Tacrolimus Versus Prednisolone for the Treatment of Minimal Change Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Tacrolimus Versus Prednisolone for the Treatment of Minimal Change Disease (NCT00982072), a randomized phase 4 study. The primary outcome is Percentage of Participants Achieving Complete Remission From Nephrotic Syndrome at 8 Weeks.

2. Clinical Question

The registry record describes the study purpose as comparing the effectiveness of tacrolimus and prednisolone for nephrotic syndrome secondary to minimal change disease. The primary outcome is a binary event: whether a participant achieved complete remission by the specified assessment. The posted results give counts for both groups and a between-group risk difference, with a confidence interval and an unadjusted recalculation of the comparison.

The primary question is limited to remission at 8 weeks. Outcomes at other times and other aspects of disease course are separate questions, not substitutes for this endpoint. The registry record also lists remission at later assessments, relapse among patients achieving remission, serious adverse events, and change in glomerular filtration rate as secondary outcomes. Detailed posted results are available for the relapse endpoint; results for the other listed secondary outcomes are not reported in the registry record.

This is a comparison of two assigned treatments in the trial population, not a general estimate for every patient with minimal change disease. The record does not report treatment doses, detailed eligibility criteria, or the basis for selecting the endpoint definition.

3. Trial Design

The study is a phase 4, randomized, parallel-group trial with no masking reported. The registry record lists an enrollment of 52 participants. Prednisolone was assigned to 25 participants and tacrolimus to 27 participants for the posted primary outcome. The sponsor is Imperial College Healthcare NHS Trust. The record gives a start date of 2009-12, primary completion on 2019-10-10, and a status of completed.

A parallel-group design assigns participants to one of the treatment groups and compares outcomes between groups. Random allocation is intended, in general, to reduce systematic differences between groups at assignment. With no masking, the record indicates that the study was not masked; further details about who knew treatment assignments or how this may have affected outcome assessment are not reported in the registry record. The record does not give the randomization procedure or allocation concealment details.

The primary outcome was complete remission from nephrotic syndrome at 8 weeks, assessed among patients at that time point on an intention-to-treat basis. The outcome description specifies normalization of serum albumin and urine PCR <50 units. The registry record does not report the treatment regimen, dose, duration beyond the endpoint timeframe, or procedures for assessing remission.

The listed secondary outcomes include remission at 16 and 26 weeks, relapse among patients achieving remission, serious adverse events, and change in baseline glomerular filtration rate. The available detailed secondary outcome results concern relapse over 2 years. Specific results for the other listed secondary outcomes are not reported in the registry record. The registry record does not report the trial's original sample-size justification, interim-analysis plan, or detailed statistical analysis plan.

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

Analysis population for the primary outcome, as defined in the registry: Patients at 8 weeks of therapy- on intention to treat basis

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.

6. Primary Endpoint

The registry lists the primary outcome as Percentage of Participants Achieving Complete Remission From Nephrotic Syndrome at 8 Weeks, measured over 8 weeks.

The primary endpoint is the number of participants achieving complete remission from nephrotic syndrome at 8 weeks. Complete remission is described as normalization of serum albumin and urine PCR <50 units. The posted endpoint population is patients at 8 weeks of therapy, analyzed on an intention-to-treat basis. The outcome is binary: each participant is counted as having achieved the defined remission or not.

A binary endpoint gives a direct count of participants meeting a clinical definition during a specified period. Comparing the proportion with the event in each assigned group can describe the absolute difference in the observed event frequency. In general, an absolute difference is often easier to interpret than a relative measure because it describes the change in event probability on the original scale. It does not, by itself, establish why the groups differ or whether the estimate is precise.

The endpoint focuses on remission at one defined assessment, rather than the duration of remission, later relapse, or change in kidney function. Those are distinct outcomes. The registry record does not report why this endpoint definition or the 8-week timeframe was selected, or how laboratory measurements were collected and adjudicated. Accordingly, interpretation should stay close to the operational definition and the population stated in the record.

7. Results Posted in the Registry

• Tacrolimus: 63.0% (17/27) versus 84.0% (21/25) on control; risk difference -21.0 percentage points (95% CI -44.2 to 2.2), risk ratio 0.75 (0.54–1.05); unadjusted two-proportion z-test p = 0.087.

8. Secondary Efficacy Endpoints

The registry also posts results for secondary outcomes. For each outcome below, the endpoint type, the test applied to the posted summary statistics, the result and its interpretation are given in turn. Secondary endpoints are usually not adjusted for multiple testing, so they support rather than confirm the primary finding.

Percentage of Patients Achieving Remission Who Then Relapse

• Endpoint type: Binary: the number of participants with the event, compared as proportions.
• Test: Unadjusted two-proportion z-test, with a Wald 95% confidence interval for the risk difference.
• Result:
  • Tacrolimus: 72.7% (16/22) versus 73.9% (17/23) on control; risk difference -1.2 percentage points (95% CI -27.0 to 24.7), risk ratio 0.98 (0.69–1.40); unadjusted two-proportion z-test p = 0.928.
• Interpretation:
  • The 95% confidence interval includes no difference, so these data do not show a statistically significant difference between Tacrolimus and the control. The interval gives the range of effects that remain compatible with the data.

The listed secondary outcomes broaden the trial questions beyond remission at 8 weeks. They include remission at 16 and 26 weeks, relapse among patients achieving remission, serious adverse events, and change in baseline glomerular filtration rate. Detailed posted results are available for relapse over 2 years, but detailed results for the other listed secondary outcomes are not reported in the registry record.

The relapse outcome addresses what happened after remission, whereas the primary endpoint asks whether complete remission was achieved at a specific earlier assessment. Those endpoints have different definitions, timeframes, and populations. The available secondary result therefore adds a separate dimension to the clinical course rather than confirming or overturning the primary comparison. The serious adverse event counts are described separately in the safety discussion.

Across the information available, the secondary outcomes do not provide a broad, complete set of corroborating estimates. Some listed outcomes have no detailed posted results in the registry record, and the detailed relapse result remains an uncertain comparison. It is not appropriate to treat a result for one secondary endpoint as a substitute for unreported outcomes or as proof of a consistent overall pattern.

General: Secondary endpoints are often interpreted with more caution than a designated primary endpoint, particularly when a study examines several outcomes or time points. Testing multiple questions can increase the chance of a nominally notable result arising by chance if no multiplicity strategy is used. Secondary results can still be informative, but they are generally better treated as supportive or exploratory unless the analysis plan establishes otherwise. A multiplicity adjustment or hierarchy is not reported in the registry record.

The registry record lists five outcome measures and one analysis. That count does not establish how many comparisons were planned across every listed time point or whether any adjustment was intended. The statistical plan for secondary outcome testing is not reported in the registry record.

Secondary outcomes registered for this trial, for reference (only those above were recalculated):

• Percentage of Patients Achieving Complete Remission From Nephrotic Syndrome at 16 and 26 Weeks
• Percentage of Patients Achieving Remission Who Then Relapse
• Number of Serious Adverse Events
• Change in Baseline Glomerular Filtration Rate

9. Statistical Interpretation

10. Statistical Methods Explained

What does a risk difference express?

General: A risk difference subtracts the event proportion in a reference group from the event proportion in the comparison group. Here, tacrolimus is compared with prednisolone. A negative estimate means the observed event proportion is lower in the tacrolimus group; a positive estimate would mean it is higher. Unlike a relative measure, the risk difference stays on an absolute probability scale. It describes the contrast in observed event frequencies, not the mechanism behind any difference.

For the primary endpoint, the posted risk difference is -0.210. The listed group proportions are 0.84 for prednisolone and 0.630 for tacrolimus. The estimate is based on the participants included in the posted group counts. It is an unadjusted comparison; an adjusted estimate is not reported in the registry record.

How should a confidence interval be understood?

General: A confidence interval expresses uncertainty around an estimated effect under the assumptions of the method used to construct it. It is not a range containing every participant's outcome, and it does not assign a probability that the true effect lies at a particular point within the interval. A wide interval signals that the estimate is not very precise. Whether a difference is important in practice requires context and, for formal equivalence or non-inferiority claims, prespecified margins; those margins are not reported in the registry record.

The primary interval runs from -0.442 to 0.022. Because it includes zero, the posted estimate is compatible with no difference as well as with differences in either direction. The interval's width also means the estimate does not pinpoint the size of a possible group difference. The record does not provide a confidence level in the primary comparison field, but the confidence interval is presented as the interval accompanying that estimate.

Why can p values differ?

General: A p value summarizes how unusual data at least as discrepant as the observed data would be under a specified null model and test procedure. It is not the probability that the null hypothesis is true, and it does not measure the magnitude or clinical importance of an effect. Different methods can produce different p values, especially with binary outcomes and modest group sizes, because they can handle the data and uncertainty differently.

The registry record reports a Fisher exact test p value of 0.12. The unadjusted recalculation is p = 0.087. Both should be read alongside the estimated risk difference and its interval. The record does not explain the difference between the two p values, and the recalculation does not replace the sponsor's reported analysis. The discrepancy flag is false.

What does Fisher's exact test do?

General: Fisher's exact test is a method for comparing categorical outcomes between groups. It calculates a p value using the observed arrangement of event and non-event counts under a null hypothesis of no association, conditional on the margins of the count table. It is one possible test for a binary endpoint. A test result alone does not communicate the absolute size or precision of a difference, which is why the event counts and risk-difference interval are also important.

The registry identifies the sponsor analysis as Fisher Exact for the primary comparison. It does not report whether the test was one-sided or two-sided, or give further details about its implementation. Those details are not reported in the registry record.

What does intention-to-treat mean in general?

General: An intention-to-treat approach analyzes participants in the groups to which they were randomized, regardless of later adherence or treatment changes, according to the prespecified definition. It aims to preserve the comparison created by randomization. The label alone does not explain how missing outcomes were handled or whether every randomized participant contributed an observed endpoint.

The primary outcome population is described as intention to treat. The record does not provide further information about missing outcome handling, treatment switching, adherence, or exclusions. Those details are not reported in the registry record and cannot be inferred from the intention-to-treat label.

11. Confidence Intervals and Precision

The primary risk-difference estimate is -0.210, with a 95% confidence interval from -0.442 to 0.022. This interval is wide relative to the point estimate and crosses zero. It therefore leaves uncertainty both about whether there is a difference and about its size and direction. It is compatible with a notably lower remission proportion for tacrolimus, a difference close to zero, or a small positive difference on the risk-difference scale.

General: Precision concerns how narrowly an estimate is determined by the data and statistical method. More precise estimates generally have narrower confidence intervals, while fewer observations or less information can lead to greater uncertainty. A confidence interval should be considered with the point estimate and outcome definition rather than interpreted as a pass-or-fail threshold. A confidence interval crossing zero does not demonstrate that two treatments are equivalent.

The posted counts are 21 of 25 for prednisolone and 17 of 27 for tacrolimus. The overall enrollment was 52. These group sizes give the context for the estimate, but do not remove uncertainty in the comparison. The registry record does not report a prespecified clinically important difference or a precision target for the primary endpoint, so the interval cannot be judged against such a target from the record.

For the secondary relapse endpoint, the risk-difference interval also includes zero and is broad. It concerns a different outcome and denominator, so its precision does not resolve uncertainty about the primary endpoint. Detailed results for other listed secondary outcomes are not reported in the registry record.

12. Planning a Similar Trial: Sample Size

This is a planning illustration, not a re-evaluation of the original trial. It asks: if a new study wanted to detect a difference like the one observed, or a smaller one, how large would it need to be? It is deliberately not “observed power” of the finished trial, which is not a meaningful quantity.

The planning figures are participants per arm, not total events. The stated illustration uses a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. It considers detecting a stated fraction of the observed effect. The trial's own original design assumptions and sample-size justification are not reported in the registry record.

For the primary comparison, planning for 100% of the observed effect gives 68 participants per arm for 80% power and 91 participants per arm for 90% power. Planning for 75% or 50% of the observed effect requires larger group sizes. The values are illustrations based on the observed effect, not a reconstruction of the trial's original design. The relapse planning figures are much larger because the observed risk difference for that endpoint is small; they should be read as sensitive to the effect size used in the calculation.

General: Power is the probability, under a specified assumed effect and analysis model, of detecting an effect at the chosen significance level. The 80% power column gives the illustrative participants per arm for that assumed probability; the 90% power column gives the group size for a higher assumed probability of detection under the same planning framework. Smaller effects require more participants because they are harder to distinguish from random variation.

These figures are not observed power. They are prospective planning illustrations using the observed effect as a starting point and the stated assumptions. The trial's actual enrollment was 52, and the record does not report that these illustrative calculations were used to design or conduct the trial.

13. How the Sample Size Is Derived

Power is the probability that a study of a given size rejects the null hypothesis when a specific true effect exists. It depends on four things: the effect to detect, the variability of the outcome, the significance level (here a two-sided α of 0.05, so z_1−α/2 = 1.960) and the desired power (z_1−β = 0.842 for 80% and 1.282 for 90%).

Formula

n is the number of participants in each arm, p₀ and p₁ are the control and treatment proportions, and p̄ is their average.

Worked calculation for this trial

1. Control proportion p₀ = 0.840; treatment proportion p₁ = 0.630; average p̄ = 0.735.
2. First term: z_1−α/2 × √[2p̄(1−p̄)] = 1.96 × 0.6243 = 1.2236.
3. Second term: z_1−β × √[p₀(1−p₀) + p₁(1−p₁)] = 0.842 × 0.6063 = 0.5103.
4. n = (1.2236 + 0.5103)² / (-0.2104)² = 67.9, rounded up to 68 participants per arm for 80% power.

The smaller-effect rows in the table above repeat the same calculation with the effect multiplied by 0.75 and 0.5 (for ratios, applied to the logarithm of the ratio).

R code

alpha <- 0.05
z_a   <- qnorm(1 - alpha / 2)          # two-sided
p0 <- 0.84                          # control proportion
p1 <- 0.62962962962963                          # treatment proportion
n_per_arm <- function(power, frac = 1) {
  q1 <- p0 + (p1 - p0) * frac               # smaller effect = fraction of the observed difference
  pbar <- (p0 + q1) / 2
  z_b <- qnorm(power)
  ceiling(round((z_a * sqrt(2 * pbar * (1 - pbar)) + z_b * sqrt(p0 * (1 - p0) + q1 * (1 - q1)))^2 / (q1 - p0)^2, 6))
}
for (f in c(1, 0.75, 0.5))
  cat(sprintf("%3.0f%% of observed effect: n per arm = %s (80%% power), %s (90%% power)\n", 100 * f,
              format(n_per_arm(0.80, f), big.mark = ","), format(n_per_arm(0.90, f), big.mark = ",")))

# Check against R's built-in function (unrounded n per arm)
cat("power.prop.test:", round(power.prop.test(p1 = p0, p2 = p1, power = 0.80)$n, 1), "\n")

# Simulation: how often does the z-test reject at the 80%-power sample size?
set.seed(982072)
n    <- n_per_arm(0.80)
reps <- 20000
x0 <- rbinom(reps, n, p0); x1 <- rbinom(reps, n, p1)
ph <- (x0 + x1) / (2 * n)
z  <- ((x1 - x0) / n) / sqrt(ph * (1 - ph) * 2 / n)
cat(sprintf("Simulated power at n = %s per arm: %.3f (%d replicates)\n", format(n, big.mark = ","), mean(abs(z) > z_a, na.rm = TRUE), reps))

Output from running the code

100% of observed effect: n per arm = 68 (80% power), 91 (90% power)
 75% of observed effect: n per arm = 114 (80% power), 152 (90% power)
 50% of observed effect: n per arm = 237 (80% power), 316 (90% power)
power.prop.test: 67.9 
Simulated power at n = 68 per arm: 0.808 (20000 replicates)

The simulation draws many pairs of event counts at the planned sample size and counts how often the two-proportion z-test rejects the null. It should land close to the target power; at very small required sample sizes it can fall below it, because the normal approximation is rough there.

14. Analyses Reported by the Sponsor

The registry record reports the sponsor's primary comparison as a Fisher exact test with p = 0.12. The unadjusted recalculation gives p = 0.087, alongside a risk difference of -0.210 and a 95% confidence interval from -0.442 to 0.022. The sponsor's analysis is the reported trial analysis; the recalculation is a separate, simple unadjusted analysis of the posted summary statistics and should not be presented as replacing it.

The two p values are not identical. The record does not give enough detail to determine the reason, so no specific explanation should be assigned. The discrepancy flag is false, and the sponsor's result remains the result to cite as the trial's own analysis. The recalculated effect estimate and interval can still help describe the observed contrast and its precision, but they are not evidence that the sponsor used the same calculation.

