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 Permeability Factor in Focal Segmental Glomerulosclerosis (NCT00007475), a na phase 1, phase 2 study. The primary outcome is Reduction in Proteinuria in Recurrent FSGS Following Renal Transplant With Plasma Exchange and Cyclophosphamide..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00007475 |
| Phase | Phase 1, Phase 2 |
| Condition | Focal Segmental Glomerulosclerosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns patients with focal segmental glomerulosclerosis (FSGS), including patients whose disease recurs following renal transplantation and patients being evaluated for elevated FSGS permeability factor (FPF). The registry describes evaluation of FPF levels and RNA expression profiles, as well as follow-up of participants with native kidneys. The primary outcome population is listed as all participants, regardless of amount of follow-up. The record does not provide a detailed eligibility definition or numerical baseline description of the enrolled participants.
Intervention
The listed treatment combines plasma exchange and cyclophosphamide. The treatment description states that plasma exchange involved a course of 5 treatments over 10 days, followed by cyclophosphamide. The cyclophosphamide regimen depended on GFR; the available description is incomplete, so the full dosing rules cannot be reconstructed from the registry record. The study also evaluates FPF levels, RNA expression and treatment-related changes in FPF.
Comparator
No randomized control group is identified. The registry classifies the study as single-group, with allocation recorded as NA and masking recorded as none. Comparisons among FPF-defined subgroups or observations over time are not equivalent to a randomized treatment comparison. They do not by themselves separate treatment effects from participant differences, the course of disease or other changes during follow-up.
Primary question
The primary outcome concerns proteinuria remission or response in FSGS occurring in native kidneys, in the context of plasma exchange and cyclophosphamide. Outcome categories are defined by proteinuria levels: complete remission is below 0.3 g/d; partial remission is between 0.3 and 2 g/d; incomplete response is between 2 and 3.5 g/d; and relapse is a return to at least 3.5 g/d. The registry describes assessment every 3 months up to a year. It does not report numerical outcome results, so the frequency of these outcomes and the precision of any treatment-associated change cannot be assessed.
3. Trial Design
The registry describes a completed phase 1 and phase 2 study sponsored by the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). It lists enrollment as 15, the study model as single-group, allocation as NA, and masking as none. The recorded start is 2000-12 and primary completion is 2014-06. These design features describe an exploratory study rather than a randomized, masked comparison of treatment strategies.
The study's stated aims extend beyond the primary proteinuria outcome. They include identifying patients with elevated FPF, examining peripheral blood mononuclear cell RNA expression profiles, describing FPF kinetics during immunomodulatory therapy or plasma exchange, identifying immunosuppressive agents associated with sustained reductions in FPF, and examining whether sustained FPF reduction is associated with recurrence after renal transplantation. These aims address biomarkers, biological patterns, treatment-associated changes and clinical outcomes. The registry record does not report numerical findings for these aims.
The intervention record lists plasma exchange and cyclophosphamide. The described plasma-exchange course consists of 5 treatments over 10 days, followed by cyclophosphamide. The dosing description refers to GFR categories, but the available text is truncated, so the complete regimen is not reported in the registry record. The treatment description does not establish a control condition.
Because the design is single-group, changes observed during follow-up cannot be interpreted as a randomized contrast with an untreated or alternative-treatment group. The record does not report a protocol-level sample-size justification, a detailed statistical analysis plan, or the procedures used to handle departures from the stated treatment course. The registry lists 5 outcome measures and 1 analysis, but that information alone does not define the analysis population or explain how the multiple study aims were addressed. Numerical estimates for the primary outcome 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.
Before assignment: Enrollment of subjects occurred at fraction of the anticipated rate despite over a decade of study conduct; the discrepancy between actual (15) and projected (100) enrollment is due to (a) lack of revising the registry entry with revisions of the protocol (n=50, excluding historical controls) and (b) lower referral rates by external nephrologists.
