TerminatedNot Applicable

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

Stopped: the study was terminated due to the primary end point being met.

United States203 participantsStarted 2013-09-25

Plain-language 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.

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

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

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

Percentage of Participants With Hyperammonemic Crisis (HAC) by Baseline Ammonia-Scavenging Medication, Retrospective Values

Timeframe: 12 months prior to enrollment (retrospective)

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).

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).

Trial details

NCT IDNCT01948427

SponsorAmgen

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2020-02-24

Results submitted2020-12-16

View on ClinicalTrials.gov More Urea Cycle Disorder trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

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

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

Percentage of Participants With Hyperammonemic Crisis (HAC) by Baseline Ammonia-Scavenging Medication, Retrospective Values

Timeframe: 12 months prior to enrollment (retrospective)

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).

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).