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 A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia (NCT02055157), a non-randomized phase 2 study. The primary outcome is Overall Summary of Adverse Events During Initial 6-Month Period.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02055157 |
| Phase | Phase 2 |
| Condition | Achondroplasia |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 35 |
| Arms in the primary outcome | n/a |
| Lead sponsor | BioMarin Pharmaceutical |
| Results posted | Yes |
2. Clinical Question
Population
Children with achondroplasia were enrolled in a Phase 2 study of BMN 111. The registry record lists enrollment as 35. The safety population is described as participants who received at least one dose of study treatment and were included in safety analysis during the initial six-month period and the entire study period.
Intervention
The intervention was daily BMN 111. The study used sequential dose-escalation cohorts. The listed cohort doses were 2.5 µg/kg, 7.5 µg/kg, 15 µg/kg, and 30 µg/kg. The study was open-label, so masking was not used.
Comparator
There was no randomized control group. The cohorts received different doses in a sequential, nonrandomized design; they are not a like-for-like control comparison. Differences between cohort results therefore cannot be interpreted as randomized treatment effects.
Primary question
The primary outcome was an overall summary of adverse events during the initial six-month period, with a timeframe of up to Month 6 ± 7 Days. The outcome records participants with adverse events, including treatment-emergent adverse events and serious adverse events. The primary objective was to assess safety and tolerability.
3. Trial Design
This was a Phase 2, open-label, sequential cohort dose-escalation study of BMN 111 in children with achondroplasia. It was nonrandomized and had no masking. Enrollment was 35. The registry lists the study as completed, with a start date of 2014-01-13 and a primary completion date of 2017-10-02.
Participants were assigned to sequential dose cohorts, with listed doses of 2.5 µg/kg, 7.5 µg/kg, 15 µg/kg, and 30 µg/kg. The intervention is described as daily BMN 111. The registry record does not report a randomized comparator, so the cohorts should not be treated as equivalent groups differing only by dose. Sequential enrollment and dose escalation also mean that cohort membership is tied to the study sequence.
The primary outcome was an overall summary of adverse events over the initial six-month period. The registry also lists secondary outcomes involving change from baseline in annualized growth velocity, height Z-scores using the Centers for Disease Control and Prevention reference standard, and upper-to-lower body ratios. Some outcomes are defined for the initial six-month period, while others are listed for the entire study period or for specified cohorts and switchers. Numerical results for those secondary outcomes are not reported in the registry record.
The registry record does not report the protocol details governing escalation decisions, cohort entry, or switching. Nor does it report a randomized comparison or a sample-size justification. These omissions limit reconstruction of the design beyond its stated sequential, nonrandomized, open-label structure.
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.
Initial 6 Months
| Stage | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| STARTED | 8 | 8 | 10 | 9 |
| COMPLETED | 7 | 7 | 10 | 8 |
| NOT COMPLETED | 1 | 1 | 0 | 1 |
Reasons for not completing this period:
| Reason | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Subject discontinued due to NeedlePhobia | 0 | 1 | 0 | 0 |
| Adverse Event | 0 | 0 | 0 | 1 |
| Consent withdrawn by subject | 1 | 0 | 0 | 0 |
18-month Extension
| Stage | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| STARTED | 7 | 7 | 10 | 8 |
| COMPLETED | 6 | 6 | 10 | 8 |
| NOT COMPLETED | 1 | 1 | 0 | 0 |
Reasons for not completing this period:
| Reason | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 |
|---|---|---|---|---|
| Investigator decision | 0 | 1 | 0 | 0 |
| Subject discontinued due to NeedlePhobia | 1 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Safety Analysis Population includes all subjects who received at least one dose of study treatment and were used for safety analysis in the initial 6-month period and entire study period.
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.
Population: Safety Analysis Population consists of all subjects who received at least one dose of study treatment and were used for safety analysis in the initial 6-month period and entire study period.
| Characteristic | Unit | Cohort 1 | Cohort 2 | Cohort 3 | Cohort 4 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 8 | 8 | 10 | 9 | 35 | |
| Age, Continuous | yrs | 7.3 (SD 1.58) | 8.3 (SD 2.19) | 8.0 (SD 1.63) | 6.9 (SD 1.17) | 7.6 (SD 1.68) |
| Sex: Female, Male: Female | Participants | 5 | 3 | 6 | 5 | 19 |
| Sex: Female, Male: Male | Participants | 3 | 5 | 4 | 4 | 16 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 1 | 1 | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 8 | 8 | 9 | 7 | 32 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 1 | 1 |
| Race/Ethnicity, Customized: White | Participants | 7 | 6 | 5 | 6 | 24 |
| Race/Ethnicity, Customized: Asian | Participants | 0 | 1 | 3 | 3 | 7 |
| Race/Ethnicity, Customized: Black or African American | Participants | 1 | 0 | 1 | 0 | 2 |
| Race/Ethnicity, Customized: Other | Participants | 0 | 1 | 1 | 0 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Overall Summary of Adverse Events During Initial 6-Month Period, measured over Up to Month 6 ± 7 Days.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is an overall summary of adverse events during the initial six-month period, with the registry timeframe stated as up to Month 6 ± 7 Days. The results are expressed as counts of participants with an event, rather than counts of every event episode. The safety population is defined as all subjects who received at least one dose of study treatment and were used for safety analysis in the initial six-month period and the entire study period.
A treatment-emergent adverse event is defined in the record as an event that newly appeared, increased in frequency, or worsened in severity following initiation of study drug administration. Serious adverse events are also reported. This endpoint is suited to the stated primary objective of assessing safety and tolerability: it describes how many participants experienced events during treatment exposure. It does not by itself establish that the drug caused an event, distinguish all event severities, or summarize the number of recurrent event episodes.
Counts are useful for describing what was recorded, but they are not a complete measure of risk on their own. Interpretation also depends on the number of participants followed, exposure duration, event definitions, and the way events were collected and classified. The registry reports participants at risk for the event summaries, but does not provide confidence intervals or a formal comparative analysis in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
For the initial six-month period, no serious adverse events were recorded in the 2.5 µg/kg, 7.5 µg/kg, 15 µg/kg, or 30 µg/kg cohorts. The corresponding numbers at risk were 8, 8, 10, and 9. Other adverse events were recorded for all participants at risk in each of those cohorts: 8 of 8, 8 of 8, 10 of 10, and 9 of 9, respectively. These summaries describe participants with events, not the total number of event episodes.
The extension-period summaries include serious adverse events in the 2.5 µg/kg, 15 µg/kg, and 30 µg/kg groups: 1 participant among 7 at risk, 1 among 22, and 1 among 8, respectively. No serious adverse events were recorded in the 7.5 µg/kg extension group, which had 14 participants at risk. Other adverse events were reported for 7 of 7 participants at risk at 2.5 µg/kg, 13 of 14 at 7.5 µg/kg, 22 of 22 at 15 µg/kg, and 8 of 8 at 30 µg/kg.
For the entire-study summaries, serious adverse events were recorded for 1 participant among 8 at risk at 2.5 µg/kg, none among 15 at risk at 7.5 µg/kg, 1 among 22 at 15 µg/kg, and 1 among 9 at 30 µg/kg. Other adverse events were recorded for 8 of 8, 14 of 15, 22 of 22, and 9 of 9 participants, respectively. The registry reports no deaths in any listed cohort-period summary.
These are descriptive safety counts, not estimates from a randomized comparison. Cohort sizes differ, and the reported numbers at risk vary across periods; participants may also be represented in more than one period summary. Consequently, comparisons across doses or periods should not be read as causal evidence that one dose is safer or less safe than another. The registry record does not report confidence intervals or inferential tests for these summaries. The counts should be read as the posted event experience in this study, with uncertainty that cannot be quantified from the listed results alone.
9. Statistical Methods Explained
What does a participant count represent?
The primary outcome uses counts of participants with an event. A participant count records whether a person experienced an event meeting the relevant definition during the stated period. It is not the same as a count of event episodes: one participant may have more than one event, but the participant-level summary does not enumerate every episode. The registry also lists participants at risk, which gives context for each group’s count. These counts should not be converted into an implied event rate unless a rate is explicitly reported.
Why separate serious and other adverse events?
Serious adverse events are presented separately from other adverse events. This distinction helps readers see whether events were classified as serious, while the overall adverse-event summary describes a broader category. The registry defines a treatment-emergent adverse event by its timing and change after treatment begins: it newly appears, increases in frequency, or worsens in severity. A treatment-emergent designation describes when an event occurred relative to treatment; by itself it does not prove that treatment caused the event.
How should cohort results be compared?
In a randomized trial, random assignment is intended to make comparison groups similar on average apart from the assigned intervention. This study was nonrandomized and sequential, so that basis for comparison is absent. Cohorts may differ for reasons connected to their place in the sequence, and the registry record does not describe enough detail to assess or adjust for such differences. A numerical contrast between the dose groups is therefore descriptive, not a randomized estimate of a dose effect.
