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 Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease (NCT00158600), a randomized phase 3 study. The primary outcome is Summary of Patients Reporting Treatment-Emergent Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00158600 |
| Phase | Phase 3 |
| Condition | Pompe Disease (Late-onset), Glycogen Storage Disease Type II (GSD-II), Acid Maltase Deficiency Disease |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 90 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Genzyme, a Sanofi Company |
| Results posted | Yes |
2. Clinical Question
Population
Patients with late-onset Pompe disease were eligible for this phase 3 study. The registry describes Pompe disease as a deficiency of acid alpha-glucosidase, an enzyme involved in breaking down glycogen in lysosomes. The study enrollment was 90. The safety population consisted of patients who received any amount of study treatment, and safety analyses assigned patients according to the treatment they actually received.
Intervention
The intervention was intravenous alglucosidase alfa at 20 milligrams per kilogram of body weight, infused every other week for 78 weeks. The registry’s overall objective was to evaluate safety, efficacy and pharmacokinetics in patients receiving alglucosidase alfa compared with placebo.
Comparator
The comparator was intravenous placebo infused every other week for 78 weeks. The registry describes the study as randomized, double-blind, placebo-controlled and parallel-group. It labels masking as quadruple, but the identities of the people covered by that masking are not reported in the registry record.
Primary question
The posted primary outcome asks how many patients reported treatment-emergent adverse events during weeks 0-78. The safety summary includes adverse events, serious adverse events, treatment-related adverse events and infusion-associated reactions. It is a safety outcome, not a single measure of functional benefit. Although the study’s overall objective also included efficacy and pharmacokinetics, the registry information does not provide a single primary efficacy comparison.
3. Trial Design
This was a phase 3 randomized, double-blind, placebo-controlled, parallel-group study. The registry reports enrollment of 90 and a completed study status. The listed start was 2005-09 and primary completion was 2007-09. These dates describe the registry record; they do not, by themselves, give details about recruitment, follow-up completion or the timing of individual assessments.
Patients were assigned to alglucosidase alfa or placebo. The alglucosidase alfa regimen was an intravenous infusion of 20 milligrams per kilogram every other week for 78 weeks. The placebo regimen was an intravenous infusion every other week for 78 weeks. The registry does not describe the allocation ratio as part of the design details. The safety results report 60 patients at risk in the alglucosidase alfa group and 30 in the placebo group; these are the posted safety-group denominators, not a substitute for a fuller account of randomization or treatment receipt.
Randomization is intended, in general, to make treatment groups comparable at the point of assignment by using a chance process. Masking is intended, in general, to limit the influence of treatment knowledge on behavior, assessment or reporting. The registry labels the study quadruple-masked, but does not report which roles were masked or how masking was maintained. The actual method of sequence generation, concealment procedures, stratification, or any changes to the study plan are not reported in the registry record.
The registry lists percent predicted proximal muscle strength of the lower limbs measured by quantitative muscle testing, and physical-component health-related quality of life measured by the MOS Short Form-36, among secondary outcomes. Those outcomes provide context for the study’s broader aims, but their numerical results and analysis details are not included in the registry record. The posted primary outcome instead summarizes treatment-emergent adverse events during weeks 0-78.
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: One hundred patients screened and 90 enrolled.
Overall Study
| Stage | Alglucosidase Alfa | Placebo |
|---|---|---|
| STARTED | 60 | 30 |
| COMPLETED | 55 | 26 |
| NOT COMPLETED | 5 | 4 |
Reasons for not completing this period:
| Reason | Alglucosidase Alfa | Placebo |
|---|---|---|
| unable to commit time to study | 0 | 1 |
| Adverse Event | 2 | 1 |
| Death | 1 | 0 |
| Withdrawal by Subject | 2 | 2 |
Analysis population for the primary outcome, as defined in the registry: All patients who received any amount of study treatment comprise the safety population. Patients were considered, for safety analysis, to be in the treatment group of the treatment they actually received. Missing or invalid safety or resource utilization data were not replaced.
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 | Alglucosidase Alfa | Placebo | Total |
|---|---|---|---|---|
| Participants analysed | 60 | 30 | 90 | |
| Age, Continuous | years | 45.3 (SD 12.37) | 42.6 (SD 11.63) | 44.4 (SD 12.14) |
| Sex: Female, Male: Female | Participants | 26 | 19 | 45 |
| Sex: Female, Male: Male | Participants | 34 | 11 | 45 |
| Race/Ethnicity, Customized: Hispanic | participants | 1 | 1 | 2 |
| Race/Ethnicity, Customized: Asian | participants | 1 | 1 | 2 |
| Race/Ethnicity, Customized: Black or African American | participants | 0 | 0 | 0 |
| Race/Ethnicity, Customized: White | participants | 57 | 27 | 84 |
| Race/Ethnicity, Customized: Unknown or not reported | participants | 1 | 1 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Summary of Patients Reporting Treatment-Emergent Adverse Events, measured over weeks 0-78.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is a summary of patients reporting treatment-emergent adverse events during weeks 0-78. The registry defines these events as adverse events that occurred after study treatment began. Its safety summary covers adverse events, serious adverse events, treatment-related adverse events and infusion-associated reactions. These categories describe different aspects of safety: seriousness concerns the event’s classification, while treatment-relatedness records an assessment of whether an event was related to treatment. An infusion-associated reaction is identified as a separate category in the registry description.
The endpoint is expressed as a number of participants, rather than a continuous measurement such as muscle strength. Counting patients who experienced an event can make the safety burden easier to interpret than a list of event descriptions alone. However, a participant count does not convey every feature of an event. It does not, on its own, describe duration, severity within a category, recurrence, timing, or the number of events experienced by each patient. The posted summary should therefore be read as a count-based safety overview, not as a complete clinical characterization of each event.
Safety outcomes are used to identify and describe unwanted events observed during exposure to study treatment. They complement efficacy outcomes rather than answering the same question. The overall study objective included safety, efficacy and pharmacokinetics, but the primary outcome stated in the registry is safety-focused. The registry’s description does not establish that any reported event was caused by treatment; an event occurring after treatment starts is treatment-emergent by the stated definition, while treatment-related events are listed as a separate category.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports 13 patients with serious adverse events among 60 at risk in the alglucosidase alfa group, and 6 among 30 at risk in the placebo group. It also reports an overall figure of 19 among 90. These are the posted safety counts. They describe the number of patients recorded with serious adverse events in each group; they do not indicate how many separate serious events occurred, because a patient may have experienced more than one event and the presented counts are patient-based.
The group denominators matter when reading these counts. The alglucosidase alfa and placebo safety groups differ in size, so the raw counts alone do not provide a like-for-like comparison of event frequency. The figures are not adjusted for follow-up, baseline characteristics or other factors. A comparative estimate with uncertainty, such as a confidence interval, would help express how compatible the observed data are with a range of possible differences between groups. Such an estimate is not reported in the registry record in the registry record. No new comparison should be inferred by subtracting counts or calculating percentages from the posted figures.
The registry also lists Abdominal pain upper, Hypersensitivity, Diverticulitis, Fall, Humerus fracture and Flank pain among the top serious adverse event terms, each reported as 3.3%. These terms identify reported event types; the list does not establish that treatment caused them. The event-term percentages should be interpreted as registry-reported summaries. The denominator and method used for each term are not described in the registry record, so they should not be converted into event counts or treated as a complete account of serious events.
The primary outcome covers several safety categories. The registry information does not reduce that endpoint to one group comparison, and the posted material described in the registry record does not provide a single inferential result for it. No p-value, confidence interval or adjusted treatment contrast for the primary safety outcome is reported in the registry record. The record’s overall objective includes efficacy, but efficacy results are not presented in the registry record. The secondary outcomes named in the registry are also not accompanied by results.
These limits mean that the reported counts support a description of the safety observations recorded during the trial, not a definitive estimate of a treatment effect on safety. In general, an observed difference between groups can reflect random variation as well as a real difference. Precision depends on the amount of information and the variability in the observations; a point estimate without an uncertainty interval does not show how wide that uncertainty may be. The registry record does not report the analysis methods needed to assess those matters for the primary outcome.
The figures also apply to the safety population as defined by the registry: patients who received any amount of study treatment, analyzed according to the treatment actually received. This is distinct from an analysis based on original randomized assignment. The registry does not report a separate intention-to-treat result in the registry record. Accordingly, the serious-event counts should be described as safety-population results, not assumed to represent every possible analysis of the randomized study.
9. Statistical Methods Explained
Clinical trial results can be summarized in different ways depending on the outcome. A count of patients who experienced an event is a binary-outcome summary: each patient is classified according to whether the event was recorded or not for the specified outcome and period. The primary outcome uses participant counts and covers treatment-emergent adverse events during weeks 0-78. That format is appropriate for reporting how many patients were recorded with an event, but it does not preserve all details about event timing, recurrence or severity.
What does a participant count tell the reader?
A count is the number of patients meeting the outcome definition. Its meaning depends on the population being counted, the event definition and the observation period. Here, the safety population includes patients who received any amount of study treatment, and patients are assigned to the group of the treatment they actually received for safety analysis. The reported at-risk counts give the group denominators associated with the posted serious adverse event results. A count is not a rate per unit of follow-up, and the registry entries do not give person-time for these results.
When comparing groups, raw counts should not be interpreted without considering the number of patients at risk in each group. In general, a proportion expresses the count relative to its denominator, while a risk difference or relative measure compares outcome frequency between groups. Those comparisons require clear outcome definitions and appropriate uncertainty estimates. The registry entries do not provide a calculated comparative estimate or confidence interval for the primary safety endpoint, so one should not be reconstructed from the counts.
Why does an uncertainty interval matter?
In general, a confidence interval expresses the precision of an estimate by giving a range of values compatible with the observed data under the assumptions of the statistical method. A narrow interval indicates greater precision than a wide interval, although interpretation also depends on the clinical question and analysis. The registry entries do not report a confidence interval for the serious adverse event comparison. Without one, the counts alone cannot communicate the full uncertainty around a group contrast.
A p-value, in general, measures how unusual data at least as incompatible with a specified null hypothesis would be under that hypothesis and the chosen statistical model. It is not the probability that the null hypothesis is true, and it does not measure the size or clinical importance of an effect. The registry record does not report a p-value for the primary safety outcome in the registry record. A p-value should not be reverse-engineered from participant counts when the analysis method and necessary details are not reported.
How should the sample-size illustration be understood?
The stated sample-size assumptions describe a planning illustration based on an observed effect, rather than the trial’s original design justification. The illustration uses a two-sided significance level of 0.05, equal allocation between arms and a normal approximation; it considers detecting a stated fraction of the observed effect. The stated convention is participants per arm for 80% power for non-time-to-event outcomes. These are planning assumptions, not evidence that the trial had a particular probability of detecting an effect and not a report of observed power.
In general, a sample-size calculation asks how much information a future study would need to detect a prespecified effect under chosen assumptions. It depends on the outcome, the effect size, variability or event frequency, significance level, allocation, and the desired power. A planning calculation based on an effect observed in a completed study is illustrative: the observed effect may itself be imprecise, and choosing a fraction of it is a design scenario rather than a claim about the true effect. The sample-size justification used to plan this trial is not reported in the registry record.
What does over-dispersion mean in general?
For count outcomes, some statistical models assume that the variability follows a particular relationship to the mean. Over-dispersion means that observed variability is greater than the model assumes. If it is ignored, standard errors may be too small and confidence intervals or tests may appear more precise than warranted. This is a general statistical consideration, not a reported feature of this trial’s analysis. The registry entries do not identify a count model or an over-dispersion assessment for the safety results.
Likewise, general methods for binary outcomes include models that estimate group-specific risks and contrasts while accounting for relevant design features. Whether adjustment is appropriate depends on the prespecified analysis and available data. The statistical model, covariate adjustment, handling of repeated events and any sensitivity analyses are not reported in the registry record. The posted participant counts therefore should not be described as adjusted estimates.
Why distinguish categories and event terms?
The registry groups safety summaries into adverse events, serious adverse events, treatment-related adverse events and infusion-associated reactions. These are not interchangeable outcomes. An event may be classified by seriousness, assessed for relation to treatment, and also considered an infusion-associated reaction according to the study’s definitions. The detailed operational definitions for these classifications are not reported in the registry record. The registry’s summary should be read using its stated categories rather than assuming that each category is mutually exclusive.
Event terms offer another level of description. The listed serious-event terms name types of reported events, while the participant counts summarize patients with serious events overall. The term percentages do not supply enough information to reconstruct patient counts, nor do they show whether patients had multiple terms. No such conversion or assumption is warranted. General interpretation of adverse-event data also distinguishes observation from causation: an event recorded after treatment begins is temporally treatment-emergent, but that timing alone does not establish that treatment caused it.
10. Multiplicity, Missing Data and Analysis Populations
The primary outcome is described as a safety summary with several categories: adverse events, serious adverse events, treatment-related adverse events and infusion-associated reactions. The registry information also indicates that the primary outcome has several categories or time points and is not reduced to one comparison. When many outcomes, categories or time points are examined, the chance of at least one apparently notable finding can increase. In general, multiplicity procedures may be prespecified to address that issue, or results may be presented as descriptive. The multiplicity strategy for this trial is not reported in the registry record.
The registry lists secondary outcomes for percent predicted proximal muscle strength of the lower limbs measured by quantitative muscle testing, and physical-component health-related quality of life measured by the MOS Short Form-36 Health Survey. The statistical plan for these outcomes, the intended hierarchy among outcomes, and any adjustment for multiple comparisons are not reported in the registry record. Their inclusion in the registry does not establish which analyses were confirmatory or how their results should be interpreted.
For missingness, the registry states that missing or invalid safety or resource-utilization data were not replaced. This means no replacement was made for those data under the stated approach; it does not explain how much data were missing, why they were missing, or how missing observations affected each analysis. The method for handling missing efficacy or secondary-outcome data is not reported in the registry record. No imputation strategy or sensitivity analysis should be assumed.
The safety population includes patients who received any amount of study treatment, with group assignment based on treatment actually received. This definition differs from an analysis retaining all patients according to randomized assignment. A separate intention-to-treat analysis, per-protocol analysis, or comparison of those analysis populations is not reported in the registry record. The details of randomization, withdrawals and follow-up completeness are also not reported in the registry record.
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 |
|---|---|---|---|
| Alglucosidase Alfa | 13 | 60 | 21.7% |
| Placebo | 6 | 30 | 20.0% |
| Overall | 19 | 90 | 21.1% |
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 |
|---|---|
| Alglucosidase Alfa | 60 of 60 |
| Placebo | 30 of 30 |
| Overall | 90 of 90 |
Most frequent serious adverse event terms
| Event term | Organ system | Alglucosidase Alfa | Placebo | Overall |
|---|---|---|---|---|
| Hypersensitivity | Immune system disorders | 2 of 60 | 0 of 30 | 2 of 90 |
| Fall | Injury, poisoning and procedural complications | 1 of 60 | 1 of 30 | 2 of 90 |
| Humerus fracture | Injury, poisoning and procedural complications | 1 of 60 | 1 of 30 | 2 of 90 |
| Coronary artery disease | Cardiac disorders | 1 of 60 | 0 of 30 | 1 of 90 |
