- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05528757
Limb-Girdle Video Assessment
December 12, 2023 updated by: The Emmes Company, LLC
Limb-Girdle Video Assessment Concept Elicitation and Feasibility Study
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.
Study Overview
Status
Terminated
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 Type
Observational
Enrollment (Actual)
45
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Massachusetts
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Kingston, Massachusetts, United States, 02360
- Casimir
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
14 years and older (Child, Adult, Older Adult)
Accepts Healthy Volunteers
No
Sampling Method
Non-Probability Sample
Study Population
Persons living with a confirmed diagnosis of LGMD 2A, 2B, or 2I.
Description
- 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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
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LGMDs Patients
Patients with a genetically confirmed diagnosis of a LGMD subtype 2A, 2B, or 2I
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Assessing the fluctuation of movement abilities of LGMDs patients via remote video capture.
Time Frame: January 2023 - March 2023
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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.
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January 2023 - March 2023
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Task Selection Survey
Time Frame: August 2022 - September 2022
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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.
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August 2022 - September 2022
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Focus Group
Time Frame: September 2022 - October 2022
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Small group discussion of participants with similar functional abilities to discuss the progression of symptoms and heterogeneity among subtypes.
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September 2022 - October 2022
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Exit Interview
Time Frame: January 2023 - March 2023
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After the feasibility video captures are complete, participants are asked about their experiences using the LGVA Video Capture Manual and the recording procedures.
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January 2023 - March 2023
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Study Director: Lauren Carroll, Casimir
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
June 20, 2022
Primary Completion (Actual)
November 2, 2022
Study Completion (Actual)
November 2, 2022
Study Registration Dates
First Submitted
August 24, 2022
First Submitted That Met QC Criteria
September 1, 2022
First Posted (Actual)
September 6, 2022
Study Record Updates
Last Update Posted (Actual)
December 18, 2023
Last Update Submitted That Met QC Criteria
December 12, 2023
Last Verified
December 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- CAS-CAS008-01
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
No
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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