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Muscular DystrophiesNCT02921321

Pilot Study of Cardiac MR in Patients With Muscular Dystrophy

An observational study of Muscular Dystrophies and Cardiac Fibrosis, run by Children's National Research Institute at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

Muscular Dystrophy can affect the skeletal muscles and also the heart and breathing muscles, causing significant morbidity and mortality. As patients are now living longer, treatment of muscular dystrophies involves drugs that help improve heart function. Read more about this study

Who is it for?
Ages 6 years and older
How many people
60 participants
Where
1 siteUnited States
Study dates
Jan 2014to 9 Dec 2024
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 6 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Study population: Male or females at least 6 years of age with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy.

Who may be able to join

  • Diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy

Who cannot join

  • Day-time users of continuous positive airway pressure (CPAP)
  • Sip ventilator users
  • Invasive ventilator dependent
  • Pregnant minors or adults (when uncertain, participants will undergo urine testing) or lactating minors and adults
  • Females who are nursing and who do not plan to discard breast milk for 24 hours
  • Decompensated congestive heart failure (unable to lie flat during CMR)
Show all 18Show fewer
  • Impaired renal excretory function (calculated Glomerular Filtration Rate less than 30 milliliters/min)
  • Contra-indications to Magnetic Resonance Imaging:
  • Cardiac pacemaker or implantable defibrillator
  • Cerebral aneurysm clip
  • Neural stimulator
  • Metallic ocular foreign body
  • 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

  • Children's National Health System

    Washington D.C., District of Columbia, 20010

Other trials in United States

About this study

Official title: Pilot Study of Cardiac Magnetic Resonance in Patients With Muscular Dystrophy

Muscular Dystrophy can affect the skeletal muscles and also the heart and breathing muscles, causing significant morbidity and mortality. As patients are now living longer, treatment of muscular dystrophies involves drugs that help improve heart function. However, better types of heart imaging studies are needed to understand how these treatments work. Researchers want to improve heart imaging to identify earlier indicators of heart dysfunction in muscular dystrophy patients and how these are changed by medical treatment. The new imaging indicators will also help identify candidates for entry into future clinical trials.

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.

Keywords
MRI, Muscular Dystrophy, Cardiac, Late gadolinium enhancement
Medical subject headings
Muscular Dystrophies, Genetic Diseases, Inborn, Musculoskeletal Diseases

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Myocardial characterization of fibrosis in patients with muscular dystrophy using contrast based magnetic resonance imaging

    Measured: At the end of each MRI scan through study completion, up to 5 years.

Secondary outcomes (3)

  1. Measure the amount of intramyocardial fibrosis using extracellular volume measurements

    Measured: At the end of each MRI scan through study completion, up to 5 years.

  2. Identification and validation of serum biomarker ST2 (Interleukin 1 receptor-like 1 protein ) in the presence of myocardial fibrosis.

    Measured: At the end of the study, up to 10 years

  3. Measure regional myocardial strain and correlate with presence of myocardial fibrosis

    Measured: At the end of each MRI scan through study completion, up to 5 years.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted3 Oct 2016 (estimated)
  2. Study startJan 2014
  3. Primary completion9 Dec 2024
  4. Study completion9 Dec 2024

Resources

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
60 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02921321
Sponsor's protocol ID
4193
Other IDs
Internal Bridge Funding
Status last verified
Dec 2024
First submitted
29 Aug 2016
Last updated
11 Dec 2024
Results posted
No
Data monitoring committee
No
Individual data sharing
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 3319190

  • Nigro 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 2312196

  • Corrado 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

  • Mendell JR, Moxley RT, Griggs RC, Brooke MH, Fenichel GM, Miller JP, King W, Signore L, Pandya S, Florence J, et al. Randomized, double-blind six-month trial of prednisone in Duchenne's muscular dystrophy. N Engl J Med. 1989 Jun 15;320(24):1592-7. doi: 10.1056/NEJM198906153202405.

    PubMed 2657428

  • Cox GF, Kunkel LM. Dystrophies and heart disease. Curr Opin Cardiol. 1997 May;12(3):329-43.

    PubMed 9243091

  • Deburgrave N, Daoud F, Llense S, Barbot JC, Recan D, Peccate C, Burghes AH, Beroud C, Garcia L, Kaplan JC, Chelly J, Leturcq F. Protein- and mRNA-based phenotype-genotype correlations in DMD/BMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene. Hum Mutat. 2007 Feb;28(2):183-95. doi: 10.1002/humu.20422.

    PubMed 17041906

  • Mathews KD, Moore SA. Limb-girdle muscular dystrophy. Curr Neurol Neurosci Rep. 2003 Jan;3(1):78-85. doi: 10.1007/s11910-003-0042-9.

    PubMed 12507416

  • Lim LE, Campbell KP. The sarcoglycan complex in limb-girdle muscular dystrophy. Curr Opin Neurol. 1998 Oct;11(5):443-52. doi: 10.1097/00019052-199810000-00006.

    PubMed 9847993

  • Turakhia MP, Schiller NB, Whooley MA. Prognostic significance of increased left ventricular mass index to mortality and sudden death in patients with stable coronary heart disease (from the Heart and Soul Study). Am J Cardiol. 2008 Nov 1;102(9):1131-5. doi: 10.1016/j.amjcard.2008.06.036. Epub 2008 Aug 27.

    PubMed 18940278

  • Devereux RB, Lutas EM, Casale PN, Kligfield P, Eisenberg RR, Hammond IW, Miller DH, Reis G, Alderman MH, Laragh JH. Standardization of M-mode echocardiographic left ventricular anatomic measurements. J Am Coll Cardiol. 1984 Dec;4(6):1222-30. doi: 10.1016/s0735-1097(84)80141-2.

    PubMed 6238987

  • Gardin JM, McClelland R, Kitzman D, Lima JA, Bommer W, Klopfenstein HS, Wong ND, Smith VE, Gottdiener J. M-mode echocardiographic predictors of six- to seven-year incidence of coronary heart disease, stroke, congestive heart failure, and mortality in an elderly cohort (the Cardiovascular Health Study). Am J Cardiol. 2001 May 1;87(9):1051-7. doi: 10.1016/s0002-9149(01)01460-6.

    PubMed 11348601

  • Haider AW, Larson MG, Benjamin EJ, Levy D. Increased left ventricular mass and hypertrophy are associated with increased risk for sudden death. J Am Coll Cardiol. 1998 Nov;32(5):1454-9. doi: 10.1016/s0735-1097(98)00407-0.

    PubMed 9809962

  • Lavie CJ, Milani RV, Ventura HO, Messerli FH. Left ventricular geometry and mortality in patients >70 years of age with normal ejection fraction. Am J Cardiol. 2006 Nov 15;98(10):1396-9. doi: 10.1016/j.amjcard.2006.06.037. Epub 2006 Oct 2.

    PubMed 17134637

  • Verdecchia P, Carini G, Circo A, Dovellini E, Giovannini E, Lombardo M, Solinas P, Gorini M, Maggioni AP; MAVI (MAssa Ventricolare sinistra nell'Ipertensione) Study Group. Left ventricular mass and cardiovascular morbidity in essential hypertension: the MAVI study. J Am Coll Cardiol. 2001 Dec;38(7):1829-35. doi: 10.1016/s0735-1097(01)01663-1.

    PubMed 11738281

  • Bluemke DA, Kronmal RA, Lima JA, Liu K, Olson J, Burke GL, Folsom AR. The relationship of left ventricular mass and geometry to incident cardiovascular events: the MESA (Multi-Ethnic Study of Atherosclerosis) study. J Am Coll Cardiol. 2008 Dec 16;52(25):2148-55. doi: 10.1016/j.jacc.2008.09.014.

    PubMed 19095132

Questions about this trial

What is this clinical trial studying?

An observational study of Muscular Dystrophies and Cardiac Fibrosis, run by Children's National Research Institute 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 1 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: finished, as of the sponsor's last update on 11 Dec 2024. 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?

Myocardial characterization of fibrosis in patients with muscular dystrophy using contrast based magnetic resonance imaging (time frame: At the end of each MRI scan through study completion, up to 5 years.). 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 NCT02921321, which also lists 51 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 11 Dec 2024.

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