European Registry of Patients With Infantile-onset Spinal Muscular Atrophy
NCT ID: NCT03339830
Last Updated: 2021-09-13
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
100 participants
OBSERVATIONAL
2017-10-10
2020-11-15
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* 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 Criteria
ALL
No
Sponsors
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Institut de Myologie, France
OTHER
Responsible Party
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Principal Investigators
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Laurent Servais, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Institute of Myology
Locations
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Hopital Morvan - CHU de Brest
Brest, , France
Service de Rééducation Pédiatrique Infantile " L'Escale " - Hôpital Femme Mère Enfant
Bron, , France
Hôpital le Bocage - CHU Dijon
Dijon, , France
Maladie Neuromusculaire de l'enfant - Service Maladies infectieuses et neurologie infantile - Hôpital Roger Salengro
Lille, , France
I-Motion Institute
Paris, , France
Unité de neurologie pédiatrique - Hôpital des enfants
Toulouse, , France
Countries
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Other Identifiers
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2017-A02291-52
Identifier Type: OTHER
Identifier Source: secondary_id
IO-SMA-Registry
Identifier Type: -
Identifier Source: org_study_id
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