Muscular DystrophyNCT01633242
Heart Imaging in Children With Muscular Dystrophy
An observational study of Muscular Dystrophy, run by National Heart, Lung, and Blood Institute (NHLBI) at 1 site in United States.
Trial summary
Background: Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function. However, better types of heart imaging studies are needed to improve treatment of heart problems related to muscular dystrophy. Read more about this study
- Who is it for?
- Ages 8 to 100 years
- Conditions the study is for
- Muscular Dystrophy
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 24 Feb 2012to 11 Oct 2016
- 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 8 to 100 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Male or females with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy.
- 8 to 100 years of age.
Who cannot join
- Day-time users of continuous positive airway pressure (CPAP)
- Sip ventilator users
- Invasive ventilator dependent
- Pregnant minors (when uncertain, participants will undergo urine testing) or lactating minors
- Decompensated congestive heart failure (unable to lie flat during CMR)
- Impaired renal excretory function (calculated Glomerular Filtration Rate less than 30mL/min)
Show all 18Show fewer
- Contra-indications to Magnetic Resonance Imaging:
- Cardiac pacemaker or implantable defibrillator
- Cerebral aneurysm clip
- Neural stimulator
- Metallic ocular foreign body
- Harrington-rod
- Any implanted device (i.e. insulin pump, drug infusion device)
- Claustrophobia
- Metal shrapnel or bullet
- Investigator assessment of inability to comply with protocol
- Unable/unwilling to lie still throughout the research procedure or who require sedation
- Persons with cognitive impairment
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, 20892
About this study
Official title: A Pilot Study of Cardiac Magnetic Resonance in Patients With Muscular Dystrophy
Background:
- Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function. However, better types of heart imaging studies are needed to improve treatment of heart problems related to muscular dystrophy. Better heart imaging methods are especially needed for children with muscular dystrophy. Researchers want to test different heart imaging methods in children with muscular dystrophy. They will look at cardiac magnetic resonance imaging (MRI) and standard heart function tests.
Read more
Objectives:
- To develop and test new methods for imaging the heart in children with muscular dystrophy.
Eligibility:
- Children and adolescents between 8 and 17 years of age who have muscular dystrophy.
Design:
- Participants will be screened with a physical exam and medical history.
- Participants will provide a blood sample at the start of the study. They will also have heart function tests before having the imaging study.
- Participants will have a cardiac MRI scan that will last up to 60 minutes. Some tests will require a MRI contrast agent (a drug that helps the image appear more clearly on the scan).
Read the detailed description
Cardiomyopathy causes significant morbidity and mortality in multiple forms of muscular dystrophy affecting children, including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and subtypes of autosomal recessive limb-girdle muscular dystrophy (LGMD2). Pharmaceutical treatments for the cardiomyopathy of muscular dystrophy, including angiotensin-converting enzyme (ACE) inhibition and beta-adrenergic receptor blockade, afford significant benefit and demonstrate cardiac remodeling in clinical studies. Further studies are needed to identify and characterize more sensitive indicators of cardiac dysfunction in muscular dystrophy subjects to better stratify subjects for entry into clinical protocols. Using the framework of the Cooperative International Neuromuscular Research Group (CINRG) and the Clinical and Translational Science Award (CTSA) consortium, this pilot study will assess cardiac outcome measures in children obtained by gadolinium enhanced cardiac Magnetic Resonance Imaging (MRI) and echocardiographic methods that can be reliably implemented across a consortium of clinical sites devoted to the study of pharmaceutical treatments for muscular dystrophy. These cardiac MRI scans will be shared with the PITT1109 research protocol in all subjects that are enrolled in both studies.
- Conditions
- Muscular Dystrophy
- Keywords
- MRI, Muscular Dystrophy, Cardiac, Myocardium, Intramyocardial fat
- Medical subject headings
- Muscular Dystrophies
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate CMR measures in muscular dystrophy patients
Secondary outcomes (3)
Early Detection
Measurement of Disease Progression
Ultimate identification of the response to developing therapies
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Jul 2012 (estimated)
- Study start24 Feb 2012
- Study completion11 Oct 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by National Heart, Lung, and Blood Institute (NHLBI)The 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
- Time perspective
- Prospective
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Andrew E Arai, M.D., Principal investigator, National Heart, Lung, and Blood Institute (NHLBI)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01633242
- Sponsor's protocol ID
- 120090
- Other IDs
- 12-H-0090
- Status last verified
- 11 Oct 2016
- First submitted
- 30 Jun 2012
- Last updated
- 13 Sep 2019
- Results posted
- No
Publications
Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 Dec 24;51(6):919-28. doi: 10.1016/0092-8674(87)90579-4.
PubMed 3319190Nigro G, Comi LI, Politano L, Bain RJ. The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy. Int J Cardiol. 1990 Mar;26(3):271-7. doi: 10.1016/0167-5273(90)90082-g.
PubMed 2312196Corrado G, Lissoni A, Beretta S, Terenghi L, Tadeo G, Foglia-Manzillo G, Tagliagambe LM, Spata M, Santarone M. Prognostic value of electrocardiograms, ventricular late potentials, ventricular arrhythmias, and left ventricular systolic dysfunction in patients with Duchenne muscular dystrophy. Am J Cardiol. 2002 Apr 1;89(7):838-41. doi: 10.1016/s0002-9149(02)02195-1.
