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Pyrimidine Analog

Uridine Triacetate to Replace Uridine for Orotic Aciduria

Phase 3
Waitlist Available
Research Sponsored by Wellstat Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up days 28 and 42
Awards & highlights

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.

Eligible Conditions
  • Orotic Aciduria

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~days 28 and 42
This trial's timeline: 3 weeks for screening, Varies for treatment, and days 28 and 42 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Patients With Stable Predetermined Principal Hematologic Parameters
Secondary study objectives
Patients With Levels of Uridine in the Plasma Consistent With Expected Therapeutic Benefit
Patients With Stable or Improved Orotic Acid and Orotidine Levels

Trial Design

1Treatment groups
Experimental Treatment
Group I: Uridine Triacetate to Replace UridineExperimental Treatment1 Intervention
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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Uridine triacetate
FDA approved

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Who is running the clinical trial?

Wellstat TherapeuticsLead Sponsor
5 Previous Clinical Trials
454 Total Patients Enrolled
Michael K. Bamat, Ph.D.Study DirectorWellstat Therapeutics
~0 spots leftby Sep 2025
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