Center for Global Research Data
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CureDuchenne
| About | CureDuchenne is recognized as the global leader in research, patient care and innovation for improving and extending the lives of children with Duchenne. |
| Will individual participant data be available (including data dictionaries)? | Yes |
| What data in particular will be shared? | All of the individual participant data collected, after deidentification, for placebo subjects from the phase 2 and 3 randomized, double-blind, placebo-controlled clinical trials of drisapersen for the treatment of Duchenne muscular dystrophy (DMD) as well as observational studies.
The PRO-DMD-01 study was a prospective observational study of disease progression of boys with Duchenne muscular dystrophy. |
| Exceptions | CureDuchenne is only able to approve the use of the data for research purposes at this time and not for use by a commercial organization or as part of a registration package. For-profit entities should contact CureDuchenne directly via heather@cureduchenne.org to verify use and arrangements. |
| What documents will be available? | For clinical trials:
Study data, in a format similar to SDTM Protocols with all amendments Annotated case report forms Dataset specifications (“define” files) For observation studies: Raw datasets Protocol Operations manual |
| For what types of analyses? | Investigation of placebo response and the outcomes of comparator (placebo) subjects in DMD clinical trials |
| By what mechanism will the data be made available? | Using the Vivli Platform via secure download |