Evolution of the Functional and Muscular State of Patients With Muscular Dystrophy 2A Belts
Launched by ASSISTANCE PUBLIQUE - HÔPITAUX DE PARIS · Apr 25, 2024
Trial Information
Current as of June 05, 2025
Not yet recruiting
Keywords
ClinConnect Summary
This clinical trial is focused on understanding how limb girdle muscular dystrophy type 2A (LGMD2A) affects the muscles and overall function of patients over time. LGMD2A is a genetic condition that primarily impacts the muscles around the pelvis and shoulders, leading to progressive weakness. The study aims to monitor changes in muscle strength and function in patients with this condition to better understand its progression and effects.
To participate in the trial, individuals must have a confirmed diagnosis of LGMD2A, which means they have specific genetic mutations related to the calpain 3 gene. Participants should be between 65 and 74 years old and able to take part in various tests, such as muscle strength assessments. This is an important opportunity for eligible patients to contribute to research that could improve understanding and treatment of this condition. However, certain individuals, like those with other significant health issues or who are currently involved in other clinical trials, may not be eligible. Overall, participants can expect to undergo a series of assessments that will help researchers learn more about LGMD2A.
Gender
ALL
Eligibility criteria
- Inclusion Criteria:
- • Confirmed diagnosis of autosomal recessive LGMD2A (two pathogenic mutations in the calpain 3 gene)
- • Ability to participate in the tests and examinations planned by the study (manual muscle tests, motor scales)
- • Informed and having signed a consent form
- • Affiliate to a social security scheme in France (beneficiary or entitled person).
- Exclusion Criteria:
- • Patient with another disease likely to significantly interfere with the interpretation of the natural history of LGMD2A
- • Participant in a clinical trial with an investigational product within 3 months preceding inclusion
- • Patient unable or unwilling to comply with protocol requirements
- • Patient under guardianship, curatorship or legal protection
- • Pregnant or breastfeeding woman
- • Patient deprived of liberty
- • Adult patient unable to express consent
- • Refusal to participate
About Assistance Publique Hôpitaux De Paris
Assistance Publique - Hôpitaux de Paris (AP-HP) is a leading public hospital system in France, renowned for its commitment to healthcare excellence and innovative medical research. As a prominent clinical trial sponsor, AP-HP plays a pivotal role in advancing medical knowledge and improving patient care through rigorous scientific investigations across a wide range of therapeutic areas. With a focus on collaboration and interdisciplinary approaches, AP-HP leverages its extensive network of hospitals and expert clinicians to facilitate high-quality clinical trials that adhere to the highest ethical and regulatory standards, ultimately aiming to translate research findings into tangible health benefits for diverse patient populations.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Patients applied
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported