Clinical trial NCT01948427: Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
Official title: Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)
Study type: OBSERVATIONAL
Overall status: TERMINATED
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
Lead sponsor: Amgen
Sponsor class: INDUSTRY
First posted: 2013-09-23
Enrollment: 203
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with a confirmed or suspected diagnosis of UCD

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.

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.

Eligibility criteria: 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.

Primary outcomes:
measure: Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication
timeFrame: 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
measure: Median Blood Ammonia Levels Over Time, by Last-Known Ammonia-Scavenging Medication
timeFrame: 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
measure: Percentage of Participants With Hyperammonemic Crisis (HAC) by Baseline Ammonia-Scavenging Medication, Retrospective Values
timeFrame: 12 months prior to enrollment (retrospective)
measure: Percentage of Participants With Hyperammonemic Crisis (HAC), Post-Baseline by Last Known Ammonia-Scavenging Medication
timeFrame: From enrollment through the end of study (mean overall duration on study was 1187.7 days).
measure: Number of Participants With Serious Adverse Events (SAEs)
timeFrame: From enrollment through the end of study (mean overall duration on study was 1187.7 days).
Secondary outcomes:
Other outcomes:
Locations:
city: Blue Bell
country: United States
facility: United BioSource Corporation
state: Pennsylvania
Study officials:
affiliation: Amgen
name: MD
role: STUDY_DIRECTOR
References: