Glycogen Storage Disease Type VNCT03844022
MRI in McArdle Disease (GSDV)
An observational study of Glycogen Storage Disease Type V, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Glycogen Storage Disease Type V
- What is being tested?
- MRI
- How many people
- 87 participants
- Where
- 1 siteDenmark
- Study dates
- 1 Mar 2018to 1 Feb 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Study population: McArdle disease (GSDV) Healthy controls
Who may be able to join
- GSDV or healthy controls
Who cannot join
- MRI contradictions
Trial locations and contacts
1 site in 1 country.
About this study
Official title: MRI in McArdle Disease (Glycogen Storage Disease Type V)
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.
- Conditions
- Glycogen Storage Disease Type V
- Being tested
- MRI
- Medical subject headings
- Glycogen Storage Disease Type V
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MRIDiagnostic test
magnetic resonance imaging
| Study group | Role | What they receive |
|---|---|---|
| McArdle disease | Glycogen storage disease Receives: Diagnostic Test: MRI | |
| Healty controls | Age and gender matched Receives: Diagnostic Test: MRI |
What is being measured
The primary outcome is the main question the study is designed to answer.
DIXON MRI
Whole body quantitative muscle fat fraction analyses (DIXON)
Measured: 1 time
Secondary outcomes (3)
Muscle strength
MRC scale
Measured: 1 time
T1 MRI
Whole body qualitative muscle fat fraction analyses (T1)
Measured: 1 time
Back pain questionnaire
Back pain questionnaire
Measured: 1 time
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Feb 2019
- Study start1 Mar 2018
- Primary completion1 Feb 2021
- Study completion1 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Glycogen Storage Disease Type VStudies of the same condition, open and under way.
- More trials run by Copenhagen University Hospital, RigshospitaletThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Enrolment
- 87 participants (actual)
Sponsor and investigators
- Lead sponsor
- Copenhagen University Hospital, Rigshospitalet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Nicoline Løkken, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03844022
- Sponsor's protocol ID
- H-18023049.1
- Status last verified
- Feb 2023
- First submitted
- 12 Dec 2018
- Last updated
- 13 Feb 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 35853747
Study at a glance
From the registry record, last updated by the sponsor on 13 Feb 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- MRI in McArdle Disease (Glycogen Storage Disease Type V)
- ClinicalTrials.gov ID
- NCT03844022
- Acronym / sponsor ID
- H-18023049.1
- Conditions
- Glycogen Storage Disease Type V
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 87 participants (actual)
- Lead sponsor
- Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
- Study start
- 1 Mar 2018 (actual)
- Primary completion
- 1 Feb 2021 (actual)
- Study completion
- 1 Feb 2021 (actual)
- First posted
- 18 Feb 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case-control
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| McArdle disease | – | Diagnostic Test: MRIGlycogen storage disease |
| Healty controls | – | Diagnostic Test: MRIAge and gender matched |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
- Study population
- McArdle disease (GSDV) Healthy controls
Key inclusion criteria (1 listed)
- GSDV or healthy controls
Key exclusion criteria (1 listed)
- MRI contradictions
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MRImagnetic resonance imaging | Diagnostic test | Healty controls, McArdle disease |
Primary outcome
DIXON MRI
Time frame: 1 time
Whole body quantitative muscle fat fraction analyses (DIXON)
3 secondary outcomes
Muscle strength
Time frame: 1 time
T1 MRI
Time frame: 1 time
Back pain questionnaire
Time frame: 1 time
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Feb 2023.
- Derived from this study: PubMed 35853747Lokken 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Glycogen Storage Disease Type V, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 13 Feb 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
DIXON MRI (time frame: 1 time). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03844022, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Feb 2023.