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 Trial of Growth Hormone Therapy in Pediatric Crohn's Disease (NCT00109473), a randomized phase 2 study. The primary outcome is Crohn's Disease Histologic Index of Severity (CDHIS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00109473 |
| Phase | Phase 2 |
| Condition | Crohn's Disease |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 22 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Children's Hospital Medical Center, Cincinnati |
| Results posted | Yes |
2. Clinical Question
Population
Children with Crohn’s disease were the intended population. The registry record lists enrollment of 22 participants but does not give further eligibility details in the record.
Intervention
The intervention arm received growth hormone alongside corticosteroid therapy. The arm description specifies Nutropin AQ at 0.075 mg/kg/day by daily subcutaneous administration.
Comparator
The comparison arm received corticosteroid therapy as prescribed by the referring gastroenterologist, without growth hormone. The registry record describes the study as randomized, open-label, and parallel-group.
Primary question
The primary outcome was the Crohn’s Disease Histologic Index of Severity (CDHIS), assessed at baseline and 12 weeks. The record’s stated purpose was to examine whether growth hormone affected intestinal healing relative to no growth hormone; however, it does not provide a single reduced comparison or numerical result for the primary outcome.
3. Trial Design
This was a phase II randomized, parallel-group trial in pediatric Crohn’s disease. The registry lists enrollment of 22 participants, no masking, and a completed status. The listed study start is 2005-04 and primary completion is 2009-07. These dates describe the registry record; they do not establish how long an individual participant was followed beyond the stated outcome time points.
The two principal arms compared growth hormone plus corticosteroid therapy with corticosteroid therapy alone. The growth hormone arm is described as Nutropin AQ, 0.075 mg/kg/day by daily subcutaneous administration. Corticosteroid therapy in the comparison arm was prescribed by the referring gastroenterologist. Because masking was listed as none, participants and study personnel were not described as masked in the registry record. Whether the pathologist who computed the histology score was masked to treatment assignment is not reported in the registry record.
The primary outcome was CDHIS at baseline and 12 weeks. The record also lists secondary outcomes relating to serum IGF-1 z score, IMPACT III, PCDAI, corticosteroid use, endoscopic severity, height velocity, and fecal calprotectin. It indicates eight outcome measures in total. The registry record does not give detailed eligibility criteria, the allocation procedure, the planned analysis model, or the analysis populations.
Random allocation can help make comparison groups similar on average, but it does not guarantee balance in a small trial. The open-label design is also relevant when outcomes involve clinical judgments or participant reports. These design features frame interpretation; they do not establish what the treatment groups’ results were.
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: 21 subjects enrolled, one withdrew consent prior to randomization.
Baseline (0 Through 12 Weeks)
| Stage | Growth Hormone Plus Corticosteroid (CTX) | Corticosteroid (CTX) |
|---|---|---|
| STARTED | 10 | 10 |
| Analyzed | 10 | 10 |
| COMPLETED | 10 | 9 |
| NOT COMPLETED | 0 | 1 |
Week 12 to Week 24
| Stage | Growth Hormone Plus Corticosteroid (CTX) | Corticosteroid (CTX) |
|---|---|---|
| STARTED | 10 | 9 |
| Analyzed | 9 | 9 |
| COMPLETED | 9 | 9 |
| NOT COMPLETED | 1 | 0 |
52 Week Extension Phase
| Stage | Growth Hormone Plus Corticosteroid (CTX) | Corticosteroid (CTX) |
|---|---|---|
| STARTED | 9 | 8 |
| Analyzed | 9 | 8 |
| COMPLETED | 5 | 3 |
| NOT COMPLETED | 4 | 5 |
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 | Growth Hormone Plus Corticosteroid (CTX) | Corticosteroid (CTX) | Total |
|---|---|---|---|---|
| Participants analysed | 10 | 10 | 20 | |
| Age, Customized: <=18 years | participants | 10 | 10 | 20 |
| Age, Continuous | years | 12 (SD 3) | 13 (SD 3) | 13 (SD 3) |
| Sex: Female, Male: Female | Participants | 2 | 2 | 4 |
| Sex: Female, Male: Male | Participants | 8 | 8 | 16 |
| Region of Enrollment: United States | participants | 10 | 10 | 20 |
6. Primary Endpoint
The registry lists the primary outcome as Crohn's Disease Histologic Index of Severity (CDHIS), measured over Baseline and 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | scores on a scale |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was the Crohn’s Disease Histologic Index of Severity, or CDHIS, assessed at baseline and 12 weeks. The index was developed and validated to assess histologic disease activity and has been used to assess mucosal healing in response to other therapies, as described in the registry record. A gastrointestinal pathologist computed the score.
