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

Approved for 5 Other Conditions

No Placebo-Only Group

Pivotal Trial

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ days 28 and 42

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

Awards & Highlights

Approved for 5 Other Conditions

This treatment demonstrated efficacy for 5 other conditions.

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

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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