Limb-Girdle Video Assessment

Terminated

• Conditions: Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I

• Registration Number: NCT05528757
• Lead Sponsor: The Emmes Company, LLC

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 Timeline

• Study Start: 2022-06-20
• Study First Submitted: 2022-08-24
• Study First Submitted QC: 2022-09-01
• Study First Posted: 2022-09-06
• Primary Completion: 2022-11-02
• Study Completion: 2022-11-02
• Status Verified: 2023-12
• Last Update Submitted: 2023-12-12
• Last Update Posted: 2023-12-18

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 45

Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Assessing the fluctuation of movement abilities of LGMDs patients via remote video capture.	January 2023 - March 2023	Using the newly developed LGVA Video Capture Manual, participants are asked to record two sets of videos of them performing assigned tasks. Participants will have one week to complete each round of video capture, with approximately one month between each round. Videos will be captured remotely and uploaded through a secure platform.

Secondary Outcome Measures

Name	Time	Method
Task Selection Survey	August 2022 - September 2022	Electronic survey asking LGMDs patients about tasks and activities they perform in their daily life, and if/ how these are impacted by their LGMD symptoms.
Exit Interview	January 2023 - March 2023	After the feasibility video captures are complete, participants are asked about their experiences using the LGVA Video Capture Manual and the recording procedures.
Focus Group	September 2022 - October 2022	Small group discussion of participants with similar functional abilities to discuss the progression of symptoms and heterogeneity among subtypes.

Trial Locations

Locations (1)

Casimir; 🇺🇸 Kingston, Massachusetts, United States