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 Treatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy (NCT00174187), a non-randomized phase 3 study. The primary outcome is Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00174187 |
| Phase | Phase 3 |
| Condition | Endocrine System Diseases |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 30 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Pfizer |
| Results posted | Yes |
2. Clinical Question
Population
Children with short stature secondary to long-term corticosteroid therapy. For the primary outcome, the registry defines the Full Analysis Set through Year 3 as participants with at least one post-baseline height measurement who received study drug for at least 1 year.
Intervention
Somatropin, identified in the study title as recombinant human growth hormone Genotonorm.
Comparator
No comparator arm is described. The study is listed as non-randomized and single-group, so it is not a like-for-like comparison of treatment with a control.
Primary question
What change from baseline in height standard deviation score according to chronological age was recorded at Year 3? The registry lists this outcome but does not report its numerical result.
The study also set out to assess linear growth, bone mineralisation, and body composition during long-term Genotonorm treatment. Its listed secondary outcomes include bone age, lean body mass, fat mass, changes in lean body mass and fat mass over time, lean body mass as a percentage of total weight, and lean body mass standard deviation score according to chronological age.
The clinical question is therefore about change over time during treatment, rather than a treatment effect estimated against a concurrent control group. Without a comparator, a change from baseline cannot by itself separate treatment-associated change from growth over time, changes in the underlying condition or corticosteroid therapy, or other influences. The registry record does not report a numerical primary outcome result, so the direction and size of the recorded change cannot be described.
3. Trial Design
The registry describes a Phase 3, non-randomized, unmasked, single-group study of somatropin. The listed enrollment is 30. Pfizer is the sponsor. The study title concerns children with short stature secondary to long-term corticosteroid therapy, and the listed condition category is endocrine system diseases.
The intervention is recorded as the drug somatropin; the study title identifies Genotonorm as recombinant human growth hormone. No separate comparator intervention or control arm is described. Because allocation is non-randomized and the model is single-group, the record does not describe a randomized treatment-versus-control comparison. The registry specifically cautions that the arms are not a like-for-like control comparison. No masking was used.
The record lists a study start of 2000-09 and a primary completion of 2011-09, and gives the status as terminated. The reason for termination and the date of termination are not reported in the registry record. The record does not state the planned follow-up schedule beyond outcome time points such as Year 3 and the adverse-event periods described as up to Year 3 and after Year 3.
The primary outcome is change from baseline in height standard deviation score according to chronological age at Year 3. The registry defines the Full Analysis Set for that outcome through Year 3 by requiring at least one post-baseline height measurement and at least 1 year of treatment. This definition describes eligibility for that analysis, not necessarily everyone enrolled. The registry record does not report the number contributing primary outcome data or the numerical primary result. It also does not report a detailed statistical analysis plan, a sample-size justification, or the reason the study was terminated.
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.
Period 1 (up to 3 Years)
| Stage | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|
| STARTED | 15 | 15 | 0 |
| COMPLETED | 15 | 11 | 0 |
| NOT COMPLETED | 0 | 4 | 0 |
Reasons for not completing this period:
| Reason | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|
| Serious adverse event | 0 | 1 | 0 |
| Non compliance | 0 | 1 | 0 |
| Withdrawal by Subject | 0 | 1 | 0 |
| Other | 0 | 1 | 0 |
Between Period 1 and Period 2
| Stage | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|
| STARTED | 0 | 0 | 26 |
| Consented | 0 | 0 | 24 |
| COMPLETED | 0 | 0 | 21 |
| NOT COMPLETED | 0 | 0 | 5 |
Reasons for not completing this period:
| Reason | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|
| Did not consent to continue treatment | 0 | 0 | 2 |
| Consented, not assigned to treatment | 0 | 0 | 3 |
Period 2 (After 3 Years)
| Stage | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|
| STARTED | 0 | 0 | 21 |
| COMPLETED | 0 | 0 | 13 |
| NOT COMPLETED | 0 | 0 | 8 |
Reasons for not completing this period:
| Reason | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|
| Lost to Follow-up | 0 | 0 | 2 |
| Withdrawal by Subject | 0 | 0 | 1 |
| Other | 0 | 0 | 5 |
Analysis population for the primary outcome, as defined in the registry: Full Analysis Set (FAS) up to Year 3: included all participants who had at least 1 post-baseline height measurement and were treated with the study drug for at least 1 year.
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 | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Total |
|---|---|---|---|---|
| Participants analysed | 15 | 15 | 30 | |
| Age Continuous | years | 11.14 (SD 3.32) | 11.96 (SD 3.80) | 11.55 (SD 3.53) |
| Sex: Female, Male: Female | Participants | 9 | 2 | 11 |
| Sex: Female, Male: Male | Participants | 6 | 13 | 19 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline in Height Standard Deviation Score According to Chronological Age (SDS/CA) at Year 3, measured over Baseline, Year 3.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Inter-Quartile Range |
| Unit | Standard Deviation Score (SDS) |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is change from baseline in height standard deviation score according to chronological age at Year 3. Height standard deviation score, abbreviated SDS in the outcome description, expresses height relative to age- and gender-appropriate population reference data. The registry says the score is obtained by subtracting the reference mean height from measured height and dividing by the standard deviation of that mean. A positive score means height is above the reference mean, and a negative score means it is below that mean.
This standardization is used to place height measurements in the context of chronological age and gender, rather than interpreting a raw height value without reference to expected values. It can make a change in relative standing over time easier to describe when children are growing. The registry specifies the median and inter-quartile range as the summary statistics for the endpoint, with baseline and Year 3 as the time points.
The registry record does not report the median change, the inter-quartile range, or another numerical result for this endpoint. It therefore does not establish from the posted results whether the score increased or decreased, how large any change was, or how precisely it was estimated. The endpoint definition explains what was intended to be measured; it is not itself evidence of an observed change.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central interpretive limitation is that the registry record does not report a numerical result for the primary endpoint. Although the endpoint is defined as change from baseline in height standard deviation score according to chronological age at Year 3, no median or inter-quartile range is included in the record. The direction, magnitude, and variability of the observed change therefore cannot be assessed from the posted results.
The design also limits what a within-group change could establish. This was a non-randomized, single-group study, with no comparator arm described. A change from baseline during follow-up would describe how measurements changed among participants meeting the analysis-population definition. It would not, on its own, identify how those measurements would have changed without somatropin. Growth over time, underlying illness, corticosteroid treatment, and other influences cannot be separated from treatment-associated change through a concurrent randomized comparison in this design.
The primary outcome uses a standardized height score based on age- and gender-appropriate reference values. That makes the outcome a relative measure rather than a raw height measurement. However, the registry does not report the numerical score change or the detailed method used to analyze repeated measurements. The outcome description states that the summary is a median with an inter-quartile range; it does not specify a statistical test, model, confidence interval, or adjustment for other variables. Those methods are not reported in the registry record.
The record lists 30 outcome measures and no analysis entries. This does not supply numerical findings for the primary endpoint or the secondary outcomes. The secondary outcome list indicates that bone age and measures of lean body mass and fat mass were included, but their results are not reported in the registry record. The stated goals concerning bone mineralisation and body composition should not be mistaken for evidence that a particular result was observed.
Safety information is reported separately for periods up to Year 3 and after Year 3, and for groups identified by juvenile idiopathic arthritis or nephrotic syndrome. Those event counts are descriptive entries in the registry. They do not provide a randomized comparative estimate, and the serious-event terms listed with percentages lack a stated denominator in the record. They should not be interpreted as evidence of a treatment effect or as directly comparable risks across the different groups and periods.
Overall, the record documents the study question, design, endpoint definition, and selected safety entries, but it does not contain the numerical primary outcome result. Statistical interpretation must remain limited to those reported features. No conclusion about the size or precision of change in height score can be drawn from an endpoint definition alone.
9. Statistical Methods Explained
Statistical interpretation begins with the distinction between a study design and an outcome estimate. The registry describes a non-randomized, single-group study with no masking. It does not describe a concurrent comparator. This design can record measurements during treatment and describe change from baseline, but does not create a randomized contrast that would separate treatment-associated change from other changes occurring over time.
What does the height standard deviation score represent?
The endpoint expresses measured height relative to an age- and gender-appropriate reference population. As described in the registry, the participant's measured height is compared with the reference mean and scaled by the standard deviation of that mean. The resulting score describes how far the measurement lies from the reference mean in standard deviation units. A positive score is above that mean, while a negative score is below it. This is a general explanation of standardization; it does not supply an observed score for this study.
A standardized score can help describe height in relation to reference values across chronological ages and genders. It is not the same as a raw height measurement, and a change in the score is not automatically the same as a change in centimeters. Interpretation depends on the reference data and the reported measurement. The registry identifies the reference approach but does not report the underlying numerical results.
Why report a median and inter-quartile range?
