NCT04177134 French Register of Patients With Spinal Muscular Atrophy
| NCT ID | NCT04177134 |
| Status | Recruiting |
| Phase | — |
| Sponsor | Assistance Publique - Hôpitaux de Paris |
| Condition | Spinal Muscular Atrophy |
| Study Type | OBSERVATIONAL |
| Enrollment | 1,000 participants |
| Start Date | 2020-01-31 |
| Primary Completion | 2029-01-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
This is an observational study. You will not receive an experimental treatment; researchers will collect data based on your existing condition or standard treatment.
This trial targets 1,000 participants in total. It began in 2020-01-31 with a primary completion date of 2029-01-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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.
Eligibility Criteria
Inclusion Criteria: * Genetically confirmed SMA 5q patients through 1 to 4 types treated in reference centers in France between September 1, 2016 and August 31, 2024. * for prospective study: inform consent form signed by patient. * for retrospective data: information form with non-opposition have been given to participant. * Covered by a health insurance. Exclusion Criteria: * Other type of SMA (not 5q). * Under guardianship or curatorship. * Unable to understand french language.
Contact & Investigator
Susana Quijano-Roy, MD, PhD
PRINCIPAL INVESTIGATOR
Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, Hôpital Raymond Poincaré, 92380 Garches, FRANCE
Frequently Asked Questions
Who can join the NCT04177134 clinical trial?
This trial is open to participants of all sexes, aged 0 Days or older, studying Spinal Muscular Atrophy. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
Is NCT04177134 currently recruiting?
Yes, NCT04177134 is actively recruiting participants. Contact the research team at susana.quijano-roy@aphp.fr for enrollment information.
Where is the NCT04177134 trial being conducted?
This trial is being conducted at Garches, France.
Who is sponsoring the NCT04177134 clinical trial?
NCT04177134 is sponsored by Assistance Publique - Hôpitaux de Paris. The principal investigator is Susana Quijano-Roy, MD, PhD at Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, Hôpital Raymond Poincaré, 92380 Garches, FRANCE. The trial plans to enroll 1,000 participants.