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Completed

NCT Number: NCT02110147

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

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.

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Key information

Conditions

Hereditary Orotic Aciduria Oroticaciduria 1

Age range

6 month–19 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Children's Hospital of Michigan - Specialty Center Detroit, Detroit, Michigan, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

(Main Study):

  • Patients with diagnosed hereditary orotic aciduria
  • Judged by the investigator to have the initiative and means to be compliant with the protocol
  • Able to take oral medications
  • Able to provide written informed consent (patient or legally authorized representative)
  • Females of childbearing potential must have a negative pregnancy test at screening
  • Females of childbearing potential or males with partners of childbearing potential are to use one of the following acceptable birth control methods:
  • Surgically sterile or partner is surgically sterile
  • Using adequate contraception (hormonal contraceptives, double barrier methods, or intra-uterine devices)
  • Patients who claim to be sexually inactive agree to use an acceptable method of contraception should she or he become sexually active from 14 days prior to first dosing, throughout the study and for 14 days after the last dose administration

Exclusion criteria

(Main Study):

  • Has a known allergy to uridine triacetate or any of its excipients
  • Known to have ornithine transcarbamoylase deficiency
  • Unable to have the initiative and means to be compliant with the protocol
  • Unable to be compliant with taking oral medications
  • Unable to provide written informed consent (patient or legally authorized representative)
  • Female who is pregnant or lactating

Inclusion criteria

(Treatment Extension)

  • Patient successfully completed the Main Study

Exclusion criteria

(Treatment Extension)

  • Patient did not successfully complete the Main Study

Treatment and study plan

uridine triacetate

Drug

Other names: PN401 (Company Code Name), 2',3',5'-tri-O-acetyluridine

Primary outcomes

  1. 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.

Secondary outcomes

  1. 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.

  2. 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.

Sponsors and collaborators

Lead sponsor

Wellstat Therapeutics

Industry

Registry information

Important dates

Study start
2014
Primary completion
2014
Study completion
2016
First posted
Apr 10, 2014
Registry last updated
Jul 31, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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