Trial Outcomes & Findings for Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria (NCT NCT02110147)

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

4 participants

Primary outcome timeframe

Days 28 and 42

Results posted on

2017-07-31

Participant Flow

Participant milestones

Participant milestones
Measure	Single Arm All patients were treated with uridine triacetate oral granules.
Overall Study STARTED	4
Overall Study COMPLETED	4
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Single Arm n=4 Participants All patients were treated with uridine triacetate oral granules.
Age, Categorical <=18 years	3 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	1 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	1 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	4 participants n=5 Participants

PRIMARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Uridine Triacetate n=4 Participants All patients were treated with uridine triacetate oral granules.
Patients With Stable Predetermined Principal Hematologic Parameters	4 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Uridine Triacetate n=4 Participants All patients were treated with uridine triacetate oral granules.
Patients With Stable or Improved Orotic Acid and Orotidine Levels Day 42	4 Participants
Patients With Stable or Improved Orotic Acid and Orotidine Levels Day 28	4 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure	Uridine Triacetate n=4 Participants All patients were treated with uridine triacetate oral granules.
Patients With Levels of Uridine in the Plasma Consistent With Expected Therapeutic Benefit Day 1	4 Participants
Patients With Levels of Uridine in the Plasma Consistent With Expected Therapeutic Benefit Day 28	4 Participants

Adverse Events

Single Arm

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Wellstat Medical Information

Wellstat Therapeutics Corporation

Phone: 800.914.0071

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place