Muscular Dystrophy, DuchenneCPINCT02195999
Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
An observational study of Muscular Dystrophy, Duchenne, run by University of Florida at 1 site in United States.
Trial summary
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well as determine whether peripheral skeletal muscle… Read more about this study
- Who is it for?
- Ages 5 to 15 years, men only
- Conditions the study is for
- Muscular Dystrophy, Duchenne
- What is being tested?
- Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT) and Metabolic Exercise Testing using stationary bicycle
- How many people
- 9 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2013to 6 Mar 2019
- 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 5 to 15 years
- Men only
- No healthy volunteers
- Child
Study population: As the focus of this study is on validating novel measures of cardiac and respiratory function in individuals with DMD, this study will utilize a single-group design with up to 60 males with DMD between 5 and 15 years old upon entry to the study. The participant selection is limited to children since the detrimental effects of DMD begin in early childhood, and the life expectancy of these boys is shortened to the early- to mid-20s. Only males will be eligible to participate in the study because DMD is an X-linked recessive genetic disorder that only leads to the characteristic disease in males. Although females may be carriers, they do not exhibit the same phenotype as males.
Who may be able to join
- Male
- 5-15 years old at the time of enrollment
- Diagnosed with DMD (as defined by parent project)
- Written parental informed consent (and assent where appropriate) before any study procedures take place
Who cannot join
- Contraindication to an MRI examination
- Presence of a secondary condition that impacts muscle function or metabolism, that leads to developmental delay or impaired motor control, or that is not stable
- Participant is unable to comply with study requirements
- Congenital structural abnormality of the heart, repaired or unrepaired
- Clinically contraindicated participation
Trial locations and contacts
1 site in 1 country.
United States1 site
Clinical and Translational Research Building
Gainesville, Florida, 32610
About this study
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well as determine whether peripheral skeletal muscle dysfunction can predict cardiopulmonary dysfunction. The central hypothesis is that non-invasive MRI measures of the heart, muscle, and peripheral skeletal muscles can sensitively predict future cardiopulmonary decline.
Read the detailed description
Magnetic Resonance Imaging (MRI) of the heart and breathing muscles, special breathing tests (called pulmonary function testing), special exercise tests (using a stationary bike), and possibly an echocardiogram (ultrasound of the heart, commonly known as an "echo") will be completed up to 4 times per year for up to 4 years. Most participants will complete testing once or twice each year; however, some participants will be asked to repeat some of the tests twice during each visit.
- Conditions
- Muscular Dystrophy, Duchenne
- Being tested
- Magnetic Resonance Imaging (MRI), Pulmonary Function Testing (PFT), Metabolic Exercise Testing using stationary bicycle, Echocardiogram
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Magnetic Resonance Imaging (MRI)Other
It is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.
Pulmonary Function Testing (PFT)Other
It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
Metabolic Exercise Testing using stationary bicycleOther
Metabolic exercise testing, including assessment of exercise capacity and MVO2, evaluates global cardiopulmonary functional status. This is performed with the use of a stationary bicycle.
EchocardiogramOther
Also called Multiple-echo Dixon
The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure.
| Study group | Role | What they receive |
|---|---|---|
| Individuals with DMD | Magnetic Resonance Imaging is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.
Pulmonary Function testing (PFT) are a series of non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
Metabolic exercise testing using stationary bicycle (exercise capacity and MVO2) evaluates global cardiopulmonary functional status.
Echocardiogram with multiple-echo Dixon method helps to assess cross-sectional and longitudinal variations in myocardial structure. Receives: Other: Magnetic Resonance Imaging (MRI); Other: Pulmonary Function Testing (PFT); Other: Metabolic Exercise Testing using stationary bicycle; Other: Echocardiogram |
What is being measured
The primary outcome is the main question the study is designed to answer.
Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)
The MRI T2 and MRS will be used as a noninvasive marker of myocardial damage/inflammation of participants of this study as an early detection for DMD.
Measured: up to 4 years
Secondary outcomes (3)
Pulmonary Function Testing (PFT)
Non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
Measured: up to 4 years
Metabolic Exercise Testing (exercise capacity and MVO2)
With the use of metabolic exercise testing, the aim is to correlate changes in cardiopulmonary function with decline in peripheral skeletal muscle function in individuals with DMD. Metabolic exercise testing includes measuring exercise capacity and maximum oxygen consumption (MVO2).
Measured: up to 4 years
Multiple-echo Dixon
The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure. This method and MRS will also be used for fat fraction determination.
Measured: up to 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jul 2014 (estimated)
- Study startDec 2013
- Primary completion6 Mar 2019
- Study completion6 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Dystrophy, DuchenneStudies of the same condition, open and under way.
