Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Information source: Horizon Pharma Ireland, Ltd., Dublin Ireland
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Urea Cycle Disorder
Phase: N/A
Status: Recruiting
Sponsored by: Horizon Pharma Ireland, Ltd., Dublin Ireland Overall contact: United Biosource Corporation, Phone: 855-UCD-2595, Email: thriveregistry@unitedbiosource.com
Summary
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE
will follow enrolled participants for up to 10 years. As an observational study, enrolled
patients will not be required to make any additional office visits or take any medicine
outside of normal care.
Clinical Details
Official title: Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)
Study design: Observational Model: Cohort, Time Perspective: Prospective
Primary outcome: Assessment of UCD specific disease characteristics
Detailed description:
UCDs disproportionately affect children and females: depending on the severity of the
defect, a UCD can manifest shortly after birth or later in life. This study will track
long-term outcomes in UCD patients and effects of ammonia-scavenging agents on
neuropsychological functions of UCD patients.
This is a non-interventional, multi-center registry to be conducted in patients with UCDs.
Investigators will prescribe treatments based on usual clinical practice, and there will be
no restrictions on the use of commercially available medications. As an observational study,
this study will not change the patient/ healthcare provider relationship, nor influence the
healthcare provider's drug prescription or the therapeutic management of the patient.
Patients with UCDs will be recruited and invited to attend a Baseline visit. After eligible
patients are enrolled, retrospective and baseline data will be collected. Patients will be
followed for up to 10 years, during which time they will be assessed by their healthcare
provider. Patients and healthcare provider will be asked to report episodes of
hyperammonemic crisis, available ammonia values, and other information.
Eligibility
Minimum age: N/A.
Maximum age: N/A.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- Confirmed or suspected diagnosis of UCD
- Signed informed consent/Health Insurance Portability and Accountability Act (HIPAA)
Authorization and medical records release
Exclusion Criteria:
- Any other reason that, in the Investigator's opinion, makes the patient unsuitable to
participate in this study.
Locations and Contacts
United Biosource Corporation, Phone: 855-UCD-2595, Email: thriveregistry@unitedbiosource.com
United BioSource Corporation, Blue Bell, Pennsylvania 19422, United States; Recruiting THRIVE Registry Team, Phone: 855-823-2595, Email: thriveregistry@unitedbiosource.com
Additional Information
THRIVE Registry Website
Starting date: September 2013
Last updated: June 8, 2015
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