MRI in McArdle Disease (GSDV) | NCT03844022

Studies
MRI in McArdle Disease (GSDV)

NCT03844022

Sponsor

Rigshospitalet, Denmark

Status

Completed

Last Update Posted

Feb 13, 2023Actual

Enrollment

87Actual

Phase

Not provided

Conditions

Glycogen Storage Disease Type V

Interventions

Countries

Denmark

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

Feb 2023

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

Mar 1, 2018Actual

Primary Completion Date

Feb 1, 2021Actual

Completion Date

Feb 1, 2021Actual

First Submitted Date

Dec 12, 2018

First Submission Date that Met QC Criteria

Feb 15, 2019

First Posted Date

Feb 18, 2019Actual

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

Feb 10, 2023

Last Update Posted Date

Feb 13, 2023Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

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 aim of this study is to describe the degree of muscle wasting in patients with McArdle disease judged by MRI, quantitative magnetic resonance (DIXON) and T1 weighted images, and muscle strength, collected across multiple European sites and compared to healthy controls.

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

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

87Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
McArdle disease		Glycogen storage disease	Diagnostic Test: MRI
Healty controls		Age and gender matched	Diagnostic Test: MRI

Interventions

Name	Type	Description	Arm Group Labels	Other Names
MRI	Diagnostic Test	magnetic resonance imaging	Healty controls McArdle disease

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
DIXON MRI	Whole body quantitative muscle fat fraction analyses (DIXON)	1 time

Secondary Outcomes

Measure	Description	Time Frame
Muscle strength	MRC scale	1 time
T1 MRI	Whole body qualitative muscle fat fraction analyses (T1)	1 time

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

GSDV or healthy controls

Exclusion Criteria:

MRI contradictions

Accepts Healthy Volunteers

Yes

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

Not provided

Standard Ages

AdultOlder Adult

Study Population

McArdle disease (GSDV) Healthy controls

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Not provided

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Copenhagen Neuromuscular Center, Rigshospitalet		Copenhagen			Denmark

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
35853747	Derived	Lokken N, Revsbech KL, Jacobsen LN, Martinuzzi A, Martin MA, Diaz-Manera J, Dominguez-Gonzalez C, Brondani G, Musumeci O, Granata F, Stefan C, Merino-Sanchez C, Peralta CN, Khawajazada T, Alonso-Perez J, Toscano A, Vissing J. Muscle MRI in McArdle Disease: A European Multicenter Observational Study. Neurology. 2022 Oct 10;99(15):e1664-e1675. doi: 10.1212/WNL.0000000000200914.

ID	Term
D006008	Glycogen Storage Disease
D002239	Carbohydrate Metabolism, Inborn Errors
D008661	Metabolism, Inborn Errors
D030342	Genetic Diseases, Inborn