BMD: Participants Sought for Registry Associated with Gene Transfer Trial

Investigators are seeking people with Becker muscular dystrophy to participate in a patient registry and potentially in a gene transfer trial

A Becker muscular dystrophy patient registry at Nationwide Children's Hospital in Columbus, Ohio, will be used to identify potential participants for a gene transfer trial and to help interpret results of that trial.
Article Highlights:
  • Investigators at Nationwide Children's Hospital in Columbus, Ohio, are conducting a trial to test the safety of injecting genes for the follistatin protein into the thigh muscles of people with Becker muscular dystrophy and sporadic inclusion-body myositis.
  • People with Becker muscular dystrophy are invited to participate in a registry that complements the follistatin gene transfer study.
  • Registry participation will help with interpretation of trial results and may lead to participation in the gene transfer trial for those who are interested.
by Margaret Wahl on July 1, 2013 - 5:00am

Update (Nov. 19, 2013):Enrollment for the follistatin gene transfer trial for patients with Becker MD is complete. Enrollment for patients with sporadic inclusion-body myositis, however, is not complete. Investigators remain interested in recruiting participants with either BMD or sIBM for the outcome measures study, also known as the patient registry. See BMD, IBM: Outcome Measures Study Seeks Participants for recent information about this study. Also see BMD, IBM: Preliminary Findings in Follistatin Gene Transfer Study Promising for more information about the follistatin gene transfer trial.

In connection with a gene transfer clinical trial, investigators at Nationwide Children's Hospital in Columbus, Ohio, are seeking people with Becker muscular dystrophy (BMD) for participation in a patient registry.

The investigators say the registry will provide information that will:

Follistatin is a protein that promotes muscle growth and strength. In 2009, researchers showed that genes for the follistatin protein increased strength when injected into the leg muscles of monkeys.

The patient registry project — Clinical Outcomes Studies in Becker Muscular Dystrophy (BMD) and Sporadic Inclusion-Body Myositis (sIBM) — involves tests of muscle strength and function. (At this time, the patient registry is seeking only people with BMD, not sporadic inclusion-body myositis.)

Prospective participants in the registry must:

  • have a diagnosis of BMD, with weakness of the quadriceps (thigh) muscles;
  • have some difficulty getting out of chairs, climbing stairs or getting up from the floor;
  • be able to walk independently without the aid of a walker or cane; and
  • be able to cooperate for muscle strength testing.

Prospective participants in the registry must not:

  • have a history of liver disease or HIV infection;
  • have a history of angina or myocardial infarction within the past six months; or
  • be taking prednisone or other immunosuppressive drugs.

To participate in the registry

Contact Lindsay Alfano in Columbus, Ohio, at (614) 722-6881 or lindsay.alfano@nationwidechildrens.org.

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