Nationwide Carrier Study

Characterization of Clinical Skeletal and Cardiac Impairment in Carriers of Duchenne and Becker

Questions and Answers

What stage is this research?

This is an actively recruiting longitudinal observational clinical study.

What is the goal or purpose of this study?

To study the neuromuscular, cardiovascular and psychological/cognitive impact of being a genetic carrier of Duchenne or Becker muscular dystrophy.

Who is the sponsor of this study?

This study is sponsored by Parent Project Muscular Dystrophy.

What are the inclusion (enrollment) criteria for this study?

  • 18 years of age and older
  • Able to complete testing in English
  • A genetically confirmed mutation in the Duchenne gene with an affected child

OR
Duchenne and Becker mothers with NO somatic mutation in the Duchenne gene (females who are not carriers of Duchenne/Becker but have an affected child)

What do I have to do if I decide to participate in this study?

The study consists of three visits which take place on an annual basis. Each visit spans the course of one to two days and includes a number of procedures that address the neuromuscular, cardiovascular, psychological, and cognitive impact that you may experience. If you are interested in taking part in this study, please contact the study coordinator at Nationwide Children’s Hospital to find out more about what’s involved and decide if you would like to schedule a study visit.

Where does the study take place?

This study takes place at Nationwide Children’s Hospital in Columbus, Ohio.

Are travel expenses to the study site reimbursed?

The study is currently able to provide up to $500 in reimbursement for travel and lodging expenses. Please contact the study coordinator for additional information about reimbursement.

Why should I consider participating in this study?

While a primary benefit of the study is in understanding the impact of being a genetic carrier of Duchenne or Becker, the study also provides and pays for several tests such as an MRI and Cardiovascular Stress test which may be of value to you or your physician.

Where can I learn more about this study?

Contact study coordinator:
Eric Camino
614-722-2715
Eric.camino@nationwidechildrens.org
www.clinicaltrials.gov (NCT02972580)

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