Limb-Girdle Video Assessment

NCT05528757 | Terminated

• 1 other identifier: CAS-CAS008-01
• Study Type: observational
• Target: 45
• Locations: 1 country
• Sites: 1

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.

Conditions

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

Keywords

Limb-Girdle Muscular Dystrophy; Outcome Measure Development; Video Captures; Remote Assessment; Concept Elicitation; Feasibility Testing

Outcome Measures

Primary Outcomes (1)

• Assessing the fluctuation of movement abilities of LGMDs patients via remote video capture.

  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.

  January 2023 - March 2023

Secondary Outcomes (3)

• Task Selection Survey

  August 2022 - September 2022

• Focus Group

  September 2022 - October 2022

• Exit Interview

  January 2023 - March 2023

Study Arms (1)

LGMDs Patients

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

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• The Emmes Company, LLC: lead
• Coalition to Cure Calpain 3 (C3): collaborator
• Jain Foundation: collaborator

Study Sites (1)

Casimir

Kingston, Massachusetts, 02360, United States

Location

MeSH Terms

Conditions

Muscular Dystrophies, Limb-Girdle

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Lauren Carroll

  Casimir

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 24, 2022
• First Posted: September 6, 2022
• Study Start: June 20, 2022
• Primary Completion: November 2, 2022
• Study Completion: November 2, 2022
• Last Updated: December 18, 2023

Record last verified: 2023-12

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)