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Trial record 35 of 103 for:    Recruiting, Not yet recruiting, Available Studies | "Muscular Dystrophies"

Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy

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ClinicalTrials.gov Identifier: NCT02470962
Recruitment Status : Recruiting
First Posted : June 12, 2015
Last Update Posted : May 24, 2018
Sponsor:
Information provided by (Responsible Party):
University Children's Hospital, Zurich

Brief Summary:
This study evaluates the function of the heart in young patients with muscular dystrophy type Duchenne or Becker. Participants have their hearts examined at regular intervals by ultrasound (echocardiography) and cardiac magnetic resonance imaging.

Condition or disease Intervention/treatment
Duchenne / Becker Muscular Dystrophy Other: Observation

Detailed Description:
Muscular dystrophy leads to progressive loss of function in all muscles during childhood and adolescence, including the heart. The usual method to evaluate the heart is echocardiography, emphasizing few parameters. Cardiac magnetic resonance imaging is not as widely available as echocardiography, but early changes can be detected before they become visible on echocardiography. In this study, the investigators compare the methods of measuring heart function in order to find the best measurements for follow up and to see how fast the degenerative changes occur in the hearts of patients with muscular dystrophy.

Study Type : Observational
Estimated Enrollment : 40 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Cardiac Involvement in Patients With Duchenne/Becker Muscular Dystrophy
Study Start Date : May 2015
Estimated Primary Completion Date : April 2020
Estimated Study Completion Date : April 2020


Group/Cohort Intervention/treatment
Patients with muscular dystrophy
Boys aged 8 to 18 years with muscular dystrophy of the Duchenne / Becker type
Children without heart disease
Children without heart disease, aged 8-18 years, as CMR comparison group
Other: Observation
Observation by serial echocardiography with extended techniques and cardiac magnetic resonance imaging




Primary Outcome Measures :
  1. Left ventricular ejection fraction [ Time Frame: 3 years per patient ]

Secondary Outcome Measures :
  1. Quantification of fibrosis by LGE/T1 mapping [ Time Frame: 3 years per patient ]
  2. NT-proBNP [ Time Frame: 3 years per patient ]


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Ages Eligible for Study:   8 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   Yes
Sampling Method:   Non-Probability Sample
Study Population
  • Boys aged 8 to 18 years with DMD/BMD confirmed genetically or by muscle biopsy
  • Control group for CMR: children without heart disease
Criteria

Inclusion Criteria:

  • Boys aged 8 to 18 years with DMD/BMD confirmed genetically or by muscle biopsy
  • Informed consent

Exclusion Criteria:

  • Other clinically significant concomitant disease states (e.g., renal failure)
  • Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, dementia, etc. of the participant or his/her parents or legal caregivers,
  • Inability to lie still for the duration of the imaging procedures (approximately 45 minutes each for echocardiography and CMR)
  • MR-incompatible implanted or accidentally incorporated metal device or claustrophobia that prohibits use of magnetic resonance imaging

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02470962


Contacts
Contact: Hilfiker Silvia +41442663339 silvia.hilfiker@kispi.uzh.ch

Locations
Switzerland
Children's Hospital Recruiting
Zürich, ZH, Switzerland, 8032
Contact: Silvia Hilfiker    +41442663339    silvia.hilfiker@kispi.uzh.ch   
Contact: Barbara EU Burkhardt, MD    +41442667062    barbara.burkhardt@kispi.uzh.ch   
Sub-Investigator: Emanuela R Valsangiacomo Büchel, MD PhD         
Sub-Investigator: Andrea Klein, MD PhD         
Sponsors and Collaborators
University Children's Hospital, Zurich
Investigators
Principal Investigator: Barbara EU Burkhardt, MD Children's Hospital Zürich, Switzerland

Responsible Party: University Children's Hospital, Zurich
ClinicalTrials.gov Identifier: NCT02470962     History of Changes
Other Study ID Numbers: DMD-Herz
First Posted: June 12, 2015    Key Record Dates
Last Update Posted: May 24, 2018
Last Verified: May 2018

Additional relevant MeSH terms:
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked