Investigators are seeking people with Becker muscular dystrophy to participate in a patient registry and potentially in a gene transfer trial
|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:
Prospective participants in the registry must not:
Contact Lindsay Alfano in Columbus, Ohio, at (614) 722-6881 or firstname.lastname@example.org.