|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 email@example.com 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:
|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|