The registry specifies the median as the primary endpoint summary and the inter-quartile range as its measure of dispersion. In general, the median is the middle of an ordered set of observations. The inter-quartile range describes the spread across the central portion of those observations. These summaries can describe a distribution without assuming that it is symmetric or well represented by a mean and standard deviation. Their use does not reveal the actual distribution, and the registry record does not include the median or inter-quartile range for the primary endpoint.
The record identifies baseline and Year 3 as the endpoint time points and calls the endpoint a change from baseline. It does not explain whether the change was calculated for each participant and then summarized, whether a model was used, or how observations at intermediate times were handled. Those analytic details are not reported in the registry record. The stated summary statistics alone are not enough to infer the statistical test or estimation method.
What does the analysis-population definition tell readers?
For the primary outcome through Year 3, the registry defines the Full Analysis Set as including participants with at least one post-baseline height measurement who received study drug for at least 1 year. This definition indicates that having a post-baseline measurement and meeting the treatment-duration condition were necessary for inclusion in that stated analysis population. It does not give the number analyzed, explain how participants who did not meet those conditions were handled, or describe whether any other analysis populations were used.
Missing-data methods matter because participants may not contribute measurements at every scheduled time. In general, different approaches to incomplete follow-up can lead to different estimates, depending on why data are missing and how the analysis handles them. The registry record does not report the amount or pattern of missing primary outcome data, any imputation method, or sensitivity analyses. It is therefore not possible to evaluate how incomplete measurements may have affected the endpoint estimate.
Why does the design matter for inference?
Random allocation, when used, can support a balanced comparison by distributing measured and unmeasured characteristics between treatment groups on average. This study is listed as non-randomized and single-group, so that kind of comparison is not available. A baseline-to-follow-up description could reflect many influences besides the study drug. General statistical reasoning therefore distinguishes a within-group change from a causal treatment contrast. The record does not report a control-group estimate that would permit such a contrast.
The registry lists a Phase 3 study, but the phase label does not supply missing outcome estimates or analysis details. The statistical plan, including any prespecified model, hypothesis test, confidence-interval method, or interim analysis, is not reported in the registry record. These methods should not be inferred from the phase designation or from the type of summary statistic.
How should planning illustrations be understood?
Any planning illustrations associated with the posted material use assumptions that differ from the design described in the registry. The stated illustration assumptions involve two arms with equal allocation, a two-sided significance level of 0.05, a normal approximation, and an effect expressed as a fraction of the observed effect. The record describes the trial itself as single-group and non-randomized. Such illustrations are hypothetical planning exercises, not observed properties of this study and not observed power. The registry record does not report a trial-specific sample-size justification or the numerical results of those illustrations in the registry record for this explanation.
In general, over-dispersion means that observed variability is greater than a model assumes. When present, it can make standard errors and confidence intervals that rely on the narrower variance assumption too small, leading to overconfident inference. The registry does not report a variance model or indicate that over-dispersion was assessed for this endpoint, so this concept should not be taken as a description of the analysis performed in this study.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 30 outcome measures, including the primary height standard deviation score outcome and secondary measures concerning bone age, lean body mass, and fat mass. Considering multiple outcomes raises a general statistical issue: when many hypotheses are tested, the chance of at least one apparently notable result can increase unless the analysis addresses multiplicity. The registry record does not report a multiplicity adjustment, a hierarchy for testing outcomes, or which outcomes were treated as confirmatory. No adjustment strategy should be inferred from the outcome list.
The registry reports no analysis entries, and numerical results for the primary and secondary outcomes are not reported in the registry record. The number of listed outcomes does not tell readers how many were analyzed, how any analyses were conducted, or whether results were omitted. The record does not report a statistical analysis plan specifying outcome-specific models, tests, confidence intervals, covariate adjustment, or handling of repeated observations.
For the primary outcome through Year 3, the Full Analysis Set is defined as participants with at least one post-baseline height measurement who received study drug for at least 1 year. That definition does not describe how missing measurements were handled, whether participants with partial follow-up were retained in a particular analysis, or whether sensitivity analyses were conducted. The amount and pattern of missing data are not reported in the registry record. It is also not reported whether the analysis used imputation, a complete-case approach, or another method.
These gaps affect interpretation of both precision and potential bias. A summary among participants meeting the stated analysis definition may not represent all enrolled participants if measurements or treatment duration differ. The registry record does not provide enough information to assess that possibility or to determine whether missingness could have influenced the results.
