Actively Recruiting
French Register of Patients With Spinal Muscular Atrophy
Led by Assistance Publique - Hôpitaux de Paris · Updated on 2025-04-06
1000
Participants Needed
1
Research Sites
469 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The primary objectives of the study are to obtain clinically meaningful data on survival and outcomes of all the patients with spinal muscular atrophy (SMA) 5q types 1 through 4 (according to international classification), being followed in the reference centers of the disease in France between September 1, 2016 and August 31, 2024. The registry will collect retrospectively and prospectively the longitudinal data of the long-term follow-up for child and adult patients, under real life conditions of current medical practice, in order to document the clinical evolution of patients (survival, motor, respiratory, orthopedic and nutritional), the conditions of use of the treatments, the mortality rates of treated and untreated patients, the tolerance of the treatments, adverse events in order to better define their places in the therapeutic strategy.
CONDITIONS
Official Title
French Register of Patients With Spinal Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Genetically confirmed SMA 5q patients of types 1 through 4 treated in reference centers in France between September 1, 2016 and August 31, 2024
- Signed informed consent form for prospective participation
- Non-opposition information given for retrospective data
- Covered by health insurance
You will not qualify if you...
- SMA types other than 5q
- Under legal guardianship or curatorship
- Unable to understand French language
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, (French neuromuscular reference network (FILNEMUS), Hôpital Raymond Poincaré,
Garches, France, 92380
Actively Recruiting
Research Team
S
Susana Quijano-Roy, MD, PhD
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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