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

NCT ID: NCT02110147

Last Updated: 2017-07-31

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 4 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-04-30
• Study Completion Date: 2016-09-08

Brief 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.

Detailed 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, 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.

Conditions

Hereditary Orotic Aciduria

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Uridine Triacetate to Replace Uridine

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.

Group Type: EXPERIMENTAL

uridine triacetate

Intervention Type: DRUG

Interventions

uridine triacetate

Intervention Type: DRUG

Other Intervention Names

PN401 (Company Code Name); 2',3',5'-tri-O-acetyluridine

Eligibility Criteria

Inclusion Criteria

• 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

• Patient successfully completed the Main Study

Exclusion Criteria

• 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

• Patient did not successfully complete the Main Study

• Minimum Eligible Age: 6 Months
• Maximum Eligible Age: 19 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Wellstat Therapeutics

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Michael K. Bamat, Ph.D.

Role: STUDY_DIRECTOR

Wellstat Therapeutics

Locations

Children's Hospital of Michigan - Specialty Center Detroit

Detroit, Michigan, United States

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, United States

Countries

United States

Other Identifiers

401.13.001

Identifier Type: -

Identifier Source: org_study_id