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.

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

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 acquired from Horizon in 2024.

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

Retrospective is defined as the 12 months preceding enrollment.

Percentage of participants experiencing HAC (reported for the 12 months preceding
enrollment).

Percentage of participants experiencing HAC (post-Baseline).

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.

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

About this trial

Study ID

Status

Type

Placebo

Accepting

Not accepting

Trial Timing

What is this trial about?

What are the participation requirements?

Locations

Location

Status