| Supraventricular tachycardia | Cardiac disorders | 1 of 60 | 0 of 30 | 1 of 90 |
| Abdominal pain | Gastrointestinal disorders | 1 of 60 | 0 of 30 | 1 of 90 |
| Abdominal pain upper | Gastrointestinal disorders | 0 of 60 | 1 of 30 | 1 of 90 |
| Chest discomfort | General disorders | 1 of 60 | 0 of 30 | 1 of 90 |
| Non-cardiac chest pain | General disorders | 1 of 60 | 0 of 30 | 1 of 90 |
| Diverticulitis | Infections and infestations | 0 of 60 | 1 of 30 | 1 of 90 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Alglucosidase Alfa | Placebo | Overall |
|---|---|---|---|---|
| Fall | Injury, poisoning and procedural complications | 39 of 60 | 20 of 30 | 59 of 90 |
| Nasopharyngitis | Infections and infestations | 25 of 60 | 16 of 30 | 41 of 90 |
| Headache | Nervous system disorders | 24 of 60 | 15 of 30 | 39 of 90 |
| Diarrhoea | Gastrointestinal disorders | 18 of 60 | 13 of 30 | 31 of 90 |
| Hypoacusis | Ear and labyrinth disorders | 20 of 60 | 7 of 30 | 27 of 90 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 18 of 60 | 9 of 30 | 27 of 90 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 15 of 60 | 7 of 30 | 22 of 90 |
| Nausea | Gastrointestinal disorders | 11 of 60 | 10 of 30 | 21 of 90 |
| Back pain | Musculoskeletal and connective tissue disorders | 14 of 60 | 7 of 30 | 21 of 90 |
| Muscle spasms | Musculoskeletal and connective tissue disorders | 14 of 60 | 6 of 30 | 20 of 90 |
Serious events with the highest arm-level rate: abdominal pain upper (3.3%), hypersensitivity (3.3%), diverticulitis (3.3%), fall (3.3%), humerus fracture (3.3%).
The serious adverse event summary reports 13 patients among 60 at risk in the alglucosidase alfa group and 6 among 30 at risk in the placebo group. The registry’s overall figure is 19 among 90. These are counts of patients reported with serious adverse events in the safety summary, not counts of all individual serious events. A patient may have experienced more than one event, and the figures do not describe recurrence or time to event.
The group denominators are unequal, so the counts should not be compared as if the groups were the same size. No additional comparison, percentage or risk estimate is calculated. The registry does not report a confidence interval or statistical test for these figures in the registry record. The serious-event terms listed include Abdominal pain upper, Hypersensitivity, Diverticulitis, Fall, Humerus fracture and Flank pain, each reported as 3.3%. The registry entries do not state the denominator or counting method for those term percentages.
These reports describe events observed during the study and do not establish that treatment caused them. Mortality figures are not reported in the registry record. The safety population and its treatment assignment rule should also be kept in mind: the results cover patients who received any amount of treatment and are grouped according to treatment actually received.
12. What the Result Does, and Does Not, Mean
The posted results establish that the registry recorded serious adverse events in both treatment groups during the study’s safety follow-up. They give patient counts and group denominators, and they list several serious-event terms with their reported percentages. This is useful descriptive information about events observed under the registry’s safety definitions. It does not, by itself, determine whether an event was caused by alglucosidase alfa or placebo.
The figures do not establish a precise comparative effect. The registry entries do not include a confidence interval, p-value or adjusted estimate for the primary safety outcome. The group sizes differ, and a raw count does not account for follow-up time or other differences in the observed patients. In general, statistical uncertainty and design details are needed before drawing a formal conclusion about a group contrast. The registry record does not report enough detail to make that assessment for the primary outcome.
The results also do not answer the study’s full efficacy question. The registry’s overall objective includes efficacy and pharmacokinetics, and it names lower-limb proximal muscle strength and physical-component health-related quality of life as secondary outcomes. Their results are not reported in the registry record. No claim about improvement, lack of improvement, or comparative efficacy follows from the safety counts alone.
Nor do the counts provide a complete description of the safety experience. They do not indicate the number of events per patient, their duration, the timing of onset, or the course of each event. The registry identifies treatment-related adverse events and infusion-associated reactions as categories, but the detailed results for those categories are not included in the registry record. Death counts are not reported in the registry record.
