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Heart Imaging in Children With Muscular Dystrophy

12. september 2019 oppdatert av: National Heart, Lung, and Blood Institute (NHLBI)

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.

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).

Studieoversikt

Status

Avsluttet

Forhold

Muskeldystrofi

Detaljert beskrivelse

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.

Studietype

Observasjonsmessig

Registrering (Faktiske)

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

Forente stater
- Maryland
  - Bethesda, Maryland, Forente stater, 20892
    - National Institutes of Health Clinical Center, 9000 Rockville Pike

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

8 år til 100 år (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Beskrivelse

INCLUSION CRITERIA:
Male or females with a diagnosis of Duchenne, Becker, or Limb Girdle Muscular Dystrophy.
8 to 100 years of age.

EXCLUSION CRITERIA:

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)
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

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Hva måler studien?

Primære resultatmål

Resultatmål
Evaluate CMR measures in muscular dystrophy patients

Sekundære resultatmål

Resultatmål
Early Detection
Measurement of Disease Progression
Ultimate identification of the response to developing therapies

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Publikasjoner og nyttige lenker

Den som er ansvarlig for å legge inn informasjon om studien leverer frivillig disse publikasjonene. Disse kan handle om alt relatert til studiet.

Generelle publikasjoner

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart

24. februar 2012

Studiet fullført

11. oktober 2016

Datoer for studieregistrering

Først innsendt

30. juni 2012

Først innsendt som oppfylte QC-kriteriene