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 Fecal Microbiota Transplantation in Pediatric Patients (NCT02108821), a na phase 1 study. The primary outcome is Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02108821 |
| Phase | Phase 1 |
| Condition | Inflammatory Bowel Diseases (IBD), Crohn's Disease (CD), Ulcerative Colitis (UC) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 23 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Children's Mercy Hospital Kansas City |
| Results posted | Yes |
2. Clinical Question
Population
The registry summary describes children with inflammatory bowel disease (IBD), including Crohn's disease and ulcerative colitis, who are in a flare and not responding to first-line therapy. The record does not report the full eligibility criteria or a more detailed description of the analyzed population. Enrollment was 23.
Intervention
The intervention was fecal microbiota transplantation (FMT), also called fecal microbiome transplantation in the arm description. The registry says the transplant was to be performed at the time of EGD and colonoscopy. Further procedural details are not reported in the registry record.
Comparator
There was no separate control group: the registry describes a single-group study, with allocation listed as NA. Consequently, the posted record does not support a like-for-like comparison between FMT and another treatment or a control condition.
Primary question
The primary outcome asks for the number of adverse-event occurrences, classified by adverse-event term, at 6 months. It is a safety-focused count outcome. The registry also lists a secondary efficacy outcome using the Pediatric Ulcerative Colitis Activity Index (PUCAI) and the Pediatric Crohn Disease Activity Index (PCDAI). Results for those activity measures are not reported in the registry record.
This structure makes the central posted question descriptive: what adverse events were recorded after FMT in the study group during the stated follow-up period? It is not a randomized test of whether FMT changes IBD activity relative to another option. A single group can describe what occurred among its participants, but it cannot by itself separate effects of the intervention from the course of disease, other care, or other influences. The registry record does not report a comparative estimate for efficacy or safety.
3. Trial Design
The study is registered as Phase 1 and is described as a single-group trial. Allocation is listed as NA and masking as none. The registry therefore describes neither randomized assignment nor a masked comparison. Enrollment was 23, and the study status is completed. The record gives a start of 2014-03 and a primary completion date of 2016-09-30.
All participants were assigned to the fecal microbiota transplantation intervention group in the registry description; no separate comparator group is listed. The arm description says the transplant would be done at the time of EGD and colonoscopy. The brief summary frames the study as an investigation of efficacy and safety in patients with IBD who were in a flare and not responding to first-line therapy. The registry does not report the full protocol, the detailed eligibility criteria, or the exact follow-up procedures beyond the primary outcome’s 6 months timeframe.
The primary outcome is the number of adverse-event occurrences by adverse-event term at 6 months. The record also lists an efficacy outcome based on PUCAI and PCDAI. Although the registry identifies two outcome measures, it lists zero analyses. Detailed statistical methods, analysis populations, and model specifications are not reported in the registry record.
These design features matter when interpreting results. A single-group study can record adverse events and changes in disease-activity measures among participants, but without a concurrent comparison group it cannot establish how those observations differ from what would have occurred under another treatment or no intervention. The lack of masking can also matter for outcomes that involve clinical assessment or reporting, although the registry record does not say how those outcomes were assessed. Any planning illustrations are not the trial’s design: the record describes them using assumptions for equal allocation between two arms, which does not match this single-group design.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Overall Study
| Stage | Fecal Microbiome Transplantation-open Label |
|---|---|
| STARTED | 23 |
| COMPLETED | 23 |
| NOT COMPLETED | 0 |
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Fecal Microbiome Transplantation |
|---|---|---|
| Participants analysed | 23 | |
| Age, Categorical: <=18 years | Participants | 19 |
| Age, Categorical: Between 18 and 65 years | Participants | 4 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 10 (8 to 21) |
| Sex: Female, Male: Female | Participants | 11 |
| Sex: Female, Male: Male | Participants | 12 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 20 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | Participants | 23 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Occurrences of Adverse Events at 6 Months According to Adverse Event Term, measured over 6 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Events |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is the number of adverse-event occurrences at 6 months, grouped according to adverse-event term. It is a count outcome: its intended unit is events, rather than a change in an IBD activity score or a direct measure of benefit. Adverse-event monitoring is used to characterize safety experiences during a study, and organizing events by term can distinguish the kinds of events recorded.
A count of occurrences and a count of participants affected are not interchangeable. One participant may experience more than one event, and the same event term may occur more than once. The registry’s adverse-event summary separately reports how many participants were affected in serious and other adverse-event categories. Those participant counts do not provide the term-by-term occurrence counts named in the primary endpoint. The event counts by term are not reported in the registry record.
The specified timeframe is 6 months. A defined observation period helps make clear which events the endpoint is intended to capture, but interpretation also depends on how events were identified, recorded, and attributed. Those operational details are not reported in the registry record. The primary outcome is safety-focused; the efficacy outcome is listed separately and uses PUCAI and PCDAI. Results for that efficacy outcome are not reported in the registry record, so it cannot be used to characterize disease-activity changes.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted registry summary gives a limited descriptive account of adverse events in a single group. It records 0 participants affected by serious adverse events among 23 listed at risk for that category, 12 participants affected by other adverse events among 23 listed at risk for that category, and 0 deaths. These figures describe participant counts in the registry’s adverse-event summary. They are not the same as the primary endpoint’s event-occurrence counts by term, which are not reported in the registry record.
The distinction between people and events is important. A participant-level count indicates whether a person was affected within the category; an event-level count can include repeated occurrences in the same person. The registry does not provide enough detail to infer how many total events occurred, whether events recurred, or which specific terms account for the participant counts. The list of top serious adverse-event terms is empty, but this should not be treated as a complete term-by-term account of all events.
These are descriptive observations, not a treatment-effect estimate. Because there is no separate control group and no randomized comparison, the figures cannot show how the adverse-event experience compares with another treatment or with the expected experience of similar patients without FMT. The record also does not report an adjusted analysis, confidence intervals, or hypothesis tests. The precision of the posted participant counts therefore cannot be assessed using inferential results in the registry record.
The study’s stated secondary outcome concerns PUCAI and PCDAI, but no outcome results for those measures are reported in the registry record. The record consequently does not establish the direction, size, or precision of any change in disease activity. The summary’s stated aim of examining efficacy and safety should not be confused with results demonstrating either.
In general, confidence intervals describe a range of values compatible with data under a specified statistical model and assumptions. They can communicate uncertainty around an estimate; a point count alone does not do so. No confidence interval is reported for the adverse-event figures. In general, a comparison between groups is needed to estimate a difference in outcomes between those groups, while a single-group count describes only the observed group. Here, the registry reports no comparator and no comparative estimate.
The registry includes planning illustrations described as being based on an observed effect, with assumptions involving two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. Those are planning assumptions, not evidence that the trial used a two-arm design, and the study itself is listed as single-group. Such illustrations should not be interpreted as observed power or as an analysis of the reported safety outcomes. The sponsor’s own analysis would take precedence if the record contained a discrepancy, but no sponsor analysis is described in the registry record.
9. Statistical Methods Explained
The registry identifies a count outcome for the primary endpoint and describes a single-group study. It lists zero analyses. Statistical methods used to estimate or compare outcomes are not reported in the registry record. The concepts below explain how these kinds of data are generally interpreted; they do not assert that a particular model or inferential procedure was used in this study.
What does a count endpoint represent?
In general, a count endpoint records how many events occur within a defined observation period. This study’s primary endpoint is described as the number of adverse-event occurrences by term at 6 months. Count data differ from measurements such as a laboratory value or activity score: counts take nonnegative integer values and may be concentrated at small values, with some participants potentially having more events than others. A clear definition of what qualifies as an event, how repeated events are handled, and how events are assigned to terms is important for interpreting such data. Those details are not reported in the registry record.
Event counts also need to be distinguished from counts of affected participants. A participant may experience multiple events, and multiple participants may experience events with the same term. The registry’s adverse-event summary reports participant counts by broad category, whereas the primary endpoint is described in event units and by term. The available summary therefore cannot be treated as a substitute for event-level results.
Why can variation in counts matter?
In general, count models often begin with assumptions about how the average count relates to its variability. Over-dispersion means that the observed variation is greater than a model’s basic assumptions allow. If it is ignored, uncertainty estimates can be too small and statistical conclusions can be overly confident. Alternative models or robust uncertainty estimates can sometimes address over-dispersion, but the appropriate choice depends on the data and analysis plan. The registry record does not report a count model, a dispersion assessment, or any alternative method.
For a single-group study, descriptive summaries can report counts and the number of participants affected. Inferential methods may also quantify uncertainty under stated assumptions, but they do not create a comparison group where none exists. A confidence interval for a single-group rate or count would address uncertainty in that group’s estimated experience, not the difference between FMT and another treatment. No such interval is reported in the registry record.
What does the design allow analysts to estimate?
In general, randomized allocation supports a comparison by distributing both measured and unmeasured influences across groups in expectation. This registry instead describes one group, with allocation listed as NA and masking as none. Descriptive summaries can characterize what was recorded among that group. They cannot establish a causal contrast with a control condition. The registry does not report an adjusted model or a causal analysis.
