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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RECRUITING
1200 participants
OBSERVATIONAL
2018-06-21
2033-12-30
Brief Summary
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Detailed Description
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* better understand the natural history of the disease in untreated patients identifying factors that can influence disease progression by combining retrospective and prospective data.
* Describe the patterns of disease progression (in terms of functional measure, adverse events, levels of care, and hospitalization) in treated and untreated patients
* Identify all the patients treated with the available therapies in Italy, providing first line details on epidemiological data in a wider network including all centers recognized as centers for spinal muscular atrophy (SMA) in each region.
As part of the activity clinical evaluators, data manager and sub-investigators are also trained and use common manuals of operation to ensure reliability across the participating centers. The registry provides accurate and reliable information on natural history including results from currently used functional measures. The registry also includes information on supportive care, hospitalization and adverse events.
Details of the electronic clinical file record including data assembly and the platform used are available as part of a peer reviewed paper
Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
Exclusion Criteria
ALL
No
Sponsors
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University of Milan
OTHER
Istituto Giannina Gaslini
OTHER
Ospedale Pediatrico Bambin Gesù
OTHER
University of Messina
OTHER
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
OTHER
Responsible Party
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Principal Investigators
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Eugenio Mercuri, MD
Role: PRINCIPAL_INVESTIGATOR
F Policlinico Gemelli IRCCS
Locations
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Istituto Gaslini
Genova, , Italy
Nemo Sud
Messina, , Italy
Centro Clinico nemo
Milan, , Italy
Ospedale Bambino gesu
Rome, , Italy
Policlinico gemelli
Rome, , Italy
Countries
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Central Contacts
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Facility Contacts
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Claudio Bruno
Role: primary
Sonia Messina
Role: primary
Valeria Sansone
Role: primary
Adele D'Amico
Role: primary
Other Identifiers
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1894
Identifier Type: -
Identifier Source: org_study_id
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