Superior Management for Multinational Rare Disease Program

Premier Research is currently managing a multicenter, multinational, pediatric and adult rare disease program for an orphan drug used in the treatment of Alkaline Phosphatase Deficiency. This program is currently being conducted in the following regions: North America, Europe, Asia, and the Middle East. In the more obscure regions of the world, critical infants who are not within close proximity to a study site are being treated under compassionate use until transportation becomes available to an approved site.

This program is comprised of five studies: one retrospective chart review, one prospective observational study and two prospective interventional studies and an ongoing extension study for which we are providing monitoring support. Services we are currently providing for this program include: Study Start-up, Project Management, Monitoring, Data Management, Data Capture in EDC, Safety Support Services and Programming of Tables and Listings for DSMB meetings.

Enrollment has already been completed for the adult interventional study. Our partnership with this small biotech company began as a two study program; and as a result of our exceptional work on these studies, this program has grown, as noted above, to include three additional studies.

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