Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria
Status:
Completed
Trial end date:
2016-09-08
Target enrollment:
Participant gender:
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.