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 Injection of Autologous Adipose-derived Stromal Vascular Fraction in the Finger of Systemic Sclerosis Patients (NCT03060551), a na early/phase 1 study. The primary outcome is Raynaud's Condition Scale.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03060551 |
| Phase | EARLY/Phase 1 |
| Condition | Systemic Sclerosis |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 20 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Suk-Ho Moon |
| Results posted | Yes |
2. Clinical Question
Population
Patients with systemic sclerosis whose hands and fingers were the focus of the intervention. The registry record does not provide further participant characteristics or eligibility details.
Intervention
Injection of autologous adipose-derived stromal vascular fraction. The record says the cells were obtained from lipoaspirates using an automated processing system and injected into the subcutaneous tissue of each finger in contact with neurovascular pedicles.
Comparator
There was no comparator group. The study is recorded as single-group, with allocation listed as not applicable and no masking.
Primary question
The study focused on safety and tolerability, with preliminary assessment of potential efficacy. Its primary outcome was the Raynaud's Condition Scale, which measures the severity of Raynaud's phenomenon. The registry lists assessments at baseline, 2 weeks, 6 weeks, 12 weeks, and 24 weeks.
The central statistical question is how the scale and other recorded outcomes changed over time among participants receiving the injection. Because all participants were in the intervention group, those changes cannot be compared with outcomes under placebo or another control treatment. Changes after treatment may be described, but this design cannot separate a treatment effect from other explanations for change, including the natural course of symptoms or measurement variability.
The registry describes preliminary assessments as a basis for considering potential efficacy that would need confirmation in a randomized placebo-controlled trial on a larger population. That framing matters: the study is primarily an early assessment of safety and tolerability, not a controlled test of efficacy. Numerical results for the primary scale are not reported in the registry record, so the magnitude, direction, and precision of any observed change cannot be assessed from the registry information.
3. Trial Design
This is a completed early-phase 1 study of autologous adipose-derived stromal vascular fraction injection in the fingers of patients with systemic sclerosis. The registry lists an enrollment of 20, a single-group model, allocation as not applicable, and no masking. There is no concurrent control group, so the design is not a randomized comparison and does not support a like-for-like comparison between treatment arms.
The intervention description states that stromal vascular fraction was obtained from lipoaspirates using an automated processing system. It was then injected into the subcutaneous tissue of each finger in contact with neurovascular pedicles. The registry identifies the intervention as a procedure. Further details about treatment delivery, participant eligibility, follow-up procedures, and the operational conduct of the study are not reported in the registry record.
The registry lists the study start as 2018-07-01, primary completion as 2019-12-30, and status as completed. Its stated purpose is to outline safety and assess tolerability, with preliminary assessments at 6 months intended to suggest potential efficacy requiring confirmation in a randomized placebo-controlled trial on a larger population. The sponsor is Suk-Ho Moon.
The main outcome is the Raynaud's Condition Scale, assessed at baseline and at 2 weeks, 6 weeks, 12 weeks, and 24 weeks. The registry specifies a median and inter-quartile range for this outcome. It also lists secondary nailfold capillary microscopy outcomes covering irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array, and capillary ramifications. The registry record does not provide a detailed protocol or a full statistical analysis plan. Sample-size justification, planned interim analyses, and details of analysis populations 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: Among the initially enrolled patients, one patient refused further procedures after baseline evaluation, while another patient dropped out due to complications during local anesthesia right before SVF injection.
Overall Study
| Stage | SVF Injection Group |
|---|---|
| STARTED | 20 |
| COMPLETED | 18 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | SVF Injection Group |
|---|---|
| Withdrawal by Subject | 1 |
| Adverse Event | 1 |
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 | SVF Injection |
|---|---|---|
| Participants analysed | 20 | |
| Age, Continuous | years | 47 (42 to 57) |
| Sex: Female, Male: Female | Participants | 15 |
| Sex: Female, Male: Male | Participants | 3 |
| Race and Ethnicity Not Collected | Participants | — |
| Region of Enrollment: South Korea | participants | 20 |
| Body mass index (BMI) kg/m2 | kg/m2 | 21.5 (18.2 to 23.1) |
| Disease duration (years) | years | 6 (1 to 17) |
| Reynaud's phenomenon duration | years | 9 (4 to 17) |
| Smoking history (Current or ex-) | Participants | 3 |
| Interstitial lung disease | Participants | 10 |
| Types of cutaneous manisfetations (diffuse or limited): Diffuse | Participants | 8 |
| Types of cutaneous manisfetations (diffuse or limited): Limited | Participants | 10 |
| Hand dominance (right) | Participants | 18 |
| Pulmonary hypertension | Participants | 1 |
| History of renal crisis | Participants | 1 |
| Diabetes mellitus | Participants | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Raynaud's Condition Scale, measured over Baseline, 2 weeks, 6 weeks, 12 weeks, 24 weeks.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Inter-Quartile Range |
| Unit | score on a scale |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the Raynaud's Condition Scale, a score intended to represent the severity of Raynaud's phenomenon. The scale ranges from 0 to 10, with higher scores indicating more severe Raynaud's phenomenon. A severity scale can make a patient-reported or clinically assessed symptom burden easier to track consistently across scheduled assessments, although the registry record does not specify how the scale was administered or who completed it.
The listed time points are baseline, 2 weeks, 6 weeks, 12 weeks, and 24 weeks. These repeated assessments permit description of the score over follow-up within the intervention group. The registry specifies that the outcome is summarized using a median and inter-quartile range. In general, the median describes the middle of the observed score distribution, while the inter-quartile range describes its spread across the central part of that distribution. These summaries can be useful when scores are skewed or contain outlying values.
The registry does not report the median or inter-quartile range at any time point, nor does it report a numerical change from baseline, a confidence interval, or a hypothesis test for this endpoint. Therefore, it is not possible to assess the observed direction or size of change, or its statistical precision, from the registry record. The scheduled assessments and scale definition explain what was intended to be measured, but do not establish an outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a small, single-group study of an injection procedure in patients with systemic sclerosis. Enrollment was 20, and the design had no control arm. This means the primary statistical evidence, if outcome values were available, would concern change within the treated group over time. Such change is not equivalent to an estimated treatment effect against an untreated, placebo, or alternative-treatment group.
The primary outcome is clearly defined: the Raynaud's Condition Scale ranges from 0 to 10, and higher scores represent more severe Raynaud's phenomenon. Assessments were planned at baseline, 2 weeks, 6 weeks, 12 weeks, and 24 weeks. However, the registry record does not report the actual score summaries or an estimate of change. Without those values, neither the direction nor magnitude of change can be described. There is also no reported confidence interval to show the precision of an estimated change, and no reported test result to assess compatibility with a specified null hypothesis.
The registry lists nailfold capillary microscopy findings as secondary outcomes, including irregularly enlarged capillaries, giant capillaries, hemorrhages, loss of capillaries, disorganization of the vascular array, and capillary ramifications. Numerical results for these outcomes are not reported in the registry record. Consequently, they cannot be used to corroborate or qualify the primary outcome from the information recorded.
For safety, the registry lists 0 serious adverse events among 20 participants at risk and 5 other adverse events among 20 participants at risk. It also lists 0 deaths. These are counts as recorded, not estimates of comparative risk: there is no control group, and the registry does not provide event descriptions, timing, severity details for the other events, or a broader account of ascertainment. A count of zero serious events in this study does not demonstrate that the procedure has no serious risks.
The registry's stated purpose is an early assessment of safety and tolerability with preliminary assessments of potential efficacy. That framing is appropriate to the design: a single-group study can help describe the experience recorded in the study, but it cannot by itself determine whether change was caused by the intervention. The registry explicitly says that potential efficacy would need confirmation in a randomized placebo-controlled trial on a larger population. Since the numerical efficacy results are absent from the registry record, even a descriptive account of the actual symptom trajectory is not possible.
9. Statistical Methods Explained
Statistical methods determine how measurements are summarized, how uncertainty is expressed, and what comparisons can be supported. The registry identifies some design and outcome features, but it does not report a complete statistical analysis plan. The distinctions below explain the general meaning of those methods and the limits of what can be inferred from the registry record.
What does the single-group design allow?
In a single-group study, participants receive the intervention and outcomes can be described within that group. Repeated measurements may show how scores vary from baseline across follow-up. However, without a concurrent comparison group, there is no direct estimate of what would have happened to similar participants over the same period without the intervention. Natural symptom variation, changes in care, expectations, and measurement variability are examples of general explanations that can affect within-group change. The registry does not report a method that addresses these possible explanations.
Allocation is listed as not applicable, and masking is listed as none. These design features are consistent with the registry's single-group description, but the record does not explain further how outcome assessments were conducted. Lack of masking can matter in general when outcomes depend on judgment or participant reporting, because expectations may influence assessments. That is a methodological consideration, not evidence that bias occurred in this study.
Why report a median and inter-quartile range?
The primary endpoint is specified as a median with an inter-quartile range. In general, the median is a measure of location that is less influenced by extreme observations than the mean. The inter-quartile range describes the spread of the central portion of a distribution. This summary can be informative when a score's distribution is asymmetric or when observations are concentrated at particular values. The registry does not state why this summary was selected, describe the score distribution, or provide the actual summaries.
A median and inter-quartile range at each scheduled assessment would describe score distributions at those times. They would not, by themselves, quantify the paired change for each participant or show uncertainty around a change estimate. Analysis of repeated observations can account for the fact that measurements from the same participant are related; the particular method used for such data is not reported in the registry record. It would therefore be inappropriate to assume that a specific repeated-measures model or test was used.
What would a confidence interval contribute?
In general, a confidence interval expresses uncertainty around an estimated quantity under the assumptions of the statistical method. It can show which effect sizes are reasonably compatible with the observed data and analysis. A narrow interval indicates greater precision than a wide interval, though precision is not the same as freedom from bias. The registry record does not report a confidence interval for the primary outcome or the secondary outcomes. The precision of any change estimate therefore cannot be evaluated from the registry record.
A hypothesis test, when used, addresses compatibility between the data and a specified null hypothesis under a statistical model. A p-value alone would not measure the size or importance of an effect. The registry record does not report a p-value or identify the hypothesis tests used. No conclusion about statistical significance can be drawn from the outcome information recorded.
How should planning calculations be read?
Sample-size calculations are planning illustrations, not observed results. The sample-size assumptions associated with this study describe a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The effect to detect is described as a stated fraction of the observed effect. For time-to-event calculations, the stated convention is to report the total number of events using the Schoenfeld method; other calculations are expressed as participants per arm. These assumptions do not change the fact that the actual study is single-group and has no like-for-like control comparison.
General planning calculations based on an observed effect illustrate how sample size may relate to a target effect under specified assumptions. They do not represent the study's observed power. Nor do such illustrations establish that the study was designed or powered for the assumed target. The registry's own sample-size justification is not reported in the registry record. The observed primary outcome summary needed to interpret an effect-based illustration is also not reported in the registry record, so no specific planning estimate should be inferred from the study description alone.
Over-dispersion is a general concern when count data vary more than an assumed model allows. If present, a model that assumes less variability than the data contain can produce uncertainty estimates that are too small. The registry lists several nailfold findings, but does not report the data distributions or statistical models used for them. It is therefore not possible to assess whether over-dispersion was relevant or how it was handled.
More broadly, an analysis method should match the outcome type, repeated-measures structure, distribution, and study question. The registry specifies the primary outcome's scale and summary format, and names the secondary outcome domains. It does not report model specifications, covariate adjustment, methods for handling missing observations, or sensitivity analyses. Those details are not reported in the registry record and cannot be reconstructed from the design description.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 16 outcome measures and 16 analyses. When a study examines many outcomes or makes multiple comparisons, the chance of at least one apparently notable result arising by chance can increase. In general, multiplicity methods may control the overall error rate, prioritize a primary outcome, or treat secondary findings as exploratory. The registry identifies the Raynaud's Condition Scale as the primary outcome and lists nailfold capillary microscopy findings among the secondary outcomes, but a multiplicity adjustment or testing hierarchy is not reported in the registry record.
