This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs) (NCT01948427), a registered phase not stated study. The primary outcome is Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01948427 |
| Phase | Phase not stated |
| Condition | Urea Cycle Disorder |
| Allocation | Not Stated |
| Design model | Not Stated |
| Masking | Not Stated |
| Enrollment | 203 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Amgen |
| Results posted | Yes |
2. Clinical Question
Population
Participants with a urea cycle disorder who had a Baseline visit and an assessment at the relevant time point. The enrollment listed in the registry record is 203.
Intervention
This was observational, not a treatment-assignment study. Participants continued care without being required by the study to take medicine or make office visits beyond normal care. Medication groups are based on the last-known ammonia-scavenging medication at a given time point.
Comparator
The record lists medication groups, including Sodium Phenylbutyrate, Ravicti, Sodium Benzoate, Carglumic Acid, and Other. It does not describe a randomized or like-for-like control comparison; allocation is not stated.
Primary question
How did mean blood ammonia levels, measured in µmol/L, vary over the specified follow-up time points according to the last-known ammonia-scavenging medication? The primary outcome is descriptive in its stated form. The registry record does not report analyzed mean values or an estimated difference between medication groups.
The study is described as a long-term registry intended to collect information on patients with urea cycle disorders. It planned to follow participants for up to 10 years. Its primary outcome includes a retrospective period covering the 12 months preceding enrollment, Baseline, and later visits through Month 72. Participants could be grouped by different last-known medications at different time points, and the record notes that a participant may have been on more than 1 therapy during the study. Those features matter when interpreting patterns: the groups are not fixed randomized arms, and a descriptive contrast cannot by itself isolate a medication effect from differences in participants, care, or observation over time.
3. Trial Design
NCT01948427 is listed as an observational study, with the official title “Long-Term Registry of Patients With Urea Cycle Disorders (UCDs).” Its brief description identifies it as THRIVE and says that it would collect information on patients with UCDs, with follow-up for up to 10 years. Participants were not required to make additional office visits or take medicine outside normal care for the study. The record lists Amgen as sponsor and an enrollment of 203.
The registry record lists a start date of 2013-09-25, a primary completion date of 2020-02-24, and a status of TERMINATED. These entries describe the record; they do not, on their own, establish how long each participant was followed or why the study ended. A reason for termination is not reported in the registry record.
The primary outcome is mean blood ammonia level over time, reported in µmol/L, with standard deviation as the stated measure of dispersion. The specified schedule runs from 12 months prior to enrollment (retrospective) through Month 72. The outcome population is defined at each time point as participants with a Baseline visit and an assessment at that time point. Medication grouping is based on the last-known ammonia-scavenging medication at that time point, rather than on a randomized assignment described in the record.
This design can describe measurements collected during usual care, but it is not described as a randomized comparison. Allocation is not stated, and the record explicitly cautions that the arms are not a like-for-like control comparison. The protocol, eligibility details beyond the outcome-population definition, and reasons for the stated enrollment target are not reported in the registry record. There are 5 outcome measures listed and 0 analyses reported. Consequently, the record offers limited information for evaluating how the planned measurements were analyzed or how the study’s overall findings were determined.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| STARTED | 49 | 89 | 4 | 1 | 60 |
| COMPLETED | 42 | 72 | 4 | 1 | 46 |
| NOT COMPLETED | 7 | 17 | 0 | 0 | 14 |
Reasons for not completing this period:
| Reason | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|
| Serious Adverse Event | 3 | 0 | 0 | 0 | 1 |
| Investigator Request | 0 | 8 | 0 | 0 | 6 |
| Lost to Follow-up | 1 | 0 | 0 | 0 | 4 |
| Withdrawal by Subject | 2 | 0 | 0 | 0 | 1 |
| Other, Not Specified | 1 | 9 | 0 | 0 | 2 |
Analysis population for the primary outcome, as defined in the registry: 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).
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other | Total |
|---|---|---|---|---|---|---|---|
| Participants analysed | 49 | 89 | 4 | 1 | 60 | 203 | |
| Age, Continuous | years | 10.9 (SD 13.5) | 16.7 (SD 15.1) | 9.3 (SD 8.7) | 2.0 (SD NA) | 14.7 (SD 16.9) | 14.5 (SD 15.3) |
| Sex: Female, Male: Female | Participants | 33 | 54 | 1 | 0 | 33 | 121 |
| Sex: Female, Male: Male | Participants | 16 | 35 | 3 | 1 | 27 | 82 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 13 | 16 | 3 | 0 | 7 | 39 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 36 | 73 | 1 | 1 | 53 | 164 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race: White | Participants | 30 | 63 | 4 | 1 | 48 | 146 |
| Race: Black or African-American | Participants | 9 | 9 | 0 | 0 | 4 | 22 |
| Race: Asian | Participants | 1 | 2 | 0 | 0 | 3 | 6 |
| Race: Native Hawaiian or Other Pacific Islander | Participants | 1 | 0 | 0 | 0 | 0 | 1 |
| Race: Other, Not Specified | Participants | 8 | 15 | 0 | 0 | 5 | 28 |
6. Primary Endpoint
The registry lists the primary outcome as Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication, measured over 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.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | µmol/L |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is titled “Mean Blood Ammonia Levels Over Time, by Last Known Ammonia-Scavenging Medication.” It measures blood ammonia as a continuous quantity in µmol/L and specifies the mean as the summary measure, with standard deviation describing dispersion. The timeframe includes a retrospective period covering the 12 months before enrollment, Baseline, and assessments from Day 7 to 30 through Month 72. The registry record defines the retrospective period as the 12 months preceding enrollment.
A mean gives a concise summary of the measured values in a group at a time point. Standard deviation describes how spread out individual measurements are around that mean; it is not a measure of precision in the same way as a confidence interval. In general, repeated measurements can help describe how a clinical measure changes across follow-up, while also making clear that the number and composition of observed participants may vary by time point.
The outcome population requires a Baseline visit and an assessment at the given time point. Participants are grouped by their last-known ammonia-scavenging medication at that time, and a participant may have been on more than 1 therapy during the study. This definition supports a time-specific descriptive summary, not a fixed-arm randomized contrast. The registry record does not report the observed means, standard deviations, participant counts at each time point, or an inferential estimate for this endpoint. Without those results, the endpoint’s observed pattern and its precision cannot be described.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a planned longitudinal summary of blood ammonia levels among participants with urea cycle disorders. It identifies the measure, unit, dispersion statistic, time points, and a population rule for each time point. It does not report the primary outcome’s numerical results. In particular, it provides no observed group means, standard deviations, confidence intervals, or tests of differences in the information available for this analysis. The record lists 0 analyses.
That distinction separates a stated endpoint from an analyzed result. Naming mean blood ammonia as an outcome does not establish what values were observed or whether they changed over time. A plotted or tabulated estimate would be needed to describe the direction, size, and uncertainty of any pattern; those values are not reported in the registry record. The listed enrollment of 203 is not a substitute for the number measured at each time point. The outcome definition restricts each time-point summary to participants with both a Baseline visit and an assessment at that time point, but the size of each such subset is not reported.
The medication categories also require cautious interpretation. Grouping is based on the last-known ammonia-scavenging medication at a given time point. The record notes that participants may have been on more than 1 therapy during the study. It does not describe random assignment, and the record cautions that the arms are not a like-for-like control comparison. Thus, even if numerical group summaries were available, a difference between groups would be descriptive and could reflect differences in who was observed, when measurements were available, or other factors not described in the record. It would not by itself identify an effect caused by a medication.
There is also no reported inferential analysis for the primary endpoint. The statistical plan, adjustment approach, model structure, and handling of repeated observations are not reported in the registry record. No confidence interval or p-value for the primary outcome is given. The absence of those details means that the precision and statistical uncertainty of any putative contrast cannot be assessed from this record.
The status is TERMINATED, but the record does not report why termination occurred or how it affected data collection. The listed primary completion date does not establish that every participant completed the planned follow-up. Nor does a planned follow-up horizon of up to 10 years show that all enrollees were observed for that duration. In sum, the record documents the observational question and measurement schedule, but the reported information does not support a conclusion about the observed course of blood ammonia levels or a comparative medication effect.
9. Statistical Methods Explained
What does a mean describe?
For a continuous measurement such as blood ammonia, a mean is a way to summarize the center of the recorded values in a group. It is useful for describing a typical level in the sense of an arithmetic average, but it does not show the full distribution. Individual observations may vary, and a mean alone does not reveal whether values are tightly clustered or widely spread. The endpoint therefore also names standard deviation, which describes the spread of observations around the mean.
These are descriptive statistics. They tell the reader how measurements in the analyzed group are summarized, but they do not by themselves quantify uncertainty about the mean or support a causal conclusion. A confidence interval is a general statistical tool that gives a range of values compatible with the data and a stated method; it would provide information about estimate precision beyond the sample mean and standard deviation. No confidence interval for this endpoint is reported in the registry record.
Why do repeated time points need careful reading?
The endpoint covers a retrospective period, Baseline, and multiple later visits. In general, repeated measurements can describe the course of a measure within a study, but a sequence of group means does not necessarily represent the same set of people at every visit. The stated outcome population requires a Baseline visit and an assessment at the particular time point. The number contributing at each time point is not reported in the registry record. If the observed group changes over follow-up, differences between time-specific means may reflect both changes in measurements and changes in who contributed data.
General methods for repeated data can account for the fact that measurements from the same person are related. The registry record does not report whether a repeated-measures model or any other model was used. It also does not report whether analyses used all available observations, a specified subset, or an adjustment for measured characteristics. Those details cannot be inferred from the listed endpoint alone.
What does medication grouping mean?
The record groups participants by last-known ammonia-scavenging medication at the given time point. This is a time-specific classification, not a stated randomized assignment. It also notes that participants may have been on more than 1 therapy during the study. In general, observational group comparisons can be affected by differences between participants and by changes in treatment or measurement over time. Without random allocation or a reported adjustment strategy, a numerical difference between groups would not automatically represent a treatment effect.
The record explicitly says the arms are not a like-for-like control comparison and that allocation is not stated. The details of how the medication groups were formed beyond the last-known-medication description, including whether exposure changes were handled in a particular way, are not reported in the registry record. Therefore, group labels should be read as categories for descriptive reporting, not as evidence of exchangeable treatment arms.
What does dispersion tell the reader?
Standard deviation is expressed in the same units as the measurement and describes variation among observations. It is not interchangeable with a standard error or confidence interval. In general, a standard deviation can be large even when a mean is estimated precisely if there are many observations, or a mean can be uncertain when relatively few observations are available. Precision depends on the amount and structure of information, not just on the spread of individual measurements. Since neither endpoint estimates nor time-specific counts are reported, the record does not permit an assessment of the observed spread or precision.
Over-dispersion is a general issue for statistical models when observed variability exceeds what a model assumes. It can lead to uncertainty estimates that are too small if ignored. The registry record does not describe a model for this endpoint, so it does not establish whether over-dispersion was assessed or relevant. This is a general statistical consideration, not a reported feature of the study analysis.
What can a p-value or confidence interval add?
In general, a p-value measures how incompatible data are with a specified null model under the assumptions of a statistical test. It is not the probability that a treatment works, nor does it describe the size or clinical importance of an effect. A confidence interval gives a range reflecting estimate uncertainty under its method and assumptions. Both depend on an explicitly defined comparison and analysis. The registry record reports no analyses for the primary outcome, and no p-value or confidence interval is reported. It is therefore not possible to characterize inferential evidence for the stated primary question from the record.
Sample-size calculations are planning tools, not evidence about what a completed study observed. The record’s planning illustration assumptions describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect defined as a stated fraction of the observed effect. It specifies that continuous-outcome illustrations are expressed as participants per arm for 80% power or other stated planning targets, while time-to-event illustrations use total events. No numerical sample-size illustration values are reported in the information available for this analysis. Such illustrations would not constitute observed power and would not replace the study’s actual endpoint results. The registry record does not report the trial’s own sample-size justification or design calculations.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 5 outcome measures, but reports 0 analyses. It identifies one primary outcome in the information available for this analysis: mean blood ammonia levels over time by last-known ammonia-scavenging medication. The record does not report a statistical plan describing how multiple outcomes or multiple time points were handled. It also does not report a multiplicity adjustment, a hierarchy of confirmatory tests, or which analyses, if any, were intended to be confirmatory. These matters are not reported in the registry record.
Multiplicity is a general concern when many outcomes, time points, groups, or statistical tests are examined. With more opportunities to identify a result, a nominal test threshold may not preserve the intended overall false-positive rate across all tests. Methods such as prespecifying a primary comparison or adjusting for multiple testing can address different forms of this issue. The appropriate approach depends on the study question and analysis plan. No particular approach can be attributed to this registry from the recorded information.
The outcome population is described as participants with a Baseline visit and an assessment at a given time point. That identifies a basic inclusion rule for each summary, but the registry record does not report the number with missing measurements, reasons for missingness, imputation methods, or sensitivity analyses. It also does not report whether all available measurements were analyzed or how withdrawal and incomplete follow-up were handled.
The record does not provide a full analysis-population definition for all outcomes. It therefore does not establish whether the listed enrollment of 203 corresponds to any specific analysis population. The details of protocol deviations, exclusions, and handling of participants with different medication histories are not reported in the registry record. Without those details, completeness and potential selection effects cannot be evaluated.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Sodium Phenylbutyrate | 30 | 49 | 61.2% |
| Ravicti | 40 | 89 | 44.9% |
| Sodium Benzoate | 2 | 4 | 50.0% |
| Carglumic Acid | 0 | 1 | 0.0% |
| Other | 16 | 60 | 26.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Sodium Phenylbutyrate | 3 of 49 |
| Ravicti | 2 of 89 |
| Sodium Benzoate | 0 of 4 |
| Carglumic Acid | 0 of 1 |
| Other | 1 of 60 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Sodium Phenylbutyrate | 0 of 0 |
| Ravicti | 0 of 0 |
| Sodium Benzoate | 0 of 0 |
| Carglumic Acid | 0 of 0 |
| Other | 0 of 0 |
Most frequent serious adverse event terms
| Event term | Organ system | Sodium Phenylbutyrate | Ravicti | Sodium Benzoate | Carglumic Acid | Other |
|---|---|---|---|---|---|---|
| Hyperammonaemia | Metabolism and nutrition disorders | 19 of 49 | 26 of 89 | 0 of 4 | 0 of 1 | 2 of 60 |
| Vomiting | Gastrointestinal disorders | 7 of 49 | 5 of 89 | 1 of 4 | 0 of 1 | 2 of 60 |
| Hyperammonaemic Crisis | Metabolism and nutrition disorders | 8 of 49 | 3 of 89 | 0 of 4 | 0 of 1 | 1 of 60 |
| Gastroenteritis Viral | Infections and infestations | 2 of 49 | 4 of 89 | 0 of 4 | 0 of 1 | 3 of 60 |
| Influenza | Infections and infestations | 2 of 49 | 2 of 89 | 0 of 4 | 0 of 1 | 2 of 60 |
| Diarrhoea | Gastrointestinal disorders | 3 of 49 | 1 of 89 | 1 of 4 | 0 of 1 | 0 of 60 |
| Pyrexia | General disorders | 3 of 49 | 2 of 89 | 0 of 4 | 0 of 1 | 0 of 60 |
| Mental Status Changes | Psychiatric disorders | 2 of 49 | 3 of 89 | 0 of 4 | 0 of 1 | 0 of 60 |
| Abdominal Pain | Gastrointestinal disorders | 0 of 49 | 3 of 89 | 1 of 4 | 0 of 1 | 0 of 60 |
| Clostridium Difficile Infection | Infections and infestations | 2 of 49 | 0 of 89 | 0 of 4 | 0 of 1 | 1 of 60 |
Serious events with the highest arm-level rate: hyperammonaemia (38.8%), abdominal pain (25.0%), diarrhoea (25.0%), nausea (25.0%), vomiting (25.0%).
The registry record lists serious adverse event counts by medication group. For Sodium Phenylbutyrate, 49 are listed at risk and 30 affected, with 3 deaths. For Ravicti, the corresponding figures are 89, 40, and 2. Sodium Benzoate has 4 at risk, 2 affected, and 0 deaths; Carglumic Acid has 1 at risk, 0 affected, and 0 deaths; Other has 60 at risk, 16 affected, and 1 death. The record also lists 0 in the other-event count fields for each group.
These are counts associated with the registry’s group listings, not randomized comparative safety estimates. The record cautions that the arms are not a like-for-like control comparison, and it does not report exposure duration, person-time, event adjudication, or adjusted comparisons. The counts alone should not be read as rates or as evidence that one medication caused an event or death. The registry record does not report the denominator details needed to interpret the listed serious-event percentages as risks.
The listed top serious adverse event terms include Hyperammonaemia (38.8%), Abdominal Pain (25.0%), Diarrhoea (25.0%), Nausea (25.0%), Vomiting (25.0%), and Medical Device Site Pain (25.0%). The denominator and method used to calculate these percentages are not reported in the registry record. These terms should therefore be read as the record’s listed event summaries, not as comparative rates or treatment effects.
12. What the Result Does, and Does Not, Mean
The registry record establishes that an observational study was intended to collect information about patients with urea cycle disorders and to follow participants for up to 10 years. It identifies mean blood ammonia level over time as the primary outcome and specifies the unit, time points, dispersion measure, and time-specific population definition. It also lists medication groups and serious adverse event counts. These details describe the study question and some recorded outcomes.
The record does not report analyzed values for the primary endpoint. It therefore does not show whether mean blood ammonia levels increased, decreased, remained similar, or differed across medication groups. It also does not report estimates of uncertainty or inferential tests for that endpoint. The listed enrollment of 203 does not reveal how many participants contributed measurements at any particular visit. No conclusion about the observed endpoint pattern or its precision can be drawn from the endpoint description alone.
