NCT03844022

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
87

participants targeted

Target at P50-P75 for all trials

Timeline
Completed

Started Mar 2018

Typical duration for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2018

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

December 12, 2018

Completed
2 months until next milestone

First Posted

Study publicly available on registry

February 18, 2019

Completed
2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2021

Completed
Last Updated

February 13, 2023

Status Verified

February 1, 2023

Enrollment Period

2.9 years

First QC Date

December 12, 2018

Last Update Submit

February 10, 2023

Conditions

Glycogen Storage Disease Type V

Outcome Measures

Primary Outcomes (1)

  • DIXON MRI

    Whole body quantitative muscle fat fraction analyses (DIXON)

    1 time

Secondary Outcomes (3)

  • Muscle strength

    1 time

  • T1 MRI

    1 time

  • Back pain questionnaire

    1 time

Study Arms (2)

McArdle disease

Glycogen storage disease

Diagnostic Test: MRI

Healty controls

Age and gender matched

Diagnostic Test: MRI

Interventions

MRIDIAGNOSTIC_TEST

magnetic resonance imaging

Healty controlsMcArdle disease

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

McArdle disease (GSDV) Healthy controls

You may qualify if:

  • GSDV or healthy controls

You may not qualify if:

  • MRI contradictions

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Copenhagen Neuromuscular Center, Rigshospitalet

Copenhagen, Denmark

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type V

Condition Hierarchy (Ancestors)

Glycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Study Design

Study Type
observational
Observational Model
CASE CONTROL
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
MD, Research fellow

Study Record Dates

First Submitted

December 12, 2018

First Posted

February 18, 2019

Study Start

March 1, 2018

Primary Completion

February 1, 2021

Study Completion

February 1, 2021

Last Updated

February 13, 2023

Record last verified: 2023-02

Data Sharing

IPD Sharing
Will not share

Locations

DK(1)