The record lists an efficacy outcome based on PUCAI and PCDAI. In general, a change score compares a later measurement with an earlier one, while an endpoint score describes a value at a specified assessment. The registry entry does not provide the analysis definition, assessment schedule, or results for these activity measures. It is therefore not possible to determine from the registry record whether the planned analysis concerned change, a threshold, or another summary.
How should planning calculations be read?
In general, sample-size calculations depend on assumptions about the outcome, the effect worth detecting, variability, significance level, power, and allocation. Planning values describe a hypothetical design under those assumptions; they are not observed results and do not show the power achieved by a completed trial. The registry’s planning illustration is described as using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Its stated two-arm allocation assumption does not match the registered single-group design. The planning assumptions should not be read as a protocol specification or as evidence of a design decision. The registry does not report a trial-specific sample-size justification.
In general, an observed-effect-based illustration can be especially uncertain because the observed effect is itself an estimate and may not represent the effect in a future study. Planning for a fraction of an observed effect is a way to explore hypothetical scenarios; it is not an independent estimate of the effect and does not resolve the limitations of a single-group design. Any calculated participants-per-arm values are planning illustrations, not numbers of participants observed in this trial.
What inferential results are available?
The registry reports zero analyses and does not list p-values, confidence intervals, model estimates, or adjusted effects. Without these, the posted information is descriptive rather than a complete inferential analysis. In general, a p-value evaluates compatibility of data with a specified null model; it does not by itself measure the size or importance of an effect. No p-value is reported for the primary endpoint or the secondary outcome in the registry record.
For a count outcome, interpretation also depends on the observation window and on whether all participants had comparable opportunity for event recording. The primary timeframe is 6 months, but the registry does not report participant-level follow-up completeness, exposure time, or how incomplete observation was handled. Those omissions limit assessment of both the count summaries and any rate-based interpretation.
10. Multiplicity, Missing Data and Analysis Populations
The registry identifies two outcome measures: a primary adverse-event count and a secondary efficacy outcome using PUCAI and PCDAI. It reports zero analyses. A statistical testing plan, a hierarchy among outcomes, and any adjustment for multiple comparisons are not reported in the registry record.
In general, testing multiple outcomes or making multiple comparisons can increase the chance of a false-positive finding if each test is interpreted without considering the collection of tests. Prespecified hierarchies or multiplicity adjustments can address that issue, but the registry does not describe such procedures. Since no analyses or efficacy results are reported, there is no basis to reconstruct which outcomes were tested or how their results were interpreted.
Missing-data methods are also not reported in the registry record. The entry does not describe the number of participants with observed endpoint data, whether any participants had incomplete follow-up, how missing assessments were handled, or whether imputation was used. The registered enrollment of 23 should not be assumed to mean that every participant contributed complete data to every outcome.
Analysis populations are not described. The record does not specify whether results were to use all enrolled participants, participants who received the intervention, or another defined group. These omissions matter because denominators and analysis populations determine which observations contribute to an estimate. No assumptions about missingness, exclusions, or analysis-set membership can be made from the registry record.
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 |
|---|---|---|---|
| Fecal Microbiota Transplantation | 0 | 23 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Fecal Microbiota Transplantation | 0 of 23 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 4% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Fecal Microbiota Transplantation | 12 of 23 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Fecal Microbiota Transplantation |
|---|---|---|
| Abdominal Pain | Gastrointestinal disorders | 11 of 23 |
| Diarrhea | Gastrointestinal disorders | 5 of 23 |
| Flatulence and bloating | Gastrointestinal disorders | 5 of 23 |
| Emesis | Gastrointestinal disorders | 3 of 23 |
| Bloody stools | Gastrointestinal disorders | 2 of 23 |
| Nausea | Gastrointestinal disorders | 2 of 23 |
| Fever | General disorders | 1 of 23 |
The adverse-event summary reports 0 participants affected by serious adverse events among 23 listed at risk for that category. It reports 12 participants affected by other adverse events among 23 listed at risk for that category, and 0 deaths. These figures describe affected participants in the registry summary, not the number of individual event occurrences.
The primary outcome is defined as adverse-event occurrences by term at 6 months. The registry does not report those term-level occurrence counts. It also does not provide a detailed account of event severity beyond the serious and other categories, timing, recurrence, or attribution. The empty list of top serious adverse-event terms does not establish that all term-level safety information is available.
In general, a count of participants affected answers how many people had at least one event in a category, while an event count can include multiple events per person. Neither alone establishes whether an intervention caused an event. With no control group, these summaries describe what was recorded in the study group; they do not estimate excess risk relative to another condition. The registry record does not report confidence intervals or a comparative safety analysis.
12. What the Result Does, and Does Not, Mean
The registry record documents a completed, single-group Phase 1 study of fecal microbiota transplantation in pediatric patients with IBD. Its primary outcome was designed to count adverse-event occurrences by term at 6 months. The posted adverse-event summary reports participant counts for serious and other adverse events and deaths, while the term-specific event counts are not reported in the registry record.
These data can be read as a descriptive account of safety observations recorded in the group. They do not, on their own, show whether FMT caused an event, whether the event frequency differed from another treatment, or whether the risk would be similar in a different population. Those questions require additional evidence and an appropriate comparison. No control group or comparative estimate is listed.
The registry also lists an efficacy outcome based on PUCAI and PCDAI. Its results are not reported in the registry record, so the listed outcome should not be mistaken for evidence of improvement or lack of improvement. The record’s stated study aims are not results, and the absence of posted efficacy estimates does not establish an effect in either direction.
More broadly, a completed study status does not mean that every planned outcome or analysis is available in the registry record. Detailed methods, analysis populations, missing-data procedures, and inferential results are not reported. Any conclusions should therefore remain within the narrow scope of the descriptive figures and registered study design. Planning illustrations, where included, concern hypothetical assumptions and are not observed power or treatment-effect findings.
13. Why This Trial Matters Statistically
This trial illustrates why outcome definitions and study design need to be read together. Its primary endpoint is a count of adverse-event occurrences by term, while the registry’s safety summary reports numbers of participants affected in broad categories. Both are useful kinds of information, but they answer different questions. Treating them as interchangeable would obscure whether a participant experienced repeated events and whether the result concerns people or event occurrences.
The single-group structure is equally important. A descriptive safety account can identify what was recorded among participants, but the absence of a comparator limits what can be inferred about relative risk or causal effects. That limitation is not repaired by a count of events, a completed status, or a planning calculation. A comparative question requires a design and analysis that provide a meaningful comparison.
The record also separates a safety-focused primary outcome from an efficacy outcome based on pediatric disease-activity indices. Since the registry does not report results for the latter, the presence of a registered efficacy measure should not be conflated with an efficacy finding. This distinction helps readers separate what investigators intended to assess from what the posted results actually describe.
Finally, the record highlights the value of transparent analysis details. Counts are easier to interpret when event definitions, follow-up completeness, analysis populations, and uncertainty estimates are reported. Those details are not reported in the registry record. Recognizing that absence is part of sound statistical interpretation: it prevents descriptive observations from being overstated as comparative or causal evidence.
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 has important limits for interpretation of this specific trial. It describes a single group, with allocation listed as NA and masking as none, and does not list a control group. Therefore, the posted summary cannot provide a like-for-like comparison or isolate the intervention’s effect from other influences.
The primary endpoint is stated in terms of event occurrences by adverse-event term at 6 months, but term-level event counts are not reported in the registry record. The posted safety summary instead gives affected-participant counts for serious and other adverse events and deaths. Details about event definitions, recurrence, timing, attribution, and ascertainment are not reported.
The registry lists an efficacy outcome using PUCAI and PCDAI but does not report results for it. It also does not describe detailed statistical methods, analysis populations, missing-data handling, outcome assessment schedules, or confidence intervals. The full eligibility criteria and detailed procedural information are not reported in the registry record. These gaps limit assessment of who contributed outcome data, how complete follow-up was, and how precisely any outcome was estimated.
The planning assumptions described in the record use equal allocation between two arms, whereas the registered study is single-group. Those assumptions are not a description of the trial’s design, and the record does not report a trial-specific sample-size justification. Together, these limitations mean that the available summary supports only a narrow descriptive reading of the posted participant-level safety figures, not broad claims about comparative safety or efficacy.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Fecal Microbiota Transplantation in Pediatric Patients — NCT02108821.
- Registry record last updated 2021-02-04; status last verified 2021-02.
- Results first posted 2021-02-04.
- 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 is a completed Phase 1, single-group study of fecal microbiota transplantation in pediatric patients with relapsed IBD. Enrollment was 23; allocation is listed as NA and masking as none. The record gives a start of 2014-03 and a primary completion date of 2016-09-30.
The primary outcome is adverse-event occurrences by term at 6 months. The registry summary reports 0 participants affected by serious adverse events among 23 at risk, 12 affected by other adverse events among 23 at risk, and 0 deaths. Term-level event counts are not reported. A secondary efficacy outcome using PUCAI and PCDAI is listed, but its results are not reported in the registry record. The record lists zero analyses and does not report detailed statistical methods, missing-data handling, or analysis populations.