Ignite Creation Date:
2025-12-24 @ 6:56 PM
Ignite Modification Date:
2026-01-20 @ 4:31 PM
Study NCT ID:
NCT05528757
Status:
TERMINATED
Last Update Posted:
2023-12-18
First Post:
2022-08-24
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Limb-Girdle Video Assessment
Sponsor:
The Emmes Company, LLC
Organization:
Study Overview
Official Title:
Limb-Girdle Video Assessment Concept Elicitation and Feasibility Study
Status:
TERMINATED
Status Verified Date:
2023-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Contract termination/lack of funding
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to develop a new remote-based video assessment outcome measure for Limb-Girdle Muscular Dystrophy (LGMD) trials. The overall objectives for this study are: 1. Identify domains and tasks meaningful to participants with a Limb-Girdle Muscular Dystrophy (LGMD) for development of the LGVA, including considerations for subtype heterogeneity and functional subgroup branching; 2. Determine the feasibility and reliability of the LGVA with test-retest of the LGVA Video Capture Manual; 3. Assess and refine the LGVA Video Capture Manual to ensure standardization and incorporate feedback from participants; 4. Collect source material videos using the LGVA Video Capture Manual to support the development of scorecards for the LGVA.
Detailed Description:
The purpose of this study is to gain insight from people living with an LGMD and gather source material videos to develop and test Limb-Girdle Video Assessment (LGVA), a new outcome measure designed to assess changes in functional abilities of participants in LGMDs clinical trials. The LGVA is being designed as a remote video capture to assess nuanced changes in compensatory movement patterns over time. This study involves two stages. In the concept elicitation stage, the tasks for the LGVA Video Capture Manual will be finalized based on feedback from participants living with an LGMD 2A, 2B, or 2I in various stages of disease progression. After finalizing the LGVA Video Capture Manual, source material videos will be collected to evaluate the feasibility and appropriateness of the LGVA tasks, standardize capture procedures, and inform the development of scorecards for the LGVA.
Study Oversight
Has Oversight DMC:
False
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?: