Autologous Cell Therapy for Female Stress Urinary Incontinence: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Autologous Cell Therapy for Female Stress Urinary Incontinence (NCT01008943), a na phase 2 study. The primary outcome is Number of Participants That Experienced Biopsy Procedure-related Adverse Events.

2. Clinical Question

3. Trial Design

This was a phase 2 prospective study with a single-group, nonrandomized design. The registry lists enrollment of 16 and identifies the intervention as Autologous Muscle Derived Cells, a biological treatment. Allocation is recorded as not applicable, masking as none, and no control arm is described. These features mean the trial is not a randomized comparison and does not provide a like-for-like control group for interpreting observed outcomes.

The study summary describes its purpose as determining the safety and potential effectiveness of a single dose of 200 million Cook MyoSite Autologous Muscle Derived Cells for stress urinary incontinence. That statement describes the study’s aim; it does not establish that every participant received the same dose or that the intended dose was administered. Dose receipt and dose-specific results are not reported in the registry record.

The registry lists a start date of 2010-06-02, a primary completion date of 2012-09-21, and a status of completed. Cook MyoSite is listed as the sponsor. The registry record identifies four outcome measures but contains no analysis entries. It does not report a statistical analysis plan, sample-size justification, interim analyses, or the criteria used to define analysis populations.

The primary outcome concerns biopsy-procedure-related events at biopsy or between biopsy and treatment, approximately 6 weeks. Since biopsy occurred before treatment, the registry presents these results independently of AMDC dose received. The adverse-event listings also give counts for serious and other adverse events in the AMDC group. These safety listings do not create a control comparison, and the registry record does not establish that every listed event was caused by the intervention.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: One patient experienced procedural dizziness.

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 Number of Participants That Experienced Biopsy Procedure-related Adverse Events, measured over at biopsy or between biopsy and treatment, approximately 6 weeks.

The primary endpoint is the number of participants who experienced biopsy procedure-related adverse events. The registry defines such events as systemic responses to the biopsy procedure or injury at the biopsy site. Biopsy was required to generate AMDC products, so it was a part of the study process even though it preceded treatment. The stated timeframe is at biopsy or between biopsy and treatment, approximately 6 weeks.

This endpoint separates events related to the tissue-collection procedure from events potentially associated with the cell treatment. Because biopsy preceded AMDC treatment, the registry presents the results independently of AMDC dose received. That distinction is important: a biopsy-related outcome cannot, by itself, describe the safety of treatment exposure.

The registry reports that one patient experienced procedural dizziness. It also states that all biopsy procedure-related events either self-resolved or were easily treated. The reported result is a participant count, not a measure of event severity across all possible outcomes, and no comparative estimate is reported. The registry record does not provide a confidence interval or an analysis entry for this endpoint. It also does not provide further details about the duration or clinical course of the reported dizziness.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The registry identifies the primary outcome as a participant count: the number of participants who experienced biopsy procedure-related adverse events. This is a binary outcome at the participant level. For each participant, the underlying status is whether at least one event meeting the endpoint definition was experienced; the reported summary is the count of participants with such an event. This is different from counting every event, because a participant could in principle experience more than one event. The registry’s stated endpoint is participants, not total event episodes.

What does a count establish?

A count is descriptive. It records how many participants met the outcome definition in the observed group. It does not, by itself, estimate what would have happened under another treatment or without the procedure. To interpret a count as a rate or compare it with another group, the relevant denominator and a suitable comparison design are needed. Even with a denominator, a single-group result does not remove differences in participant characteristics, care, or event ascertainment that could affect comparisons with other settings.

The primary endpoint is restricted by its definition and timeframe. It includes systemic responses to biopsy or injury at the biopsy site, at biopsy or between biopsy and treatment, approximately 6 weeks. This makes the outcome relevant to the biopsy procedure, but not interchangeable with all adverse events during the study. The registry explicitly notes that biopsy was required to generate AMDC products and occurred before treatment; the results are therefore presented independently of AMDC dose received.

Why does the design matter?

In general, random allocation helps make groups comparable with respect to both measured and unmeasured characteristics, on average. This trial is recorded as single group and nonrandomized, with allocation not applicable and no masking. It has no like-for-like randomized control comparison. Consequently, the observed counts describe events in the study group but cannot establish what the event count would have been under a different intervention or without AMDC treatment. This is a design limitation, not a statistical adjustment that can be resolved from the counts alone.

The absence of masking can also matter in general, since awareness of treatment or study procedures may influence reporting or assessment. The registry lists masking as none, but it does not describe how adverse events were elicited, judged, or verified. It is therefore not possible to characterize the impact of ascertainment procedures from the registry record.

What would an inferential analysis add?

In general, a confidence interval expresses uncertainty around an estimated quantity under a statistical model. For a participant count, an interval might be used to convey uncertainty in an underlying event probability, provided that the denominator, analysis population, and method are specified. A narrow interval would indicate greater precision under those assumptions, while a wide interval would indicate less precision. The registry record contains no analysis entries and gives no confidence interval for the primary outcome, so the precision of an estimated event probability is not reported.

In general, a hypothesis test can assess compatibility between observed data and a specified null hypothesis, subject to the model and assumptions used. A p-value does not measure the probability that a treatment works, and a non-significant result would not establish equivalence or absence of risk. Here, the registry record does not report a test or a p-value for the primary endpoint, and there is no randomized comparator for a treatment-effect test. No inferential conclusion should be inferred from the descriptive count.

How should planning calculations be read?

In general, sample-size calculations depend on assumptions about the effect to detect, variability or event frequency, significance level, allocation, and analysis method. The registry’s planning description specifies a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; it describes the effect as a stated fraction of the observed effect. It also says that calculations for time-to-event outcomes use the total number of events, while other calculations use participants per arm. These are planning assumptions, not evidence that the trial itself used that design.

Any sample-size figure based on an observed effect is a planning illustration, not observed power and not a retrospective confirmation that the study was adequately powered. The trial itself is recorded as single group, while the planning description assumes equal allocation between two arms. The record does not provide a trial-specific sample-size justification, and no numerical planning estimates are stated in the registry record for interpretation. The planning assumptions therefore should not be treated as a description of the completed study’s actual statistical design.

Finally, over-dispersion is a general concern for count outcomes when observed variability exceeds what a chosen model expects. It can make standard errors too small if the model fails to account for that extra variability. The primary outcome is a participant count rather than an event-rate model, and the registry gives no analysis method or model diagnostics. There is no basis for claiming that over-dispersion was assessed or that a particular adjustment was used.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Autologous Muscle-Derived Cells (AMDC)")
n      <- c(16)
events <- c(1)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Autologous Muscle-Derived Cells (AMDC)  16  1  6.2  0.2  30.2  1.1  28.3  1.5  28.7

11. Multiplicity, Missing Data and Analysis Populations

The registry identifies four outcome measures, but it does not report analysis entries. It does not state whether the outcomes were tested as a family, whether one outcome was designated for formal inference beyond the recorded primary label, or whether any multiplicity adjustment was planned. Without a reported testing strategy, the possibility of selective emphasis across several outcomes cannot be evaluated statistically. No adjusted or unadjusted inferential result is reported in the registry record.

Missing-data methods are not reported in the registry record. The record does not describe the amount or pattern of missing outcome information, how missing values were handled, or whether any sensitivity analysis was conducted. It is therefore not possible to determine whether outcome counts reflect complete follow-up or a specified approach to missing observations.

The analysis populations are not reported in the registry record. It does not specify whether participants were analyzed according to enrollment, treatment receipt, or another population definition. The primary endpoint’s description says biopsy-related results are presented independently of AMDC dose received, but it does not supply a full analysis-population definition. This should not be read as proof that every enrolled participant contributed complete data to every outcome.

These reporting gaps limit the ability to assess how outcome selection, missingness, and population definitions may have affected the reported results. They do not justify assuming that data were missing or that analyses were biased; the relevant information is simply not reported in the registry record.

12. Safety Results

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

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: fibula fracture (6.2%).

The AMDC group safety figures list 16 participants at risk for other adverse events, with 12 affected, and 16 at risk for serious adverse events, with 1 affected. These are participant counts, not counts of every event episode. The registry also lists fibula fracture as the top serious-event term, with 6.2%. The term and percentage should be read as part of the serious-event listing, not as a treatment-effect estimate.

The primary biopsy-related outcome is separate from these overall adverse-event figures. The registry reports one patient with procedural dizziness and says that biopsy procedure-related events either self-resolved or were easily treated. It does not establish that the serious-event listing is biopsy-related, nor does the listed serious-event term establish that the event was caused by AMDC treatment. Causal attribution, timing, duration, and individual clinical context are not reported in the registry record for interpretation.

Because the study is a single-group, nonrandomized trial, these safety figures describe what was recorded in the AMDC group without a control comparison. They cannot establish whether event occurrence was higher or lower than it would have been under another treatment or in the absence of treatment. The registry record does not report an inferential safety analysis or a confidence interval for these counts.

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

The registry record documents a completed prospective study in which biopsy was required to generate autologous muscle-derived cell products. For the primary biopsy-procedure outcome, it reports one patient with procedural dizziness and states that all biopsy procedure-related events either self-resolved or were easily treated. The stated timeframe is at biopsy or between biopsy and treatment, approximately 6 weeks. This is a description of the reported procedural events, not a comparison of treatment strategies.

