Adipose-derived SVF for the Treatment of Knee OA: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Adipose-derived SVF for the Treatment of Knee OA (NCT02726945), a randomized na study. The primary outcome is Safety - Number of Participants With Treatment-Emergent Serious Adverse Events.

2. Clinical Question

The trial was intended to examine safety and efficacy for pain, function, and stiffness in people with knee osteoarthritis. Its listed primary outcome addresses safety by counting participants with treatment-emergent serious adverse events. The record also lists a secondary efficacy outcome: percent change in WOMAC score from baseline to 6 months. No result for that secondary outcome is reported in the registry record.

The comparison of interest is among the low-dose SVF, high-dose SVF, and placebo groups. Random assignment and double masking are recorded, but the record does not describe the allocation procedure, who was masked, or whether masking was maintained. The registry record does not report a specific statistical comparison for the primary safety outcome. The posted counts therefore describe the recorded safety outcome by group, rather than establishing a comparative effect.

3. Trial Design

This is a randomized, double-blinded, parallel-group clinical trial of a device-based SVF intervention for osteoarthritis of the knee. The listed intervention is GID SVF-2, and the comparator is placebo. The arm descriptions identify low-dose SVF, high-dose SVF, and placebo groups. The record does not state the dose amounts, the allocation ratio, or the details of how randomization was conducted.

The sponsor is GID BIO, Inc. The trial is marked completed, with a reported start of 2016-04 and primary completion date of 2018-04-01. Enrollment is listed as 39. The record identifies the phase as NA; it does not provide further phase information. The stated purpose is to examine safety and efficacy for pain, function, and stiffness in the knees of participants with osteoarthritis.

The primary outcome is the number of participants with treatment-emergent serious adverse events, with a timeframe of up to 1 year. A description says subjects were monitored for serious and device-related adverse events and that baseline MRIs would be compared with 1 year for abnormal findings. The record says 38 subjects were monitored to 1 year and one subject was monitored for 6 weeks, after withdrawing to receive a total knee replacement. It does not explain how that difference in monitoring duration was handled in the analysis.

The listed secondary outcome is percent change in WOMAC score from baseline to 6 months. No efficacy results or details of the WOMAC analysis are reported in the registry record. The record also does not describe a statistical analysis plan, a sample-size justification, interim analyses, or the analysis populations. These omissions limit what can be said about the planned and performed comparisons beyond the stated design features and posted outcome counts.

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: Thirty-eight subjects were monitored to 1 year. One subject was monitored for 6 weeks, when the subject withdrew from the study to receive a total knee replacement.

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.

Population: All subjects

6. Primary Endpoint

The registry lists the primary outcome as Safety - Number of Participants With Treatment-Emergent Serious Adverse Events, measured over up to 1 year.

The primary endpoint is the number of participants with treatment-emergent serious adverse events during monitoring up to 1 year. It is a count of participants, not a count of all event episodes. If one participant experiences more than one event, a participant-based outcome is intended to count that person as a participant with an event rather than treating every event as a separate person; the registry record does not give further counting rules.

Safety outcomes are used to describe adverse experiences observed during a trial. Serious adverse events are distinguished from other adverse events, but the registry record does not provide a definition of seriousness or list serious-event terms. It states that serious and device-related adverse events would be monitored. It also describes comparing baseline MRIs with 1 year for abnormal findings, but does not report MRI results in the registry record for the posted outcome.

A count is straightforward to interpret as the number of participants recorded with the outcome. It is less informative without a clear denominator, follow-up completeness, event definitions, and a comparison method. The adverse-event record lists 13 participants at risk in each group, with zero participants recorded as having serious adverse events in each. The primary outcome description also reports monitoring of 38 participants to 1 year and one participant to 6 weeks. How those monitoring details relate to the posted at-risk counts is not reported in the registry record.

