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French Register of Patients With Spinal Muscular Atrophy

NCT04177134 · Status: RECRUITING · Type: OBSERVATIONAL · Enrollment: 1000

Last updated 2025-04-06

No results posted yet for this study

Summary

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

Sponsors & Collaborators

  • Assistance Publique - Hôpitaux de Paris

    lead OTHER

Principal Investigators

  • Susana Quijano-Roy, MD, PhD · Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, Hôpital Raymond Poincaré, 92380 Garches, FRANCE

Eligibility

Min Age
0 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2020-01-31
Primary Completion
2029-01-31
Completion
2029-01-31

Countries

  • France

Study Locations

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Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04177134 on ClinicalTrials.gov