The design also limits what a medication-group contrast could mean. This is an observational registry, not a reported randomized trial, and the record says that the arms are not a like-for-like control comparison. The medication grouping is based on the last-known therapy at each time point; participants may have been on more than 1 therapy during the study. In general, observed differences in such groups can reflect characteristics or care patterns that differ between participants as well as any medication-related variation. The registry record does not report an adjusted analysis that could address such differences.
The safety counts are descriptive entries, not causal evidence. They do not provide a basis for ranking medications by safety because group comparability, exposure duration, and the relevant event-rate denominators are not reported. Likewise, the status TERMINATED and the listed completion date do not explain why the study ended or what effect that had on completeness. The record supports an account of the registry’s design and reported listings, but not a conclusion about treatment benefit, harm, or comparative effectiveness.
13. Why This Trial Matters Statistically
Longitudinal registries can be statistically valuable because they collect information during routine care rather than requiring the study to assign a treatment. For conditions such as urea cycle disorders, a record that tracks measurements over time can describe what was collected and how participants’ observed care was categorized. The intended follow-up of up to 10 years and the range of specified visits make the registry’s measurement schedule an important part of its design.
The statistical challenge is that long-term observation is not the same as complete follow-up for every participant. The analysis population is defined separately at each time point, while participant counts at those times are not reported. As a general matter, changing participation can affect how time-specific averages should be interpreted. Reporting counts, dispersion, uncertainty intervals, and methods for incomplete data would help readers judge both the pattern and its precision.
The medication grouping also illustrates a central distinction in clinical statistics: descriptive comparisons are not necessarily causal comparisons. A registry can show how measured outcomes are distributed across recorded categories, but an observational design does not create randomized, interchangeable groups. The record’s explicit warning that the arms are not a like-for-like control comparison is therefore important. Clear reporting of analysis methods and participant flow would be needed to assess whether any adjusted association was estimated and what assumptions it required.
For this record, the primary outcome is specified, but 0 analyses are reported. That gap limits what can be learned about the observed blood ammonia trajectory from the registry entry alone. The statistical importance lies both in the questions the registry set out to track and in the distinction between a planned measurement framework and reported evidence. The entry also demonstrates why safety counts, percentages, denominators, and group definitions must be interpreted together rather than treated as standalone comparisons.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The registry record is limited by the absence of reported primary-outcome analyses. Although it specifies mean blood ammonia levels and standard deviation across a detailed schedule, it gives no observed values, participant counts at each time point, confidence intervals, or statistical tests. The record lists 0 analyses, so the primary question cannot be answered from the reported analysis information.
The design is observational and the record states that the arms are not a like-for-like control comparison; allocation is not stated. Medication categories are based on last-known medication at a time point, and participants may have been on more than 1 therapy during the study. The registry record does not report how confounding, treatment changes, or repeated measurements were handled. This prevents assessment of the extent to which group differences, if any, could be interpreted as medication-related rather than descriptive associations.
Follow-up completeness is also unclear. The planned duration is up to 10 years, but the record does not report participant-level follow-up, visit-specific counts, missing-data reasons, or methods for incomplete measurements. The stated status is TERMINATED, and the reason for termination is not reported in the registry record. The listed primary completion date does not establish that all planned observations were obtained.
Safety listings lack reported denominator definitions, exposure duration, and comparative analysis methods. The record’s serious adverse event terms and percentages therefore cannot be interpreted as comparative risks. Details of eligibility, the complete protocol, analysis populations, sample-size justification, and multiplicity handling are also not reported in the registry record. These limitations concern the available record; they do not establish what information may exist outside it.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs) — NCT01948427.
- Registry record last updated 2024-07-01; status last verified 2024-06.
- Results first posted 2021-02-18.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
NCT01948427 is an observational registry titled “Long-Term Registry of Patients With Urea Cycle Disorders (UCDs),” also described as THRIVE. The registry record lists Amgen as sponsor, enrollment of 203, status TERMINATED, a start date of 2013-09-25, and a primary completion date of 2020-02-24. It planned follow-up for up to 10 years without requiring additional office visits or medicine beyond normal care.
The primary outcome is mean blood ammonia level over time, measured in µmol/L and summarized with standard deviation. It includes a retrospective period, Baseline, and follow-up through Month 72, with population eligibility defined at each time point. The record lists 5 outcome measures and 0 analyses. It also lists serious adverse event counts by medication group, but does not report comparative safety estimates. The primary endpoint’s analyzed values, the analysis methods, and the reason for termination are not reported in the registry record.