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 Uric Acid Lowering Trial in Youth Onset T2D (NCT03899883), a na phase 2 study. The primary outcome is Systolic Blood Pressure.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03899883 |
| Phase | Phase 2 |
| Condition | Diabetic Kidney Disease, Hyperuricemia, Diabetes |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 11 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Colorado, Denver |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns adolescents and young adults with youth-onset type 2 diabetes. The registry summary describes participants aged 18-25 whose diabetes was diagnosed before age 21. The enrollment field records 11 participants; the summary describes a proposed pilot and feasibility trial in ten participants.
Intervention
The intervention was a single administration of pegloticase: 8 mg IV in 250 mL 0.9% normal saline. The study aimed to evaluate uric acid lowering and markers of cardiovascular and kidney health.
Comparator
The registry describes a single-group study, with no masking and allocation recorded as NA. No separate control group or randomized comparison is reported. Baseline measurements are part of the stated primary outcome timeframe, but they are not a concurrent control group.
Primary question
The primary outcome was mean systolic blood pressure, measured at baseline and one week after treatment. The clinical question is whether the recorded blood pressure measure changed over that period after pegloticase administration. The registry record does not report the outcome values or an estimate of change, so the size and precision of any change cannot be described.
3. Trial Design
This completed Phase 2 study is registered as a single-group trial of pegloticase in youth-onset type 2 diabetes. Allocation is recorded as NA, masking as none, and the study model as single group. The registry therefore does not describe a randomized comparison between treatment and control arms. A before-and-after assessment within one group is not equivalent to a concurrent controlled comparison: changes over time cannot be separated from the effects of treatment using a control arm that is not present in the recorded design.
The intervention is described as one administration of pegloticase, 8 mg IV in 250 mL 0.9% normal saline. The primary outcome is systolic blood pressure measured by sphygmomanometer at baseline and one week post treatment. Serum uric acid is listed as a secondary outcome. The registry lists nine outcome measures, but no outcome analysis is recorded.
The enrollment field reports 11 participants. The study summary instead describes a pilot and feasibility trial proposal involving ten participants. These figures refer to different parts of the registry record: one is the enrollment entry and the other is the summary's description of the proposed study. The record does not explain the difference. The study is marked completed, with a start date of 2020-01-01 and primary completion date of 2023-04-13.
The target population is described in the summary as young people aged 18-25 with type 2 diabetes diagnosed before age 21. Further eligibility criteria, recruitment details, adherence information, and the timing of any additional measurements are not reported in the registry record. The design is described as a pilot and feasibility study intended to evaluate uric acid lowering and cardiorenal markers, rather than as a randomized efficacy comparison.
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 | Men With Youth-onset T2D |
|---|---|
| STARTED | 11 |
| COMPLETED | 9 |
| NOT COMPLETED | 2 |
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 | Men With Youth-onset T2D |
|---|---|---|
| Participants analysed | 11 | |
| Age, Continuous | Years | 22 (SD 5) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 11 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 6 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 3 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 8 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 3 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Mean arterial pressure | mmHg | 98 (SD 10) |
6. Primary Endpoint
The registry lists the primary outcome as Systolic Blood Pressure, measured over Baseline and 1 week post treatment.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | mmHg |
| Endpoint classification | Continuous Endpoint |
Systolic blood pressure is the primary outcome. It is a continuous measurement reported in mmHg, and the registry specifies that the outcome is a mean with standard deviation. The stated method is measurement by sphygmomanometer. The timeframe compares baseline with one week post treatment.
Blood pressure is included among the markers the investigators sought to evaluate in relation to cardiovascular health. As a measured physiological quantity, it can be summarized across participants and compared across the two stated time points. The choice of a one-week assessment captures a short-term measurement after the single pegloticase administration described in the registry. It does not, by itself, establish whether any observed change would persist beyond that timeframe or reflect a longer-term clinical outcome.
The registry record does not report the baseline or post-treatment mean, their standard deviations, the number of participants contributing measurements at each time point, or an estimated within-participant change. It also does not report a confidence interval or a statistical test for this endpoint. Accordingly, the endpoint's intended measurement and timing are clear, but its observed result and precision cannot be assessed from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry identifies systolic blood pressure as the primary outcome, with measurements planned at baseline and one week post treatment. However, the registry record does not report the outcome values, an estimated change, a measure of uncertainty, or a test result. There is therefore no posted primary-outcome estimate to interpret. Neither the direction nor the magnitude of a blood pressure change can be established from the record.
