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

Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional

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

Brief Summary: This protocol has two parts - the Main Study which is 42 days in length and the Treatment Extension Study 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.

Detailed Summary:

Data to be collected during the Main Study include demographic, baseline disease information and medical history including all prior disease-directed therapy. In addition, vital signs, laboratory values and adverse events information will be collected and recorded. Urine samples will be obtained and measured for orotic acid and orotidine levels. Systemic levels of uridine will be evaluated from plasma samples collected at set timepoints.

Upon successful completion of the Main Study and entry into the Treatment Extension of the study, physical exams and vital signs will be performed every six (6) months. Additionally, plasma samples to measure systemic levels of uridine and urine samples to measure levels of orotic acid and orotidine will be collected every (6) six months.


Sponsor: Wellstat Therapeutics

Current Primary Outcome: Stability of predetermined principal hematologic parameters [ Time Frame: 6 weeks ]

Hereditary orotic aciduria patients will be taking uridine triacetate as replacement therapy for uridine. The primary outcome measure will be based on predetermined principal hematologic parameter(s) based on the patient's response(s) to oral uridine when dosing is switched from oral uridine to oral uridine triacetate. The primary outcome measure in patients not previously receiving uridine replacement therapy will be improvement in the patient's principal affected hematologic parameter(s) on Days 28 and 42 compared to baseline (Day 0) of the Main Study.


Original Primary Outcome: Levels of orotidine and orotic acid in urine [ Time Frame: 6 weeks ]

Hereditary orotic aciduria patients will be taking uridine triacetate as replacement therapy for uridine. Levels of orotidine and orotic acid in the urine will be measured during both the main study as well as the extension study.


Current Secondary Outcome:

  • Levels of orotic acid and orotidine in urine [ Time Frame: Days 0, 1, 28 and 42 ]
    The patients' levels of orotic acid and orotidine in the urine will be measured on Days 0, 1, 28 and 42 of the Main Study.
  • Levels of uridine in the plasma [ Time Frame: Days 0, 1 and 28 ]
    Plasma levels of uridine in treated patients will be measured on Days 0, 1 and 28 of the Main Study.


Original Secondary Outcome:

Information By: Wellstat Therapeutics

Dates:
Date Received: April 1, 2014
Date Started: April 2014
Date Completion: August 2016
Last Updated: May 12, 2016
Last Verified: May 2016