Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)

Urea Cycle Disorder Clinical Trial

— THRIVE  

Official title:

Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01948427
• Other study ID #: HPN-100-014
• Status: Terminated
• Start date: September 25, 2013
• Est. completion date: February 24, 2020

Study information

• Verified date: February 2021
• Source: Horizon Pharma Ireland, Ltd., Dublin Ireland
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 203
• Est. completion date: February 24, 2020
• Est. primary completion date: February 24, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Disease
• Urea Cycle Disorder
• Urea Cycle Disorders, Inborn

Locations

Country	Name	City	State
United States	United BioSource Corporation	Blue Bell	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Horizon Therapeutics, LLC

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication	Retrospective is defined as the 12 months preceding enrollment.	12 months prior to enrollment (retrospective), Baseline, Day 7 to 30, Month 6, Month 12, Month 18, Month 24, Month 30, Month 36, Month 42, Month 48, Month 54, Month 60, Month 66, Month 72
Primary	Median Blood Ammonia Levels Over Time, by Last-Known Ammonia-Scavenging Medication	Retrospective is defined as the 12 months preceding enrollment.	12 months prior to enrollment (retrospective), Baseline, Day 7 to 30, Month 6, Month 12, Month 18, Month 24, Month 30, Month 36, Month 42, Month 48, Month 54, Month 60, Month 66, Month 72
Primary	Percentage of Participants With Hyperammonemic Crisis (HAC) by Baseline Ammonia-Scavenging Medication, Retrospective Values	Percentage of participants experiencing HAC (reported for the 12 months preceding enrollment).	12 months prior to enrollment (retrospective)
Primary	Percentage of Participants With Hyperammonemic Crisis (HAC), Post-Baseline by Last Known Ammonia-Scavenging Medication	Percentage of participants experiencing HAC (post-Baseline).	From enrollment through the end of study (mean overall duration on study was 1187.7 days).
Primary	Number of Participants With Serious Adverse Events (SAEs)	An SAE is an adverse event that: is fatal or life-threatening; results in persistent or significant disability or incapacity. Disability is defined as a substantial disruption of a person's ability to conduct normal life functions; requires inpatient hospitalization or prolongation of an existing hospitalization; is a congenital anomaly/birth defect; any other important medical event that may jeopardize the patient and may require medical or surgical intervention to prevent one of the outcomes listed above.	From enrollment through the end of study (mean overall duration on study was 1187.7 days).

External Links

•   THRIVE Registry Website