Prosensa, GSK Conducting DMD Natural History Study

Prosensa and GlaxoSmithKline aim to enroll 250 participants in a study to determine the usual progression of Duchenne muscular dystrophy and aid clinical trials

Article Highlights:
  • Natural history studies help researchers design and interpret the results of clinical trials.
  • The natural history study in Duchenne MD has enrolled 100 participants out of a planned 250.
  • The study is taking place at 16 sites in 10 countries; three sites are in the U.S.
by Margaret Wahl on November 14, 2013 - 11:01am

Update (June 25, 2014): Prosensa announced in a June 25, 2014, press release that it has completed enrollment for this natural history study. The study is no longer open to new participants. The study is expected to be completed in November 2017, but an interim analysis is expected later in 2014.

Update (Jan. 27, 2014): Prosensa is now the sole sponsor of this study.

original story:

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). (Editor's note: Prosensa later became the sole sponsor of this study.)

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:

  • determine the muscle strength and function of children and adolescents with DMD and its evolution over time;
  • assess the quality of life for DMD patients; and
  • measure proteins found in the blood and urine that may be linked to disease progression.

During each study visit, participants are asked to:

  • perform muscle testing with a physical therapist to see how well they can walk, run, jump and climb stairs, move their arms, legs, hands and fingers, and breathe in a machine;
  • answer surveys about quality of life and how they think they can perform daily tasks; and
  • provide blood and urine samples.

Prospective participants must:

  • have a diagnosis of DMD with any type of dystrophin gene mutation (exon deletion, duplication, stop codon, etc.); and
  • be 3-18 years old.

Prospective participants must not take any investigational drugs during the study, although other medications are permitted.

To participate

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

U.S. study sites and contact information:


  • Erica Goude, University of California at Davis Health System, Sacramento, (916) 734-0968,


  • Sarah Jacobson, Cincinnati Children's Hospital Medical Center, (513) 803-9036,
  • Susan Gailey, Nationwide Children's Hospital, Columbus, (614) 355-2897,
  • Krista Kunkler, Nationwide Children's Hospital, Columbus, (614) 722-2238,
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