Ignite Creation Date:
2024-05-06 @ 10:48 AM
Last Modification Date:
2024-10-26 @ 12:35 PM
Study NCT ID:
NCT03351270
Status:
COMPLETED
Last Update Posted:
2023-03-02
First Post:
2017-11-14
Brief Title:
Prospective Natural History Study of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies
Sponsor:
Institut de Myologie France
Organization:
Institut de Myologie France
Study Overview
Official Title:
Prospective Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy and Other CentroNuclear Myopathies
Status:
COMPLETED
Status Verified Date:
2023-03
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:
NatHis-CNM
Brief Summary:
This is the 2 years extension of the prospective and longitudinal study of the natural history and functional status of patients with myotubular myopathy and other centronuclear CNM sponsored by Dynacure including ten additional pediatric patients with mutation in MTM1 or DNM2 genes the patients are planned to be enrolled in one year leading to an expected total number of 70 patients followed at least over 1 year period Data from the study will be used to characterize the disease course of CNM and determine which outcome measures will be the best to assess the efficacy of potential therapies
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