Prosensa and GlaxoSmithKline aim to enroll 250 participants in a study to determine the usual progression of Duchenne muscular dystrophy and aid clinical trials
Update (Jan. 27, 2014): Prosensa is now the sole sponsor of this study.
Dutch biotechnology company Prosensa and multinational pharmaceutical company GlaxoSmithKline (GSK) are seeking participants with Duchenne muscular dystrophy (DMD) who are 3-18 years old for a large-scale study of the disorder's "natural history" (usual course over time).
Prosensa is the developer of drisapersen and other experimental compounds to treat DMD, and GSK has been involved in drisapersen's development.
"The goal of this observational study is to characterize DMD at various stages of progression using the same measures used in ongoing clinical studies, such as the 'six-minute walk test,' said a Nov. 7, 2013, press release from Prosensa, in which the company announced it had enrolled 100 participants out of its anticipated 250.
In the same release, Giles Campion, Prosensa's chief medical officer, said, "This study will foster greater understanding of the progression of this debilitating disease and will help explore new endpoints that could be used to expedite drug development."
About the DMD natural history study
The study will take place at 16 sites in 10 countries. U.S. sites are in Cincinnati and Columbus, Ohio, and in Sacramento, Calif.
Participants will be seen every six months for three years (a total of seven visits), but they can choose to stop participating at any time and can leave the natural history study and move into a drug study if one is available. (There is no medication being tested in the natural history study.)
The natural history study will:
During each study visit, participants are asked to:
Prospective participants must:
Prospective participants must not take any investigational drugs during the study, although other medications are permitted.
For additional study details and for contact information for sites in Argentina, Belgium, Brazil, France, Germany, Italy, the Netherlands, Sweden and Turkey, see A Prospective Natural History Study of Progression of Subjects with Duchenne Muscular Dystrophy (or enter NCT01753804 in the search box at ClinicalTrials.gov).
U.S. study sites and contact information: