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

NCT01948427 | Terminated Results

• 1 other identifier: HPN-100-014
• Study Type: observational
• Target: 203
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Urea Cycle Disorder

Keywords

UCD; 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; Citrulline; Ammonia

Outcome Measures

Primary Outcomes (5)

• 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).

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with a confirmed or suspected diagnosis of UCD

You may qualify if:

• Confirmed or suspected diagnosis of UCD
• Signed informed consent/Health Insurance Portability and Accountability Act (HIPAA) Authorization and medical records release

You may not qualify if:

• Any other reason that, in the Investigator's opinion, makes the patient unsuitable to participate in this study.

Sponsors & Collaborators

• Amgen: lead

Study Sites (1)

United BioSource Corporation

Blue Bell, Pennsylvania, 19422, United States

Location

Related Links

• THRIVE Registry Website

MeSH Terms

Conditions

Urea Cycle Disorders, Inborn; Ornithine Carbamoyltransferase Deficiency Disease

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases; Genetic Diseases, X-Linked

Limitations and Caveats

Due to data collection complexities in this observational study (e.g., switching of medications, incomplete data on medications, and missing SAE dates) AEs could not be reliably summarized according to a participant's treatment at the time of SAE, and was therefore summarized by treatment at baseline.

Results Point of Contact

• Title: Horizon Therapeutics, LLC
• Organization: Horizon Therapeutics, LLC

clinicaltrials@horizontherapeutics.com

866-479-6742

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Target Duration: 10 Years
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2013
• First Posted: September 23, 2013
• Study Start: September 25, 2013
• Primary Completion: February 24, 2020
• Study Completion: February 24, 2020
• Last Updated: July 1, 2024
• Results First Posted: February 18, 2021

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.
• 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.

Locations

US (1)