Ignite Creation Date:
2024-05-06 @ 2:04 PM
Last Modification Date:
2024-10-26 @ 1:24 PM
Study NCT ID:
NCT04202627
Status:
COMPLETED
Last Update Posted:
2023-03-29
First Post:
2019-12-11
Brief Title:
Biomarker Development in LGMD2i
Sponsor:
ML Bio Solutions Inc
Organization:
ML Bio Solutions Inc
Study Overview
Official Title:
Biomarker Development in LGMD2i
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:
MLB-01-001
Brief Summary:
The overall goal of this natural history study is to define the key LGMD2i phenotypes as measured by standard clinical outcome assessments COAs and to validate a muscle biomarker for LGMD2i to support therapeutic development
Detailed Description:
Limb Girdle Muscular Dystrophy LGMD 2i is an autosomal recessive form of LGMD that is due to missense mutations in the Fukutin-related protein FKRP gene Patients develop progressive proximal muscle weakness that leads to loss of ambulation Patients will also commonly develop a cardiomyopathy and respiratory compromise
There are promising new therapies that have been developed and as a result therapeutic trials are approaching
The rationale for this study is to define appropriate COAs for LGMD2i which will facilitate therapeutic development and ensure properly powered clinical trials In addition measurement of dystroglycan in muscles represents a potential muscle biomarker that could be used in early phase clinical trials as a measure of target engagement The clinical utility of changes in dystroglycan has not been validated in human samples
There are promising new therapies that have been developed and as a result therapeutic trials are approaching
The rationale for this study is to define appropriate COAs for LGMD2i which will facilitate therapeutic development and ensure properly powered clinical trials In addition measurement of dystroglycan in muscles represents a potential muscle biomarker that could be used in early phase clinical trials as a measure of target engagement The clinical utility of changes in dystroglycan has not been validated in human samples
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