Overall Study
| Stage | FPF NOT Assayed, Plasma Exchange + Cyclophosphamide Completed | FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed | FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed | Historical Controls |
|---|---|---|---|---|
| STARTED | 12 | 1 | 2 | 0 |
| FSGS Permeability Factor (FPF) Assayed | 0 | 1 | 2 | 0 |
| Initial Transplant | 12 | 1 | 2 | 0 |
| Post-initial Transplant Proteinuria | 12 | 1 | 2 | 0 |
| Initial Plasma Exchange | 12 | 1 | 2 | 0 |
| Initiate Cyclophosphamide | 12 | 1 | 2 | 0 |
| COMPLETED | 8 | 1 | 2 | 0 |
| NOT COMPLETED | 4 | 0 | 0 | 0 |
Reasons for not completing this period:
| Reason | FPF NOT Assayed, Plasma Exchange + Cyclophosphamide Completed | FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed | FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed | Historical Controls |
|---|---|---|---|---|
| Withdrawal by Subject | 1 | 0 | 0 | 0 |
| Adverse Event | 3 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: all participants, regardless of amount of follow-up
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 | FPF NOT Assayed Provisionally, PE + Cyclophosphamide Completed | FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed | FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed | Total |
|---|---|---|---|---|---|
| Participants analysed | 12 | 1 | 2 | 15 | |
| Age, Continuous | years at consent | 39.5 (32.25 to 47.25) | 63 (63 to 63) | 19 (18.5 to 19.5) | 37 (25 to 48.5) |
| Sex: Female, Male: Female | Participants | 8 | 0 | 1 | 9 |
| Sex: Female, Male: Male | Participants | 4 | 1 | 1 | 6 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 3 | 0 | 0 | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 3 | 0 | 0 | 3 |
| Race (NIH/OMB): White | Participants | 6 | 1 | 2 | 9 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 12 | 1 | 2 | 15 |
| Treated for Prior Recurrence: Yes | participants | 2 | 1 | 0 | 3 |
| Treated for Prior Recurrence: No | participants | 10 | 0 | 2 | 12 |
| Time from onset/diagnosis of Focal Segmental Glomerulosclerosis to study enrollment/consent | years | 10 (7.75 to 14.75) | 6 (6 to 6) | 1.5 (1.25 to 1.75) | 8 (4 to 13.5) |
6. Primary Endpoint
The registry lists the primary outcome as Reduction in Proteinuria in Recurrent FSGS Following Renal Transplant With Plasma Exchange and Cyclophosphamide., measured over every 3 months up to a year followed with native kidneys.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | 95% Confidence Interval |
| Unit | proportion of participants with outcome |
| Endpoint classification | Continuous Endpoint |
The primary outcome is described as reduction in proteinuria in recurrent FSGS following renal transplant with plasma exchange and cyclophosphamide. Its operational description specifies outcomes for FSGS occurring in native kidneys, with assessment every 3 months up to a year. It separates proteinuria status into complete remission, partial remission, incomplete response and relapse. These categories make the outcome clinically interpretable in terms of proteinuria levels rather than treating all changes as interchangeable.
The registry labels the measure as a mean, with a 95% confidence interval, and describes the unit as the proportion of participants with an outcome. It explains that each category may be treated as a binary outcome: a participant either meets that category or does not. The mean of such a zero/one measure is a proportion. The record specifies an exact binomial approach for the confidence interval.
As a general statistical point, categorical outcome definitions can clarify what counts as remission or response, while the proportion communicates how common that category was in the analyzed group. A confidence interval describes uncertainty around an estimate; it does not correct for the absence of a comparison group or establish that treatment caused an observed outcome. No proportions or confidence-interval limits for the primary outcome are reported in the registry record. The listed outcome population includes all participants, regardless of amount of follow-up, but the record does not explain how incomplete follow-up was handled in estimating those proportions.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group study, not a randomized treatment comparison. Its treatment and outcome descriptions can identify what was intended to be studied, but they do not supply numerical primary-outcome results. In particular, the record does not report the proportion of participants with complete remission, partial remission, incomplete response or relapse, nor any corresponding confidence-interval limits. The size and precision of a primary-outcome association therefore cannot be evaluated from the registry outcome information.
The primary outcome categorizes proteinuria in FSGS occurring in native kidneys. Complete remission is defined as proteinuria below 0.3 g/d, partial remission as between 0.3 and 2 g/d, incomplete response as between 2 and 3.5 g/d, and relapse as a return to at least 3.5 g/d. Assessment is described every 3 months up to a year. These definitions state how outcome categories are distinguished; they do not tell the reader how many participants met any category or how outcomes changed over follow-up.
The study also has biomarker and exploratory aims concerning FPF levels, RNA expression profiles, treatment-associated FPF kinetics and recurrence after transplantation. The record gives these aims and names secondary outcomes, but it does not report numerical findings for them. It is therefore not possible to establish from this record whether FPF levels changed, whether a change was sustained, or whether any such change was associated with a clinical outcome.
As a general statistical point, an estimate and its confidence interval are both needed to judge the observed magnitude and its precision. A wide interval would indicate substantial uncertainty, while a narrow interval would indicate greater precision under the analysis assumptions. Neither can be assessed for the primary outcome because the numerical estimates are not reported in the registry record. Even if estimates were available, precision would not by itself resolve causal limitations inherent in a single-group design.
The primary completion date and completed status indicate that the study is recorded as completed, but completion is not evidence that every planned analysis was reported. The enrollment entry is 15; the record does not provide the primary-outcome denominators or explain how participants with varying follow-up contributed to each category. The outcome description explicitly includes all participants regardless of amount of follow-up, but the handling of missing observations is not reported in the registry record.
The adverse-event record contains counts for named treatment groups and lists serious-event terms at 8.3%. Those safety figures describe reported events, not proteinuria efficacy. The safety information also cannot substitute for the missing primary-outcome estimates. Overall, the available information establishes the study question, design features, outcome definitions and some safety counts, while leaving the numerical clinical findings and their uncertainty unreported.
9. Statistical Methods Explained
What does the listed mean represent?
The primary measure is labelled a mean, but its unit is a proportion of participants with an outcome. The registry explains the connection: if each participant is represented by a zero/one value for whether a defined outcome occurred, the average of those values is the proportion meeting the definition. This is a standard way to express a binary outcome. It is not a mean proteinuria concentration, and it does not describe the magnitude of proteinuria change for an individual participant.
The outcome description applies this approach separately to categories such as complete remission, partial remission, incomplete response and relapse. A participant meeting one category contributes to that category's count; the registry notes that calculations may be expressed relative to the complement of the whole group count. Because no actual category proportions are reported, the registry record does not show how common any category was or whether category estimates were based on the same follow-up information.
What does the confidence interval contribute?
The record specifies 95% confidence intervals calculated using an exact binomial distribution. As a general statistical explanation, a confidence interval expresses the uncertainty associated with an estimated proportion under the chosen model. The exact binomial method is designed for a binary outcome and uses the binomial sampling framework rather than relying on a large-sample normal approximation for the interval. An interval is not a guarantee that the true value lies within its particular limits, and it does not account for sources of bias that are not part of the calculation.
The interval limits are not reported in the registry record. Consequently, neither the point estimates nor their precision can be reviewed. Without the denominators used for each outcome estimate, it is also not possible to assess how much information contributed to each proportion. The listed enrollment does not establish the denominator for every analysis, particularly when the outcome population is described as including participants regardless of amount of follow-up.
Why does the study design matter to interpretation?
The study is recorded as single-group, allocation NA and unmasked. As a general statistical point, a single-group study can describe outcomes observed during treatment and can examine patterns over time. It does not provide the randomized concurrent comparison needed to isolate a treatment effect from other explanations for change. For example, changes may coincide with treatment without being attributable solely to treatment. This is a design limitation, not a calculation problem that a confidence interval can fix.
The registry lists plasma exchange followed by cyclophosphamide. It also sets out aims involving FPF and RNA expression, but the record does not state a detailed statistical plan for modeling repeated measurements, comparing subgroups or dealing with potential confounding. Those procedures are not reported in the registry record. The lack of masking is recorded, but the record does not explain how outcome assessment was standardized or whether assessors were independent of treatment decisions.
How should planning illustrations be read?
The sample-size assumptions describe a planning illustration based on an observed effect, not the trial's own design. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect expressed as a stated fraction of the observed effect. As a general statistical point, such calculations explore the sample size that might be needed under specified assumptions. They are not evidence that the completed study had that sample size, and they are not observed power.
The registry's design is single-group, whereas the planning illustration assumes two-arm equal allocation. That illustration therefore should not be mistaken for a description of the enrolled study or a randomized comparison. The values of any planning estimates are not reported in the registry record. A planning calculation also depends on the effect assumed, variability and analysis method; it cannot replace a protocol-level justification, which is not reported in the registry record.
What does over-dispersion mean in general?
As a general statistical point, over-dispersion means that observed variation is greater than a model assumes. When present, standard errors based on the narrower variance assumption may understate uncertainty. It can arise when observations are more heterogeneous or dependent than the model allows. The registry record does not report an over-dispersion assessment or a model for repeated observations. The primary outcome is described as a binary proportion with an exact binomial interval, so the record does not give a basis to claim that over-dispersion was found or addressed.
More generally, repeated observations from the same participant are not automatically independent. A method that analyzes each visit separately differs from a method that models within-participant change. The registry gives an assessment schedule but does not report which approach was used for the primary analysis. No model specification, covariate adjustment, or sensitivity analysis is reported in the registry record. These omissions limit assessment of how the stated outcome definitions were translated into estimates.
10. Multiplicity, Missing Data and Analysis Populations
The study describes several distinct aims: FPF identification and kinetics, RNA expression profiles, treatment-associated changes in FPF, proteinuria outcomes and recurrence after transplantation. The registry lists 5 outcome measures and 1 analysis. It does not report a multiplicity strategy, such as whether any outcome was designated as confirmatory, whether comparisons were prioritized, or whether statistical thresholds were adjusted across outcomes. The registry record does not explain how results across the different aims should be interpreted together.
As a general statistical point, examining multiple outcomes or subgroup contrasts can increase the chance that some findings appear noteworthy by chance. A prespecified hierarchy or adjustment can address particular forms of this issue, but neither is reported in the registry record. The listed number of analyses alone does not establish that only one hypothesis or comparison was evaluated.
The primary outcome population is described as all participants, regardless of amount of follow-up. The registry does not report how missing measurements, incomplete follow-up, withdrawals or unavailable outcome classifications were handled. It also does not provide outcome-specific denominators. Thus, the stated population description does not clarify whether a participant with limited follow-up contributed to every scheduled assessment or only to available observations.
The record does not report an intention-to-treat definition, a per-protocol definition, or another analysis-population rule. Since the study is single-group and the treatment description does not give a complete dosing regimen, the handling of incomplete treatment is also not reported in the registry record. These gaps prevent assessment of whether the analyzed participants corresponded to the enrolled population or whether missingness could have influenced the observed proportions.
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 |
|---|---|---|---|
| Plasma Exchange + Cyclophosphamide, Complete Treatment | 3 | 12 | 25.0% |
| FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed | 0 | 1 | 0.0% |
| FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed | 0 | 2 | 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 |
|---|---|
| Plasma Exchange + Cyclophosphamide, Complete Treatment | 0 of 12 |
| FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed | 0 of 1 |
| FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed | 0 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Plasma Exchange + Cyclophosphamide, Complete Treatment | FPF Assayed Pre-Tx as Low, PE + Cyclophosphamide Completed | FPF Assayed Pre-Tx as High, PE + Cyclophosphamide Completed |
|---|---|---|---|---|
| Rejection of Kidney Graft | Immune system disorders | 1 of 12 | 0 of 1 | 0 of 2 |
| Hemorrhagic cystitis | Infections and infestations | 1 of 12 | 0 of 1 | 0 of 2 |
| Adenocarcinoma of the Prostate Gland | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 12 | 0 of 1 | 0 of 2 |
| Neutropenia | Blood and lymphatic system disorders | 1 of 12 | 0 of 1 | 0 of 2 |
Serious events with the highest arm-level rate: rejection of kidney graft (8.3%), hemorrhagic cystitis (8.3%), adenocarcinoma of the prostate gland (8.3%), neutropenia (8.3%).
The registry reports serious and other adverse-event counts for three named treatment groups. For “Plasma Exchange + Cyclophosphamide, Complete Treatment,” 12 participants are listed as at risk for serious events and 3 as affected; the other-event fields list 12 at risk and 0 affected. The low-pre-treatment-FPF completed group lists 1 at risk and 0 affected in both fields. The high-pre-treatment-FPF completed group lists 2 at risk and 0 affected in both fields. These groups have different descriptions, and the registry does not establish that they should be added together.
The serious-event terms listed are rejection of kidney graft, hemorrhagic cystitis, adenocarcinoma of the prostate gland and neutropenia, each at 8.3%. The record does not provide enough detail to assign those terms to a particular group or to establish whether an individual could contribute to more than one term. The figures should therefore be read as registry-reported event information, not as a comparative safety estimate.
As a general statistical point, event counts and percentages depend on the population at risk, duration and completeness of observation, and the rules used to code events. The registry record does not report follow-up time for safety, attribution to treatment, or a comparative control group. Death counts are not reported in the registry record; the corresponding fields are null. These data do not provide a basis for comparing risk between treatments.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a completed phase 1 and phase 2 single-group study evaluated plasma exchange and cyclophosphamide in the context of FSGS, proteinuria and FPF. It defines a primary outcome based on proteinuria categories and describes follow-up every 3 months up to a year for FSGS occurring in native kidneys. It also states biomarker and recurrence-related objectives. These details clarify the questions the study was intended to address.
The record does not report numerical results for the primary endpoint. It therefore does not show the proportion of participants meeting each proteinuria category, the size of any change, or the precision of an estimate. Numerical findings for FPF levels, RNA expression profiles, FPF kinetics and transplant recurrence are also not reported in the registry record. The absence of these outcome results means that the treatment-associated clinical and biomarker findings cannot be assessed from the record.
