Thrive

Overview

Status: Not yet recruiting
Keywords: UCD , Ravicti , Urea Cycle Disorder , Thrive
IRB Number: 00072336
Specialty: Pediatric Genetics
Sub Specialties: Medical Genetics

Brief Summary

Objectives
To characterize the demographics and clinical course of the patient population diagnosed with UCD
To track growth and neurocognitive outcomes for patients with UCDs.
To generate comparative effectiveness data in UCD patients.
 
Study Design
This is a multi-center, prospective, non-interventional study designed to collect data on safety and outcomes in patients with UCDs. It is designed to permit all interested healthcare providers to participate as Investigators, and all interested patients to participate. No controls are planned. Patients enrolled in this registry will be required to provide a select set of data at baseline and to undergo regular office visits and data collection, as indicated in Appendix A.

Principal Investigator: Nicola Longo
Department: Pediatric Genetics
Co Investigator: Lorenzo Botto

Contact Information

Name:Carrie Bailey
Phone: 8015873605
Email: carrie.bailey@hsc.utah.edu

Inclusion Criteria

A patient must meet all of the following criteria to be eligible for participation in the study.
1. Confirmed or suspected diagnosis of UCD.
2. Signed informed consent and HIPAA Authorization, by the patient or a legally acceptable representative, obtained before any study-related activities are undertaken. Assent should be obtained from pediatric patients according to local regulations.
3. Signed medical records release form, by the patient or a legally acceptable representative, permitting abstraction of the patient’s medical records at Baseline and during participation in the study.

Exclusion Criteria

A patient who meets any of the following criteria is not eligible for participation in the study.
1. Any other reason that, in the Investigator's opinion, makes the patient unsuitable to participate in this study.