Limb-Girdle Video Assessment | NCT05528757

Studies
Limb-Girdle Video Assessment

NCT05528757

Sponsor

The Emmes Company, LLC

Status

Terminated

Last Update Posted

Dec 18, 2023Actual

Enrollment

45Actual

Phase

Not provided

Conditions

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

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Dec 2023

Overall Recruitment Status or Expanded Access Status

Terminated

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Contract termination/lack of funding

Expanded Access Info

Start Date

Jun 20, 2022Actual

Primary Completion Date

Nov 2, 2022Actual

Completion Date

Nov 2, 2022Actual

First Submitted Date

Aug 24, 2022

First Submission Date that Met QC Criteria

Sep 1, 2022

First Posted Date

Sep 6, 2022Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Dec 12, 2023

Last Update Posted Date

Dec 18, 2023Actual

Name	Class
Coalition to Cure Calpain 3 (C3)	UNKNOWN
Jain Foundation	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

45Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
LGMDs Patients		Patients with a genetically confirmed diagnosis of a LGMD subtype 2A, 2B, or 2I

Interventions

Not provided

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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

Measure	Description	Time Frame
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.	August 2022 - September 2022
Focus Group

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

14 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Persons living with a confirmed diagnosis of LGMD 2A, 2B, or 2I.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Lauren Carroll	Casimir	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Casimir		Kingston	Massachusetts	02360	United States

IPD Sharing Statement Module

Plan to Share IPD

Description

Not provided

Types

Not provided

Time Frame

Not provided

Access Criteria

Not provided

URL

Not provided

Condition Browse Module

MeSH Terms

ID	Term
D049288	Muscular Dystrophies, Limb-Girdle

Ancestor Terms

ID	Term
D009136	Muscular Dystrophies
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases

Browse Leaves

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Browse Branches

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