CASE STUDY Creating Solutions to Ensure Endpoint Protection in Challenging Rare Disease TrialRegions France, Netherlands, Sweden, United Kingdom, United States STUDY DESCRIPTION A sponsor engaged with PRA to implement the first clinical trial in a specific rare disease. Study Duration 44 months No. of Clinical Sites 18 sites Patient Population 24 patients Creating Solutions to Ensure Endpoint Protection in Challenging Rare Disease Trial Primary Endpoint: Safety, tolerability, and efficacy of medical product Study Phase: Phase II The collection of high-quality endpoint data was paramount to the success of this Phase II program. Because this was the first clinical trial ever done for this dise ase, it was necessary to develop non- disease-specific clinical assessments, in co

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