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

Completed · Phase 3

Conditions studied: Hereditary Orotic Aciduria

In brief

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.

Key facts

Study ID
NCT02110147
Run by
Wellstat Therapeutics
People needed
4
Starts
2014-04-01
Expected to finish
2016-09-08
Last updated by the study team
2017-07-31

Who can join

Age: 1 and older, up to 19. Sex: any. Healthy volunteers: not accepted.

Where it is running

Full record on ClinicalTrials.gov

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