Motor Parameters in Patients With Limb Girdle Muscular Dystrophy

NCT04772027 | Unknown

• 2 other identifiers: APHP2001412020-A02090-39
• Study Type: observational
• Target: 80
• Locations: 1 country
• Sites: 1

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.

Conditions

Limb Girdle Muscular Dystrophies

Keywords

Limb Girdle Muscular Dystrophies (LGMD); motricity; instrumental assessment

Outcome Measures

Primary Outcomes (4)

• 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

Secondary Outcomes (32)

• Change from baseline on muscular parameters

  Assessed at 6 months, 12 months, 18 months and 24 months

• Joint kinematic parameters: change from baseline on gait speed

  Assessed at 6 months, 12 months, 18 months and 24 months

• Joint kinematic parameters: change from baseline on step length

  Assessed at 6 months, 12 months, 18 months and 24 months

• Joint kinematic parameters: change from baseline on cadence

  Assessed at 6 months, 12 months, 18 months and 24 months

• Joint kinematic parameters: change from baseline on step width

  Assessed at 6 months, 12 months, 18 months and 24 months

Study Arms (2)

Muscular dystrophies group

40 patients with limb girdle muscular dystrophie

Comparator group

40 healthy volunteers without neuromuscular or squeletic disorder

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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.

You may not qualify if:

• § 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.

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Laboratoire d'analyse du mouvement, Service d'exploration fonctionnelles, Hôpital Raymond Poincaré, APHP

Garche, 92380, France

RECRUITING

MeSH Terms

Conditions

Muscular Dystrophies, Limb-Girdle

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• 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

  STUDY DIRECTOR

Central Study Contacts

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

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 7, 2021
• First Posted: February 25, 2021
• Study Start: April 1, 2021
• Primary Completion: January 1, 2025
• Study Completion: January 1, 2025
• Last Updated: September 6, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (1)