The open-label design means masking was not used. General statistical considerations are that knowledge of treatment can affect reporting, assessment, or decisions about whether an event meets a classification. The record does not report procedures intended to limit these possibilities, so their influence cannot be assessed from the registry information.
What does a confidence interval add?
In general, a confidence interval expresses the precision of an estimate by giving a range of values compatible with the data under the method’s assumptions. A point count alone does not communicate that precision. No confidence intervals are reported for the adverse-event summaries in the registry record. The listed results therefore support descriptive statements about observed participant counts, but not a precise estimate of how those counts would vary across comparable populations.
What can a formal comparison establish?
In general, a statistical test or model can quantify evidence about a specified comparison, but its meaning depends on the design, assumptions, endpoint, and analysis plan. The registry record lists no analysis records for the outcomes. It does not report a formal test comparing dose cohorts, an adjusted model, or an inferential estimate for the adverse-event results. It would not be appropriate to infer statistical significance or a dose-response relationship from the counts alone.
The study’s sequential dose-escalation structure is relevant to interpretation. Dose groups are not randomized arms, and participant counts at risk differ across cohorts and periods. The extension and entire-study summaries also cover different observation periods from the initial six-month summary. In general, longer or differing exposure and follow-up can affect the opportunity to observe an event. The registry does not provide person-time or exposure-adjusted event rates, so those quantities cannot be reconstructed from the participant counts.
The registry record lists secondary outcomes concerning growth velocity, height Z-scores, and body proportions. These are distinct outcomes from the primary safety endpoint and can require different statistical summaries. Their numerical results and analysis methods are not reported in the registry record. No conclusions about their magnitude, precision, or relationship to dose can therefore be drawn from the listed adverse-event counts.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple secondary outcomes, including changes from baseline in annualized growth velocity, height Z-scores, and upper-to-lower body ratios. Several are specified for different periods or cohort groupings. When multiple outcomes are examined, general statistical practice distinguishes the primary question from additional or exploratory questions, because looking across many outcomes can increase the chance of apparently notable findings. The registry record does not report whether any multiplicity adjustment, hierarchy, or other approach to multiple testing was planned or used.
Missing-data handling is not reported in the registry record. The posted safety summaries include numbers at risk, but do not describe whether participants with incomplete follow-up were excluded, how incomplete event information was handled, or whether any imputation method was used. The record also does not report the amount or pattern of missing outcome data for the listed secondary outcomes.
The safety population is described as participants who received at least one dose and were included in safety analysis for the initial six-month period and the entire study period. The record does not give further analysis-population rules, such as detailed inclusion criteria for each outcome, or explain how participants who switched cohorts were handled analytically. It does list certain secondary outcomes for switchers, but numerical results for those outcomes are not reported in the registry record.
No statistical analysis plan, interim-analysis approach, or sample-size justification is reported in the registry record. These omissions matter because they prevent readers from determining in advance which outcomes were prioritized, how repeated testing was addressed, and what rules governed incomplete observations or analysis populations.
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 |
|---|---|---|---|
| Initial 6-Months Period 2.5 µg/kg. | 0 | 8 | 0.0% |
