Limb Girdle Muscular Dystrophy (LGMD) Natural History | NCT01783509

Studies
Limb Girdle Muscular Dystrophy (LGMD) Natural History

NCT01783509

Sponsor

Wake Forest University Health Sciences

Status

Completed

Last Update Posted

Apr 28, 2022Actual

Enrollment

13Actual

Phase

Not provided

Conditions

Muscular Dystrophies, Limb-Girdle (GENETICALLY CONFIRMED)

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

Jul 2017

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2011

Primary Completion Date

Jul 2017Actual

Completion Date

Jul 2017Actual

First Submitted Date

Jan 18, 2013

First Submission Date that Met QC Criteria

Feb 1, 2013

First Posted Date

Feb 5, 2013Estimated

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

Apr 20, 2022

Last Update Posted Date

Apr 28, 2022Actual

Name	Class
Muscular Dystrophy Association	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

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 understand more about limb-girdle muscular dystrophy. Therefore, the investigators would like to track the following information collected once a year from patients with GENETICALLY CONFIRMED LGMD: quality of life questionnaires, muscle strength, motor function, routine examination, assessment of patient (or parent) understanding of LGMD, and serum (blood) for growth factors, cytokines, and biomarkers (all parts of your blood). By tracking this information, we hope to be able to understand more about the diagnosis, progression and natural history of this disorder.

Detailed Description

Not provided

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Yes

Target Follow-Up Duration

20 Years

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

Retention

Enrollment

13Actual

Label	Type	Description	Intervention Names
LGMD		Patients with GENETICALLY CONFIRMED limb girdle muscular dystrophy

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Comprehensive clinical evaluation of individuals with GENETICALLY CONFIRMED LGMD, according to the study protocol, in order to evaluate disease progression		yearly up to 10 years

Secondary Outcomes

Not provided

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

You have a GENETICALLY CONFIRMED diagnosis of Limb Girdle Muscular Dystrophy
You must be at least 6 years of age or older (if under 18 you will need Parental consent)
You must be able to travel to the study site
You must be able to provide a DNA/Gene testing report that confirms a diagnosis of LGMD

Exclusion Criteria:

You or your child do not have a diagnosis of LGMD
Your child is under age 6
You or your child are not able to travel to the study site

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Years

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

Patients with a diagnosis of GENETICALLY CONFIRMED LGMD

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Ibrahim Binalsheikh, MD	Carolinas Medical Center - Pediatrics	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Carolinas Medical Center - Pediatrics		Charlotte	North Carolina	28203	United States

Condition Browse Module

MeSH Terms

ID	Term
D009136	Muscular Dystrophies

Ancestor Terms

ID	Term
D020966	Muscular Disorders, Atrophic
D009135	Muscular Diseases
D009140	Musculoskeletal Diseases
D009468	Neuromuscular Diseases

Browse Leaves

Not provided

Browse Branches

Not provided