Overview

This trial is active, not recruiting.

Condition hereditary orotic aciduria
Treatment uridine triacetate
Phase phase 2
Sponsor Wellstat Therapeutics
Start date April 2014
End date December 2014
Trial size 10 participants
Trial identifier NCT02110147, 401.13.001

Summary

This protocol has two parts - the Main Study which is 42 days in length and the Treatment Extension Study 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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
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.
uridine triacetate PN401 (Company Code Name)

Primary Outcomes

Measure
Stability of predetermined principal hematologic parameters
time frame: 6 weeks

Secondary Outcomes

Measure
Levels of orotic acid and orotidine in urine
time frame: Days 0, 1, 28 and 42
Levels of uridine in the plasma
time frame: Days 0, 1 and 28

Eligibility Criteria

Male or female participants from 6 months up to 19 years old.

Inclusion Criteria (Main Study): - Age equal to or less than 19 years - 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 Study) - Patient did not successfully complete the Main Study

Additional Information

Official title Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria
Description 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 of the study, 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.
Trial information was received from ClinicalTrials.gov and was last updated in May 2016.
Information provided to ClinicalTrials.gov by Wellstat Therapeutics.