CureDuchenne Teams Up with Vivli to Share DMD Clinical Trial Data

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by Vanessa Pataia |

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Seeking to improve treatment of Duchenne muscular dystrophy (DMD), CureDuchenne has teamed up with Vivli to make clinical trial data available to researchers around the world.

The Vivli platform, which currently hosts information from more than 4,700 clinical trials, works as a neutral broker between data contributors such as CureDuchenne, users, and the wider data-sharing community.

On the platform, researchers can find a data repository, an in-depth search engine, and a secure research environment with cloud-based analytical tools. Vivli, an independent non-profit organization, also harmonizes governance, policy and processes to simplify data-sharing from completed clinical trials.

“We’re committed to sharing data as a way to fuel treatment breakthroughs for muscular dystrophy. Vivli makes that possible.” Debra Miller, founder and CEO of CureDuchenne, said in a press release.

CureDuchenne is a leading nonprofit in the U.S. that re-invests 90% of its earnings into multiple research programs, with the goal of developing different approaches to treat DMD, which is the most common and lethal form of muscular dystrophy. 

The non-profit has been involved in the development of BioMarin Pharmaceutical‘s Kyndrisa (drisapersen), a medication intended to promote the production of a shorter, but functional, version of dystrophin. People with DMD lack or have too little of this key protein for muscle integrity due to mutations to the DMD gene.

Although 24-week treatment with Kyndrisa improved the physical capacity of boys with DMD in Phase 2 clinical trials (NCT01153932 and NCT01462292), it failed to stop disease progression effectively in the DEMAND III Phase 3 study (NCT01254019). A subsequent analysis of this trial found that Kyndrisa could still be beneficial for younger, less-impaired patients, underscoring the importance of early treatment.

BioMarin halted development of Kyndrisa in 2016 after the U.S. Food and Drug Administration refused to approve the therapy. GlaxoSmithKline had backed out several years earlier.

CureDuchenne will anonymize individual data from patients who participated in the Phase 2 and Phase 3 clinical trials of drisapersen and in other observational studies to make it available to scientists worldwide on the Vivli platform.

“We’re delighted to have CureDuchenne join as a member of Vivli and make this valuable data available. We welcome researchers interested in Duchenne to visit the Vivli platform to access these datasets and potentially uncover new insights in muscular dystrophy,” said Rebecca Li, Vivli’s executive director.