The adverse-event listings provide additional descriptive counts for the AMDC group, including participants with serious and other adverse events. The top serious-event term is fibula fracture, listed with 6.2%. These entries should be interpreted as safety information recorded in the registry. The registry entries do not provide sufficient context to attribute the listed serious event to biopsy or treatment, or to characterize its clinical course.

The observations do not establish treatment effectiveness. Although the study summary describes potential effectiveness as an objective, no effectiveness result is included in the registry record for analysis. Nor does the primary endpoint measure continence improvement; it measures participants with biopsy-related adverse events. The record also has no control group, no analysis entries, and no reported inferential estimate for the primary outcome.

Accordingly, the data support a descriptive account of events recorded in this study, but not a causal conclusion about AMDC safety relative to an alternative or a conclusion about efficacy. The limited enrollment and single-group design also constrain generalization. These distinctions are central to interpreting a small, nonrandomized clinical study: observed counts are informative about what was recorded, while comparative treatment effects require evidence the registry record does not report.

14. Why This Trial Matters Statistically

This trial illustrates why the endpoint’s definition and timing matter. The primary outcome concerns the biopsy needed to generate the cell products, not the effects of receiving the cell treatment. Because the biopsy preceded treatment, the registry presents biopsy-related results independently of AMDC dose received. Keeping those events distinct avoids attributing a procedural observation to an intervention that had not yet been given.

It also demonstrates the difference between descriptive safety reporting and comparative inference. Counts can document how many participants were recorded with events in a study group, and the registry provides both a primary biopsy-related count and broader adverse-event figures. But a single-group, nonrandomized design does not supply a control condition against which to estimate a treatment-related increase or decrease. A reader should not turn event counts into causal conclusions.

The record’s absence of analysis entries is statistically important. Without a reported inferential method, uncertainty estimates, missing-data approach, or analysis-population definition, readers cannot assess the precision and robustness of an estimated effect. The registry’s description of planning assumptions also shows why a planning calculation must be distinguished from the design actually used: its assumptions include equal allocation between two arms, whereas the completed study is recorded as single group.

Finally, a study can contribute useful procedural and safety observations without resolving effectiveness. This distinction is especially important when the primary outcome is an adverse-event count and the study summary includes a broader therapeutic aim. Statistical interpretation should follow the question actually measured, the design that generated the data, and the analyses that are reported.

15. 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 principal design limitation is the absence of a randomized comparator. The registry records a single group, nonrandomized design with no masking. As a result, the reported event counts do not support a like-for-like comparison, and other explanations for observed events cannot be separated from effects of study procedures or treatment.

Enrollment was 16. With a small study group, each participant’s outcome can have substantial influence on the descriptive count, and estimates of event frequency would generally be imprecise. However, the registry record supplies no confidence interval or other precision estimate for the primary endpoint, so its statistical uncertainty is not quantified.

The primary endpoint is limited to biopsy procedure-related adverse events within a defined period: at biopsy or between biopsy and treatment, approximately 6 weeks. It does not capture all possible safety outcomes over all periods, and it is not an effectiveness measure. The broader serious and other adverse-event listings are distinct from the primary biopsy endpoint; their relationship to the biopsy or AMDC treatment is not reported in the registry record for interpretation.

Important analysis details are also absent. The registry record does not report a statistical analysis plan, analysis populations, missing-data methods, outcome ascertainment procedures, or inferential analyses. It lists four outcome measures but no analysis entries. Details of eligibility criteria and actual dose receipt are not reported in the registry record either. These gaps prevent a fuller evaluation of precision, completeness, and generalizability, but they should not be filled by assumptions about what the protocol may have specified.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Autologous Cell Therapy for Female Stress Urinary Incontinence — NCT01008943.
• Registry record last updated 2021-07-01; status last verified 2021-06.
• Results first posted 2015-11-03.
• 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.

19. Record Summary

The registry identifies NCT01008943 as Autologous Cell Therapy for Female Stress Urinary Incontinence, with the official title A Canadian Prospective Nonrandomized Study of Autologous Cell Therapy for Female Stress Urinary Incontinence. It is listed as a completed phase 2 study sponsored by Cook MyoSite, with enrollment of 16 and a single-group, nonrandomized design. The intervention is Autologous Muscle Derived Cells.

The primary outcome is the number of participants with biopsy procedure-related adverse events at biopsy or between biopsy and treatment, approximately 6 weeks. The registry reports one patient with procedural dizziness and states that biopsy-related events either self-resolved or were easily treated. The broader safety listing includes serious and other adverse-event counts in the AMDC group and identifies fibula fracture as the top serious-event term, with 6.2%.

The registry lists four outcome measures and no analysis entries. It does not report a randomized comparison, statistical analysis plan, analysis populations, or missing-data methods. The reported information supports a descriptive reading of the recorded outcomes, not a comparative estimate of treatment effect.