General: A p value depends on the test and its assumptions, while an effect estimate and confidence interval describe magnitude and uncertainty on a scale that can be clinically interpretable. When a registry analysis and an unadjusted recalculation are both available, readers should keep their provenance clear, avoid selecting one merely because it is smaller, and consider the reported effect estimate and interval as well as the test result.

The registry record does not provide further analysis specifications, such as adjustment variables or additional model details. Those details are not reported in the registry record.

15. Multiplicity, Missing Data and Analysis Populations

The registry record lists a primary outcome and several secondary outcomes, including remission at later time points, relapse, serious adverse events, and glomerular filtration rate change. When several endpoints or time points are examined, multiplicity becomes relevant because repeated testing can increase the chance of at least one apparently unusual result under a set of null hypotheses. General: Common approaches include adjusting significance thresholds, using a hierarchy of hypotheses, or clearly treating secondary analyses as exploratory. The registry record does not report a multiplicity strategy, a testing hierarchy, or whether the secondary outcomes were prespecified in the final analysis plan.

The record identifies one reported analysis for the primary endpoint and reports a sponsor Fisher exact test. It does not give details about interim analyses, subgroup analyses, adjustment variables, or other model choices. These elements are not reported in the registry record and should not be assumed absent or present.

The primary endpoint population is described as intention to treat. That statement does not specify how missing outcomes were handled. The record does not report the number of missing primary assessments, imputation methods, sensitivity analyses, or participant flow by reason for exclusion. Those details are not reported in the registry record. The group denominators posted for the endpoint provide the analysis counts but do not, by themselves, establish the completeness of follow-up or explain any difference between enrollment and analyzed populations.

The relapse endpoint has its own denominators among patients achieving remission. The record does not explain the selection or follow-up process for that analysis, or how missing relapse information was handled. The denominators should therefore be understood as the posted analysis groups, not as evidence about why participants are not represented in that endpoint.

The original sample-size rationale, planned effect, and planned analysis assumptions are not reported in the registry record. Without them, it is not possible to assess whether the trial's design was intended to provide a particular precision or power for the primary or secondary outcomes.

16. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: diverticualr disease (4.0%), headache (4.0%), fracture radius (4.0%), respiratory infection (4.0%), hypertension (3.7%).

The registry record reports serious adverse events for 4 of 25 participants in the prednisolone group and 3 of 27 in the tacrolimus group. It reports zero deaths in each group. Other adverse events are listed for 18 of 25 participants receiving prednisolone and 20 of 27 receiving tacrolimus. These are counts within the recorded groups; they do not, on their own, establish comparative safety.

General: Safety counts are descriptive unless accompanied by a specified comparative analysis and appropriate information about event definitions, timing, severity, and follow-up. A count of participants with at least one event is different from a count of all event occurrences. The registry record does not provide enough detail to determine the full event-level pattern, exposure time, or how events were attributed to treatment. It also does not report a comparative statistical analysis of these safety counts.

The listed serious-event terms and percentages are not a substitute for a complete account of event definitions and group-specific denominators. The posted figures should be read as the recorded counts, not as proof that one treatment is safer or that either treatment has a particular safety profile.

17. What the Result Does, and Does Not, Mean

The primary result describes complete remission at 8 weeks in this randomized trial. The posted counts were 21 of 25 participants receiving prednisolone and 17 of 27 receiving tacrolimus. The estimated risk difference for tacrolimus relative to prednisolone is -0.210, and its 95% confidence interval extends from -0.442 to 0.022. Thus, the observed group contrast is negative, but the interval leaves substantial uncertainty about the size and direction of the underlying difference.

The result does not establish that the treatments are equivalent. An interval that includes zero is not evidence of equivalence, particularly without a prespecified equivalence margin. Nor does the result establish that either treatment is superior. The sponsor's reported Fisher exact test has p = 0.12, while the unadjusted recalculation gives p = 0.087; neither p value should be interpreted as the probability that a treatment effect is absent.

The finding is specific to the population, endpoint definition, and 8-week assessment described in the registry record. It does not answer questions about remission at every later time, relapse for all patients, long-term kidney outcomes, or other clinical settings. The record lists further outcomes, but detailed results for several are not reported in the registry record. The available relapse endpoint is a separate analysis among patients achieving remission.

