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

NCT02110147 | Completed Phase 3 Results

• 1 other identifier: 401.13.001
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 2

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.

Conditions

Hereditary Orotic Aciduria

Outcome Measures

Primary Outcomes (1)

• Patients With Stable Predetermined Principal Hematologic Parameters

  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.

  Days 28 and 42

Secondary Outcomes (2)

• Patients With Stable or Improved Orotic Acid and Orotidine Levels

  Days 28 and 42

• Patients With Levels of Uridine in the Plasma Consistent With Expected Therapeutic Benefit

  Days 1 and 28

Study Arms (1)

Uridine Triacetate to Replace Uridine

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.

Drug: uridine triacetate

Interventions

uridine triacetate DRUG

Also known as: PN401 (Company Code Name), 2',3',5'-tri-O-acetyluridine

Uridine Triacetate to Replace Uridine

Eligibility Criteria

• Age: 6 Months - 19 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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 successfully completed the Main Study
• Patient did not successfully complete the Main Study

Sponsors & Collaborators

• Wellstat Therapeutics: lead

Study Sites (2)

Children's Hospital of Michigan - Specialty Center Detroit

Detroit, Michigan, 48201, United States

Location

Children's Hospital of Pittsburgh of UPMC

Pittsburgh, Pennsylvania, 15224, United States

Location

MeSH Terms

Conditions

Oroticaciduria 1

Interventions

uridine triacetate

Results Point of Contact

• Title: Wellstat Medical Information
• Organization: Wellstat Therapeutics Corporation

medinfo@wellstat.com

800.914.0071

Study Officials

• Michael K. Bamat, Ph.D.

  Wellstat Therapeutics

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 1, 2014
• First Posted: April 10, 2014
• Study Start: April 1, 2014
• Primary Completion: December 1, 2014
• Study Completion: September 8, 2016
• Last Updated: July 31, 2017
• Results First Posted: June 28, 2017

Record last verified: 2017-06

Data Sharing

• IPD Sharing: Will share

Locations

US (2)