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Limb-Girdle Video Assessment

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05528757
Recruitment Status : Active, not recruiting
First Posted : September 6, 2022
Last Update Posted : May 31, 2023
Coalition to Cure Calpain 3 (C3)
Jain Foundation
Information provided by (Responsible Party):
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.

Condition or disease
Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I

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.

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Study Type : Observational
Actual Enrollment : 45 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Limb-Girdle Video Assessment Concept Elicitation and Feasibility Study
Actual Study Start Date : June 20, 2022
Actual Primary Completion Date : March 1, 2023
Estimated Study Completion Date : January 31, 2024

LGMDs Patients
Patients with a genetically confirmed diagnosis of a LGMD subtype 2A, 2B, or 2I

Primary Outcome Measures :
  1. Assessing the fluctuation of movement abilities of LGMDs patients via remote video capture. [ Time Frame: 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 :
  1. Task Selection Survey [ Time Frame: 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.

  2. Focus Group [ Time Frame: September 2022 - October 2022 ]
    Small group discussion of participants with similar functional abilities to discuss the progression of symptoms and heterogeneity among subtypes.

  3. Exit Interview [ Time Frame: 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   14 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Persons living with a confirmed diagnosis of LGMD 2A, 2B, or 2I.
  • Person with confirmed diagnosis of an LGMD 2A, 2B, or 2I
  • ≥14 years of age at the timing of consent (if age 14 - 17, consent from the parent/ legal guardian is also needed)
  • Able to read, speak, and understand English
  • Residing in the USA
  • Willing and able to complete an electronic survey, which includes questions about personal characteristics and medical history as well as review of tasks
  • Willing and able to discuss their experiences as a person living with an LGMD in a group setting
  • Willing and able to complete 2 rounds of video capture following instructions in the LGVA Video Capture Manual in a 30-day period
  • Willing and able to complete an exit interview to discuss their experiences capturing and recording videos with the LGVA Video Capture Manual

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05528757

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United States, Massachusetts
Kingston, Massachusetts, United States, 02360
Sponsors and Collaborators
The Emmes Company, LLC
Coalition to Cure Calpain 3 (C3)
Jain Foundation
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Study Director: Lauren Carroll Casimir
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Responsible Party: The Emmes Company, LLC
ClinicalTrials.gov Identifier: NCT05528757    
Other Study ID Numbers: CAS-CAS008-01
First Posted: September 6, 2022    Key Record Dates
Last Update Posted: May 31, 2023
Last Verified: May 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by The Emmes Company, LLC:
Limb-Girdle Muscular Dystrophy
Outcome Measure Development
Video Captures
Remote Assessment
Concept Elicitation
Feasibility Testing
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophies, Limb-Girdle
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn