Ignite Creation Date:
2025-12-24 @ 7:12 PM
Ignite Modification Date:
2025-12-29 @ 5:06 PM
Study NCT ID:
NCT06774703
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-01-14
First Post:
2024-03-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Nemaline Myopathy Clinical Research Network (NM-CTRN)
Sponsor:
Stanford University
Organization:
Study Overview
Official Title:
Nemaline Myopathy Clinical Research Network (NM-CTRN)
Status:
NOT_YET_RECRUITING
Status Verified Date:
2025-01
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:
None
Brief Summary:
The goal of this study is to establish a research network to help define the natural disease history and clinical outcome measures for Nemaline Myopathy (NM).
Detailed Description:
The long-term aim of this study is to incorporate these outcome measures into clinical trials for NM therapies. Outcome measures to be assessed will be dependent on the participant's age and functional status. Follow-up visits will be conducted either every 3 or 6 months, dependent on age, for a total of 3 years.
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?:
False
Is an FDA AA801 Violation?: