Ignite Creation Date:
2024-05-06 @ 1:18 PM
Last Modification Date:
2024-10-26 @ 1:11 PM
Study NCT ID:
NCT03981289
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2024-07-01
First Post:
2019-06-03
Brief Title:
Defining Clinical Endpoints in Limb Girdle Muscular Dystrophy LGMD
Sponsor:
Virginia Commonwealth University
Organization:
Virginia Commonwealth University
Study Overview
Official Title:
GRASP-LGMD Defining Clinical Endpoints in LGMD
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-09
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:
GRASP-01-001
Brief Summary:
Limb Girdle Muscular Dystrophy comprise a group of disorders made up of over 30 mutations which share a common phenotype of progressive weakness of the shoulder and hip girdle muscles While the individual genetic mutations are rare as a cohort LGMDs are one of the four most common muscular dystrophies The overall goal of project 1 is to define the key phenotypes as measured by standard clinical outcome assessments COAs for limb girdle muscular dystrophies LGMD to hasten therapeutic development
Detailed Description:
The genetic heterogeneity has been a barrier to broad natural history efforts with prior investigations often limited to single gene mutations Much attention is paid to the variability within individual mutations eg distal presentations as opposed to defining the best strategy for measuring change in overall LGMD disease burden This presents a major dilemma for LGMD rare disease research how to balance diverse genes leading to overlapping phenotypes versus variants in the same gene leading to divergent phenotypes What is clear is as a group LGMDs are chronic and progressive leading to significant lifetime morbidity and represent a large unmet clinical need
Recent developments in the investigators genetic understanding of LGMD and molecular approaches to therapy have led to proposed gene replacement therapies for at least three of the LGMD mutations Several of these gene replacement therapies are currently in pre-clinicalphase 1 testing leading to an urgent need for natural history data In addition non-specific therapies which target muscle mass or function are being tested in other muscular dystrophies and may prove beneficial for LGMD
Recent developments in the investigators genetic understanding of LGMD and molecular approaches to therapy have led to proposed gene replacement therapies for at least three of the LGMD mutations Several of these gene replacement therapies are currently in pre-clinicalphase 1 testing leading to an urgent need for natural history data In addition non-specific therapies which target muscle mass or function are being tested in other muscular dystrophies and may prove beneficial for LGMD
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| GRASP-LGMD | OTHER | Virginia Commonwealth University | None |