NCT04694547

Brief Summary

The purpose of this survey is to report experiences with variants of a ketogenic diet in a group of individuals diagnosed with McArdle disease (glycogen storage disease type V). Further more the survey will report on physical activity, fatigue, sleep and quality of life in this cohort.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
200

participants targeted

Target at P75+ for all trials

Timeline
Completed

Started Jan 2021

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

First Submitted

Initial submission to the registry

October 5, 2020

Completed
3 months until next milestone

First Posted

Study publicly available on registry

January 5, 2021

Completed
5 days until next milestone

Study Start

First participant enrolled

January 10, 2021

Completed
1.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2022

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2022

Completed
Last Updated

February 13, 2023

Status Verified

February 1, 2023

Enrollment Period

1.8 years

First QC Date

October 5, 2020

Last Update Submit

February 10, 2023

Conditions

McArdle Disease

Outcome Measures

Primary Outcomes (5)

  • Questions on experiences with a ketogenic diet

    Survey

    Baseline

  • IPAQ (INTERNATIONAL PHYSICAL ACTIVITY QUESTIONNAIRE) short form

    Survey

    Baseline

  • MFI-20 (MULTIDIMENSIONAL FATIGUE INVENTORY)

    Survey

    Baseline

  • ISI (insomnia severity index)

    Survey

    Baseline

  • MGQOL 15 score (Myasthenia Gravis Quality Of Life)

    Survey

    Baseline

Interventions

There is no intervention, as this is a survey studyOTHER

Survey

Eligibility Criteria

Age18 Years - 100 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

McArdle disease

You may qualify if:

  • Verified GSDV diagnosis

You may not qualify if:

  • Age under 18 years

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Copenhagen Neuromuscular Center, Rigshospitalet

Copenhagen, Denmark

Location

Related Publications (1)

  • Reason SL, Lokken N, Voermans N. International patient group harnesses social media to help inform rare disease research: use of a low carbohydrate ketogenic diet in McArdle disease. Curr Opin Endocrinol Diabetes Obes. 2021 Oct 1;28(5):441-445. doi: 10.1097/MED.0000000000000663.

    PMID: 34269712DERIVED

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 ONLY
Time Perspective
CROSS SECTIONAL
Sponsor Type
OTHER
Responsible Party
SPONSOR INVESTIGATOR
PI Title
Principal Investigator, MD

Study Record Dates

First Submitted

October 5, 2020

First Posted

January 5, 2021

Study Start

January 10, 2021

Primary Completion

November 1, 2022

Study Completion

December 1, 2022

Last Updated

February 13, 2023

Record last verified: 2023-02

Locations

DK(1)