The endpoint addresses safety, not efficacy. Although the trial’s summary names pain, function, and stiffness as objectives, those concepts are not directly measured by this primary count. The listed WOMAC outcome is separate and no result for it is reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

A binary safety outcome records whether each participant did or did not have the specified event during the defined monitoring period. The posted primary endpoint is expressed as a count of participants with treatment-emergent serious adverse events. Reporting the count by randomized group makes the observed data easy to describe, but a count by itself does not provide the full context of risk. The denominator and the observation period matter, as do event definitions and the way missing follow-up is treated.

The adverse-event record lists 13 participants at risk in each group. It records zero participants with serious adverse events in each group. These are the observed counts. They should not be converted into a claim that the true chance of an event is zero. General statistical principle: even when no events are observed, uncertainty remains about the underlying event frequency, especially when the number of observed participants is limited. A confidence interval can communicate that uncertainty. No confidence interval is reported in the registry record.

A comparative analysis could, in general, estimate the difference or another contrast between groups and quantify uncertainty around that contrast. The registry record does not report a particular contrast, a test statistic, a p-value, a confidence interval, or a model for the serious-adverse-event outcome. It is therefore not possible to characterize a reported inferential comparison from the registry entries. The descriptive counts should remain distinct from any recalculated or modeled result that might use additional assumptions.

Randomization is a design method intended, in general, to reduce systematic differences between groups at allocation. Double masking is also recorded. Masking can, in general, reduce the influence of knowledge about assignment on participant experience, clinical management, or outcome assessment. The record does not identify which people were masked, how the random sequence was generated, how assignment was concealed, or whether masking success was assessed. The presence of randomization and masking labels does not supply those missing procedural details.

Over-dispersion is a general concern for count outcomes when variability is greater than a chosen model expects. It may matter for counts of repeated events, particularly when events cluster within participants. This endpoint is described as the number of participants with an event, rather than a total count of repeated events, and no model is reported. There is no basis in the registry record to say that over-dispersion was assessed or that a particular count model was used. The distinction between participants affected and event episodes is important when reading adverse-event summaries.

For planning, a sample-size illustration is described as based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes the detectable effect as a stated fraction of the observed effect. This is a planning convention, not evidence about the trial’s original design or its actual statistical plan. The record does not report the trial’s sample-size justification. The illustrated assumptions also do not, by themselves, identify which groups or outcome are intended for a comparison. A planning sample size is not observed power, and it does not add information to the observed safety counts.

General explanation: power is a design property describing the chance that a specified analysis will detect an effect of a specified size under its assumptions. It is not a measure of whether an observed result is reliable, and a post hoc power calculation does not replace an interval estimate or a clear account of the analysis. The sample-size illustration described in the record should be read as a planning exercise rather than as evidence that the trial was adequately powered for any specific safety or efficacy comparison.

The efficacy outcome listed is percent change in WOMAC score from baseline to 6 months. A change score compares a participant’s follow-up measurement with that participant’s baseline measurement. General statistical practice may analyze such outcomes in several ways, including comparing follow-up values with baseline adjustment or comparing change scores. The registry record does not state which approach was used, how WOMAC data were summarized, or whether any adjustment was made. It reports no result for this outcome in the registry record for the posted results.

Precision is conveyed by estimates and their uncertainty, not by a count alone. General explanation: a confidence interval gives a range of values compatible with the data and the method used, under its assumptions. A narrow interval usually indicates greater precision than a wide interval. Because no interval estimates are reported for the primary safety endpoint, readers cannot assess its precision from the registry record beyond noting the observed counts and listed at-risk group sizes.

Finally, statistical interpretation depends on consistency between the prespecified endpoint, the analysis population, and the reported data. The record gives a primary outcome title and a monitoring description, but it does not report the protocol’s analysis rules, handling of incomplete follow-up, or definitions for serious events. Without those details, the counts can be described accurately, but their inferential meaning and relationship to a planned hypothesis test remain unclear.

10. Multiplicity, Missing Data and Analysis Populations

Multiplicity arises when a study evaluates more than one outcome, comparison, time point, or analysis. In general, the more statistical tests conducted, the greater the chance that at least one result may appear notable by chance if no adjustment or prespecified testing hierarchy is used. The registry record lists the serious-adverse-event outcome and a secondary WOMAC percent-change outcome from baseline to 6 months. It does not report whether multiplicity was addressed, whether outcomes had a hierarchy, or which comparisons were prespecified for formal testing.

