Status:
COMPLETED
European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
Lead Sponsor:
Institut de Myologie, France
Conditions:
Spinal Muscular Atrophy
Eligibility:
All Genders
Brief Summary
IO-SMA-Registry is a prospective, longitudinal and observational study which objective is to collect prospectively information on longevity, psychomotor development and respiratory function of patient...
Eligibility Criteria
Inclusion
- Spinal Muscular Atrophy diagnosed in childhood (before 18 months) and genetically confirmed.
- For patients with SMA type 1: Never acquired independent sitting position (more than 30 seconds, without hand support or any external support)
- For patients with SMA type 2 or 3: Patient treated with a market approved treatment for SMA or with a treatment in an expanded access program
- Any age
- Patients over 18 years of age or parent(s)/legal guardian(s) of patients \< 18 years of age not opposed to data collection for research purposes
Exclusion
- None
Key Trial Info
Start Date :
October 10 2017
Trial Type :
OBSERVATIONAL
Allocation :
ACTUAL
End Date :
November 15 2020
Estimated Enrollment :
100 Patients enrolled
Trial Details
Trial ID
NCT03339830
Start Date
October 10 2017
End Date
November 15 2020
Last Update
September 13 2021
Active Locations (6)
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1
Hopital Morvan - CHU de Brest
Brest, France
2
Service de Rééducation Pédiatrique Infantile " L'Escale " - Hôpital Femme Mère Enfant
Bron, France
3
Hôpital le Bocage - CHU Dijon
Dijon, France
4
Maladie Neuromusculaire de l'enfant - Service Maladies infectieuses et neurologie infantile - Hôpital Roger Salengro
Lille, France