Assessing motor function in patients with limb girdle muscular dystrophy
Characterization and Identification of Motor Parameters Using Instrumental Assessment in Patients With Limb Girdle Muscular Dystrophy
Assistance Publique - Hôpitaux de Paris · NCT04772027
This study is tracking how motor skills change over two years in people with limb girdle muscular dystrophy to see how it affects their daily life compared to healthy individuals.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 80 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris (other) |
| Locations | 1 site (Garche) |
| Trial ID | NCT04772027 on ClinicalTrials.gov |
What this trial studies
This observational study aims to follow patients with limb girdle muscular dystrophy (LGMD) over two years to evaluate changes in motor parameters using instrumental assessments. Participants will undergo five visits, including a baseline and four follow-ups, to monitor their motor function and compare it with healthy volunteers. The study will also explore the relationships between various muscular and biomechanical parameters, providing insights into the progression of LGMD and its impact on quality of life.
Who should consider this trial
Good fit: Ideal candidates include adults aged 18 to 70 diagnosed with limb girdle muscular dystrophy who can maintain an upright position and walk independently.
Not a fit: Patients with additional neuromuscular disorders or severe respiratory issues may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to better understanding and management of limb girdle muscular dystrophy, improving patient care and outcomes.
How similar studies have performed: While similar studies have been conducted, this specific approach focusing on instrumental assessments in LGMD is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Garche
- Laboratoire d'analyse du mouvement, Service d'exploration fonctionnelles, Hôpital Raymond Poincaré, APHP — Garche, France (RECRUITING)
Study contacts
- Principal investigator: Céline BONNYAUD, PhD — Laboratoire d'analyse du mouvement, Service d'explorations fonctionnelles, Hôpital Raymond Poincaré, APHP
- Study coordinator: Céline BONNYAUD, PhD
- Email: celine.bonnyaud@aphp.fr
- Phone: +33 (0)1 71 14 49 21
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Limb Girdle Muscular Dystrophies, motricity, instrumental assessment