- More trials run by University of FloridaThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Florida
- Sponsor type
- University, hospital or other
- Collaborators
- CureDuchenne
- Responsible party
- Sponsor
- Investigators
- Barry Byrne, MD, PhD, Principal investigator, University of Florida
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02195999
- Sponsor's protocol ID
- IRB201300420
- Acronym
- CPI
- Status last verified
- Jul 2019
- First submitted
- 17 Jul 2014
- Last updated
- 8 Jul 2019
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Assessment of Cardiopulmonary Function in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02195999
- Acronym / sponsor ID
- CPI / IRB201300420
- Conditions
- Muscular Dystrophy, Duchenne
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- University of FloridaUniversity, hospital or other
- Collaborators
- CureDuchenne
- Study start
- Dec 2013
- Primary completion
- 6 Mar 2019 (actual)
- Study completion
- 6 Mar 2019 (actual)
- First posted
- 21 Jul 2014 (estimated)
- 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.
This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well as determine whether peripheral skeletal muscle dysfunction can predict cardiopulmonary dysfunction. The central hypothesis is that non-invasive MRI measures of the heart, muscle, and peripheral skeletal muscles can sensitively predict future cardiopulmonary decline.
Detailed description
Magnetic Resonance Imaging (MRI) of the heart and breathing muscles, special breathing tests (called pulmonary function testing), special exercise tests (using a stationary bike), and possibly an echocardiogram (ultrasound of the heart, commonly known as an "echo") will be completed up to 4 times per year for up to 4 years. Most participants will complete testing once or twice each year; however, some participants will be asked to repeat some of the tests twice during each visit.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Individuals with DMD | – | Other: Magnetic Resonance Imaging (MRI), Other: Pulmonary Function Testing (PFT), Other: Metabolic Exercise Testing using stationary bicycle, Other: EchocardiogramMagnetic Resonance Imaging is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction. Pulmonary Function testing (PFT) are a series of non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology. Metabolic exercise testing using stationary bicycle (exercise capacity and MVO2) evaluates global cardiopulmonary functional status. Echocardiogram with multiple-echo Dixon method helps to assess cross-sectional and longitudinal variations in myocardial structure. |
Who may qualify
- Ages
- 5 to 15 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- As the focus of this study is on validating novel measures of cardiac and respiratory function in individuals with DMD, this study will utilize a single-group design with up to 60 males with DMD between 5 and 15 years old upon entry to the study. The participant selection is limited to children since the detrimental effects of DMD begin in early childhood, and the life expectancy of these boys is shortened to the early- to mid-20s. Only males will be eligible to participate in the study because DMD is an X-linked recessive genetic disorder that only leads to the characteristic disease in males. Although females may be carriers, they do not exhibit the same phenotype as males.
Key inclusion criteria (4 listed)
- Male
- 5-15 years old at the time of enrollment
- Diagnosed with DMD (as defined by parent project)
- Written parental informed consent (and assent where appropriate) before any study procedures take place
Key exclusion criteria (5 listed)
- Contraindication to an MRI examination
- Presence of a secondary condition that impacts muscle function or metabolism, that leads to developmental delay or impaired motor control, or that is not stable
- Participant is unable to comply with study requirements
- Congenital structural abnormality of the heart, repaired or unrepaired
- Clinically contraindicated participation
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 |
|---|---|---|
| Magnetic Resonance Imaging (MRI)It is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction. | Other | Individuals with DMD |
| Pulmonary Function Testing (PFT)It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology. | Other | Individuals with DMD |
| Metabolic Exercise Testing using stationary bicycleMetabolic exercise testing, including assessment of exercise capacity and MVO2, evaluates global cardiopulmonary functional status. This is performed with the use of a stationary bicycle. | Other | Individuals with DMD |
| EchocardiogramAlso: Multiple-echo DixonThe echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure. | Other | Individuals with DMD |
Primary outcome
Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)
Time frame: up to 4 years
The MRI T2 and MRS will be used as a noninvasive marker of myocardial damage/inflammation of participants of this study as an early detection for DMD.
3 secondary outcomes
Pulmonary Function Testing (PFT)
Time frame: up to 4 years
Metabolic Exercise Testing (exercise capacity and MVO2)
Time frame: up to 4 years
Multiple-echo Dixon
Time frame: up to 4 years
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 8 Jul 2019.
- 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.
Barry Byrne, MD, PhD principal investigator
University of Florida
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 Muscular Dystrophy, Duchenne, run by University of Florida at 1 site in United States.
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 4 inclusion and 5 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 8 Jul 2019. 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 United States. 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?
Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS) (time frame: up to 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 8 Jul 2019.