The same limitation applies to serum uric acid, which is listed as a secondary outcome. The record does not provide its measured values or an estimate of change. While the study summary describes an aim to lower uric acid and evaluate cardiorenal markers, an aim or hypothesis is not an outcome result. The registry does not establish whether the intervention changed uric acid, blood pressure, or any other marker.
The design also shapes what could be concluded even if numerical before-and-after results were reported. This is a single-group study with no randomized control arm listed. A difference between baseline and follow-up could describe change among participants with observations at both times, but would not by itself show that pegloticase caused the change. Other time-related influences, measurement variation, and changes in participant circumstances cannot be distinguished from treatment effects through a concurrent comparison in this design.
The registry records enrollment of 11. It does not state how many participants had complete measurements for the primary outcome or whether the analysis population differed from enrollment. The number of observations contributing to each outcome is not reported in the registry record. That missing information matters because incomplete follow-up can affect both the interpretation of a mean and the precision of an estimated change.
Safety entries should be read separately from evidence about the primary outcome. The registry records zero participants with a serious adverse event and five participants with other adverse events among 11 at risk. Those entries describe reported event counts; they do not supply efficacy evidence or establish the absence of every possible harm. The record does not list serious adverse event terms.
Overall, the posted registry information describes the intended population, treatment, outcome, and study structure, but supplies no numerical primary or secondary outcome results. The completed status indicates the registry's study status, not that a particular outcome was achieved. Without outcome estimates and their uncertainty, the degree of change and its precision cannot be judged.
9. Statistical Methods Explained
The registry record describes a continuous primary outcome summarized as a mean and standard deviation. These statistics answer different questions. A mean describes the average measured value in the analyzed group, while a standard deviation describes the spread of participant measurements around that average. A standard deviation is not the same as the uncertainty around the mean: uncertainty also depends on how many participants contribute data and on the analysis structure.
What would a change estimate describe?
General statistical explanation: For a before-and-after study, an estimated change can summarize the difference between measurements at the two time points. If the same participants contribute both measurements, the paired nature of observations matters. A statistical analysis can use the within-participant relationship, which is not recoverable from separate group means and standard deviations alone. The registry record does not report a change estimate or the method used to analyze the repeated measurements, so no specific method can be attributed to this study.
A confidence interval, when reported, gives a range of values compatible with the data and the method's assumptions. It communicates precision rather than proving that a result is important or causal. A narrow interval indicates greater precision than a wide interval, all else equal. No confidence interval for systolic blood pressure or serum uric acid is reported in the registry record. A p-value, if reported, would address compatibility with a specified statistical null model; it would not measure the size or practical importance of a change. No primary-outcome p-value is reported.
Why does the study structure matter?
General statistical explanation: A single-group before-and-after design can describe how measurements differ across time among observed participants. It cannot use a concurrent untreated or alternative-treatment group to distinguish treatment-related change from other explanations. This is a design limitation, not a problem that a more complicated statistical test can automatically remove. Because the registry lists no randomized comparison, any interpretation should remain within the limits of a one-group assessment.
The registry characterizes the intervention as a single administration and gives the primary assessment timeframe as baseline and one week post treatment. That pairing identifies when the outcome was intended to be measured, but does not reveal whether every participant contributed both measurements. The number of participants with observed data at each time point, the handling of missing measurements, and the analysis population are not reported in the registry record.
What do the planning assumptions mean?
General statistical explanation: The registry's planning description refers to a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It also describes planning for a stated fraction of an observed effect. These are assumptions for an illustrative two-arm sample-size calculation, not a description of the actual single-group design. A two-sided significance level sets the convention for evaluating evidence in either direction under a specified model; it is not a probability that a hypothesis is true. Equal allocation is an assumed split of participants across two groups, not the allocation used in this single-group study.
The planning note says that the effect used for illustration is based on the observed effect. Such calculations are planning illustrations only. They do not represent observed power, do not establish that the study was adequately powered, and do not substitute for outcome estimates with uncertainty. Numerical planning estimates are not reported in the registry record. No calculation can be interpreted as the number of participants actually enrolled or as evidence that a clinical effect was detected.
The note also distinguishes calculations expressed as participants per arm from time-to-event calculations expressed as total events. The listed primary outcome is continuous, not time-to-event. The registry does not give an analysis model for the primary outcome or report a time-to-event endpoint. More detailed information about the intended statistical plan, any assumptions for an analysis of repeated measurements, or any adjustments is not reported in the registry record.
Why can over-dispersion matter?
