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 Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome (NCT01113385), a na na study. The primary outcome is Focal Segmental Glomerulosclerosis Permeability Factor (FSPF).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01113385 |
| Phase | NA |
| Condition | Focal Segmental Glomerulosclerosis, Steroid Resistant Nephrotic Syndrome |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 7 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Children's National Research Institute |
| Results posted | Yes |
2. Clinical Question
The study asks whether oral galactose is associated with a change in focal segmental glomerulosclerosis permeability factor (FSPF) in children with steroid-resistant nephrotic syndrome. Its rationale is the investigators’ hypothesis that galactose may bind the factor thought to contribute to kidney permeability changes, with a resulting reduction in albumin permeability and proteinuria. That rationale is a hypothesis, not a finding reported by the registry.
Population
Children with steroid-resistant nephrotic syndrome and focal segmental glomerulosclerosis. The registry lists an enrollment of 7. It does not report further participant characteristics in this record.
Intervention
Oral D-Galactose at 0.2gm/kg/dose twice a day, to a maximum of 15 gm BID, for 16 weeks. These are the registered regimen details, not evidence that the treatment changed the endpoint.
Comparator
The study is listed as single-group, with allocation recorded as NA. No control group or comparator treatment is listed, so the design does not provide a like-for-like concurrent comparison.
Primary question
The primary endpoint is FSPF, expressed as Palb, at 16 weeks. The registry’s stated clinically significant response criteria are reduction to <0.5 Palb or a decrease by > 0.3 Palb. The result for this endpoint is not reported in the registry record.
3. Trial Design
NCT01113385 is a completed pilot study sponsored by Children's National Research Institute. Its title concerns oral galactose in children with steroid-resistant nephrotic syndrome; its official title specifies the effect on FSPF and proteinuria. The registry lists the start as 2009-10 and primary completion as 2013-03.
The design is recorded as single-group, with allocation listed as NA and masking as NONE. The intervention is D-Galactose, described as oral treatment for 16 weeks. Enrollment is listed as 7. The record does not describe a concurrent control group, random assignment, or a masked comparison. Consequently, the design is not a randomized treatment comparison, and changes over time cannot be separated from other explanations using a control group within this study.
The primary outcome is FSPF at 16 weeks, measured as Palb. The record also lists the number of participants achieving complete or partial remission at 16 weeks as a secondary outcome, and states that there are 2 outcome measures and 1 analysis. The reported information does not include the result for either outcome. The study phase is listed as NA. Further details about the protocol, participant eligibility criteria, or how the analysis was carried out are not reported in the registry record.
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 | Galactose |
|---|---|
| STARTED | 7 |
| COMPLETED | 7 |
| NOT COMPLETED | 0 |
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 | Galactose |
|---|---|---|
| Participants analysed | 7 | |
| Age, Continuous | years | 7.7 (SD 4) |
| Age, Categorical: <=18 years | Participants | 7 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Sex: Female, Male: Female | Participants | 3 |
| Sex: Female, Male: Male | Participants | 4 |
| Region of Enrollment: United States | participants | 7 |
6. Primary Endpoint
The registry lists the primary outcome as Focal Segmental Glomerulosclerosis Permeability Factor (FSPF), measured over 16 weeks.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Palb |
| Endpoint classification | Continuous Endpoint |
The primary endpoint is focal segmental glomerulosclerosis permeability factor (FSPF), expressed as Palb, at 16 weeks. The registry describes Palb as the induction of glomerular albumin permeability in isolated glomeruli. Its scale runs from 0 to 1, with 0 indicating normal glomeruli and 1 indicating injury to the permeability barrier.
This is a continuous laboratory measure: it represents a value along a scale rather than a category such as remission or no remission. The record defines a clinically significant response as a reduction in FSPF to <0.5 Palb or a decrease in FSPF by > 0.3 Palb in response to oral galactose at week 16. These are criteria for judging a response, not reported findings. The registry record does not report the observed FSPF value, change from any earlier measurement, or the number of participants meeting either criterion.
FSPF and the secondary remission outcome address different aspects of the study question: one is a permeability measure, while the other counts participants meeting remission categories. Neither endpoint’s result is reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes the study question and its endpoint, but it does not provide the primary outcome result. There is no reported FSPF mean at 16 weeks, no reported change from an earlier measurement, and no standard deviation or confidence interval for the endpoint in the record. The stated response thresholds therefore cannot be used to determine how participants’ measurements compared with those criteria.
The primary endpoint is continuous and is measured on the Palb scale from 0 to 1. The registry’s response definition—reduction to <0.5 Palb or a decrease by > 0.3 Palb—specifies how a potentially meaningful response was to be characterized. A threshold in an endpoint definition does not itself show that any participant reached it. The number of participants meeting the criteria is not reported in the registry record.
The study is listed as a single-group pilot with enrollment of 7. Without a registered concurrent comparator, the study design cannot distinguish a change during treatment from changes that might occur for other reasons. This is a design limitation, not evidence for or against an effect. The registry record also does not provide enough outcome information to assess the size or precision of any observed change.
The registry lists complete or partial remission at 16 weeks as a secondary outcome, but does not report its result. Nor does it provide an analysis estimate, a measure of uncertainty, or a statistical test result for that outcome. The stated FSPF response criteria and the remission outcome should not be treated as observed results.
In general, estimates describe the observed data, while uncertainty measures indicate how precisely an estimate is determined. Without a reported estimate and its uncertainty for the primary endpoint, the direction, magnitude, and precision of the observed FSPF result cannot be characterized from this record. The posted information supports description of the intended endpoint and study design only; it does not support a conclusion about the treatment’s effect.
9. Statistical Methods Explained
The registry identifies FSPF as the primary outcome and describes it as a continuous measure reported as Palb. It also lists the parameter as a mean and the dispersion measure as a standard deviation. These labels describe the intended summary of the endpoint; the record does not include the actual mean or standard deviation.
What does a mean describe?
In general, a mean represents the arithmetic average of the measured values in the analysis population. For a continuous endpoint such as Palb, it can provide a compact description of the measurements. A mean alone does not show how widely participant measurements vary or whether the mean change reflects a consistent pattern across participants. The record does not report the primary outcome mean or define the analysis population for that endpoint.
What does a standard deviation describe?
In general, a standard deviation describes how dispersed individual measurements are around their mean. It is not the same as the uncertainty of the mean. A standard deviation can help describe variation among measurements, while a confidence interval is commonly used to express uncertainty about an estimate. The registry lists standard deviation as the dispersion measure, but no numerical dispersion result or confidence interval is reported in the registry record.
What would a confidence interval add?
In general, a confidence interval gives a range of values compatible with an estimate under a specified statistical method and assumptions. Its width reflects precision: a narrower interval indicates greater precision than a wider interval, all else being comparable. It does not guarantee that the true value lies within the interval. No confidence interval for FSPF or remission is reported in the registry record, so precision cannot be assessed from the posted results.
How should the single-group design be considered?
The registry lists a single-group study and no like-for-like control comparison. In general, repeated measurements in one group can describe change over time, but without a concurrent comparator, such change alone does not establish that the intervention caused it. Other influences, including the course of the condition and changes in care, may also be relevant. The registry record does not report the analysis structure or any adjustment for other factors, so the specific method used to analyze the endpoint is not reported in the registry record.
What do the response thresholds mean?
The protocol description sets out two ways to classify a clinically significant FSPF response at week 16: a value below 0.5 Palb or a decrease greater than 0.3 Palb. These criteria are distinct from the continuous outcome summary. In general, a threshold can make an endpoint easier to interpret as a category, but categorization also discards information about the exact measurements. The registry does not report the observed values or the number meeting either criterion.
What do the planning assumptions establish?
The record describes a planning illustration based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and a target effect defined as a stated fraction of the observed effect. Those assumptions describe a hypothetical planning exercise, not the registered single-group trial design. Sample-size illustrations are planning calculations, not observed power, and they do not substitute for results. The record does not report the trial’s own sample-size justification or design rationale.
What analysis details are unavailable?
The registry does not report the specific statistical test or model, how repeated measurements were handled, whether adjustments were made, or how the analysis treated missing observations. It also does not report the analysis population, the number contributing to the primary outcome, or the result estimate and its uncertainty. Those details are needed to assess how the posted outcome was derived; they should not be inferred from the endpoint labels alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 2 outcome measures and 1 analysis. The primary outcome is FSPF at 16 weeks, and the listed secondary outcome is the number of participants achieving complete or partial remission at 16 weeks. The results for both are not reported in the registry record.
Multiplicity arises when several outcomes, time points, or statistical comparisons are evaluated. In general, assessing multiple results can increase the chance that an apparently notable result occurs by chance. A prespecified testing hierarchy or adjustment can address some multiplicity concerns. The registry record does not report a multiplicity strategy, such as an adjustment, hierarchy, or designation of outcomes for confirmatory testing. The number of listed outcomes alone does not establish how they were handled statistically.
Missing-data handling and analysis populations are also not reported in the registry record. Enrollment is listed as 7, but the record does not state how many participants contributed measurements to the primary endpoint or the remission outcome, whether any measurements were missing, or how missing observations were treated. It does not report whether the analysis included all enrolled participants or a defined subset.
The registry record also does not report interim analyses or a detailed statistical analysis plan. Without these details, it is not possible to assess the planned approach to multiplicity, missing observations, or analysis-population selection. These are reporting gaps and should not be filled by assumptions.
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 |
|---|---|---|---|
| Galactose | 0 | 7 | 0.0% |
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 |
|---|---|
| Galactose | 1 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Galactose |
|---|---|---|
| Diarrhea | Gastrointestinal disorders | 1 of 7 |
For the Galactose group, the registry reports 7 participants at risk for serious adverse events and 0 affected. For other adverse events, it reports 7 participants at risk and 1 affected. These are the registry’s reported counts; they do not describe the primary efficacy endpoint.
The serious adverse event terms list is empty. The death field is null, so death information is not reported in the registry record; null should not be interpreted as a confirmed count of zero. The record does not provide further event descriptions, timing, severity details beyond the serious-event category, or an assessment of whether events were related to the intervention.
In general, a count of affected participants is a descriptive safety summary for the period and definitions used in a study. It does not establish that an event was caused by treatment, and a small study may provide limited information about uncommon harms. The safety figures should therefore be read as the counts recorded for this study, not as a complete estimate of safety in a broader population.
12. What the Result Does, and Does Not, Mean
The registry record establishes that this was a completed, single-group pilot study of oral D-Galactose in children with steroid-resistant nephrotic syndrome and focal segmental glomerulosclerosis. It describes a 16-week intervention and identifies FSPF, measured as Palb, as the primary endpoint. It also specifies response criteria and lists remission as a secondary outcome.
What the record does not establish is the observed result. It does not report the FSPF measurement at 16 weeks, a change from an earlier measurement, or the number meeting the stated FSPF criteria. It also does not report the remission result. Thus, the registered endpoint definitions cannot be read as evidence that a response occurred or did not occur.
Even if a within-group change were reported, a single-group design without a concurrent comparator would limit causal interpretation. In general, change over time may reflect the intervention, but it may also reflect other influences. The registry record does not provide the outcome estimates or details needed to evaluate those possibilities for this study.
The enrollment of 7 and the posted safety counts describe this pilot record, but do not by themselves establish the size or precision of an effect. The serious adverse event count is 0 among 7 at risk, and the other adverse event count is 1 among 7 at risk. These counts are not a substitute for outcome results, and death information is not reported in the registry record.
The appropriate interpretation is therefore limited: the record documents the study’s question, intervention, design, endpoint definitions, and certain safety counts. It does not report enough efficacy information to characterize the observed treatment effect or its uncertainty.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of how a pilot question, a continuous endpoint, and a single-group design need to be interpreted together. The registry defines a biological permeability measure, FSPF in Palb, and provides thresholds for describing a potentially important response. Such prespecified definitions can clarify what investigators intended to regard as meaningful, but they cannot replace reporting the measured values and their uncertainty.
The single-group design is central to interpretation. In general, a study without a concurrent comparator can describe what happened among participants receiving the intervention, but it cannot isolate the intervention’s contribution to change in the same way as a suitable controlled comparison. This distinction matters when a study’s rationale proposes a causal mechanism.
The record also illustrates why complete outcome reporting is essential. The primary and secondary outcomes are identified, but their results are not reported in the registry record. Without those results, readers cannot assess the direction, magnitude, variability, or precision of the findings, or determine how participants compared with the stated response criteria.
Finally, safety counts and efficacy outcomes answer different questions. The reported adverse-event counts provide a limited descriptive view of events recorded in the group; they do not supply evidence about the primary endpoint. For statistical interpretation, each type of evidence must be considered according to what it measures and what the design can support.
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 main trial-specific limitation is the single-group design. The registry lists allocation as NA and does not describe a control arm. As a result, the design does not provide a concurrent comparison for interpreting change during treatment. This limits causal conclusions even if outcome changes had been reported.
The registry lists enrollment as 7, but does not report the number of participants included in each outcome analysis. It also does not report baseline or week-16 FSPF results, the number meeting the response criteria, or the remission outcome. Without those results, the size and precision of any observed change cannot be evaluated.
The record specifies the measurement scale and clinically significant response criteria, but does not report how the analysis was conducted, how missing measurements were handled, or what analysis population was used. It does not report a multiplicity approach, sample-size justification for the trial, or detailed statistical analysis plan. These omissions constrain assessment of the analysis and the reliability of any outcome interpretation.
The safety record includes counts for serious and other adverse events, but provides no serious-event terms and no death count; the death field is null. Further event details and attribution to the intervention are not reported in the registry record. These limitations apply to interpretation of the registered information and should not be replaced with assumptions about unreported data.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Oral Galactose in Children With Steroid Resistant Nephrotic Syndrome — NCT01113385.
- Registry record last updated 2014-09-15; status last verified 2014-09.
- Results first posted 2014-09-15.
- 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
NCT01113385 is a completed pilot study sponsored by Children's National Research Institute. It enrolled 7 and evaluated oral D-Galactose in children with steroid-resistant nephrotic syndrome and focal segmental glomerulosclerosis. The registered regimen was 0.2gm/kg/dose twice a day, to a maximum of 15 gm BID, for 16 weeks.
The design is listed as single-group, with allocation NA and masking NONE. The primary endpoint is FSPF, measured as Palb at 16 weeks; the secondary outcome concerns complete or partial remission at 16 weeks. The registry defines FSPF response criteria but does not report either outcome result. Safety counts list 0 affected for serious adverse events and 1 affected for other adverse events, each among 7 at risk. Death information is not reported in the registry record.