A new study to determine the best "outcome measure" (measurable activity) with which to assess thigh-muscle (quadriceps) strength in men with Becker muscular dystrophy (BMD) is seeking participants.
The study, taking place at Nationwide Children's Hospital in Columbus, Ohio, is a necessary prelude to a planned trial of gene therapy involving injections of genes for the follistatin protein in people with BMD.
Follistatin interferes with the actions of the muscle growth-limiting protein myostatin and has shown promise in animal studies.
About the new studies
For the outcome measure study, Nationwide investigators are seeking men with BMD who are at least 18 years old, able to walk for six minutes or more and meet other study criteria. Subjects will be asked to walk for six minutes, will have their strength measured, and will be timed while doing other activities. Use of a cane is permitted for the walking test.
The study is being conducted at Nationwide and is expected to take about two hours of the participants' time during a single visit. Some help with lodging expenses is available for those traveling to Columbus.
The gene therapy trial is not yet approved to recruit participants. When it is, the investigators say, information will be posted at Nationwide Children's Hospital Center for Gene Therapy.
Although these studies have not received MDA support, Nationwide Children's Hospital is part of the MDA/DMD Clinical Research Network (see Speeding the Course of Clinical Trials), which is conducting related studies.
Meaning for people with BMD
There is no direct benefit for people with BMD who participate in the outcome measure study. However, the information will be used to move the follistatin gene therapy study forward. Ultimately, follistatin gene therapy could have benefit for people with BMD.