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 |
|---|---|---|---|
| Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | 13 | 15 | 86.7% |
| Somatropin- Up To Year 3 (Nephrotic Syndrome) | 14 | 15 | 93.3% |
| Somatropin- After Year 3 | 19 | 21 | 90.5% |
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 |
|---|---|
| Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | 13 of 15 |
| Somatropin- Up To Year 3 (Nephrotic Syndrome) | 14 of 15 |
| Somatropin- After Year 3 | 21 of 21 |
Most frequent serious adverse event terms
| Event term | Organ system | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|---|
| Nephrotic syndrome | Renal and urinary disorders | 0 of 15 | 9 of 15 | 4 of 21 |
| Juvenile arthritis | Musculoskeletal and connective tissue disorders | 2 of 15 | 0 of 15 | 6 of 21 |
| Abdominal pain | Gastrointestinal disorders | 2 of 15 | 1 of 15 | 4 of 21 |
| Condition aggravated | General disorders | 3 of 15 | 2 of 15 | 0 of 21 |
| Diabetes mellitus | Metabolism and nutrition disorders | 1 of 15 | 2 of 15 | 2 of 21 |
| Gastroenteritis | Infections and infestations | 2 of 15 | 1 of 15 | 2 of 21 |
| Rheumatoid arthritis | Musculoskeletal and connective tissue disorders | 3 of 15 | 0 of 15 | 1 of 21 |
| Biopsy kidney | Investigations | 0 of 15 | 1 of 15 | 2 of 21 |
| Osteonecrosis | Musculoskeletal and connective tissue disorders | 1 of 15 | 0 of 15 | 2 of 21 |
| Ovarian cyst | Reproductive system and breast disorders | 0 of 15 | 1 of 15 | 2 of 21 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Somatropin- Up To Year 3 (Juvenile Idiopathic Arthritis) | Somatropin- Up To Year 3 (Nephrotic Syndrome) | Somatropin- After Year 3 |
|---|---|---|---|---|
| Nephrotic syndrome | Renal and urinary disorders | 0 of 15 | 11 of 15 | 7 of 21 |
| Red blood cell sedimentation rate increased | Investigations | 5 of 15 | 0 of 15 | 4 of 21 |
| Proteinuria | Renal and urinary disorders | 0 of 15 | 6 of 15 | 3 of 21 |
| Joint effusion | Musculoskeletal and connective tissue disorders | 4 of 15 | 0 of 15 | 4 of 21 |
| Juvenile arthritis | Musculoskeletal and connective tissue disorders | 3 of 15 | 0 of 15 | 5 of 21 |
| Headache | Nervous system disorders | 2 of 15 | 3 of 15 | 3 of 21 |
| Abdominal pain | Gastrointestinal disorders | 2 of 15 | 1 of 15 | 3 of 21 |
| Insulin-like growth factor increased | Investigations | 0 of 15 | 3 of 15 | 3 of 21 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 1 of 15 | 1 of 15 | 4 of 21 |
| Nausea | Gastrointestinal disorders | 2 of 15 | 1 of 15 | 2 of 21 |
Serious events with the highest arm-level rate: nephrotic syndrome (60.0%), juvenile arthritis (28.6%), condition aggravated (20.0%), rheumatoid arthritis (20.0%), abdominal pain (19.0%).
The registry reports serious adverse-event entries for somatropin during periods up to Year 3 and after Year 3. Up to Year 3, the juvenile idiopathic arthritis group lists 15 participants at risk and 13 affected; the nephrotic syndrome group lists 15 at risk and 14 affected. After Year 3, the record lists 21 at risk and 19 affected. These are descriptive counts for the registry's named groups and periods, not a randomized comparison. The record does not explain whether participants or events could appear across periods, so the entries should not be added together.
The record also lists serious-event terms with percentages: nephrotic syndrome, 60.0%; juvenile arthritis, 28.6%; condition aggravated, 20.0%; rheumatoid arthritis, 20.0%; abdominal pain, 19.0%; and hip arthroplasty, 14.3%. A denominator and further details about these term percentages are not reported in the registry record. They should not be treated as rates directly comparable across terms or groups. Death entries are null, so death counts are not reported in the registry record.
These entries describe reported safety events; they do not establish that somatropin caused them. The underlying conditions named in the groups are relevant context, and there is no control group for comparison. The record does not report event-level timing, severity details beyond the serious-event category, or an adjusted safety analysis.
12. What the Result Does, and Does Not, Mean
The record identifies a study of somatropin in children with short stature secondary to long-term corticosteroid therapy and specifies a primary outcome focused on change in height standard deviation score by Year 3. It also describes secondary outcomes involving bone age and body composition, and includes selected serious adverse-event entries. These elements show what the study set out to assess and what kinds of information the registry records.
The record does not report a numerical result for the primary endpoint. It therefore cannot establish the size, direction, or precision of any change in height score. The stated median and inter-quartile range are the planned or recorded summary format, not actual results. Secondary outcome results are also not reported in the registry record, so their direction and magnitude cannot be assessed either.
Even if a within-group change were available, this design would not provide a randomized comparison against a control group. Change during treatment is not, by itself, an estimate of what would have happened in the absence of treatment. Growth over time, corticosteroid exposure, underlying disease, and other factors may influence measurements. The registry record does not provide a comparison that would distinguish these influences.
The safety entries should likewise be read descriptively. They list serious events in named groups and periods but do not provide a control-group comparison or establish causation. The percentages attached to serious-event terms are not accompanied by a stated denominator in the record.
Accordingly, the posted information supports a description of the trial design, endpoint definition, analysis-population criteria, and selected safety entries. It does not support a conclusion about the magnitude or precision of the primary outcome, a causal treatment effect, or a comparative safety effect. Those conclusions require results and analytic details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial illustrates why a clearly defined clinical endpoint and a reported numerical estimate are both needed for statistical interpretation. The registry gives a detailed definition of height standard deviation score, including its reference to chronological age and gender, and specifies a median with an inter-quartile range. Yet without the numerical summary, readers cannot assess the observed change or its variability. A precise endpoint definition does not substitute for an outcome estimate.
It also illustrates the difference between documenting change and estimating a treatment contrast. A single-group study can describe measurements over time among participants meeting the analysis definition. Without randomization and a comparator, however, those changes cannot isolate the contribution of the intervention from other influences. This distinction is important when interpreting studies in which natural development and concurrent therapies may also affect outcomes.
The registry's Full Analysis Set definition is another important statistical detail. Requiring a post-baseline height measurement and at least 1 year of treatment clarifies who qualified for the stated analysis, but it does not show how many participants contributed data or how missing measurements were handled. A population definition and a missing-data method answer different questions; the registry record provides only the former.
Finally, the listed secondary outcomes and safety information point to a broad assessment, but the record does not provide outcome results or a multiplicity strategy. Readers need to distinguish the scope of outcomes from evidence about those outcomes. The record is informative about design and measurement, while leaving key elements needed for statistical conclusions unreported.
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 for interpretation is the absence of numerical outcome results in the registry record. The primary endpoint is defined, but its median change and inter-quartile range are not reported. Results for the listed secondary outcomes are also not reported. Consequently, the size, direction, and precision of changes cannot be evaluated from the posted results.
The non-randomized, single-group design provides no like-for-like control comparison. Any within-group change would be difficult to attribute specifically to somatropin because the record does not describe a concurrent control group. The study is also unmasked, which can matter for outcomes involving measurement or assessment; the registry does not report additional safeguards or blinded outcome assessment.
The primary analysis population requires a post-baseline height measurement and at least 1 year of treatment. The number meeting that definition is not reported in the registry record. The record also does not describe the amount or pattern of missing data, reasons for missingness, imputation, sensitivity analyses, or how participants outside that definition were treated.
The statistical plan is not reported in the registry record. In particular, the record does not specify a hypothesis test or model for the primary outcome, confidence-interval procedures, covariate adjustment, an interim-analysis plan, or multiplicity handling. The reason for termination and the date of termination are not reported. These omissions limit assessment of precision, potential bias, and the extent to which the posted outcomes reflect prespecified analyses.
Safety interpretation is also limited by the descriptive nature of the entries, the lack of a comparator, and the absence of a stated denominator for serious-event term percentages. The registry does not provide enough detail to establish causality or compare event risks across the different groups and periods.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Treatment With Recombinant Human Growth Hormone (GH) in Children With Short Stature Secondary to a Long Term Corticoid Therapy — NCT00174187.
- Registry record last updated 2012-12-04; status last verified 2012-11.
- Results first posted 2012-12-04.
- 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
The registry record describes a Phase 3, non-randomized, unmasked, single-group study of somatropin in children with short stature secondary to long-term corticosteroid therapy. The listed enrollment is 30, the sponsor is Pfizer, and the study status is terminated. The record lists a start of 2000-09 and a primary completion of 2011-09.
The primary outcome is change from baseline in height standard deviation score according to chronological age at Year 3, summarized by a median and inter-quartile range. The Full Analysis Set definition requires at least one post-baseline height measurement and at least 1 year of treatment. The numerical primary outcome result and secondary outcome results are not reported in the registry record. Serious adverse-event entries are descriptive and do not constitute a randomized comparison.