Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria
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|ClinicalTrials.gov Identifier: NCT02110147|
Recruitment Status : Completed
First Posted : April 10, 2014
Results First Posted : June 28, 2017
Last Update Posted : July 31, 2017
|Condition or disease||Intervention/treatment||Phase|
|Hereditary Orotic Aciduria||Drug: uridine triacetate||Phase 3|
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, 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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria|
|Actual Study Start Date :||April 2014|
|Actual Primary Completion Date :||December 2014|
|Actual Study Completion Date :||September 8, 2016|
Experimental: Uridine Triacetate to Replace Uridine
Replacement therapy for oral 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. The starting dose of uridine triacetate will be 60 mg/kg/day which may be escalated to 300 mg/kg/day of oral uridine triacetate. The dose may be given once a day or as equally divided doses twice a day.
Drug: uridine triacetate
- Patients With Stable Predetermined Principal Hematologic Parameters [ Time Frame: Days 28 and 42 ]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.
- Patients With Stable or Improved Orotic Acid and Orotidine Levels [ Time Frame: Days 28 and 42 ]Significantly elevated urine orotic acid levels are characteristic of patients with HOA. Therefore, urinary orotic acid and orotidine levels were assessed in patients at baseline (on uridine or uridine naïve) and on Day 28 and Day 42 following the switch to uridine triacetate. The table below shows the number of participants with stable or improved orotic acid and orotidine levels at Day 28 and Day 42 compared to baseline.
- Patients With Levels of Uridine in the Plasma Consistent With Expected Therapeutic Benefit [ Time Frame: Days 1 and 28 ]HOA is principally a chronic uridine deficiency disorder. The purpose of uridine triacetate administration is to provide an exogenous source of uridine. Therefore, plasma uridine levels were assessed at various time points (prior to dosing, 30 min, 1 hour, 2 hours, 4 hours, 6 hours and 8 hours) following uridine triacetate dosing to ensure levels of uridine consistent with previously observed symptomatic improvement from administration of uridine were achieved. The table below shows the number of participants with uridine levels consistent with or exceeding previously observed symptomatic improvements.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02110147
|United States, Michigan|
|Children's Hospital of Michigan - Specialty Center Detroit|
|Detroit, Michigan, United States, 48201|
|United States, Pennsylvania|
|Children's Hospital of Pittsburgh of UPMC|
|Pittsburgh, Pennsylvania, United States, 15224|
|Study Director:||Michael K. Bamat, Ph.D.||Wellstat Therapeutics|