The primary outcome is assessed at baseline, 2 weeks, 6 weeks, 12 weeks, and 24 weeks. Repeated assessment times create choices about which contrasts are central to interpretation, such as change from baseline at a specified follow-up or an overall pattern over time. The registry does not report which contrasts were prespecified, whether all assessment times were tested, or how any resulting multiplicity was handled. Numerical results are not reported, so the practical effect of these choices cannot be evaluated.
Missing-data handling is not reported in the registry record. The record does not state how many participants contributed measurements at each time point, how incomplete observations were handled, or whether any sensitivity analyses were conducted. The analysis population and any rules for inclusion or exclusion from analyses are also not reported in the registry record. These omissions limit assessment of whether the outcome summaries, if available, represent all enrolled participants or a subset with observed measurements.
Because the study is single-group, multiplicity considerations do not resolve the absence of a control group. Statistical adjustment for multiple testing can address one type of uncertainty, but it cannot create a causal comparison that the design did not include.
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 |
|---|---|---|---|
| SVF Injection Group | 0 | 20 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| SVF Injection Group | 0 of 20 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| SVF Injection Group | 5 of 20 |
Most frequent non-serious adverse event terms
| Event term | Organ system | SVF Injection Group |
|---|---|---|
| Transient pallor in finger after injection | Injury, poisoning and procedural complications | 3 of 20 |
| Transient paresthesia in liposuction lesions | Injury, poisoning and procedural complications | 1 of 20 |
| Dizziness after lidocaine injection | Injury, poisoning and procedural complications | 1 of 20 |
The registry lists 0 serious adverse events among 20 participants at risk in the SVF Injection Group. It lists 5 other adverse events among 20 participants at risk and 0 deaths. The record does not list serious adverse event terms. These figures are counts reported in the registry, not a comparison with another treatment or a measure of excess risk attributable to the injection.
A zero count of serious adverse events means that none were recorded in this group under the study's reporting and follow-up process. It does not establish that serious events cannot occur. In general, interpretation of safety counts depends on how events were defined, actively sought, assessed, and followed over time. Those details are not reported in the registry record. The record also does not describe the five other adverse events, their severity, timing, duration, or relationship to the procedure.
Because there was no control group, the event counts cannot establish whether adverse events were more or less common than under another approach. The enrollment was 20, so the safety experience recorded applies to a limited group of participants. The registry figures are useful as a compact account of what was recorded, but the missing event descriptions and lack of comparative data limit interpretation.
12. What the Result Does, and Does Not, Mean
The registry record identifies a completed early-phase 1 study examining autologous stromal vascular fraction injection in the fingers of patients with systemic sclerosis. Its design and outcome definitions can be described, as can the recorded adverse event counts. However, the record does not report numerical results for the primary Raynaud's Condition Scale outcome or for the listed nailfold capillary findings. The actual pattern of change and its precision therefore cannot be assessed from the registry information.
Even if within-group outcome summaries were available, a single-group study would describe change over time among participants receiving the injection, not the difference between the injection and a control treatment. Without a control group, alternative explanations for change cannot be separated from an intervention effect. The registry itself describes the efficacy assessments as preliminary and says potential efficacy requires confirmation in a randomized placebo-controlled trial on a larger population.
The safety record lists 0 serious adverse events, 5 other adverse events, and 0 deaths, with 20 participants at risk in each adverse event category. These counts do not prove that serious adverse events are impossible, and the registry does not supply descriptions of the other adverse events. They should be interpreted as recorded events in this study, not as comparative safety estimates.