The score contains eight items reflecting epithelial injury, mucosal inflammation, and extent of involvement. It ranges from 0 to 16; higher scores indicate worsening disease, and 0 is the lowest possible score. The record says that patients with moderate to severely active Crohn’s disease typically have scores from 6 to 12. This contextual range is not a result for the trial participants.
Histology is one way to characterize tissue-level disease activity, which is distinct from symptoms or endoscopic appearance. A measure that reflects several histologic features may help describe changes in tissue findings over time. General interpretation still depends on the observed scores, their uncertainty, and how the trial compared treatment groups. Although the registry specifies the outcome and its time points, it does not report a single reduced comparison for the primary outcome. The magnitude, direction, and precision of a treatment-group difference therefore cannot be determined from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The trial record establishes the design and the intended primary outcome, but does not provide a single reduced primary-outcome comparison. The registry lists CDHIS scores at baseline and 12 weeks, with a 95% confidence interval as the stated dispersion. It does not report a numerical treatment-group contrast, its confidence interval, or a p-value in a form that the record reduces to one comparison. The direction and magnitude of the primary result are therefore not reported in the registry record.
A confidence interval is a way to express statistical uncertainty around an estimate. In general, a narrower interval indicates greater precision, while a wider interval indicates less precision. A 95% confidence interval does not mean that there is a 95% probability that a particular fixed treatment effect lies inside the interval. It describes the long-run performance of the interval procedure under its assumptions. Without the estimate and interval bounds, the precision of this trial’s primary comparison cannot be assessed.
The registry lists enrollment of 22 participants. That enrollment alone does not establish how informative the results were: interpretation also depends on the number analyzed, the variability of scores, missing observations, and the size and precision of the treatment contrast. Those details are not reported in the registry record in a way that permits a single primary comparison to be interpreted.
The record lists seven secondary outcomes in addition to the primary measure, including clinical activity, endoscopic severity, height velocity, and fecal calprotectin. A collection of outcomes may provide different perspectives, but it also raises the general issue of multiplicity: when many comparisons are examined, the chance of at least one apparently notable result can increase. Whether the trial used any adjustment, hierarchy, or other multiplicity strategy is not reported in the registry record. No secondary-outcome estimates are included in the record summarized by these registry entries.
The safety listing reports serious adverse events affecting 1 participant in the growth hormone plus corticosteroid group and 0 in the corticosteroid group among 10 at risk in each group. The extension phase lists 2 affected among 17 at risk. These are descriptive counts, not a precise estimate of comparative risk. The registry also lists terms and percentages for serious events, but does not provide enough detail to establish their timing, overlap, or relationship to treatment. No deaths are reported as counts; the death fields are not populated in the record.
Overall, the registry supports a description of the study design and endpoint, but not a conclusion about the size or precision of the primary treatment contrast. A missing reduced comparison should not be treated as evidence of no effect, nor as evidence of benefit. The available information does not support either conclusion.
9. Statistical Methods Explained
The registry record identifies a randomized, parallel-group design and a quantitative primary outcome. It does not report a complete statistical analysis plan or a single reduced comparison for CDHIS. The explanations below distinguish general statistical principles from methods documented for this trial.
What does randomization contribute?
In general, randomization assigns participants to treatment groups using a chance-based process. It can reduce systematic differences between groups at the start of a study, on average, including differences that investigators may not have measured. It does not ensure exact balance in every trial, particularly when enrollment is limited. The registry lists 22 participants, but the allocation sequence, allocation concealment, and group sizes for the primary analysis are not reported in the registry record. Randomization therefore describes the design, not a guarantee that the groups were alike in every relevant respect.
What does the open-label design mean?
The record lists masking as none. In general, when treatment assignment is known, expectations or behavior can influence outcomes that depend on reporting or judgment. CDHIS is a histologic score computed by a gastrointestinal pathologist, which makes the pathologist’s assessment part of how the endpoint was obtained. Whether that pathologist was masked to treatment assignment is not reported in the registry record. The available description does not establish that assessments were biased, but it also does not let readers judge whether masking of the outcome assessor was used.
How should the confidence interval be understood?
The stated dispersion for the primary outcome is a 95% confidence interval. In general, a confidence interval communicates uncertainty around an estimated quantity, such as a group mean or treatment contrast. Its interpretation depends on what quantity the interval surrounds and on the method used to calculate it. The registry identifies the interval level but does not supply a reduced primary comparison with interval bounds in the record summarized by these registry entries. Consequently, neither the estimate nor its precision can be read from the outcome description alone. The model and calculation details used for any interval are not reported in the registry record.
What can be inferred from the endpoint scale?
CDHIS ranges from 0 to 16, with higher scores indicating worsening disease. In general, a scale’s direction is important because a larger value does not necessarily mean improvement. A score’s clinical meaning also depends on how score changes relate to disease status and on the comparison being made. The registry provides a typical score range for moderate to severely active disease, but does not report the trial participants’ scores or a treatment-group change. No change score or treatment contrast should be inferred from the endpoint definition.
What do the sample-size assumptions describe?
The sample-size planning illustration is described as using the observed effect, a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It is a planning illustration, not evidence of the trial’s original design justification. The registry’s own sample-size justification is not reported. Planning numbers based on an observed effect do not show the probability that this completed trial detected an effect, and they are not observed power. The assumptions and resulting illustration also cannot replace a confidence interval for the actual trial comparison.
Why can count outcomes need different methods?
The primary endpoint is described as a number on a bounded scale, while some secondary outcomes are scores and others concern counts or measurements. In general, an analysis method should reflect the outcome’s measurement properties, distribution, and study design. Count outcomes can show over-dispersion when variability exceeds what a simple Poisson model expects; in that situation, a model that allows greater variability may better represent the data. This is a general statistical point, not a claim about the trial’s data or analysis. The analysis model for CDHIS and the methods used for secondary outcomes are not reported in the registry record.
What analysis details remain unavailable?
The registry record does not report the primary analysis population, the handling of missing observations, covariate adjustment, assumptions checks, or the exact analysis model. It lists one analysis, but that count does not identify its specification or establish that no other analyses were conducted. Interim analyses and stopping rules are not reported in the registry record. These gaps limit reproducibility and make it important to distinguish a design description from a fully documented analysis.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary outcome and seven secondary outcomes. The secondary measures cover serum IGF-1 z score, IMPACT III, PCDAI, total corticosteroid use, CDEIS, height velocity, and fecal calprotectin. Looking at multiple outcomes can be useful because each may capture a different aspect of disease or treatment experience. As a general statistical matter, however, making many comparisons can increase the chance that at least one result appears notable by chance alone.
The registry record does not report whether the outcomes were arranged in a testing hierarchy, whether a multiplicity adjustment was planned, or how any secondary analyses were interpreted relative to the primary endpoint. The record indicates one analysis, but does not specify what that analysis involved. No multiplicity strategy should be assumed from the number of outcomes alone.
The record gives the primary endpoint time points as baseline and 12 weeks, but it does not report a single reduced comparison or the number of participants with usable observations at each time point. Missing-data procedures, reasons for missingness, and sensitivity analyses are not reported in the registry record. The analysis population—such as whether all randomized participants were analyzed in their assigned groups—is also not reported. These details matter because excluding participants or handling incomplete measurements in different ways can change the estimate and its uncertainty.
Interim analyses and stopping rules are not reported in the registry record. The absence of those details is not evidence that such procedures were or were not used. Without a fuller analysis description, readers cannot determine how multiplicity, incomplete outcome data, or analysis-population decisions were addressed.
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 |
|---|---|---|---|
| Growth Hormone Plus Corticosteroid (CTX) | 1 | 10 | 10.0% |
| Corticosteroid (CTX) | 0 | 10 | 0.0% |
| Extension Phase | 2 | 17 | 11.8% |
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 |
|---|---|
| Growth Hormone Plus Corticosteroid (CTX) | 10 of 10 |
| Corticosteroid (CTX) | 10 of 10 |
| Extension Phase | 17 of 17 |
Most frequent serious adverse event terms
| Event term | Organ system | Growth Hormone Plus Corticosteroid (CTX) | Corticosteroid (CTX) | Extension Phase |
|---|---|---|---|---|
| Bruising at injection site | Skin and subcutaneous tissue disorders | 1 of 10 | 0 of 10 | 0 of 17 |
| Abdominal Pain | General disorders | 0 of 10 | 0 of 10 | 1 of 17 |
| Pancreatitis | Hepatobiliary disorders | 0 of 10 | 0 of 10 | 1 of 17 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Growth Hormone Plus Corticosteroid (CTX) | Corticosteroid (CTX) | Extension Phase |
|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | 3 of 10 | 1 of 10 | 10 of 17 |
| Fever | General disorders | 2 of 10 | 2 of 10 | 8 of 17 |
| Bruising at injection site | Skin and subcutaneous tissue disorders | 4 of 10 | 2 of 10 | 5 of 17 |
| Vomiting | Gastrointestinal disorders | 2 of 10 | 3 of 10 | 6 of 17 |
| Cushingoid | Skin and subcutaneous tissue disorders | 6 of 10 | 2 of 10 | 2 of 17 |
| Nausea | Gastrointestinal disorders | 2 of 10 | 3 of 10 | 5 of 17 |
| Joint Pain | General disorders | 6 of 10 | 1 of 10 | 3 of 17 |
| Abdominal Pain | General disorders | 1 of 10 | 1 of 10 | 7 of 17 |
| Creatnine decreased | Metabolism and nutrition disorders | 4 of 10 | 1 of 10 | 3 of 17 |
| Headache | General disorders | 1 of 10 | 4 of 10 | 3 of 17 |
Serious events with the highest arm-level rate: bruising at injection site (10.0%), abdominal pain (5.9%), pancreatitis (5.9%).
The serious adverse event listing reports 10 participants at risk in each principal group. It records 1 participant affected in the growth hormone plus corticosteroid group and 0 affected in the corticosteroid group. The extension phase lists 17 at risk and 2 affected. These figures are counts within the listed groups; they should not be read as a direct comparison between the principal randomized groups and the extension phase.
The record lists bruising at injection site at 10.0%, abdominal pain at 5.9%, and pancreatitis at 5.9% among the top serious-event terms. It does not provide enough information in the registry record to establish how terms map to participants, whether participants experienced more than one term, or the timing and treatment attribution of each event. The percentages should therefore be read as registry-listed event-term figures, not as a complete account of event frequency or causality.
Serious adverse events are distinct from other adverse events. The record lists other adverse events affecting all 10 at-risk participants in each principal group and all 17 at-risk participants in the extension phase. Death counts are not populated in the record. These safety figures are descriptive and, with limited group counts and unavailable event detail, do not by themselves provide a precise comparative safety estimate.
12. What the Result Does, and Does Not, Mean
The record describes a randomized phase II comparison of growth hormone plus corticosteroid therapy against corticosteroid therapy alone in pediatric Crohn’s disease. It identifies a histologic primary outcome, CDHIS, and specifies assessment at baseline and 12 weeks. It also describes a score with a defined range and direction. These registry entries clarify what the study set out to measure, but they do not establish the result of the comparison.
The key limitation for interpreting efficacy is that the record does not reduce the primary outcome to a single comparison. It does not report an estimate of the difference between groups, the associated confidence interval bounds, or a p-value for a single primary contrast. Accordingly, the magnitude, direction, and statistical precision of the primary result are not reported in the registry record. This absence cannot be interpreted as showing that the groups were the same, and it cannot be interpreted as showing that one approach was better.
Likewise, the registry’s endpoint description is not itself an outcome. The score’s scale and meaning explain how a result could be interpreted if reported, but no trial score or score change can be inferred from those definitions. The enrollment figure also does not establish whether the study was adequately powered, because the planned effect, variability assumptions, analysis population, and original sample-size justification are not reported in the registry record.
The adverse-event counts provide a limited descriptive account. They identify affected participants in the listed groups, but do not establish precise comparative risks or treatment causation. The extension phase is a separate registry listing and should not be conflated with the randomized comparison. More complete event descriptions and analysis details would be needed to characterize safety fully.
The most defensible reading is therefore about the trial’s question and structure, rather than an unreported treatment effect. The registry documents a small, open-label randomized trial with a specified histologic endpoint and several secondary measures. It does not supply enough primary-outcome or analysis detail to determine how large or precise any treatment contrast was.
13. Why This Trial Matters Statistically
This trial is statistically notable for asking a question that spans different kinds of outcomes. Its primary measure is a histologic severity score, while the secondary list includes clinical activity, an endoscopic index, height velocity, corticosteroid use, and a biomarker. Such measures address different domains, so their interpretation depends on clearly defining a primary question and explaining how other outcomes relate to it.
The randomized design creates a basis for comparing assigned groups, but the open-label designation and limited enrollment make transparent reporting especially important. In general, randomization addresses some sources of confounding, while masking and consistent outcome assessment can help reduce other sources of bias. The record specifies that a gastrointestinal pathologist computed CDHIS but does not report whether the pathologist was masked. Reporting that detail would help readers assess the measurement process without assuming that the score was either biased or unbiased.
The endpoint itself illustrates why statistical reporting needs both a measure and an estimate. A bounded scale with a stated direction tells readers what higher or lower values mean, but a treatment comparison and its uncertainty are needed to assess the evidence. Here, the registry describes the CDHIS and its assessment times but does not provide a single reduced primary contrast. This distinction between a prespecified question and a reported result is central to interpreting trial records.
The record also makes multiplicity relevant: several secondary outcomes accompany the primary endpoint. Clear reporting of outcome hierarchy, analysis populations, missing-data handling, and multiplicity methods would help readers understand how to weigh findings across measures. Those analysis details are not reported in the registry record. Thus, the statistical importance of the trial lies not in a result that can be inferred, but in the need to connect design, endpoint definition, analysis, and transparent numerical reporting.
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 important limitation for interpretation is that the registry record does not report a single reduced primary-outcome comparison. Although CDHIS is specified at baseline and 12 weeks and its scale is described, the record does not give trial scores, a treatment contrast, or the bounds of a primary confidence interval. The primary result’s direction, size, and precision therefore cannot be assessed.
Enrollment is listed as 22, but the number analyzed for the primary outcome and the group sizes at each time point are not reported in the registry record. The record also does not give eligibility criteria, baseline group characteristics, missing-data procedures, analysis populations, or sensitivity analyses. These omissions limit assessment of who contributed data and how incomplete measurements might affect comparisons.
The study is listed as unmasked. The registry says a gastrointestinal pathologist computed the histologic score, but does not report whether the pathologist was masked to treatment assignment. The exact analysis model, covariate adjustment, and assumptions are not reported. Neither interim analyses nor stopping rules are described. The registry’s sample-size justification is also not reported, and the listed planning illustration is not a substitute for the original design rationale.
For safety, the record gives group-level counts and a short list of serious-event terms but not detailed timing, attribution, participant-level overlap, or event narratives. The extension phase is listed separately and does not provide a direct substitute for the randomized comparison. These limitations constrain conclusions about both effectiveness and safety; they do not establish that an unreported effect or event was absent.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Trial of Growth Hormone Therapy in Pediatric Crohn's Disease — NCT00109473.
- Registry record last updated 2020-10-30; status last verified 2020-10.
- Results first posted 2011-09-16.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry record describes NCT00109473, a completed phase II randomized, open-label, parallel-group trial of growth hormone therapy in pediatric Crohn’s disease. Enrollment was 22. The listed study start is 2005-04 and primary completion is 2009-07.
The main comparison was growth hormone plus corticosteroid therapy versus corticosteroid therapy alone. The primary outcome was CDHIS at baseline and 12 weeks, scored from 0 to 16 with higher scores indicating worsening disease. The record lists seven secondary outcomes and indicates eight outcome measures overall.
The registry does not report a single reduced comparison for the primary outcome, the primary estimate and confidence interval bounds, or detailed analysis methods. Serious adverse event counts are listed for the principal groups and an extension phase, but event details are limited. The record supports describing the trial’s design and intended measurements, not determining the magnitude or precision of its primary treatment contrast.