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

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

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.

Study Overview

• Status: Terminated
• Conditions: Urea Cycle Disorder

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.

Study acquired from Horizon in 2024.

• Study Type: Observational
• Enrollment (Actual): 203

Contacts and Locations

Study Locations

• United States
  • Pennsylvania
    • Blue Bell, Pennsylvania, United States, 19422
      • United BioSource Corporation

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with a confirmed or suspected diagnosis of UCD

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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)
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).
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).

Collaborators and Investigators

• Sponsor: Amgen
• Investigators: Study Director: MD, Amgen

Publications and helpful links

Helpful Links

• THRIVE Registry Website

Study record dates

Study Major Dates

• Study Start (Actual): September 25, 2013
• Primary Completion (Actual): February 24, 2020
• Study Completion (Actual): February 24, 2020

Study Registration Dates

• First Submitted: September 16, 2013
• First Submitted That Met QC Criteria: September 18, 2013
• First Posted (Estimated): September 23, 2013

Study Record Updates

• Last Update Posted (Estimated): July 1, 2024
• Last Update Submitted That Met QC Criteria: June 18, 2024
• Last Verified: June 1, 2024

More Information

Terms related to this study

• Keywords: UCD; Ammonia; Citrulline; BUPHENYL; RAVICTI; Urea; Urea cycle disorder; Glycerol phenylbutyrate; Benzoate; Sodium phenylbutyrate; Ornithine transcarbamylase (OTC); Argininosuccinate synthetase (ASS); Argininosuccinate lyase (ASL); Carbamyl phosphate synthetase (CPS); Arginine
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Amino Acid Metabolism, Inborn Errors; Urea Cycle Disorders, Inborn
• Other Study ID Numbers: HPN-100-014

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.
• IPD Sharing Time Frame: Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study.
• IPD Sharing Access Criteria: Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below.
• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR