Ignite Creation Date:
2025-12-24 @ 11:45 PM
Ignite Modification Date:
2025-12-25 @ 9:38 PM
Study NCT ID:
NCT05755451
Status:
RECRUITING
Last Update Posted:
2023-03-06
First Post:
2023-02-08
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Natural History of SMA
Sponsor:
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Organization:
Study Overview
Official Title:
Natural History of SMA: Long Term Longitudinal Study and Development of a Registry
Status:
RECRUITING
Status Verified Date:
2023-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
iSMAR
Brief 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.
Detailed 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
* 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 Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: