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 Effect of Increlex® on Children With Crohn Disease (NCT00764699), a na phase 2, phase 3 study. The primary outcome is The Primary Outcome Variable for the Monitoring Study (Baseline, Six Months and Disease Exacerbation) Will be Longitudinal Growth as Measured by Height Velocity.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00764699 |
| Phase | Phase 2, Phase 3 |
| Condition | Crohn Disease |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 3 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Nationwide Children's Hospital |
| Results posted | Yes |
2. Clinical Question
Population
Children with Crohn disease were the intended population. The registry description says participants would have had the diagnosis for at least one year. The record lists three enrolled participants, whereas the brief summary describes a plan to recruit 20.
Intervention
The intervention was treatment with recombinant IGF-1, identified as rhIGF (Increlex). The registry rationale proposes that replacing IGF-1 might address growth concerns associated with Crohn disease.
Comparator
No comparator group is described. The study is listed as a single-group study, with allocation marked NA. It therefore does not provide a like-for-like control comparison.
Primary question
The primary outcome was longitudinal growth measured by height velocity, with assessment planned at baseline, six months and disease exacerbation. The listed timeframe is six months and 1 year. The registry record says no analysis could be performed, so it does not answer whether height velocity changed during treatment.
The registry rationale frames the study around poor weight gain and short stature among patients with Crohn disease, and a hypothesis about abnormal IGF-1 generation and growth. It also describes earlier concerns about glucose intolerance with growth hormone in chronically ill children, particularly those receiving corticosteroids. Those statements explain the study’s motivation; they are not findings from this trial.
The primary question is descriptive as well as therapeutic: how would height velocity change over follow-up in children treated with rhIGF? Without a control group, even an observed change would not by itself separate treatment-associated change from other influences over time. In this record, no outcome values or analysis are reported, so neither the size nor the direction of any change can be assessed.
3. Trial Design
The registry classifies the study as a single-group study and lists no allocation method. Masking is listed as none. The intervention was rhIGF (Increlex), and the arm description is treatment with rhIGF. No untreated or active-comparator arm is described, so the study was not designed in the registry as a randomized comparison.
The record lists Phase 2 and Phase 3 and an enrollment of 3. The brief summary describes an intended recruitment of 20 patients from a pediatric gastroenterology practice. It also describes assessments at baseline and six-month intervals for one year while on treatment. The primary outcome listing names baseline, six months and disease exacerbation, with a timeframe of six months and 1 year. The record does not explain the difference between the intended recruitment and the recorded enrollment.
The study started in 2008-10, had a primary completion date of 2010-05, and is listed as terminated. The reason for termination is not reported in the registry record. The primary outcome description states that the principal investigator left the institution suddenly and participant records remained with the institution, so no analysis could be performed.
These design details matter for interpretation. A single group can describe outcomes among treated participants if measurements and analyses are available, but it cannot provide a direct comparison with a concurrent control group. Here, the registry record does not report analyzed growth measurements. The design and the absence of analysis therefore leave no treatment-effect estimate to interpret.
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 | rhIGF |
|---|---|
| STARTED | 3 |
| COMPLETED | 0 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | rhIGF |
|---|---|
| PI left and study terminated | 3 |
Analysis population for the primary outcome, as defined in the registry: The PI left the institution suddenly and participant records stayed with the institution, so no analysis was able to be performed.
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 | rhIGF |
|---|---|---|
| Participants analysed | 3 | |
| Age, Categorical: <=18 years | Participants | 3 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 12.67 (SD 2.52) |
| Sex: Female, Male: Female | Participants | 1 |
| Sex: Female, Male: Male | Participants | 2 |
| Region of Enrollment: United States | participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as The Primary Outcome Variable for the Monitoring Study (Baseline, Six Months and Disease Exacerbation) Will be Longitudinal Growth as Measured by Height Velocity, measured over Six months and 1 year.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was longitudinal growth measured by height velocity. Height velocity expresses growth over time, rather than a single height measurement. Repeated measurements can therefore describe the pace of linear growth during follow-up. The registry lists assessment at baseline, six months and disease exacerbation, and gives a timeframe of six months and 1 year.
In general, a longitudinal growth measure is relevant when the research question concerns whether growth changes during treatment. Tracking the measure over time can describe the pattern within participants, provided the measurements and their timing are available. The registry rationale links Crohn disease with concerns about poor growth and proposes that rhIGF replacement might improve growth. That is the study hypothesis, not a result.
The registry record supplies no height-velocity values, summary statistics, or analysis. It states that no analysis could be performed because the principal investigator left the institution and participant records stayed with the institution. As a result, the endpoint cannot be interpreted for magnitude, direction, variability, or precision. The record also does not report how height velocity was calculated or how measurements were handled when follow-up was incomplete.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central statistical finding is the absence of an analyzed primary outcome. Although the registry identifies height velocity as the primary endpoint and lists a follow-up timeframe, it reports that no analysis was performed. There is consequently no posted estimate of height-velocity change, no measure of variability, and no confidence interval or hypothesis-test result to interpret. The registry record does not establish whether the outcome changed in either direction.
The record lists three enrolled participants. This is a small recorded enrollment, but enrollment alone is not an outcome result. It cannot indicate how participants’ growth changed, whether measurements were complete, or how precisely an effect might have been estimated. The brief summary describes a plan to recruit 20 patients, but the record does not explain why enrollment was lower than that planned figure.
The study has no comparator arm: it is listed as single group, with allocation marked NA. Even if within-participant measurements had been analyzed, a change over time would not be a controlled contrast. In general, changes can reflect multiple influences, and a single-group design cannot directly distinguish a treatment-associated change from other changes during follow-up. In this record, the more basic limitation is that the measurements were not analyzed at all.
The terminated status does not itself explain why the study ended. The primary outcome description gives an administrative explanation for the lack of analysis: the principal investigator left the institution suddenly, and participant records remained there. The reason for termination, the status of follow-up, and the availability of individual measurements are not reported in the registry record.
Safety reporting is also limited. For the rhIGF group, the record lists three participants at risk and zero serious adverse events and zero other adverse events. Deaths are not reported in the registry record. These counts describe the posted safety entries; they do not resolve the absence of an analyzed growth outcome, and they should not be interpreted as proof that no harms occurred.
Any sample-size figures associated with planning are illustrations, not observed power and not evidence that the study was adequately powered. The stated illustration assumptions include a two-sided significance level of 0.05 and equal allocation between two arms. Those assumptions do not describe this single-group design. With no reported primary outcome analysis, there is no observed effect estimate in the record that can support a trial-specific conclusion about treatment effects.
Overall, the posted information documents the intended question, intervention, design, recorded enrollment, and reason no analysis was available. It does not provide evidence for estimating the effect of rhIGF on height velocity in this study. No efficacy conclusion, positive or negative, can be drawn from the absence of an analysis.
9. Statistical Methods Explained
The registry record identifies a primary outcome but reports no analysis. Statistical methods can clarify what an analysis might ordinarily communicate and why the information is unavailable, but they cannot recover results that are not reported. The general explanations below are not claims about analyses performed in this trial.
What would an outcome estimate describe?
In general, an estimate summarizes the observed outcome in a defined set of participants. For longitudinal growth, an analysis could describe measured height velocity over the stated follow-up or summarize changes between measurement times. The exact choice depends on the analysis plan and available data. The registry record does not report such an estimate, nor does it describe an analysis method for the endpoint.
An estimate is more informative when accompanied by a measure of uncertainty. A confidence interval gives a range of values compatible with the data under the assumptions of the method. A narrow interval indicates greater precision than a wide interval, although precision alone does not establish that a result is free from bias or that a treatment caused an observed change. No confidence interval is reported for the primary outcome.
What would a hypothesis test add?
In general, a hypothesis test evaluates how compatible the data are with a specified null hypothesis, given the model and assumptions used. A p-value is not the probability that the null hypothesis is true, and it does not measure the size or clinical importance of an effect. The registry record reports no primary outcome p-value or test statistic. It also does not report the trial’s planned testing procedure, so no specific hypothesis test can be attributed to the study.
The planning illustration describes a two-sided significance level of 0.05. That is an assumption for the illustration, not evidence that a trial analysis used this threshold. The illustration also assumes equal allocation between two arms, while the registry identifies the study as single group. The illustration’s assumptions therefore should not be mistaken for the trial’s design or statistical analysis plan.
How does the single-group design affect comparison?
In general, an outcome measured in one treated group can describe what happened among those participants if data are available. It does not directly estimate a contrast against what would have happened over the same period without the intervention or under another intervention. A randomized control group can help make such a contrast, subject to the trial’s conduct and analysis. No control group is listed for this study.
Repeated measurements within participants can be useful because they describe change over time, but the interpretation still depends on measurement timing, completeness, and the analysis approach. A comparison of measurements at different times is not automatically a causal treatment effect. The registry record does not report the participant-level measurements, the number with complete follow-up, or a model for repeated observations.
What can a planning illustration establish?
In general, a sample-size calculation is conditional on assumptions about the target effect, variability, statistical test, allocation, and desired power. Changing those assumptions can change the calculated sample size. Such calculations are planning aids; they are not outcomes and do not establish that a trial had a particular probability of success after it was conducted.
The registry’s illustration is described as based on a stated fraction of an observed effect, with normal approximation and equal allocation. It is explicitly a planning illustration rather than the trial’s own design. The assumptions do not match the listed single-group design. The registry record does not report a protocol-based sample-size justification, and the illustration should not be called observed power. No numerical illustration results are stated in the record described for this analysis.
Why do missing analysis details matter?
Statistical interpretation depends on more than the endpoint name. It also depends on which participants were included, how outcomes were measured, how missing observations were handled, and what analysis was planned. These details can affect both the estimate and its uncertainty. The registry record says no analysis could be performed because the principal investigator left the institution and participant records stayed there. It does not report an analysis population, a missing-data method, or a statistical model.
Because there are no outcome estimates, there is no basis for recalculating a result from posted summary statistics. Nor is there a sponsor analysis to compare with a recalculation. General statistical methods explain what evidence would usually be needed, but they cannot turn the reported enrollment or safety counts into an estimate of growth change.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary outcome measure, longitudinal growth measured by height velocity, and reports zero analyses. It does not report a statistical plan describing how that endpoint would be analyzed, which time point would govern the primary inference, or how measurements at baseline, six months, disease exacerbation, and the listed follow-up timeframe would be incorporated. The registry record does not describe secondary outcomes or a hierarchy of outcomes.
Multiplicity arises in general when several hypotheses or outcome comparisons are tested. Repeated testing can increase the chance of obtaining a statistically notable result by chance unless the analysis plan accounts for the testing structure. The record does not report whether multiplicity adjustments were planned. With no reported analyses, there are no reported p-values to assess for multiplicity.
Missing-data handling is also not reported. The registry statement that no analysis could be performed because participant records remained with the institution explains why results are absent, but it does not describe whether measurements were missing, how many participants completed follow-up, or what data were available. The record does not report an imputation method, a sensitivity analysis, or an approach to incomplete longitudinal measurements.
An analysis population is not specified in the posted outcome information. The record does not say whether an intended analysis would include all enrolled participants, only participants with follow-up measurements, or another group. It therefore does not support a claim about an intention-to-treat population or a per-protocol population. These omissions limit reproducibility and prevent assessment of how participant inclusion or missing data might have affected an estimate.
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 |
|---|---|---|---|
| rhIGF | 0 | 3 | 0.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| rhIGF | 0 of 3 |
The registry lists the rhIGF group with three participants at risk. It reports zero serious adverse events and zero other adverse events for that group. These are the posted event counts, not estimates of comparative risk: the study has no comparator group listed, and no risk comparison can be made from these figures.
The record lists deaths as null, so deaths are not reported in the registry record. A missing or unreported value is not the same as a reported count of zero. The record also provides no serious adverse-event terms, no event descriptions, and no details about how long participants were observed for safety reporting.
In general, a count of zero events in a small group does not establish that an event is impossible or that the intervention is free of risk. The amount of information depends on the size of the observed group, follow-up, event definitions, and completeness of reporting. The listed three participants at risk provide limited information, and the registry does not report further detail to assess safety ascertainment.
These safety entries should be read separately from the primary growth outcome. They do not supply evidence about height velocity, and they do not compensate for the absence of an outcome analysis.
12. What the Result Does, and Does Not, Mean
The record establishes that a study of rhIGF (Increlex) in children with Crohn disease was registered as a single-group study, listed three enrolled participants, and was terminated. It identifies longitudinal growth measured by height velocity as the primary outcome. It also states why no primary analysis was available: the principal investigator left the institution suddenly and participant records stayed with the institution.
Those details do not establish whether height velocity increased, decreased, or remained unchanged. No outcome values, treatment-effect estimate, precision measure, or hypothesis-test result are reported. The absence of a reported analysis is not a finding of no effect; it is an absence of evidence from the primary outcome in this registry record.
The intended recruitment of 20 described in the brief summary and the recorded enrollment of 3 are not interchangeable. The record does not explain the difference, and it does not report how many participants had measurements at each planned assessment. Consequently, the completeness of follow-up cannot be determined from the posted outcome information.
The lack of a comparator also limits what a within-group change, if it had been reported, could establish. In general, changes observed over time may have explanations other than treatment. A controlled comparison can address that question more directly, whereas this design is listed as single group. No causal treatment effect can be inferred from the design description alone.
The safety counts show zero serious and zero other adverse events among three participants at risk in the listed group, while deaths are not reported in the registry record. These counts should be interpreted narrowly and do not establish absence of risk.
In short, the registry record documents a research question and an incomplete evidentiary record, not an answer about the effect of rhIGF on growth. It supports neither a conclusion that treatment improved growth nor a conclusion that it did not.
13. Why This Trial Matters Statistically
This trial matters statistically because it illustrates the difference between a registered research question and an analyzable result. The registry identifies an endpoint and a follow-up framework, but it also states that no analysis could be performed. Without outcome measurements or estimates, readers cannot assess the direction, size, or precision of a result.
The recorded enrollment is 3, while the brief summary describes an intention to recruit 20. That contrast makes the distinction between planned recruitment and actual enrollment important. The record does not explain the difference, and it supplies no outcome analysis from which to judge the consequences. A planned sample size, an enrolled sample, and a completed analysis are distinct parts of the evidence chain.
The single-group design also makes the comparison question consequential. In general, repeated measurements can characterize change among treated participants, but they do not provide the same direct contrast as a control group. The registry’s planning illustration assumes equal allocation between two arms, whereas the trial is listed as single group. Keeping planning assumptions separate from the actual design helps prevent an illustration from being mistaken for the study’s statistical plan.
The administrative explanation for the missing analysis is also relevant to transparency. The record says the principal investigator left the institution and participant records remained there. It does not report how the records were handled afterward or whether an analysis was later completed. Stating what is absent, rather than filling the gap with assumptions, preserves the distinction between missing evidence and a null finding.
For biostatistical interpretation, the case emphasizes that an endpoint label, enrollment count, and safety count cannot substitute for outcome estimates. A treatment effect requires analyzed outcome data and a clear account of the comparison and analysis. Here, those elements are not reported, so the principal statistical conclusion is about the limits of what can be inferred.
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 primary limitation is that no analysis of the primary outcome was performed, according to the registry record. The record provides no height-velocity values, summary statistics, treatment-effect estimate, confidence interval, or hypothesis-test result. It is therefore impossible to evaluate the outcome’s magnitude, direction, or precision from the posted information.
The study is listed as single group, with allocation marked NA and masking listed as none. No comparator is described. This means the design does not provide a like-for-like control comparison, and any within-group change, if available, would require cautious interpretation. In this record, outcome results are unavailable in any case.
The registry lists three enrolled participants, while the brief summary describes intended recruitment of 20. The reason for this difference is not reported in the registry record. The record also does not say how many participants completed follow-up or had measurements at the stated assessment times. Completeness of longitudinal outcome data is therefore unknown.
The primary outcome description attributes the lack of analysis to the principal investigator leaving the institution suddenly while participant records stayed with the institution. The reason for termination is not reported. The record does not provide a statistical analysis plan, an analysis population, a missing-data approach, or details of measurement procedures.
Safety reporting is limited to the posted counts, with deaths not reported in the registry record. The record does not give event terms or additional follow-up detail. Finally, the planning illustration is not the trial’s own design, and its equal-allocation, two-arm assumptions do not match the listed single-group structure. It cannot remedy the lack of an outcome analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Effect of Increlex® on Children With Crohn Disease — NCT00764699.
- Registry record last updated 2018-03-02; status last verified 2018-02.
- Results first posted 2018-03-02.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
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18. Record Summary
The study, titled Effect of Increlex® on Children With Crohn Disease, is listed as Phase 2 and Phase 3 and sponsored by Nationwide Children's Hospital. It started in 2008-10, had a primary completion date of 2010-05, and has a terminated status. The registry lists three enrolled participants.
The design is single group, with no allocation method and no masking. The intervention was rhIGF (Increlex). The primary outcome was longitudinal growth measured by height velocity, with the listed timeframe of six months and 1 year. The brief summary describes intended recruitment of 20 and follow-up over one year.
The registry record states that no primary outcome analysis could be performed because the principal investigator left the institution and participant records remained there. It reports zero serious and zero other adverse events among three participants at risk; deaths are not reported in the registry record. No treatment-effect estimate for growth is available.