The trial's randomized design supports a comparative assessment, but no masking is reported. The registry record does not provide details about treatment adherence, outcome assessment procedures, missing data, or the full analysis plan. Those unknowns constrain interpretation. The findings should be treated as estimates with uncertainty, rather than as a definitive claim about treatment effects or individual outcomes.

General: A trial result is one contribution to an evidence base. Its estimate, precision, design, endpoint relevance, and limitations all matter. The registry data alone do not provide a basis for medical advice.

18. Why This Trial Matters Statistically

This trial illustrates why a treatment comparison should be read through both the effect estimate and its uncertainty. The primary risk difference gives an absolute-scale contrast between the groups, while its interval shows that the estimate is not sufficiently precise to settle the direction or magnitude of the underlying difference. A p value alone would not convey that range of uncertainty.

The comparison also illustrates the distinction between a trial's reported analysis and a recalculation based on posted summary statistics. The sponsor reports a Fisher exact test; the recalculation gives a different unadjusted p value. Keeping those analyses distinct protects against presenting a derived calculation as if it were the trial's own reported result.

The outcome structure matters as well. Remission at 8 weeks, later remission, relapse after remission, safety events, and change in glomerular filtration rate are related but not interchangeable questions. A result for one endpoint cannot fill in for unreported results on another. The registry record's incomplete detail on multiplicity, missing outcomes, and analysis planning further shows why transparent methods are important when interpreting a set of endpoints.

Finally, planning illustrations demonstrate how sample-size requirements depend on the effect assumed for detection and the desired power. They are not a measure of what this completed trial achieved, and they do not reveal the original design rationale. Treating those distinctions carefully helps readers separate observed findings, statistical uncertainty, and hypothetical planning choices.

19. 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 primary comparison is based on 52 enrolled participants, with endpoint counts of 25 in the prednisolone group and 27 in the tacrolimus group. The confidence interval is broad and crosses zero, limiting precision about the size and direction of the difference. The record does not report a clinically important difference against which to judge the estimate.

The study was randomized but masking was none. The registry record does not describe allocation concealment, the randomization procedure, outcome assessment procedures, treatment adherence, or any treatment changes. These details are not reported in the registry record. Their absence from the record limits assessment of potential sources of bias but does not establish that any particular bias occurred.

The primary endpoint is specific to complete remission at 8 weeks under the stated laboratory definition. The record does not explain how the definition was selected, whether assessments were independently reviewed, or how missing measurements were handled. The intention-to-treat label is reported, but missing-data methods and participant flow are not reported in the registry record.

Several secondary outcomes are listed, while detailed posted results are available only for the relapse endpoint among them. Multiplicity handling, secondary analysis plans, and interim analyses are not reported in the registry record. This makes the scope and confirmatory status of secondary findings difficult to assess. The original sample-size justification and design assumptions are also not reported. Planning illustrations based on the observed effect do not answer whether the trial was adequately designed for its intended question.

The safety information gives participant counts but limited detail for interpreting comparative risk, event timing, or exposure. The registry record also does not provide detailed eligibility criteria or the full treatment regimen. These limitations narrow the conclusions that can be drawn from the posted information.

20. Related Tutorials

21. Related Statistical Calculators

22. Sources

• ClinicalTrials.gov: Tacrolimus Versus Prednisolone for the Treatment of Minimal Change Disease — NCT00982072.
• Registry record last updated 2021-05-04; status last verified 2021-04.
• Results first posted 2021-05-04.
• 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.

23. Record Summary

NCT00982072 is a completed, phase 4, randomized, parallel-group study comparing tacrolimus tablets with prednisolone tablets for minimal change disease and nephrotic syndrome. The registry record lists an enrollment of 52 and reports no masking. The primary endpoint was complete remission at 8 weeks, defined by normalization of serum albumin and urine PCR <50 units, with the primary population described as intention to treat.

The posted counts were 21 of 25 participants for prednisolone and 17 of 27 for tacrolimus. The risk difference for tacrolimus relative to prednisolone was -0.210, with a 95% confidence interval from -0.442 to 0.022. The sponsor reports a Fisher exact test p = 0.12; the unadjusted recalculation gives p = 0.087.

Listed secondary outcomes include later remission, relapse, serious adverse events, and glomerular filtration rate change. Detailed results are not reported in the registry record for several of these outcomes. The record does not report the original sample-size rationale, multiplicity strategy, or missing-data methods.