Urea Cycle DisorderTHRIVENCT01948427

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

An observational study of Urea Cycle Disorder, run by Amgen at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Trial 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. Read more about this study

Who is it for?: Any age
Conditions the study is for: Urea Cycle Disorder
How many people: 203 participants
Where: 1 siteUnited States
Study dates: 25 Sep 2013to 24 Feb 2020
How long will I take part?: 10 Years
Phase: Observational
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages Any age
All sexes
No healthy volunteers
Child, Adult, Older adult

Study population: Patients with a confirmed or suspected diagnosis of UCD

Who may be able to join

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

Who cannot join

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

Trial locations and contacts

1 site in 1 country.

United States1 site

United BioSource Corporation
Blue Bell, Pennsylvania, 19422

Other trials in United States

About this study

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

Read the 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.

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
Medical subject headings: Urea Cycle Disorders, Inborn, Ornithine Carbamoyltransferase Deficiency Disease

What is being measured

The primary outcome is the main question the study is designed to answer.

Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication
Retrospective is defined as the 12 months preceding enrollment.
Measured: 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.
Measured: 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).
Measured: 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).
Measured: 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.
Measured: From enrollment through the end of study (mean overall duration on study was 1187.7 days).

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

25 Sep 201324 Feb 2020

First posted23 Sep 2013 (estimated)
Study start25 Sep 2013
Primary completion24 Feb 2020
Study completion24 Feb 2020

Why it stopped: the study was terminated due to the primary end point being met.

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Urea Cycle DisorderStudies of the same condition, open and under way.
More trials run by AmgenThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Observational
Observational model: Cohort
Time perspective: Prospective
Enrolment: 203 participants (actual)
Target duration: 10 Years

IDs, dates and publications

ClinicalTrials.gov ID: NCT01948427
Sponsor's protocol ID: HPN-100-014
Acronym: THRIVE
Status last verified: Jun 2024
First submitted: 16 Sep 2013
Last updated: 1 Jul 2024
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: Yes: De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
RecruitingObservational study
Abbott Vascular Medical Device Registry
Acute Myocardial Infarction, Restenoses, Coronary
RecruitingPhase 1/2
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
Active, not recruitingPhase 3
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease

All Urea Cycle Disorder trials

Study at a glance

From the registry record, last updated by the sponsor on 1 Jul 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Long-Term Registry of Patients With Urea Cycle Disorders (UCDs)
ClinicalTrials.gov ID: NCT01948427
Acronym / sponsor ID: THRIVE / HPN-100-014
Conditions: Urea Cycle Disorder
Keywords: UCD, BUPHENYL, RAVICTI, Urea, Urea cycle disorder, Glycerol phenylbutyrate, Benzoate, Sodium phenylbutyrate
Study type: Observational
Recruitment status: TerminatedReason stopped: the study was terminated due to the primary end point being met.
Enrolment: 203 participants (actual)
Lead sponsor: AmgenIndustry (pharma, biotech, devices)
Study start: 25 Sep 2013 (actual)
Primary completion: 24 Feb 2020 (actual)
Study completion: 24 Feb 2020 (actual)
First posted: 23 Sep 2013 (estimated)
Results: Posted 18 Feb 2021

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study acquired from Horizon in 2024.

Study design

Observational model: Cohort
Time perspective: Prospective
Target follow-up: 10 Years

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Patients with a confirmed or suspected diagnosis of UCD

Key inclusion criteria (2 listed)

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

Key exclusion criteria (1 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Primary outcome

Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication
Time frame: 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
Retrospective is defined as the 12 months preceding enrollment.
Median Blood Ammonia Levels Over Time, by Last-Known Ammonia-Scavenging Medication
Time frame: 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
Retrospective is defined as the 12 months preceding enrollment.
Percentage of Participants With Hyperammonemic Crisis (HAC) by Baseline Ammonia-Scavenging Medication, Retrospective Values
Time frame: 12 months prior to enrollment (retrospective)
Percentage of participants experiencing HAC (reported for the 12 months preceding enrollment).
Percentage of Participants With Hyperammonemic Crisis (HAC), Post-Baseline by Last Known Ammonia-Scavenging Medication
Time frame: From enrollment through the end of study (mean overall duration on study was 1187.7 days).
Percentage of participants experiencing HAC (post-Baseline).
Number of Participants With Serious Adverse Events (SAEs)
Time frame: From enrollment through the end of study (mean overall duration on study was 1187.7 days).
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.

Results and safety

Results posted to the registry on 18 Feb 2021, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Sodium Phenylbutyrate	49	42
Ravicti	89	72
Sodium Benzoate	4	4
Carglumic Acid	1	1
Other	60	46

Primary outcome: Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication

Time frame: 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. Mean (Standard Deviation). Unit: µmol/L

Measure	Sodium Phenylbutyrate n = 40	Ravicti n = 67	Sodium Benzoate n = 6	Carglumic Acid n = 2	Other n = 5
Retrospective	86.5 (87.13)	43.8 (27.59)	23.0 (NA)	13.0 (NA)	963.7 (721.39)
Baseline	64.5 (52.32)	49.1 (43.58)	48.0 (29.73)	21.0 (NA)	–
Day 7 to 30	64.0 (20.53)	79.6 (72.23)	55.0 (NA)	–	143.5 (98.29)
Month 6	48.7 (53.77)	45.0 (30.26)	190.3 (257.80)	48.0 (25.46)	129.4 (110.54)
Month 12	42.4 (20.87)	39.4 (24.43)	51.0 (16.97)	20.0 (NA)	79.0 (57.98)
Month 18	54.5 (53.89)	45.9 (28.26)	29.0 (11.79)	13.0 (NA)	106.0 (110.31)
Month 24	53.9 (38.49)	52.8 (78.14)	43.3 (31.26)	25.0 (NA)	–
Month 30	42.0 (18.18)	47.5 (33.27)	79.8 (85.85)	–	114.5 (127.99)

Population analysed: Participants with a Baseline visit and an assessment at given time point. Participants are grouped by last-known ammonia-scavenging medication at given time point (participants may have been on more than 1 therapy during the study).

