A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease (NCT04002830), a na phase 4 study. The primary outcome is Percent Change From Baseline in Spleen Volume Measured by MRI.

2. Clinical Question

This is a within-group question, not a comparison of taliglucerase alfa with another treatment or with a control condition. Consequently, any observed change during the study would describe participants over time; without a comparator, it would not isolate the effect of treatment from other explanations for change. The registry record names liver volume measured by MRI, hemoglobin, platelet count, and Lyso-GB1 as secondary outcomes. It does not give numerical results for those outcomes either.

The study is described as multicenter and includes participants who had not previously received treatment. The registry record does not report how participants were selected for enrollment, how many centers enrolled participants, or the eligibility criteria beyond the disease and treatment history described in the summary. It also does not report the primary endpoint estimate, its precision, or the analysis population used. The clinical question can therefore be identified from the planned endpoint, but the posted registry information does not establish the magnitude or precision of any change in spleen volume.

3. Trial Design

The registry record classifies this as a Phase 4, completed, single-group study with no masking. Allocation is listed as not applicable. It reports enrollment of 14 participants and describes the study as multicenter. There is no randomized comparison, and no separate control arm is described; the study therefore cannot provide a like-for-like contrast between treatment groups.

The intervention is taliglucerase alfa, also identified as Elelyso. The study summary describes intravenous infusion at 60 units/kg every 2 weeks for 12 months in previously untreated subjects of any age with type 3 Gaucher disease. The arm description likewise identifies intravenous taliglucerase alfa for treatment-naive patients. The registry record does not report whether every participant received the planned regimen for the full period.

The summary states that participants who tolerated infusions well, and were treated at centers where home therapy was the standard of care, could switch from site-based to home treatment at the principal investigator’s discretion. Such a switch was allowed only after no less than 3 uneventful infusions at the site. The record does not report how many participants switched, when any switch occurred, or whether treatment exposure differed among participants.

The recorded study start is 2020-11-20 and the primary completion date is 2023-07-30. The registry status is completed. The primary outcome is percent change from baseline in spleen volume measured by MRI, from baseline to month 12. Listed secondary outcomes are percent change from baseline in liver volume measured by MRI, hemoglobin, platelet count, and Lyso-GB1. Although the record names these endpoints, it does not report their numerical results or describe the detailed statistical analysis plan.

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: If a patient did not meet the inclusion criteria or meet any of the exclusion criteria listed in the protocol, the patient was excluded at screening visits and was not enrolled.

Overall Study

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.

6. Primary Endpoint

The registry lists the primary outcome as Percent Change From Baseline in Spleen Volume Measured by MRI, measured over from baseline to month 12.

The primary endpoint is percent change from baseline in spleen volume measured by MRI, with the stated timeframe from baseline to month 12. It describes how spleen volume at the later assessment differs from the participant’s baseline volume, expressed as a percentage change. MRI is the stated measurement method; the registry record does not give details of image acquisition, reading, or quality control.

A change-from-baseline measure uses each participant’s starting value as a reference, so the endpoint describes change over time rather than only the volume at month 12. Expressing change as a percentage provides a relative scale tied to baseline. These are general properties of change-from-baseline outcomes; they do not determine whether a change is clinically important or attributable to treatment.

The registry record specifies the summary parameter as a median and the dispersion as a standard error. A median describes the middle of the observed values when they are ordered. A standard error describes uncertainty in an estimate, but the record does not explain its calculation or how it was paired with the median. The numerical median, standard error, and any confidence interval for the primary endpoint are not reported in the registry record.

The endpoint is relevant to the stated study question because it measures change in spleen volume over the planned treatment period. However, the record does not provide a threshold for interpreting the size of a change, nor does it report results that would allow readers to judge the observed direction, magnitude, or precision.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does the primary summary describe?

The primary outcome is expressed as percent change from baseline in spleen volume measured by MRI, from baseline to month 12. The registry record specifies the median as the summary parameter and standard error as the dispersion measure. A median represents the central value after observations are ordered. Unlike a mean, it does not use the numerical distance of every observation from every other observation in the same way. It can be useful for describing a distribution when a typical value is of interest, but it does not by itself show how spread out individual observations are.

A standard error concerns the uncertainty of an estimated summary, rather than the variability among individual participants. General statistical practice often presents uncertainty alongside an estimate so readers can judge precision. The record does not report the actual median or standard error, nor does it explain the procedure used to derive a standard error for the median. No confidence interval or hypothesis-test result for the primary endpoint is reported in the registry record.

Why does the single-group design matter?