General statistical explanation: Over-dispersion occurs when observed variability is greater than a statistical model expects. If a model understates variability, its uncertainty estimates can be too small and its tests can appear more conclusive than warranted. Conversely, a model that allows for extra variability can produce more cautious uncertainty estimates. This general issue is relevant when selecting models for outcomes whose variation does not fit simple assumptions, but the registry does not report an over-dispersion assessment or a model for this study's primary outcome. There is no basis to claim that over-dispersion occurred or that a particular adjustment was used.
More broadly, interpretation depends on the estimate, its uncertainty, the measurement completeness, and the study design. The registry's outcome descriptions alone do not provide these elements. The central statistical conclusion is therefore about what can be evaluated: the intended outcome and timing are recorded, but the numerical results and analysis details needed to assess the magnitude, precision, and statistical evidence are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists nine outcome measures and identifies systolic blood pressure as the primary outcome. Serum uric acid is listed as a secondary outcome. The record does not describe how multiple outcomes were handled statistically, whether any hierarchy among secondary outcomes was specified, or whether any adjustment for multiple testing was planned or used. The multiplicity plan is not reported in the registry record.
General statistical explanation: When several outcomes or comparisons are tested, the chance of at least one apparently unusual result can increase if each is assessed separately using the same decision threshold. A prespecified outcome hierarchy or an adjustment can help define how to interpret multiple tests. Without a reported plan and results, it is not possible to assess whether multiplicity affected the interpretation of this study. This is a general consideration, not a claim that the study conducted any particular set of tests.
The registry records zero analyses. It does not report participant-level outcome completeness, the number analyzed for each endpoint, reasons for missing measurements, or a method for handling missing data. It is therefore not possible to determine whether all enrolled participants contributed to the primary outcome, whether analysis was restricted to participants with paired observations, or whether any imputation or other method was used.
The analysis population is likewise not described in detail. The record reports enrollment of 11, while the summary characterizes the planned pilot as involving ten participants; it does not explain this difference or state the number included in any outcome analysis. These omissions prevent a clear assessment of the relationship between enrollment, follow-up, and the population underlying any estimate. Since outcome analyses are not reported, there is no basis for evaluating the impact of missing data or multiplicity on a result.
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 |
|---|---|---|---|
| Men With Youth-onset T2D | 0 | 11 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Men With Youth-onset T2D | 0 of 11 |
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 |
|---|---|
| Men With Youth-onset T2D | 5 of 11 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Men With Youth-onset T2D |
|---|---|---|
| Pegloticase Infusion Reaction | General disorders | 2 of 11 |
| Back and neck pain | General disorders | 1 of 11 |
| Post-infusion hyperglycemia requiring insulin administration | Metabolism and nutrition disorders | 1 of 11 |
| Superficial blood clot after IV placement | Injury, poisoning and procedural complications | 1 of 11 |
| GI discomfort | Gastrointestinal disorders | 1 of 11 |
| Weakness | General disorders | 1 of 11 |
The registry reports safety counts for the group titled Men With Youth-onset T2D. It records 11 participants at risk for serious adverse events, with zero participants affected and zero deaths. For other adverse events, 11 participants were at risk and five were affected. No serious adverse event terms are listed.
These entries are counts of participants, not a complete description of event severity, duration, timing, or relationship to treatment. The record does not report the individual event details behind the other adverse event count, nor does it list serious adverse event terms. The entries should therefore be read narrowly as the safety figures recorded in the registry, rather than as a detailed account of all events experienced during the study.
A zero count for participants affected by serious adverse events does not establish that no adverse effects of any kind occurred: the registry separately records five participants affected by other adverse events. Nor do these figures establish comparative safety, because the study has no reported control group. The counts do not describe whether participants had complete safety follow-up, and the registry record does not provide further information on safety ascertainment. These safety entries do not answer the primary question about change in systolic blood pressure.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed single-group Phase 2 study of pegloticase in young people with youth-onset type 2 diabetes. It specifies the single administration, the primary systolic blood pressure measure, its measurement method, and the baseline-to-one-week timeframe. It also lists serum uric acid as a secondary outcome. These details describe what the study set out to assess.
The record does not report numerical results for the primary outcome or the secondary serum uric acid outcome. It therefore does not show the direction, magnitude, or precision of any measured change. The study's stated hypothesis and rationale describe why the investigators considered uric acid lowering and cardiorenal markers important, but a hypothesis is not a finding. No conclusion about an effect on blood pressure, uric acid, cardiovascular health, or kidney health can be drawn from the posted results.