The record does not report an analysis population, such as whether the primary analysis included all randomized participants or only those with available follow-up. It also does not describe how participants who withdrew, missed assessments, or had incomplete monitoring were handled. These details are important because analyses restricted to observed data can differ from analyses that account for all randomized participants according to a defined rule.

One participant is described as monitored for 6 weeks after withdrawing to receive a total knee replacement, while 38 subjects were monitored to 1 year. The record does not state how that participant’s later safety status or any missing efficacy measurements were handled. The reason for withdrawal is reported, but its impact on the outcome analysis is not.

Missing-data methods, outcome definitions, multiplicity procedures, and analysis populations are not reported in the registry record. Without them, it is not possible to determine whether the posted results reflect all randomized participants, a defined subset, or a particular prespecified testing strategy.

11. 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 5% reporting threshold.

Most frequent non-serious adverse event terms

For the primary safety endpoint, the record lists 13 participants at risk in each group. The number with treatment-emergent serious adverse events is zero in the low-dose SVF group, zero in the high-dose SVF group, and zero in the placebo group. The record also lists zero deaths in each group. These are counts, not a demonstration that the possibility of serious harm is absent.

The record lists other adverse events separately: one participant in the low-dose group, two in the high-dose group, and zero in the placebo group. It does not give the event terms, severity, timing, relationship to treatment, or whether participants experienced more than one event. Those counts therefore should not be interpreted as a complete account of the kinds of adverse events observed.

The stated timeframe for the primary safety outcome is up to 1 year. The monitoring description says 38 subjects were monitored to 1 year and one was monitored for 6 weeks, after withdrawing to receive a total knee replacement. The relationship between that follow-up description and the 13 participants listed at risk in each group is not explained in the registry record. Since the outcome is a participant count, it is also important not to confuse the number of participants with the number of event episodes.

In general, safety interpretation considers event definitions, exposure and follow-up, denominators, and uncertainty. The record does not report confidence intervals or a statistical comparison for the serious-event counts. The figures support a factual description of what was recorded, but they do not by themselves establish equivalence among groups or quantify the range of plausible underlying risks.

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

The result establishes that the registry record posts zero participants with treatment-emergent serious adverse events in each of the low-dose SVF, high-dose SVF, and placebo groups, with 13 participants at risk in each group. It also records zero deaths in each group. These observations are specific to the reported endpoint and monitoring information. They are not a complete statement about all possible safety outcomes, all event types, or outcomes beyond the reported follow-up.

The counts do not show that the groups have identical safety profiles. A result with no observed serious events in any group cannot, by itself, rule out differences in underlying event risk. The registry record does not report an inferential comparison or uncertainty interval, and it does not describe a formal equivalence or non-inferiority analysis. The counts should therefore be interpreted descriptively rather than as proof that any groups are the same.

The other-adverse-event counts are distinct from the primary serious-adverse-event endpoint. The record lists one other adverse event in the low-dose group, two in the high-dose group, and zero in the placebo group, but does not give the event descriptions. Without terms, timing, severity, and attribution, those counts cannot explain what kinds of events occurred or how they were judged.

These safety results do not answer whether SVF changed knee pain, function, or stiffness. The registry identifies WOMAC percent change from baseline to 6 months as a secondary outcome, but no result for that outcome is reported in the registry record. It would be inappropriate to infer efficacy from the safety endpoint or from the trial’s stated objectives alone.

General statistical interpretation distinguishes absence of an observed event from evidence of absence of risk. The uncertainty around a result depends on the amount and completeness of information and on the analysis method. Here, follow-up duration is described as up to 1 year, with one participant monitored for 6 weeks and 38 monitored to 1 year; the record does not explain how that difference was incorporated into the reported counts. These details constrain how broadly the safety result can be interpreted.

13. Why This Trial Matters Statistically

This trial is statistically notable for the distinction between a safety endpoint and an efficacy question. The primary endpoint counts participants with treatment-emergent serious adverse events, while the stated study purpose also includes pain, function, and stiffness. The listed WOMAC outcome is an efficacy measure, but no result for it is reported in the registry record. Keeping these questions separate prevents a safety count from being mistaken for evidence about symptom outcomes.

The design records randomization, double masking, and a parallel structure, with low-dose SVF, high-dose SVF, and placebo groups. These features describe how the study was organized, but do not replace details about allocation, masking roles, analysis methods, or participant follow-up. A rigorous statistical reading asks both what the design labels imply in general and which procedural details are actually documented.

The serious-event counts are zero across all groups, with 13 participants at risk in each. This makes uncertainty especially important: identical observed counts are not the same as a demonstration of identical risks. A clear account of denominators, event ascertainment, follow-up, and uncertainty estimates would help readers understand how much the data can distinguish among groups. Those estimates and analysis details are not reported in the registry record.

The follow-up description also matters. Thirty-eight subjects were monitored to 1 year, and one was monitored for 6 weeks before withdrawing to receive a total knee replacement. The record does not explain how the shorter monitoring period affected endpoint ascertainment or analysis. Reporting such details helps readers evaluate the completeness of safety information and the population represented by the results.

More broadly, this record illustrates why a posted outcome count should be read alongside the outcome definition and analysis plan. Statistical interpretation depends not only on the observed event count but also on how events were classified, how missing observations were handled, and whether comparisons were prespecified. Several of these details are not reported in the registry record.

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 registry record gives limited detail about the trial procedures. It identifies randomization and double masking but does not describe the randomization method, allocation concealment, the people who were masked, or the masking procedures. Dose amounts, eligibility criteria, and further details of the placebo are also not reported in the registry record. These omissions limit assessment of how closely the assigned groups and interventions can be characterized.

The primary endpoint is a participant count for treatment-emergent serious adverse events, but the record does not provide a definition of a serious event, a list of serious-event terms, or event-level details. It says serious and device-related adverse events would be monitored. The posted counts therefore do not reveal what events were assessed, how relatedness was determined, or whether multiple events occurred in any participant.

The monitoring description gives differing follow-up durations: 38 subjects were monitored to 1 year and one subject was monitored for 6 weeks after withdrawing to receive a total knee replacement. How that observation period was incorporated into the safety analysis is not reported in the registry record. The connection between these monitoring details and the group-specific at-risk counts is also not explained.

No inferential results for the primary safety endpoint are reported in the registry entries: no statistical test, confidence interval, or adjusted comparison. The record also does not report an analysis population, missing-data method, multiplicity procedure, interim analyses, or sample-size justification. The secondary WOMAC outcome is named, but its results and analysis are not reported. These limitations mean that the posted information supports descriptive interpretation, while leaving important questions about precision, completeness, and planned inference unresolved.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Adipose-derived SVF for the Treatment of Knee OA — NCT02726945.
• Registry record last updated 2025-04-25; status last verified 2020-02.
• Results first posted 2019-08-21.
• 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.

18. Record Summary

The record describes a completed, randomized, double-blinded, parallel trial of GID SVF-2 for knee osteoarthritis, with low-dose SVF, high-dose SVF, and placebo groups. Enrollment is listed as 39. The sponsor is GID BIO, Inc.; the reported start is 2016-04 and primary completion date is 2018-04-01.

The primary endpoint is the number of participants with treatment-emergent serious adverse events during monitoring up to 1 year. Each group has 13 participants at risk and zero participants recorded with a serious adverse event. The record also lists zero deaths in each group and other-adverse-event counts of one, two, and zero for the low-dose, high-dose, and placebo groups, respectively.

The record says 38 subjects were monitored to 1 year and one was monitored for 6 weeks after withdrawing to receive a total knee replacement. WOMAC percent change from baseline to 6 months is listed as a secondary outcome, but its result is not reported in the registry record. Statistical analysis details, uncertainty estimates, missing-data handling, and the sample-size justification are not reported in the registry record.