Ignite Creation Date:
2024-05-06 @ 8:18 PM
Last Modification Date:
2024-10-26 @ 3:24 PM
Study NCT ID:
NCT06321965
Status:
RECRUITING
Last Update Posted:
2024-03-20
First Post:
2024-02-06
Brief Title:
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Sponsor:
Hospices Civils de Lyon
Organization:
Hospices Civils de Lyon
Study Overview
Official Title:
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Status:
RECRUITING
Status Verified Date:
2024-07
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:
PHENO SMART
Brief Summary:
With the advent of new treatments for ASI new phenotypes are emerging The investigators propose to describe these new phenotypes by prospectively following children with ASI of all types treated with TRS and aged under 16 for 2 years
The investigators also propose to evaluate potential assessment tools to determine whether they are relevant for monitoring this population either routinely or for future clinical trials The investigators also aim to collect the total costs associated with ASI in order to propose a first prospective medico-economic study in France
The investigators also propose to evaluate potential assessment tools to determine whether they are relevant for monitoring this population either routinely or for future clinical trials The investigators also aim to collect the total costs associated with ASI in order to propose a first prospective medico-economic study in France
Detailed Description:
None
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