Primary outcome: Median Blood Ammonia Levels Over Time, by Last-Known Ammonia-Scavenging Medication

Measure	Sodium Phenylbutyrate n = 40	Ravicti n = 67	Sodium Benzoate n = 6	Carglumic Acid n = 2	Other n = 5
Retrospective	46.0 (18.0 to 304.0)	38.0 (11.0 to 95.0)	23.0 (23.0 to 23.0)	13.0 (13.0 to 13.0)	1360.0 (131.0 to 1400.0)
Baseline	50.0 (12.0 to 247.0)	37.0 (11.0 to 247.0)	46.5 (20.0 to 99.0)	21.0 (21.0 to 21.0)	–
Day 7 to 30	60.0 (32.0 to 93.0)	61.5 (25.0 to 249.0)	55.0 (55.0 to 55.0)	–	143.5 (74.0 to 213.0)
Month 6	33.0 (9.0 to 281.0)	34.0 (9.0 to 158.0)	44.0 (39.0 to 488.0)	48.0 (30.0 to 66.0)	96.0 (22.0 to 312.0)
Month 12	42.0 (10.0 to 83.0)	34.5 (9.0 to 122.0)	51.0 (39.0 to 63.0)	20.0 (20.0 to 20.0)	79.0 (38.0 to 120.0)
Month 18	36.0 (20.0 to 226.0)	45.0 (10.0 to 123.0)	26.0 (19.0 to 42.0)	13.0 (13.0 to 13.0)	51.0 (34.0 to 233.0)
Month 24	47.0 (10.0 to 158.0)	34.5 (9.0 to 575.0)	48.0 (10.0 to 72.0)	25.0 (25.0 to 25.0)	–
Month 30	43.0 (19.0 to 81.0)	35.0 (9.0 to 147.0)	52.0 (10.0 to 205.0)	–	114.5 (24.0 to 205.0)

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

Time frame: 12 months prior to enrollment (retrospective). Number. Unit: percentage of participants

Measure	Sodium Phenylbutyrate n = 49	Ravicti n = 89	Sodium Benzoate n = 4	Carglumic Acid n = 1	Other n = 60
Value	44.9	15.7	25.0	100.0	8.3

Population analysed: Participants with a baseline visit.

Primary outcome: Percentage of Participants With Hyperammonemic Crisis (HAC), Post-Baseline by Last Known Ammonia-Scavenging Medication

Time frame: From enrollment through the end of study (mean overall duration on study was 1187.7 days).. Number. Unit: percentage of participants

Measure	Sodium Phenylbutyrate n = 61	Ravicti n = 118	Sodium Benzoate n = 10	Carglumic Acid n = 2	Other n = 22
Value	42.6	37.3	40.0	0	72.7

Population analysed: Participants with a baseline visit and at least 1 measurement of this assessment while on therapy. Participants are grouped by last-known ammonia-scavenging medication; participants may have been on more than 1 therapy during the study and the number of participants analyzed may be greater than the overall number of participants.

Adverse events

Time frame: From enrollment through the end of study (mean overall duration on study was 1187.7 days).. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Sodium Phenylbutyrate	30 of 49 (61.2%)	0 of 0	3 of 49
Ravicti	40 of 89 (44.9%)	0 of 0	2 of 89
Sodium Benzoate	2 of 4 (50%)	0 of 0	0 of 4
Carglumic Acid	0 of 1 (0%)	0 of 0	0 of 1
Other	16 of 60 (26.7%)	0 of 0	1 of 60

Limitations and caveats (from the sponsor): 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.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 1 Jul 2024.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
THRIVE Registry WebsiteLink provided by the sponsor.
Individual participant dataSharing planned: Yes. De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request.

Related trials for Urea Cycle Disorder

RecruitingObservational study
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Rare Disorders, Undiagnosed Disorders
Not yet recruitingObservational study
MENA/Levant Epidemiological, Registry of Immunological & Clinical Traits (MERIT)
Antiphospholipid (aPL)-Positive, Rheumatic Arthritis
RecruitingObservational study
Abbott Vascular Medical Device Registry
Acute Myocardial Infarction, Restenoses, Coronary
RecruitingPhase 1/2
Phase 1/2 Study of KRRO-121 in Healthy Volunteers and Patients With UCD
Urea Cycle Disorders, Inborn, Urea Cycle Disorders
Active, not recruitingPhase 3
An Open-label Study to Investigate ECUR-506 in Male Babies Less Than 9 Months of Age With Neonatal Onset OTC Deficiency
Ornithine Transcarbamylase Deficiency, Ornithine Transcarbamylase Deficiency Disease

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

MD study director

Amgen

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Urea Cycle Disorder, run by Amgen at 1 site in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 2 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 1 Jul 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

Yes. Results are posted in the registry (first posted 18 Feb 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication (time frame: 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). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01948427.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jul 2024.