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 Efficacy and Safety Study of Pamoate of Triptorelin in Children With Precocious Puberty (NCT00564850), a na phase 3 study. The primary outcome is Number of Participants With a GnRH-stimulated LH Level ≤3 IU/L.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00564850 |
| Phase | Phase 3 |
| Condition | Precocious Puberty |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 37 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Ipsen |
| Results posted | Yes |
2. Clinical Question
Population
Children with precocious puberty: girls younger than 9 years and boys younger than 10 years, as described in the study summary. The record does not provide further eligibility details or a breakdown of enrolled participants by age or sex.
Intervention
Triptorelin pamoate 11.25 mg, identified in the registry as Decapeptyl® SR. The primary assessment was scheduled 3 months after the first injection.
Comparator
No comparison group is described. The study is registered as non-comparative, open-label, and single-group, so the record does not set out a concurrent control treatment or placebo comparison.
Primary question
At the scheduled assessment, how many participants had a GnRH-stimulated LH level at or below 3 IU/L? The registry describes this as the main measure for assessing suppression of the LH response after treatment. It does not report the observed count or proportion.
This question is narrower than whether the treatment changes the course of puberty or produces a particular clinical outcome. The registered primary endpoint is a laboratory response to a stimulation test, measured at a specified follow-up time. The record also lists other hormone measurements and pubertal-stage assessment as secondary outcomes, but their numerical results are not reported in the registry record.
Because the design has a single group, the primary question is descriptive: what response was observed among the participants assessed under the study’s endpoint definition? Without a comparator, the record cannot estimate a treatment-versus-control difference. And without the posted primary outcome result, it is not possible to describe the observed response rate or its precision.
3. Trial Design
The registry identifies NCT00564850 as a completed Phase III study of triptorelin pamoate 11.25 mg in children with precocious puberty. Its official title describes it as multicentre, non-comparative, open, and single-stage. The study summary focuses on girls younger than 9 years and boys younger than 10 years. The registry lists an enrollment of 37.
The study used a single-group design and lists no allocation to different treatment groups. Masking is recorded as none. The intervention was triptorelin pamoate 11.25 mg, also identified as Decapeptyl® SR. The primary assessment was scheduled 3 months after the first injection. The registry gives a study start of 2007-10 and primary completion of 2010-10, and lists the status as completed.
These design features matter when reading the endpoint. An open, single-group study can describe the measurements recorded after treatment, but it does not make a concurrent comparison with an untreated or differently treated group. Changes over time, if reported, would not by themselves distinguish treatment-related change from other possible explanations. In this record, the numerical primary outcome is not reported, so even the descriptive response cannot be assessed.
The record lists 15 outcome measures, including the primary LH response and secondary measures involving stimulated and basal hormone levels, girls’ oestradiol and inhibin B levels, testosterone, and change in pubertal stage using the Tanner method. The record does not report a detailed protocol, a full statistical analysis plan, an explanation of the sample-size choice, or details of any interim analyses. Those matters should not be inferred from the study title or its completed status.
The sponsor is Ipsen. The registered design and enrollment describe the study’s structure, not its outcome. Completion indicates a registry status; it does not substitute for a reported analysis or establish what the participants’ endpoint measurements 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.
Overall Study
| Stage | Triptorelin Pamoate 11.25 mg |
|---|---|
| STARTED | 37 |
| COMPLETED | 35 |
| NOT COMPLETED | 2 |
Reasons for not completing this period:
| Reason | Triptorelin Pamoate 11.25 mg |
|---|---|
| Protocol Violation | 2 |
Analysis population for the primary outcome, as defined in the registry: Analyses performed on: * Intention to Treat (ITT): all patients having received ≥1 injection. Any subject with missing data is considered a non-responder. * Modified ITT (mITT): all ITT patients with ≥ Month 3 post-baseline assessment of primary efficacy criterion. * Per Protocol (PP): all mITT patients without major protocol deviations.
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 | Triptorelin Pamoate 11.25 mg |
|---|---|---|
| Participants analysed | 37 | |
| Age, Categorical: <=18 years | Participants | 37 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 0 |
| Age, Continuous | years | 8.2 (SD 1.1) |
| Sex: Female, Male: Female | Participants | 36 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: France | participants | 37 |
| Weight | Kg | 32.76 (SD 7.36) |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With a GnRH-stimulated LH Level ≤3 IU/L, measured over 3 months after the first injection of triptorelin pamoate 11.25 mg.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants whose GnRH-stimulated LH level was at or below 3 IU/L, assessed 3 months after the first injection of triptorelin pamoate 11.25 mg. In plain terms, the endpoint classifies each assessed participant according to whether the LH response to a GnRH test meets the registered threshold. The study summary describes the intended use of this measure as assessing suppression of the LH response.
A binary endpoint such as this is useful because it gives a defined rule for classifying a participant’s result. The count can be expressed as a proportion when the number assessed is known, and that proportion can be accompanied by an uncertainty interval. The threshold and timing make the endpoint specific to this study; they do not, by themselves, show the size or precision of the observed response.
Interpretation depends on knowing how many participants were assessed and how many met the threshold, along with the analysis population used. The record describes intended population definitions, including how missing primary data are handled in the ITT population, but it does not report the primary count, proportion, or a confidence interval. Thus, the endpoint is defined, while its observed result cannot be evaluated from the registry record.
The endpoint is a laboratory response to a stimulation test, not a direct measure of every aspect of puberty or a comparison of treatment strategies. Its meaning should be kept within that scope. The registry also lists other hormone and pubertal-stage outcomes, but those do not replace the registered primary endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record gives the study question and endpoint definition but not the numerical primary result. It therefore does not establish how many participants met the LH threshold, what proportion met it, or how uncertain that estimate would be. No effect estimate, confidence interval, or primary-outcome analysis is reported in the registry record. The completed status does not fill this gap.
The primary outcome is a participant count tied to a binary threshold: an LH response at or below 3 IU/L after GnRH stimulation, at the assessment scheduled 3 months after the first injection. To interpret a response proportion, a reader would need both the number meeting the threshold and the relevant analysis denominator. Neither is reported for the primary endpoint. The enrollment of 37 is not a substitute for the endpoint denominator, since the record describes analysis populations and an assessment requirement that may differ from enrollment.
Even if the numerical response were available, the single-group design would limit the question it could answer. It could describe the recorded response among participants in the study under the registered definition. It could not directly estimate how that response differs from a concurrent comparison group, because no such group is described. The open-label design also means masking is not part of the registered study structure. These features concern interpretation of the design; they do not supply missing outcome results.
The registry lists secondary outcomes, including additional LH and FSH measurements, oestradiol and inhibin B measures in girls, testosterone, and pubertal-stage change. It reports no numerical results for these outcomes in the record at hand. Nor does it identify which analyses, if any, were considered primary within each secondary measure. The registry lists no analysis entries.
Uncertainty cannot be judged from a point estimate alone, and no point estimate is reported. In general, a confidence interval describes the range of values compatible with the observed data under a stated method; it is not a guarantee about the true value. For a binary response, its width is influenced by the number assessed and the observed proportion. Since the primary count, denominator, and interval are absent, precision cannot be characterized for this study’s main endpoint.
There is also no basis in the registry record for attributing a hypothetical response to treatment relative to what would have happened without treatment. A within-group endpoint may be clinically or scientifically informative, but it is not a randomized treatment contrast. The most defensible reading is therefore limited: the registry describes a completed study and a prespecified-looking endpoint, while the posted information does not contain the numerical efficacy result needed to characterize the observed response.
9. Statistical Methods Explained
The registry defines the main outcome as a count: participants whose GnRH-stimulated LH level was at or below 3 IU/L at the assessment 3 months after the first injection. This is a binary classification at the participant level. A value either meets the stated threshold or does not. The endpoint is therefore different from an average change in LH, and it should not be interpreted as one.
What does a count-based endpoint tell the reader?
A count describes how many assessed participants meet the outcome definition. If the number assessed is also known, the count can be expressed as a proportion. The proportion communicates the observed frequency in the analysis population, while the count keeps visible the scale of the data. Neither alone describes the precision of the estimate. A confidence interval can convey sampling uncertainty around a proportion, with its interpretation depending on the statistical method and assumptions used.
No primary count, proportion, confidence interval, or test result is reported in the registry record. The endpoint is thus defined, but the data needed to quantify the result are absent. The enrollment of 37 should not be read as the endpoint denominator: the registry describes populations based on injection and assessment status, and does not give the number included in each population.
Why do analysis populations matter?
The registry describes an ITT population as patients who received at least one injection, with a participant missing primary endpoint data counted as a non-responder. It describes a modified ITT population as ITT patients with a Month 3 post-baseline assessment of the primary efficacy criterion. The per-protocol description is incomplete in the registry record, so its full definition is not reported there. These populations can lead to different denominators and different endpoint counts. No population-specific counts or results are reported.
Counting missing endpoint data as non-response is one explicit rule for the stated ITT definition. As a general statistical matter, such a rule makes missingness consequential: the assigned outcome is determined by the analysis convention rather than by an observed test result. It may be useful to examine whether conclusions are sensitive to alternative reasonable assumptions about missing outcomes, but no such sensitivity analysis is reported in the registry record. The record also does not give the extent or reasons for missing primary endpoint data.
What can a single-group design estimate?
A single-group study can estimate an outcome frequency in the group assessed, if the results and denominator are reported. It has no concurrent control group in this registry description, so it cannot produce a randomized between-group contrast. General explanations for changes over time or differences from an external expectation cannot be ruled out by the design alone. This is a design limitation, not a calculation that can be resolved by using a different statistical test.
The open-label designation means the record reports no masking. The registry does not describe an allocation mechanism, consistent with its non-comparative, single-group design. The absence of a comparison is especially important when interpreting secondary outcomes collected at different times: a change from baseline would describe change within the observed group, not an estimated difference from a control condition.
How should the planning calculations be understood?
The stated planning framework is an illustration based on an observed effect, not the trial’s own design justification. It assumes a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the target effect defined as a stated fraction of the observed effect. Those assumptions do not describe the registered single-group study. The registry record does not report the protocol’s sample-size justification or confirm that these planning assumptions were used by the investigators.
Planning sample sizes are not observed power. They are hypothetical calculations about the design of a future study under specified assumptions. The illustration’s two-arm framework should not be mistaken for evidence that this trial randomized participants or included a comparator. No planning output values are reported in the registry record, and no observed primary effect is available there to interpret.
In general, over-dispersion means that variability is greater than a model’s assumed variability. When present, an analysis that ignores it can understate uncertainty. That general issue does not establish that over-dispersion occurred in this study: the primary endpoint is a binary participant count, and the registry does not report a model, diagnostic assessment, or adjustment for over-dispersion.
What statistical plan is reported?
The registry record does not report a full statistical analysis plan, the interval method for the primary proportion, a hypothesis test, covariate adjustment, or interim-analysis procedures. It also lists no analysis entries. These omissions prevent a reader from determining which statistical procedures were applied to the primary endpoint or how any multiplicity was handled. No procedure should be inferred from the Phase III designation or the endpoint format alone.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 15 outcome measures, including the primary LH response and multiple secondary hormone and pubertal-stage measures. When a study examines multiple outcomes or multiple analyses, the chance of at least one apparently noteworthy result can increase. General approaches to this issue include identifying a single primary outcome in advance, adjusting inferential procedures, or treating secondary findings as exploratory. The registry record does not report a multiplicity strategy, hierarchy among secondary outcomes, or adjustment method.
The record describes an ITT population consisting of patients who received at least one injection. For that population, missing primary endpoint data are considered non-response. It also describes a modified ITT population restricted to ITT participants with a Month 3 post-baseline assessment of the primary criterion. These rules clarify how the populations are intended to differ, but the record does not give their sizes or the number of missing assessments.
The per-protocol population description is cut off in the registry record. Its complete definition is not reported, and no per-protocol denominator or outcome is listed. The record also does not provide a missing-data sensitivity analysis, reasons for missing observations, or an account of how participants without follow-up assessments were handled outside the stated ITT rule.
The registry lists no analysis entries, and numerical results for the primary or secondary outcomes are not reported in the registry record. Details of interim analyses and a complete statistical plan are also not reported in the registry record. Consequently, readers cannot assess how many analyses were conducted, whether the stated populations were used as intended, or how uncertainty and multiplicity were handled in the final analysis.
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 |
|---|---|---|---|
| Triptorelin Pamoate 11.25 mg | 1 | 37 | 2.7% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Triptorelin Pamoate 11.25 mg | 7 of 37 |
Most frequent serious adverse event terms
| Event term | Organ system | Triptorelin Pamoate 11.25 mg |
|---|---|---|
| Foot fracture | Injury, poisoning and procedural complications | 1 of 37 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Triptorelin Pamoate 11.25 mg |
|---|---|---|
| Abdominal pain | Gastrointestinal disorders | 4 of 37 |
| Injection site pain | General disorders | 2 of 37 |
| Hot flush | Vascular disorders | 2 of 37 |
Serious events with the highest arm-level rate: foot fracture (2.7%).
The registry lists a Triptorelin Pamoate 11.25 mg safety group with 37 at risk. It reports 1 participant affected by a serious adverse event and 7 affected by other adverse events. These are separate registry figures; they should not be added to estimate a total, since the record does not say whether participants could appear in both categories. The listed top serious adverse event term is foot fracture, reported as 2.7%.
The record does not establish from these figures whether an event was caused by the intervention. A registry safety count describes reported events within the stated group and is not, on its own, a comparison of risk between treatment strategies. Because the study is single-group, the safety information does not include a concurrent control group for estimating a comparative event rate.
Deaths are recorded as null. That is not a reported numerical zero; the number of deaths is not reported in the registry record. The record also does not provide event-level descriptions, timing, duration, severity beyond the serious classification, or a causal assessment. The top-term listing should not be treated as a complete account of all serious adverse events.
These safety figures are limited to the registry entries. They do not describe the primary efficacy endpoint, and they should be read separately from the hormone-response outcomes.
12. What the Result Does, and Does Not, Mean
The registry record supports a description of the study’s design and intended primary measure: a single group of children with precocious puberty received triptorelin pamoate 11.25 mg, and the registered primary endpoint was the number with a GnRH-stimulated LH response at or below 3 IU/L at the scheduled assessment 3 months after the first injection. The record lists enrollment of 37 and marks the study completed.
It does not report the numerical primary outcome. Readers therefore cannot determine from this record how many participants met the threshold, what proportion met it, or how precisely that proportion was estimated. The registry also does not report numerical results for the listed secondary outcomes. Without these data, the posted information does not support a quantitative conclusion about the observed response.
Even with a reported response, the design would answer a descriptive question about the study group, not a randomized comparison of treatment with a control. A single-group result cannot by itself show what would have occurred under a different treatment or without the intervention. The open-label design and absence of a described comparator are important context for any interpretation of measurements over time.
The registry’s safety entries report affected-participant counts for serious and other adverse events, along with a serious-event term. Those entries are not a controlled comparison, and they do not establish causation. Deaths are not reported as a numerical result in the record.
In short, the registry record tells readers what was studied and how the primary response was defined, but it does not include the efficacy results needed to characterize that response. It also leaves important analysis details unreported, including the complete per-protocol definition, the multiplicity approach, and the statistical procedures used. The study’s completed status should not be mistaken for a posted result or a treatment-effect estimate.
13. Why This Trial Matters Statistically
This study illustrates why an endpoint definition and an outcome result are different kinds of information. The registry gives a clear threshold-based primary measure and a follow-up time, but it does not report the number meeting the criterion. A reader can understand what the investigators intended to count without being able to estimate the observed response or its uncertainty.
It also illustrates the interpretive limits of a single-group design. Such a design may describe outcomes in the participants studied, but it does not provide a concurrent treatment contrast. This distinction matters when readers encounter a Phase III label: the phase designation does not, by itself, mean that a study was randomized or comparative. In this record, the design is explicitly non-comparative, open, and single-group.
The analysis-population descriptions add another important statistical point. ITT, modified ITT, and per-protocol populations can include different participants, particularly when follow-up assessments are missing. The registry gives part of the population definitions and specifies a missing-data rule for ITT, but does not report the population counts or endpoint results. Without those details, readers cannot tell how the choice of population affected the result.
Finally, the registry’s description of planning calculations should not be confused with the study’s actual design or observed statistical evidence. The stated assumptions concern an illustrative two-arm calculation, while the registered study is single-group. Planning sample sizes are not observed power and cannot replace a reported effect estimate with its uncertainty. These distinctions help keep design, planning, and outcome evidence separate.
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 main limitation for interpreting efficacy is that the registry record does not report a numerical primary outcome. It provides neither the number meeting the LH threshold nor the relevant analysis denominator, proportion, confidence interval, or test result. The listed enrollment of 37 cannot be assumed to be the number with an evaluable Month 3 assessment.
The single-group, non-comparative design limits interpretation to a description of participants in the study, even if the outcome data were available. There is no concurrent control group described, and the study is open-label with no masking. The registry does not provide information that would permit a treatment-versus-control estimate.
Analysis details are incomplete. The ITT and modified ITT definitions are described, and the ITT rule treats missing primary data as non-response. However, population sizes, missing-data counts, reasons for missing assessments, sensitivity analyses, and primary results by population are not reported. The per-protocol definition is truncated, so its complete eligibility rules cannot be assessed.
The registry lists 15 outcome measures but does not report a multiplicity strategy or the numerical results for the secondary outcomes. A complete statistical analysis plan, details of interim analyses, and the trial’s sample-size justification are not reported in the registry record. The planning assumptions stated for an illustrative calculation are not evidence of the investigators’ design choices.
Safety interpretation is also limited. The record gives counts for serious and other adverse events, but no comparative safety group, event-level detail, or causal assessment. Deaths are not reported as a numerical result. These limitations reflect what the registry record contains and do not establish what may be available elsewhere.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Efficacy and Safety Study of Pamoate of Triptorelin in Children With Precocious Puberty — NCT00564850.
- Registry record last updated 2022-10-12; status last verified 2022-09.
- Results first posted 2011-12-01.
- 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
NCT00564850 is a completed Phase III, multicentre, open, non-comparative, single-group study sponsored by Ipsen. It enrolled 37 children with precocious puberty and evaluated triptorelin pamoate 11.25 mg. The study summary describes girls younger than 9 years and boys younger than 10 years. The registry gives a start of 2007-10 and primary completion of 2010-10.
The primary endpoint was the number of participants with a GnRH-stimulated LH level at or below 3 IU/L, assessed 3 months after the first injection. The registry record does not report the numerical primary result or results for the listed secondary outcomes. It describes ITT and modified ITT populations, including an ITT missing-data rule, but the per-protocol definition is incomplete.
Safety entries list 1 participant affected by a serious adverse event and 7 by other adverse events among 37 at risk; the top serious event term is foot fracture at 2.7%. Deaths are not reported numerically. The record lists no analysis entries and does not report a complete statistical plan or multiplicity approach.