NCT02470962

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
59

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Jun 2015

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2015

Completed
9 days until next milestone

First Submitted

Initial submission to the registry

June 10, 2015

Completed
2 days until next milestone

First Posted

Study publicly available on registry

June 12, 2015

Completed
6.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 22, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 22, 2022

Completed
Last Updated

March 20, 2025

Status Verified

March 1, 2025

Enrollment Period

6.8 years

First QC Date

June 10, 2015

Last Update Submit

March 17, 2025

Conditions

Outcome Measures

Primary Outcomes (1)

  • Left ventricular ejection fraction

    3 years per patient

Secondary Outcomes (2)

  • Quantification of fibrosis by LGE/T1 mapping

    3 years per patient

  • NT-proBNP

    3 years per patient

Study Arms (2)

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

Interventions

Observation by serial echocardiography with extended techniques and cardiac magnetic resonance imaging

Children without heart disease

Eligibility Criteria

Age8 Years - 18 Years
Sexmale
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-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

You may qualify if:

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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Children's Hospital

Zurich, Canton of Zurich, 8032, Switzerland

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Observation

Condition Hierarchy (Ancestors)

Muscular DystrophiesMuscular Disorders, AtrophicMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

MethodsInvestigative Techniques

Study Officials

  • Barbara EU Burkhardt, MD

    Children's Hospital Zürich, Switzerland

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
CASE ONLY
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 10, 2015

First Posted

June 12, 2015

Study Start

June 1, 2015

Primary Completion

March 22, 2022

Study Completion

March 22, 2022

Last Updated

March 20, 2025

Record last verified: 2025-03

Locations