The study is recorded as single-group, with allocation not applicable and no masking. These design features mean there is no separate randomized comparator described in the registry record. A within-group estimate can describe the participants’ observed change from baseline, but it cannot by itself distinguish treatment-associated change from other influences over time. General statistical reasoning treats a comparator as important when the aim is to estimate a difference attributable to an intervention. This registry record does not report such a between-group estimate.

The sample-size assumptions describe a planning illustration based on an observed effect, using a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. These assumptions do not describe the single-group design recorded for this study. The assumptions are planning conditions, not outcomes, and any resulting sample-size illustration is not observed power. The registry record does not report that these assumptions were the study’s own design rationale or sample-size justification.

What do the adverse-event categories mean?

The safety record separates participants affected by serious adverse events from participants affected by other adverse events and records deaths separately. General reporting convention distinguishes seriousness from the intensity of a symptom: seriousness concerns defined outcomes or consequences, whereas intensity describes how severe a symptom feels. The registry record does not give individual event narratives or an event-by-event clinical assessment. The counts therefore describe the recorded categories, not a causal comparison or a complete account of each event.

The record reports 14 participants at risk in both event categories, 1 participant affected by a serious event, 9 affected by other events, and 0 deaths. It also lists shortness of breath at 7.1% among the top serious-event terms. That percentage is presented as recorded; the registry information does not supply further detail about its denominator or the event’s course. The reported numbers should not be treated as evidence that the intervention caused or prevented an event.

What analyses are not available?

The registry record lists 0 analyses and does not report a statistical analysis plan, an interim-analysis plan, the handling of missing observations, or the analysis population for the efficacy outcomes. It also does not report p-values, confidence intervals, adjusted estimates, or sensitivity analyses. These omissions prevent readers from checking which participants contributed to each endpoint, how incomplete data were handled, or whether alternative analytical choices were examined.

The record names one primary outcome and four secondary outcomes. It does not report whether any multiplicity procedure was planned for interpreting several endpoints. General statistical methods recognize that testing multiple outcomes can affect the interpretation of nominal significance levels; a prespecified approach can help clarify how such results are to be read. No such approach is reported. Since numerical efficacy results are absent, no significance or multiplicity conclusion can be drawn.

10. Multiplicity, Missing Data and Analysis Populations

The registry record names the primary endpoint as percent change from baseline in spleen volume measured by MRI and lists four secondary outcomes: liver volume measured by MRI, hemoglobin, platelet count, and Lyso-GB1, each expressed as percent change. It does not report a multiplicity plan describing how evidence across these outcomes would be interpreted. In general, examining several outcomes can make isolated findings harder to interpret; a prespecified strategy can define which comparisons are primary and how additional results are assessed. The registry identifies the primary endpoint, but does not provide further detail on such a strategy.

Missing-data handling is not reported in the registry record. There is no description of how absent baseline or month-12 measurements would be treated, whether participants with incomplete follow-up would contribute to an analysis, or whether any sensitivity analyses were planned. The analysis population is also not reported. These details matter because conclusions can depend on which participants and measurements are included and on assumptions made about missing observations.

The registry record lists 0 analyses and does not report an interim-analysis plan, a statistical analysis plan, or the method for estimating uncertainty around the median. It also gives no p-values or confidence intervals for the primary or secondary outcomes. The stated sample-size assumptions are described as planning illustrations and do not establish the analysis procedures used in the completed study. Without these details and the numerical outcome results, the statistical handling of the efficacy data cannot be evaluated from the registry record.

11. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: shortness of breath (7.1%).

The safety record lists 14 participants at risk for both serious and other adverse events. It reports 1 participant affected by a serious adverse event, 9 participants affected by other adverse events, and 0 deaths. Shortness of breath appears among the top serious-event terms, listed at 7.1%. These figures are aggregate registry entries; they do not show when events occurred, how long they lasted, whether they resolved, or whether the same participant experienced events in more than one category.

The terms “serious” and “other” refer to the categories used in the registry record and should not be read as a full description of event severity. General safety interpretation distinguishes seriousness classification from symptom intensity, and the record does not provide details that would allow individual events to be assessed. It also does not report a comparison group, so the figures cannot be used to compare event frequency against an untreated or alternative-treatment group.

These counts describe events recorded among participants in the study; they do not establish that treatment caused an event or that an event was unrelated to treatment. The registry information does not provide further attribution details, narratives, or analyses of exposure and event timing. The listed figures should therefore be interpreted as descriptive safety reporting rather than as a causal estimate.

12. What the Result Does, and Does Not, Mean

The registry record establishes that this was a completed, single-group study of taliglucerase alfa in previously untreated subjects with type 3 Gaucher disease, with enrollment of 14 participants. It specifies a primary outcome based on MRI-measured spleen volume from baseline to month 12. It also records aggregate adverse-event information. These registry entries describe the study’s setup and some safety reporting, but they do not provide the primary efficacy result.

