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

Amgen

Status: Terminated

Conditions: Urea Cycle Disorder

Study type: Observational

Funder types: Industry

Identifiers

NCT01948427

HPN-100-014

Details and patient eligibility

About

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.

Full 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.

Enrollment: 203 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design