Expertise In Pediatric Clinical Trials: Fabry Disease

This case study illustrates how Chiltern’s knowledge of rare diseases and expertise in pediatric trials enabled the implementation of successful strategies to produce high quality data in a small patient population.

PROJECT OVERVIEW:

• Multicenter, multinational, phase I/II open-label study in pediatric patients with Fabry disease.
• Primary objective – evaluation of safety, efficacy and pharmacokinetics of a biweekly replacement therapy.
• 16 randomized patients.
• Recruitment period – 15 months. 
• Study duration – 12 week baseline observation period, 48 weeks treatment and 2 weeks follow-up.
• Sites and participating countries – 7 sites in France, Italy, Poland and UK.