The primary endpoint’s numerical result, standard error, and confidence interval are not reported in the registry record. Numerical results for the secondary outcomes are also not reported. Therefore, the posted information does not establish the size, direction, or precision of change in spleen volume or the other listed measures. Completion status alone does not supply these missing results.

Because the study has no separate comparator group, any within-group change would not by itself demonstrate that the intervention caused that change. A concurrent comparison can help account for changes that might occur over time for reasons unrelated to treatment; such a comparison is not described. This is a limitation on causal interpretation, not a finding about whether treatment did or did not have an effect.

The safety figures report participants affected by serious and other adverse events and deaths, but do not provide detailed event histories or establish causation. Nor does the record provide the analysis procedures needed to assess missing measurements, the analysis population, multiplicity, or uncertainty. The appropriate reading is consequently bounded: the registry describes the planned endpoint, design, enrollment, and selected safety counts, while leaving the primary statistical result and important analysis details unreported.

13. Why This Trial Matters Statistically

This study is statistically notable because its primary question concerns change from baseline in an MRI-measured organ volume, while the recorded design is single-group. That distinction matters: a study can describe changes among participants over time, but without a comparator it cannot directly estimate how those changes differ from what would have occurred under another condition. The design determines the kind of inference the data can support.

The endpoint is specified with a median summary and standard error dispersion. Reporting both an estimate and a measure of uncertainty would help readers assess not only the direction and magnitude of the observed change but also its precision. The registry record does not report those numerical results, so this assessment cannot be made from the posted information. It also does not explain the standard-error calculation for the median.

The record gives an enrollment of 14 and aggregate safety counts, but does not report detailed outcome analyses. In a study with a limited enrolled cohort, the distinction between descriptive observations and evidence about a broader population is especially important as a general statistical consideration. No population-level conclusion should be inferred without the outcome estimates, uncertainty, and analysis details.

Finally, the sample-size assumptions are explicitly planning illustrations and differ from the study’s recorded single-group structure by assuming equal allocation between two arms. Keeping design details separate from planning calculations prevents hypothetical sample-size outputs from being mistaken for observed results, actual study power, or the study’s own statistical plan. Here, the registry record supports a description of the design and reported safety counts, but not a conclusion about the primary endpoint.

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 principal limitation for interpretation is the absence of numerical efficacy results. The registry record identifies the primary endpoint and its timeframe but does not report the median percent change, its standard error, or a confidence interval. It also does not report numerical results for the listed secondary outcomes. Consequently, readers cannot assess the observed changes or their precision from the registry information.

The single-group design has no like-for-like control comparison. This limits causal interpretation of any change that might have occurred during follow-up. The record also reports no masking, and no allocation to a separate comparator. These are design features recorded in the registry, not evidence that outcomes did or did not change.

Enrollment was 14. The registry record does not describe the analysis population, missing-data methods, participant-level follow-up, or sensitivity analyses. It therefore does not show how incomplete endpoint measurements, if any, were handled, or whether all enrolled participants contributed to each outcome analysis. The statistical analysis plan and any interim-analysis plan are not reported.

The safety information is aggregate and does not include event narratives, timing, duration, resolution, or detailed attribution. The record lists serious and other adverse events separately, but does not provide information to reconstruct individual event histories or compare risks with another group. The study summary also allows a discretionary switch to home treatment under stated conditions, but the registry record does not report how many participants switched or whether this affected exposure or assessment. Together, these omissions constrain interpretation of both efficacy and safety reporting.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease — NCT04002830.
• Registry record last updated 2024-12-17; status last verified 2024-11.
• Results first posted 2024-12-17.
• 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.

18. Record Summary

The registry record describes NCT04002830, a Phase 4, completed, multicenter, single-group study of taliglucerase alfa in previously untreated subjects of any age with type 3 Gaucher disease. It records enrollment of 14 participants, no masking, and allocation listed as not applicable. The study summary describes intravenous infusion at 60 units/kg every 2 weeks for 12 months.

The primary outcome is percent change from baseline in spleen volume measured by MRI, from baseline to month 12, summarized by a median with standard error dispersion. The result is not reported in the registry record. Secondary outcomes include percent change in liver volume measured by MRI, hemoglobin, platelet count, and Lyso-GB1; their numerical results are also not reported.

The safety record lists 1 participant affected by a serious adverse event, 9 affected by other adverse events, and 0 deaths, among 14 at risk in each category. Shortness of breath is listed among the top serious-event terms at 7.1%. No comparator group or detailed statistical analysis is reported.