NCT01712152

Brief Summary

Carriers of Duchenne muscular dystrophy (DMD) often have no severe symptoms of the scelet muscles, but they may develop a poor heart function due to the involvement of the heart muscle. Ultrasound of the heart is recommended, but it can detect a cardiac affection only after the heart has already become weaker. Cardiac magnetic resonance tomography can detect myocardial fibrosis already at preserved heart function and may facilitate early therapy. In our study, diagnosed carriers of DMD will undergo examinations of the heart by blood tests, EKG, heart ultrasound and magnetic resonance at enrollment and after one year in order to assess the extent of cardiac affection and its association with the clinical development.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
20

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Oct 2012

Typical duration 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

October 1, 2012

Completed
18 days until next milestone

First Submitted

Initial submission to the registry

October 19, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 23, 2012

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2014

Completed
1.4 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2015

Completed
Last Updated

October 4, 2016

Status Verified

October 1, 2016

Enrollment Period

1.5 years

First QC Date

October 19, 2012

Last Update Submit

October 3, 2016

Conditions

Keywords

Duchenne muscular dystrophycarriercardiac MRImyocardial fibrosisfemale

Outcome Measures

Primary Outcomes (1)

  • T1 mapping

    T1 mapping by MOLLI sequences will be performed and compared between study entry and one year follow-up

    1 year

Secondary Outcomes (1)

  • left ventricular function

    1 year

Eligibility Criteria

Age18 Years+
Sexfemale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

data base of carriers of Duchenne muscular dystrophy

You may not qualify if:

  • Claustrophobia Excessive obesity to an extent where CMR cannot be performed Chronic renal failure with a GFR \<30 ml/min/1,73m² Implanted pacemakers/defibrillators Severe arrhythmia Inability to cooperate during the CMR Known intolerance to gadolinium Positive pregnancy test Unable or unwilling to conform to the study protocol

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

2nd Medical Dept., Rudolfstiftung

Vienna, A-1030, Austria

Location

Related Publications (1)

  • Wexberg P, Avanzini M, Mascherbauer J, Pfaffenberger S, Freudenthaler B, Bittner R, Bernert G, Weidinger F. Myocardial late gadolinium enhancement is associated with clinical presentation in Duchenne muscular dystrophy carriers. J Cardiovasc Magn Reson. 2016 Sep 22;18(1):61. doi: 10.1186/s12968-016-0281-y.

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

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

Study Officials

  • Paul Wexberg, MD

    Hospital Rudolfstiftung

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Priv.-Doz.

Study Record Dates

First Submitted

October 19, 2012

First Posted

October 23, 2012

Study Start

October 1, 2012

Primary Completion

April 1, 2014

Study Completion

September 1, 2015

Last Updated

October 4, 2016

Record last verified: 2016-10

Locations