| Initial 6-Months Period 7.5 µg/kg. | 0 | 8 | 0.0% |
| Initial 6-Months Period 15 µg/kg. | 0 | 10 | 0.0% |
| Initial 6-Months Period 30 µg/kg. | 0 | 9 | 0.0% |
| Extension Period 2.5 µg/kg. | 1 | 7 | 14.3% |
| Extension Period 7.5 µg/kg. | 0 | 14 | 0.0% |
| Extension Period 15 µg/kg. | 1 | 22 | 4.5% |
| Extension Period 30 µg/kg. | 1 | 8 | 12.5% |
| Entire Study Period 2.5 µg/kg. | 1 | 8 | 12.5% |
| Entire Study Period 7.5 µg/kg. | 0 | 15 | 0.0% |
| Entire Study Period 15 µg/kg. | 1 | 22 | 4.5% |
| Entire Study Period 30 µg/kg. | 1 | 9 | 11.1% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Initial 6-Months Period 2.5 µg/kg. | 0 of 8 |
| Initial 6-Months Period 7.5 µg/kg. | 0 of 8 |
| Initial 6-Months Period 15 µg/kg. | 0 of 10 |
| Initial 6-Months Period 30 µg/kg. | 0 of 9 |
| Extension Period 2.5 µg/kg. | 0 of 7 |
| Extension Period 7.5 µg/kg. | 0 of 14 |
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 |
|---|---|
| Initial 6-Months Period 2.5 µg/kg. | 8 of 8 |
| Initial 6-Months Period 7.5 µg/kg. | 8 of 8 |
| Initial 6-Months Period 15 µg/kg. | 10 of 10 |
| Initial 6-Months Period 30 µg/kg. | 9 of 9 |
| Extension Period 2.5 µg/kg. | 7 of 7 |
| Extension Period 7.5 µg/kg. | 13 of 14 |
Most frequent serious adverse event terms
| Event term | Organ system | Initial 6-Months Period 2.5 µg/kg. | Initial 6-Months Period 7.5 µg/kg. | Initial 6-Months Period 15 µg/kg. | Initial 6-Months Period 30 µg/kg. | Extension Period 2.5 µg/kg. | Extension Period 7.5 µg/kg. |
|---|---|---|---|---|---|---|---|
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | 0 of 8 | 0 of 8 | 0 of 10 | 0 of 9 | 1 of 7 | 0 of 14 |
| Tonsillar hypertrophy | Respiratory, thoracic and mediastinal disorders | 0 of 8 | 0 of 8 | 0 of 10 | 0 of 9 | 0 of 7 | 0 of 14 |
| Thyroglossal cyst | Congenital, familial and genetic disorders | 0 of 8 | 0 of 8 | 0 of 10 | 0 of 9 | 0 of 7 | 0 of 14 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Initial 6-Months Period 2.5 µg/kg. | Initial 6-Months Period 7.5 µg/kg. | Initial 6-Months Period 15 µg/kg. | Initial 6-Months Period 30 µg/kg. | Extension Period 2.5 µg/kg. | Extension Period 7.5 µg/kg. |
|---|---|---|---|---|---|---|---|
| Injection site erythema | General disorders | 4 of 8 | 7 of 8 | 9 of 10 | 9 of 9 | 1 of 7 | 5 of 14 |
| Injection site reaction | General disorders | 2 of 8 | 6 of 8 | 8 of 10 | 9 of 9 | 2 of 7 | 5 of 14 |
| Hypotension | Vascular disorders | 3 of 8 | 4 of 8 | 4 of 10 | 2 of 9 | 1 of 7 | 1 of 14 |
| Headache | Nervous system disorders | 4 of 8 | 2 of 8 | 2 of 10 | 3 of 9 | 1 of 7 | 1 of 14 |
| Injection site swelling | General disorders | 4 of 8 | 2 of 8 | 4 of 10 | 2 of 9 | 0 of 7 | 0 of 14 |
| Cough | Respiratory, thoracic and mediastinal disorders | 4 of 8 | 0 of 8 | 2 of 10 | 4 of 9 | 0 of 7 | 2 of 14 |
| Pyrexia | General disorders | 4 of 8 | 1 of 8 | 3 of 10 | 2 of 9 | 0 of 7 | 1 of 14 |
| Injection site urticaria | General disorders | 1 of 8 | 1 of 8 | 2 of 10 | 3 of 9 | 1 of 7 | 1 of 14 |
| Nasopharyngitis | Infections and infestations | 1 of 8 | 3 of 8 | 3 of 10 | 1 of 9 | 0 of 7 | 1 of 14 |
| Ear infection | Infections and infestations | 0 of 8 | 3 of 8 | 2 of 10 | 2 of 9 | 1 of 7 | 1 of 14 |
Serious events with the highest arm-level rate: sleep apnoea syndrome (14.3%), thyroglossal cyst (12.5%), tonsillar hypertrophy (4.5%).
The serious adverse-event figures are participant counts for each dose and study period. During the initial six-month period, the recorded serious-event count was zero in every listed cohort. The extension-period summaries report 1 participant with a serious adverse event among 7 at risk at 2.5 µg/kg, none among 14 at 7.5 µg/kg, 1 among 22 at 15 µg/kg, and 1 among 8 at 30 µg/kg. The entire-study summaries report 1 among 8 at 2.5 µg/kg, none among 15 at 7.5 µg/kg, 1 among 22 at 15 µg/kg, and 1 among 9 at 30 µg/kg.
These figures are not event rates adjusted for time at risk, and the period summaries should not be added together. The same participants may contribute to more than one period, and the registry does not provide participant-level linkage or exposure-adjusted analyses. A zero count means no serious event was recorded in that group and period; it does not establish that the underlying risk is zero. Likewise, counts from groups with different numbers at risk are not direct evidence of relative safety. No deaths are listed in the reported cohort-period summaries.
