, 18 months and 24 months
Change from baseline on number of sports practice per week
Time frame: at baseline, 6 months, 12 months, 18 months and 24 months
Change from baseline on Individualized Neuromuscular Quality of Life Questionnaire (INQoL)
Time frame: Assessed at 6 months, 12 months, 18 months and 24 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Sep 2023.
- Individual participant dataSharing planned: No
Related trials for Limb Girdle Muscular Dystrophies
Not yet recruitingPhase 1
BBM-L101 Gene Therapy in Participants With LGMD2D/R3
Limb Girdle Muscular Dystrophy Type 2D/R3
Not yet recruitingPhase 2
Labyrinthin Vaccination in Veterans With Lung Adenocarcinoma
Type and Grade of Toxicities, Demographic and Background Characteristics, Advanced, Metastatic, Recurrent Adenocarcinoma and Malignant Solid Neoplasm
RecruitingObservational study
Natural History Study for Charcot Marie Tooth Disease
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth
Active, not recruitingPhase 3
Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I)
Active, not recruitingPhase 1/2
A Study to Evaluate the Safety of AB-1003 (Previously LION-101) in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1)
Limb Girdle Muscular Dystrophy, Limb-Girdle Muscular Dystrophy Type 2
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Céline BONNYAUD, PhD principal investigator
Laboratoire d'analyse du mouvement, Service d'explorations fonctionnelles, Hôpital Raymond Poincaré, APHP
Samuel POUPLIN study director
Plateforme Nouvelles Technologies, Hôpital Raymond Poincaré, APHP
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Limb Girdle Muscular Dystrophies, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 6 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Céline BONNYAUD, PhD (+33 (0)1 71 14 49 21, celine.bonnyaud@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline on maximal strength (time frame: Assessed at 6 months, 12 months, 18 months and 24 months). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Sep 2023.