Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs) (THRIVE)
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|ClinicalTrials.gov Identifier: NCT01948427|
Recruitment Status : Active, not recruiting
First Posted : September 23, 2013
Last Update Posted : May 15, 2019
|Condition or disease|
|Urea Cycle Disorder|
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.
|Study Type :||Observational [Patient Registry]|
|Actual Enrollment :||209 participants|
|Target Follow-Up Duration:||10 Years|
|Official Title:||Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)|
|Actual Study Start Date :||September 2013|
|Estimated Primary Completion Date :||September 2026|
|Estimated Study Completion Date :||September 2026|
- Assessment of UCD specific disease characteristics [ Time Frame: Patients will be followed for the duration of the registry. The expected duration is 10 years. ]Assessment of: Blood chemistry, neurocognitive outcomes, growth and development and medications related to the management of UCD.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01948427
|United States, Pennsylvania|
|United BioSource Corporation|
|Blue Bell, Pennsylvania, United States, 19422|