12. What the Result Does, and Does Not, Mean
The registry results describe adverse events recorded among participants treated in a nonrandomized, open-label, sequential dose-escalation study. During the initial six-month period, no serious adverse events were recorded in the listed cohorts, while other adverse events were recorded for every participant at risk in each cohort. Later summaries report serious adverse events in some groups, and other adverse events in most or all participants at risk across the extension and entire-study periods. Those statements are limited to the counts and definitions in the registry record.
The results do not establish that BMN 111 caused any particular event. The treatment-emergent definition is based on timing or worsening after treatment began; causal attribution requires more than temporal association. The summaries also do not show that a dose is safer or less safe than another, because the cohorts were not randomized control groups and differed in their listed numbers at risk and observation periods.
The adverse-event counts do not provide a complete account of event frequency over time, recurrent events, severity beyond the serious-event classification, or uncertainty around the estimates. The registry record does not report confidence intervals, formal comparative tests, or exposure-adjusted rates. The results therefore support a descriptive account, not a precise comparison or a population-wide risk estimate.
The record lists growth-related secondary outcomes, including annualized growth velocity and height Z-scores, but numerical findings for these outcomes are not reported in the registry record. The safety summaries cannot answer questions about the magnitude or precision of changes in growth measures. In general, one endpoint should not be used as a substitute for another: safety counts and growth outcomes address different questions.
13. Why This Trial Matters Statistically
This study illustrates why trial design must be considered alongside numerical results. A dose-escalation study can describe adverse events observed as participants receive different doses, but a sequential nonrandomized design does not provide the same basis for causal comparison as random assignment. The group counts are informative as safety descriptions, while contrasts between cohorts remain vulnerable to differences associated with sequence, participant composition, and observation.
It also highlights the distinction between participant counts and event rates. The registry reports how many participants had events and how many were at risk in each period, but does not give exposure-adjusted rates or confidence intervals. A count can communicate what was observed without showing the precision of an estimate or accounting for different exposure durations.
The study’s stated primary objective was safety and tolerability, and the primary outcome is an adverse-event summary. The record also identifies growth-related secondary outcomes. Keeping these questions separate is important: safety summaries cannot stand in for results on growth, and the listed outcome names alone do not establish what those secondary results were.
Finally, the registry’s omissions around analysis methods, missing data, multiplicity, and sample-size justification constrain interpretation. Clear reporting of those elements helps readers assess how outcomes were selected and analyzed and how much uncertainty remains. In this record, the adverse-event counts can be read descriptively, but stronger comparative or inferential conclusions are not supported by the reported information.
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 central limitation for interpreting the dose groups is the nonrandomized, sequential design. The cohorts are not like-for-like randomized controls, so differences in their adverse-event counts cannot be attributed to dose from these summaries. The open-label design also means masking was not used; the registry record does not describe safeguards against possible effects of treatment awareness on event reporting or classification.
The adverse-event results are participant counts. The registry does not report event episodes, exposure-adjusted rates, or confidence intervals. Group sizes at risk vary across doses and periods, and extension and entire-study summaries represent different observation windows from the initial six-month period. The record does not provide enough information to assess how follow-up duration or switching affected each summary.
Only limited detail on event definitions is reported. Treatment-emergent events and serious adverse events are described, but the registry record does not provide a full account of event ascertainment, adjudication, or severity categories beyond the serious-event distinction. The reported counts alone cannot establish causality.
Further limitations are the absence of reported statistical analysis methods, multiplicity procedures, missing-data handling, and a sample-size justification. Numerical results for the listed secondary growth outcomes are not reported in the registry record, so the record does not support interpretation of those outcomes. These limitations do not negate the descriptive counts, but they restrict comparisons, precision assessment, and broader inference.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia — NCT02055157.
- Registry record last updated 2021-01-15; status last verified 2020-12.
- Results first posted 2021-01-15.
- 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
NCT02055157 is a completed Phase 2, open-label, nonrandomized, sequential cohort dose-escalation study of daily BMN 111 in children with achondroplasia. The registry lists enrollment as 35 and doses of 2.5 µg/kg, 7.5 µg/kg, 15 µg/kg, and 30 µg/kg. The primary objective was to assess safety and tolerability; the primary outcome summarized adverse events during the initial six-month period.
The posted safety results report participant counts for serious and other adverse events across initial, extension, and entire-study periods. No serious adverse events were recorded in the initial-period cohorts; later summaries include serious adverse events in some dose groups. The design does not support randomized comparisons between cohorts. The registry record does not report formal comparative analyses, confidence intervals, missing-data methods, multiplicity procedures, or numerical results for the listed growth-related secondary outcomes.