Actively Recruiting
A Prospective, Longitudinal Natural History Study of Children With LAMA2-related Dystrophies in France
Led by Institut de Myologie, France · Updated on 2024-12-12
40
Participants Needed
4
Research Sites
3 weeks
Total Duration
On this page
Sponsors
I
Institut de Myologie, France
Lead Sponsor
A
Association Française contre les Myopathies (AFM), Paris
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying children with LAMA2-related dystrophies (LAMA2-RD), a rare muscular condition, to better understand its natural course and characteristics. This observational study aims to create a well-defined group of patients in France for ongoing follow-up and to prepare for future clinical trials. The research focuses on multiple aspects of the disease including muscle function, breathing, cognitive abilities, quality of life, growth, and biomarkers.
CONDITIONS
Brief Title
Natural History Study of Children With LAMA2-related Dystrophies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent by a legal guardian and assent from the child if 6 years or older
- Confirmed diagnosis of LAMA2-RD by genetic testing or muscle biopsy
- No other confirmed neurological genetic diseases
- Willingness to maintain current exercise and physical therapy during the study
- Willingness to comply with study procedures and visits
- Affiliation with or beneficiary of a French social security scheme
You will not qualify if you...
- Developmental quotient below 70 or behavioral disorder requiring anesthesia for MRI
- Acute illness or hospitalization within 30 days before consent
- Participation in other investigational drug trials for LAMA2-RD within 30 days prior to consent
- Other significant medical conditions or frailty that may affect study results
- Pregnant or breastfeeding females
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Initial assessment at enrollment
Participants undergo evaluations including motor function tests, cognitive assessments, pulmonary and cardiac function tests, spinal X-rays, muscular MRI, and biomarker sample collections to assess the clinical characteristics of LAMA2-related dystrophies.
1 baseline visit (in-person)
Duration - Approximately 2 years
Participants are followed prospectively to monitor disease progression and changes in motor, cognitive, pulmonary, cardiac, and quality of life measures over time.
Regular visits during the study period (visit frequency determined by study protocol)
Trial Site Locations
Total: 4 locations
1
Centre de Référence GNMH, Pédiatrie Hôpital Raymond-Poincaré
Garches, France
Not Yet Recruiting
2
Service de MPR pédiatrique L'Escale - HCL
Lyon, France
Not Yet Recruiting
3
Département de neuropédiatrie Pôle Femme Mère Enfant CHU de Montpellier - Hôpital Gui de Chauliac
Montpellier, France
Not Yet Recruiting
4
Plateforme d'essais cliniques pédiatriques iMotion
Paris, France
Actively Recruiting
Research Team
A
Andreea SEFERIAN, Dr
E
Erwan GASNIER, PhD
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
1
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