Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria

Summary

This protocol has two parts - the Main Study which is 42 days in length and the Treatment Extension which allows the patients who complete the Main Study to continue receiving treatment with uridine triacetate. The purpose of this study is to replace oral administration of uridine with oral administration of uridine triacetate in patients with hereditary orotic aciduria who have received (or would reasonably be expected to receive) clinical benefit from treatment with exogenous uridine. It is also to document the continued clinical benefit of exogenous uridine when patients are switched from oral administration of uridine to oral administration of uridine triacetate.

Timeline

Start

2014-04

Primary completion

2014-12

Completion

2016-09-08

Drugs

Indications