Even if the outcome estimates were available, the single-group design would restrict causal interpretation. As a general statistical point, an outcome observed after treatment is not by itself evidence that treatment caused it. Without a randomized comparison, observed outcomes may reflect the underlying disease course, participant characteristics, concurrent care or other factors. The record's lack of masking is also relevant to interpretation, although details of outcome assessment procedures are not reported.
The serious adverse-event counts and listed terms provide limited safety information, but they do not supply a treatment-versus-control comparison. The registry does not report deaths in the listed fields. Nor does the record provide enough information to infer the relationship between each listed event and treatment. Safety figures should not be treated as proof of either presence or absence of a causal risk.
Accordingly, the registry supports a description of study design, intervention, outcome definitions and selected adverse-event entries. It does not support a conclusion about the magnitude, precision or causality of a proteinuria response. It also does not establish whether sustained FPF reduction was associated with reduced recurrence after transplantation. Those findings are not reported in the registry record.
13. Why This Trial Matters Statistically
This study is statistically notable because its objectives link a biological marker, treatment-associated changes and clinical outcomes within a single-group design. FPF levels and RNA expression profiles are not the same type of measurement as proteinuria categories or recurrence after transplantation. The questions require distinct definitions and analysis approaches, yet the registry record provides limited information about how those analyses were conducted.
The primary outcome is expressed as a proportion for categories of proteinuria status, with an exact binomial confidence interval specified. This is a recognizable approach for binary classifications. It also makes clear why the outcome definition matters: each participant's status depends on the stated proteinuria thresholds, and the estimate represents the share meeting a category rather than the average quantity of proteinuria.
The record also illustrates the importance of separating estimation from causal inference. A proportion and its interval can describe observed outcomes, but a single-group study cannot by itself show what would have happened under another treatment or without treatment. A precise estimate, if reported, would still need to be interpreted in light of the design and the population observed.
Completeness of reporting is central to statistical interpretation. The registry gives enrollment, study status, outcome definitions and some safety counts, but numerical primary outcome estimates, denominators, confidence-interval limits and missing-data procedures are not reported in the registry record. Without these, readers cannot judge the observed magnitude or the uncertainty around the clinical outcomes.
Finally, the study's broad set of aims raises questions about prioritization and multiplicity. The registry lists 5 outcome measures and 1 analysis, but does not explain how multiple biological and clinical questions were handled statistically. The example underscores that a clear endpoint definition is useful, while a complete account of analysis populations, missing observations and comparisons is needed to support a fuller interpretation.
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 largest limitation for interpreting the clinical outcome is that numerical primary-outcome results are not reported in the registry record. No category proportions, confidence-interval limits or outcome-specific denominators are given. The reader therefore cannot judge the observed frequency of remission, response or relapse, or the precision of any estimate.
The study is single-group, with allocation recorded as NA and masking as none. There is no randomized comparator identified in the registry record. As a result, treatment-associated changes cannot be separated from other explanations for outcomes. The record does not report a detailed protocol, criteria for treatment completion, or the complete cyclophosphamide dosing description; the available dosing text is truncated.
The primary outcome description refers to all participants regardless of amount of follow-up and gives an assessment schedule, but missing-data handling is not reported in the registry record. There is no explanation of how incomplete follow-up affected the outcome denominator, how multiple visits were analyzed, or whether participants contributed to each scheduled assessment. The analysis population and any sensitivity analyses are also not reported.
The study's biomarker, RNA-expression and transplant-recurrence aims involve multiple kinds of outcomes, but the registry does not describe a multiplicity plan or a hierarchy of hypotheses. Numerical results for these secondary questions are not reported. The listed safety information has limited comparative interpretation because the record does not report a control group, exposure duration or event attribution. Death counts are not reported in the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Permeability Factor in Focal Segmental Glomerulosclerosis — NCT00007475.
- Registry record last updated 2016-02-10; status last verified 2016-02.
- Results first posted 2016-02-10.
- 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 record identifies NCT00007475, “Permeability Factor in Focal Segmental Glomerulosclerosis,” as a completed phase 1 and phase 2 single-group study sponsored by NIDDK. Enrollment is listed as 15; the recorded start is 2000-12 and primary completion is 2014-06. Allocation is NA and masking is none.
The intervention includes plasma exchange followed by cyclophosphamide. The primary outcome concerns proteinuria categories in FSGS occurring in native kidneys, with assessment every 3 months up to a year. Other objectives concern FPF, RNA expression, treatment-associated changes and transplant recurrence. Numerical primary-outcome findings and their confidence intervals are not reported in the registry record.
The record lists serious adverse-event terms and group counts, but does not provide a randomized comparison. Missing-data procedures, outcome denominators, multiplicity handling and a complete statistical analysis plan are not reported in the registry record.