Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2INCT05528757
Limb-Girdle Video Assessment
An observational study of Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I, run by The Emmes Company, LLC at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 years and older
- Conditions the study is for
- Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I
- How many people
- 45 participants
- Where
- 1 siteUnited States
- Study dates
- 20 Jun 2022to 2 Nov 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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.
Read the 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.
- Keywords
- Limb-Girdle Muscular Dystrophy, Outcome Measure Development, Video Captures, Remote Assessment, Concept Elicitation, Feasibility Testing
- Medical subject headings
- Muscular Dystrophies, Limb-Girdle
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| LGMDs Patients | Patients with a genetically confirmed diagnosis of a LGMD subtype 2A, 2B, or 2I |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: January 2023 - March 2023
Secondary outcomes (3)
Task Selection Survey
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.
Measured: August 2022 - September 2022
Focus Group
Small group discussion of participants with similar functional abilities to discuss the progression of symptoms and heterogeneity among subtypes.
Measured: September 2022 - October 2022
Exit Interview
After the feasibility video captures are complete, participants are asked about their experiences using the LGVA Video Capture Manual and the recording procedures.
Measured: January 2023 - March 2023
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Sep 2022
- Study start20 Jun 2022
- Primary completion2 Nov 2022
- Study completion2 Nov 2022
Why it stopped: Contract termination/lack of funding
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2IStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Emmes Company, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Coalition to Cure Calpain 3 (C3), Jain Foundation
- Responsible party
- Sponsor
- Investigators
- Lauren Carroll, Study director, Casimir
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05528757
- Sponsor's protocol ID
- CAS-CAS008-01
- Status last verified
- Dec 2023
- First submitted
- 24 Aug 2022
- Last updated
- 18 Dec 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Limb-Girdle Video Assessment Concept Elicitation and Feasibility Study
- ClinicalTrials.gov ID
- NCT05528757
- Acronym / sponsor ID
- CAS-CAS008-01
- Keywords
- Limb-Girdle Muscular Dystrophy, Outcome Measure Development, Video Captures, Remote Assessment, Concept Elicitation, Feasibility Testing
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Contract termination/lack of funding
- Enrolment
- 45 participants (actual)
- Lead sponsor
- The Emmes Company, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Coalition to Cure Calpain 3 (C3), Jain Foundation
- Study start
- 20 Jun 2022 (actual)
- Primary completion
- 2 Nov 2022 (actual)
- Study completion
- 2 Nov 2022 (actual)
- First posted
- 6 Sep 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LGMDs Patients | – | –Patients with a genetically confirmed diagnosis of a LGMD subtype 2A, 2B, or 2I |
Who may qualify
- Ages
- 14 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Persons living with a confirmed diagnosis of LGMD 2A, 2B, or 2I.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
3 secondary outcomes
Task Selection Survey
Time frame: August 2022 - September 2022
Focus Group
Time frame: September 2022 - October 2022
Exit Interview
Time frame: January 2023 - March 2023
Results and safety
The study was stopped early (reason given: Contract termination/lack of funding). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Dec 2023.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lauren Carroll study director
Casimir
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Limb-Girdle Muscular Dystrophy ; Subtypes 2A, 2B, and 2I, run by The Emmes Company, LLC at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 18 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Contract termination/lack of funding). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Assessing the fluctuation of movement abilities of LGMDs patients via remote video capture. (time frame: January 2023 - March 2023). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Dec 2023; AzTrials last refreshed its copy on 27 Oct 2026.