Even if before-and-after values were available, a single-group design would limit causal interpretation. Without a concurrent control group, any change across the short follow-up could not be separated from other influences using this design alone. The registry also does not report the number with complete measurements or the analysis method, further limiting assessment of precision and completeness.
The safety record reports zero participants affected by serious adverse events, zero deaths, and five participants affected by other adverse events among 11 at risk. These counts are useful descriptive information, but they are not a controlled comparison and do not establish comparative safety. The registry's completed status and enrollment figure likewise do not constitute evidence of a treatment effect.
The appropriate reading is that the registry establishes the study's design and intended measurements, while leaving the outcome findings unavailable in the registry record. The information supports no claim that pegloticase improved or failed to improve any measured outcome. It also does not establish the durability of any possible change, since the primary follow-up timeframe is one week and longer-term outcome results are not reported.
13. Why This Trial Matters Statistically
This study is statistically notable because its intended question concerns change in a continuous measure over time, but the registered design is single-group. The primary outcome is systolic blood pressure measured at baseline and one week post treatment. Such a structure can be useful for describing measurements in a small pilot context, but it does not provide a randomized treatment-control contrast. The distinction between describing change and attributing change is central to interpreting findings from an uncontrolled before-and-after study.
The enrollment entry is 11, while the study summary describes a proposed trial in ten participants. The registry does not explain the difference, and it reports no outcome analyses. This makes it especially important to distinguish the number enrolled from the number analyzed and to avoid inferring that everyone enrolled contributed to each outcome. Analysis counts and missing-data handling are not reported in the registry record.
The record's planning note describes an illustrative calculation with assumptions for a two-arm comparison, while the registered trial is single group. The juxtaposition shows why planning calculations need to be read in context: an assumed allocation for an illustration does not change the actual study design, and planning values are not results. Numerical planning estimates are not reported in the registry record, and no observed primary outcome estimate is available.
Finally, the registry identifies nine outcome measures but provides no analyses and no description of a multiplicity strategy. Without outcome estimates, uncertainty measures, and analysis details, readers cannot assess statistical precision or the consistency of findings across outcomes. The case therefore illustrates how a registry can describe a clinically motivated question and a defined measurement plan while not reporting the information needed to evaluate the resulting statistical evidence.
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 most direct limitation for interpreting this trial is that the registry does not report numerical results for the primary systolic blood pressure outcome or the secondary serum uric acid outcome. No means, standard deviations, changes, confidence intervals, or p-values for these outcomes are reported. As a result, neither the size nor the precision of an outcome change can be assessed from the registry record.
The single-group design has no randomized comparator listed. Consequently, a before-and-after difference, if available, would not by itself distinguish a treatment effect from other influences over time. The stated primary timeframe is one week post treatment, so the record does not establish longer-term changes. Further details about outcome measurement schedules are not reported in the registry record.
The record reports enrollment of 11 but describes a proposed study of ten participants in its summary. It does not explain this discrepancy or report the number of participants contributing to each outcome. Eligibility details beyond the summary's description of age and diagnosis timing, participant flow, missing measurements, and analysis populations are not reported. These gaps make it difficult to evaluate completeness and the representativeness of any outcome analysis.
Although nine outcome measures are listed, no analyses are reported and no multiplicity approach is described. The statistical analysis plan, including any model for repeated measurements, is not reported in the registry record. The planning description refers to an illustrative two-arm calculation, but numerical planning estimates are absent and the actual study is single group. Finally, safety reporting is limited to counts; the registry does not provide detailed descriptions for the five participants with other adverse events. These are limitations of what can be evaluated from the registry information, not evidence that unreported analyses or events did or did not occur.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Uric Acid Lowering Trial in Youth Onset T2D — NCT03899883.
- Registry record last updated 2026-07-28; status last verified 2026-06.
- Results first posted 2026-07-28.
- 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
The registry describes a completed Phase 2, single-group study of pegloticase in youth-onset type 2 diabetes. The intervention was a single administration of 8 mg IV in 250 mL 0.9% normal saline. Enrollment is recorded as 11, while the study summary describes a proposed pilot in ten participants; the difference is not explained.
The primary outcome was mean systolic blood pressure in mmHg, measured by sphygmomanometer at baseline and one week post treatment. Serum uric acid is listed as a secondary outcome, and nine outcome measures are recorded. The registry reports zero analyses and does not report numerical outcome results or an analysis plan.
Safety entries record zero participants with serious adverse events, five with other adverse events, and zero deaths, with 11 at risk in each category. The study has no randomized comparison listed. Outcome precision and the effect of missing data cannot be assessed because outcome estimates, analysis counts, and missing-data methods are not reported in the registry record.