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Natural History of SMA — iSMAR  

Muscular Atrophy, Spinal Clinical Trial

Official title:
Natural History of SMA: Long Term Longitudinal Study and Development of a Registry

Clinical Trial Summary

This is an investigator initiated observational study with the aim to record several aspects of function, care and adverse events in a large cohort of SMA patients followed longitudinally by using a structured academic disease registry.

Clinical Trial Description

This is an observational multicenter retrospective and prospective study involving high quality data collected in 5 academic centers in Italy, the registry aims to - 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 ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05755451
Study type Observational [Patient Registry]
Source Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Contact Eugenio Mercuri, MD
Phone 06 3015
Email eugeniomaria.mercuri@policlinicogemelli.it
Status Recruiting
Phase
Start date June 21, 2018
Completion date December 30, 2033
View Details
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