PubMed 11909570
Study at a glance
From the registry record, last updated by the sponsor on 13 Sep 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of Cardiac Magnetic Resonance in Patients With Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01633242
- Acronym / sponsor ID
- 120090
- Conditions
- Muscular Dystrophy
- Keywords
- MRI, Muscular Dystrophy, Cardiac, Myocardium, Intramyocardial fat
- Study type
- Observational
- Recruitment status
- Terminated
- Enrolment
- 2 participants (actual)
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)US National Institutes of Health
- Study start
- 24 Feb 2012
- Study completion
- 11 Oct 2016
- First posted
- 4 Jul 2012 (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.
Background:
- Muscular dystrophy can affect the muscles used for heart function and breathing. Treatment usually involves drugs that help improve heart function. However, better types of heart imaging studies are needed to improve treatment of heart problems related to muscular dystrophy. Better heart imaging methods are especially needed for children with muscular dystrophy. Researchers want to test different heart imaging methods in children with muscular dystrophy. They will look at cardiac magnetic resonance imaging (MRI) and standard heart function tests.
Objectives:
- To develop and test new methods for imaging the heart in children with muscular dystrophy.
Eligibility:
- Children and adolescents between 8 and 17 years of age who have muscular dystrophy.
Design:
- Participants will be screened with a physical exam and medical history.
- Participants will provide a blood sample at the start of the study. They will also have heart function tests before having the imaging study.
- Participants will have a cardiac MRI scan that will last up to 60 minutes. Some tests will require a MRI contrast agent (a drug that helps the image appear more clearly on the scan).
Detailed description
Cardiomyopathy causes significant morbidity and mortality in multiple forms of muscular dystrophy affecting children, including Duchenne muscular dystrophy (DMD), Becker muscular dystrophy (BMD) and subtypes of autosomal recessive limb-girdle muscular dystrophy (LGMD2). Pharmaceutical treatments for the cardiomyopathy of muscular dystrophy, including angiotensin-converting enzyme (ACE) inhibition and beta-adrenergic receptor blockade, afford significant benefit and demonstrate cardiac remodeling in clinical studies. Further studies are needed to identify and characterize more sensitive indicators of cardiac dysfunction in muscular dystrophy subjects to better stratify subjects for entry into clinical protocols. Using the framework of the Cooperative International Neuromuscular Research Group (CINRG) and the Clinical and Translational Science Award (CTSA) consortium, this pilot study will assess cardiac outcome measures in children obtained by gadolinium enhanced cardiac Magnetic Resonance Imaging (MRI) and echocardiographic methods that can be reliably implemented across a consortium of clinical sites devoted to the study of pharmaceutical treatments for muscular dystrophy. These cardiac MRI scans will be shared with the PITT1109 research protocol in all subjects that are enrolled in both studies.
Study design
- Time perspective
- Prospective
Who may qualify
- Ages
- 8 to 100 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Male or females with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy.
- 8 to 100 years of age.
Key exclusion criteria (18 listed)
- Day-time users of continuous positive airway pressure (CPAP)
- Sip ventilator users
- Invasive ventilator dependent
- Pregnant minors (when uncertain, participants will undergo urine testing) or lactating minors
- Decompensated congestive heart failure (unable to lie flat during CMR)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Evaluate CMR measures in muscular dystrophy patients
3 secondary outcomes
Early Detection
Measurement of Disease Progression
Ultimate identification of the response to developing therapies
Results and safety
The study was stopped early. Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Sep 2019.
- Background: PubMed 3319190Hoffman EP, Brown RH Jr, Kunkel LM. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 1987 Dec 24;51(6):919-28. doi: 10.1016/0092-8674(87)90579-4.
- Background: PubMed 2312196Nigro G, Comi LI, Politano L, Bain RJ. The incidence and evolution of cardiomyopathy in Duchenne muscular dystrophy. Int J Cardiol. 1990 Mar;26(3):271-7. doi: 10.1016/0167-5273(90)90082-g.
- Background: PubMed 11909570Corrado G, Lissoni A, Beretta S, Terenghi L, Tadeo G, Foglia-Manzillo G, Tagliagambe LM, Spata M, Santarone M. Prognostic value of electrocardiograms, ventricular late potentials, ventricular arrhythmias, and left ventricular systolic dysfunction in patients with Duchenne muscular dystrophy. Am J Cardiol. 2002 Apr 1;89(7):838-41. doi: 10.1016/s0002-9149(02)02195-1.
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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.
Andrew E Arai, M.D. principal investigator
National Heart, Lung, and Blood Institute (NHLBI)
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, run by National Heart, Lung, and Blood Institute (NHLBI) 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 2 inclusion and 18 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: stopped early, as of the sponsor's last update on 13 Sep 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 was stopped early. Results are not available in the source record.
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?
Evaluate CMR measures in muscular dystrophy patients. 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 NCT01633242, which also lists 3 linked publications.
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 Sep 2019.