The design supports a limited descriptive account of an early investigation. It does not, from the registry information, support a conclusion about the size or certainty of symptom change, a causal claim about the injection, or a comparison with placebo or another treatment. No treatment recommendation follows from these registry data. The central statistical limitation is not a complicated result but the lack of numerical outcome results and the lack of a comparator.
13. Why This Trial Matters Statistically
This study illustrates how a trial's design determines the claims its data can support. The registry identifies a single-group early-phase study focused on safety and tolerability, with preliminary assessment of potential efficacy. That is a distinct purpose from a randomized comparison designed to estimate a treatment difference. The absence of a comparator is not a minor technical detail: it limits causal interpretation of any change over time.
The record also makes clear why endpoint definition and result reporting both matter. The Raynaud's Condition Scale is described as a score from 0 to 10, with higher scores representing more severe Raynaud's phenomenon, and the assessment schedule is specified. Yet numerical summaries are not reported. Without results, readers cannot evaluate the actual magnitude, direction, or precision of change, regardless of how clearly the outcome is defined.
The study is also a useful example of keeping safety counts in proportion. The registry lists no serious adverse events among 20 participants at risk, alongside 5 other adverse events and no deaths. Those observations can inform a descriptive account, but they do not provide a controlled estimate of comparative risk or rule out uncommon harms.
Finally, the registry's own framing emphasizes the need for confirmation in a randomized placebo-controlled study on a larger population. Statistically, this recognizes the difference between an early signal-seeking investigation and a confirmatory design. Careful interpretation preserves that distinction: design, observed outcomes, uncertainty, and causal evidence are related but not interchangeable.
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 most direct limitation of interpretation is that numerical results for the primary outcome are not reported in the registry record. The record gives the scale definition, summary format, and assessment times, but not the medians, inter-quartile ranges, estimates of change, confidence intervals, or test results. Numerical results for the secondary nailfold capillary findings are also not reported in the registry record.
The study has a single-group design, with allocation listed as not applicable and masking listed as none. With no control group, observed change cannot be separated from other influences over time. The absence of masking may matter in general when outcomes involve judgment or participant reporting, but the registry does not provide enough assessment detail to determine how this may have affected measurement.
The registry lists 16 outcome measures and 16 analyses. The analysis plan for handling multiple outcomes and assessment times is not reported in the registry record. Missing-data methods, analysis populations, model specifications, covariate adjustment, and sensitivity analyses are likewise not reported. The number of participants with available measurements at each assessment time is not reported in the registry record.
The safety account is limited to event counts. The record does not provide descriptions of the 5 other adverse events, details of their timing or severity, or additional information about event ascertainment and follow-up. The enrollment of 20 also limits how broadly the recorded safety experience can be generalized. These limitations do not establish that a particular bias or problem occurred; they define what cannot be evaluated from the registry record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Injection of Autologous Adipose-derived Stromal Vascular Fraction in the Finger of Systemic Sclerosis Patients — NCT03060551.
- Registry record last updated 2020-10-08; status last verified 2020-10.
- Results first posted 2020-10-08.
- 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
This completed early-phase 1 study examined autologous adipose-derived stromal vascular fraction injection in the fingers of patients with systemic sclerosis. The registry lists an enrollment of 20, a single-group design, allocation as not applicable, and no masking. The intervention involved processing lipoaspirates and injecting the resulting stromal vascular fraction into the subcutaneous tissue of each finger in contact with neurovascular pedicles.
The primary outcome was the Raynaud's Condition Scale, with a range of 0 to 10 and higher scores representing more severe Raynaud's phenomenon. Assessments were listed at baseline, 2 weeks, 6 weeks, 12 weeks, and 24 weeks, with the median and inter-quartile range specified as the summary. Numerical outcome results are not reported in the registry record. The registry lists 16 outcome measures and 16 analyses, including secondary nailfold capillary microscopy findings.
The adverse event record lists 0 serious adverse events, 5 other adverse events, and 0 deaths among 20 participants at risk. The registry describes efficacy assessment as preliminary and states that confirmation would require a randomized placebo-controlled trial on a larger population.