Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of DMD and BMD

Running, not enrolling

Conditions studied: Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

In brief

Longitudinal prospective observational study. This is a 24-month study with the possibility of extending the data time points. Initially baseline, then 12 and 24 months follow up studies will be completed.

Key facts

Study ID

NCT02972580

Run by

Nationwide Children's Hospital

People needed

250

Starts

2016-06-01

Expected to finish

2030-12-01

Last updated by the study team

2024-08-27

Who can join

Age: 18 and older. Sex: female. Healthy volunteers: accepted.

You may qualify if…

• Age >18 years
• Cohort A requires a genetically confirmed mutation in the DMD gene with an affected child
• Cohort B includes DMD/BMD mothers with NO somatic mutation in the DMD gene
• Cohort C age-matched healthy controls with a normal CK level
• Cohort D requires a genetically confirmed mutation in the DMD gene without an affected child
• Able to complete testing in English
• Able to consent

You may not qualify if…

• Subjects with a contraindication to cardiac or skeletal muscle MRI
• Subjects on heart failure medication at time of enrollment
• Subjects on steroid treatment
• Presence of an inherited neurologic disease or comorbidity that may affect their ability to complete this study
• Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability

Where it is running

• Nationwide Children's Hosptial — Columbus, Ohio, United States

Full record on ClinicalTrials.gov

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