Ignite Creation Date:
2024-05-06 @ 8:23 PM
Last Modification Date:
2024-10-26 @ 3:26 PM
Study NCT ID:
NCT06366815
Status:
RECRUITING
Last Update Posted:
2024-04-16
First Post:
2023-02-08
Brief Title:
phenotypeS in Non Ambulant Duchenne Muscular Dystrophy
Sponsor:
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Organization:
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Study Overview
Official Title:
Characterizing Phenothypes in Non Ambulant Duchenne Muscular Dystrophy
Status:
RECRUITING
Status Verified Date:
2024-10
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:
GUP21003
Brief Summary:
The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor respiratory and cardiac function
Detailed Description:
The aims of the study are to prospectively collect information on several aspects of function in non-ambulant DMD patients by using a structured battery of tests including motor respiratory and cardiac function to retrospectively review similar information on the data collected in the last decade and to establish the effect of steroids after loss of ambulation on different aspects of function
We also aim to use this integrated approach to identify patterns of severity and progression the most appropriate outcome measures and endpoints in each group and possible genotypephenotype correlations
We also aim to use this integrated approach to identify patterns of severity and progression the most appropriate outcome measures and endpoints in each group and possible genotypephenotype correlations
Study Oversight
Has Oversight DMC:
None
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?:
None