The appropriate reading is therefore bounded: these are registry-posted, count-based safety observations for the stated safety population and study period. They can inform a description of what was recorded, but the registry entries do not support broader causal or efficacy conclusions. Further analysis would require additional outcome results and statistical details that are not reported in the registry record.
13. Why This Trial Matters Statistically
This trial is statistically notable for the distinction between its broad objective and the primary outcome reported in the registry. The overall objective includes safety, efficacy and pharmacokinetics, while the primary outcome is a multi-category summary of treatment-emergent adverse events. Recognizing that distinction prevents safety counts from being mistaken for a functional efficacy result. The registry also names muscle strength and physical-component quality of life as secondary outcomes, but their numerical results are not reported in the registry record.
The trial is described as randomized, double-blind, placebo-controlled and parallel-group, features that define the basic comparison. The registry labels masking as quadruple but does not identify the masked roles. The safety analysis uses a population defined by receipt of any amount of treatment and groups patients according to treatment actually received. That population definition is important because it is not automatically the same as an analysis by original randomized assignment.
The posted serious adverse event counts demonstrate why denominators and uncertainty matter. The groups have different numbers of patients at risk, and the raw counts are not an adjusted or time-based comparison. Without an inferential estimate and its uncertainty, readers should distinguish descriptive reporting from evidence about the size and precision of a treatment contrast. General statistical reasoning cannot fill in an analysis that the registry record does not report.
The trial also provides a useful example of why safety summaries are not a single homogeneous outcome. Seriousness, treatment-relatedness and infusion association are separate classifications, and the record names each as part of the safety summary. Event terms add detail but do not establish causation. Finally, the planning assumptions described for sample-size illustrations should not be confused with the trial’s original design rationale or with observed power. The original sample-size justification is not reported in the registry record.
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 registry entries do not include a single numerical comparison for the primary safety endpoint, an uncertainty interval, or a p-value. The primary outcome contains several categories or time points and is not reduced to one comparison. As a result, the posted serious adverse event counts support description but do not provide a complete inferential account of the primary endpoint.
The safety population is defined by receipt of any amount of treatment, with patients analyzed in the group of treatment actually received. The registry entries do not provide a separate analysis by randomized assignment, details about treatment discontinuation, or follow-up completeness. The amount and reasons for missing data are not reported. Although missing or invalid safety or resource-utilization data were not replaced, that statement alone does not show whether missingness could have affected the observed summaries.
The event-term percentages are reported without a denominator or counting method in the registry record. They should not be converted into counts or interpreted as mutually exclusive categories. The registry also does not report event timing, recurrence, duration or severity details for the listed terms. Mortality figures are not reported in the registry record.
The statistical plan is not reported in the registry record. This includes the model, adjustment strategy, multiplicity approach, prespecified analysis hierarchy, handling of repeated events and sensitivity analyses. The masking label is given, but the masked roles are not. The original sample-size justification is not reported; the stated sample-size assumptions describe planning illustrations rather than the trial’s own design rationale.
Finally, efficacy and secondary-outcome results are not included in the registry record. The registry’s overall objective and listed secondary outcomes do not substitute for numerical results or an analysis plan. These limitations restrict interpretation to the posted safety counts and definitions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Placebo-Controlled Study of Safety and Effectiveness of Myozyme (Alglucosidase Alfa) in Patients With Late-Onset Pompe Disease — NCT00158600.
- Registry record last updated 2015-04-28; status last verified 2015-04.
- Results first posted 2010-07-23.
- Data retrieved from ClinicalTrials.gov on 2026-11-09.
- Group sizes, means, event counts and sponsor-reported p-values are reproduced as posted. Effect estimates, confidence intervals and p-values not attributed to the sponsor are independently calculated from the posted summary statistics.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT00158600 is a completed phase 3 randomized, double-blind, placebo-controlled, parallel-group study of alglucosidase alfa versus placebo in patients with late-onset Pompe disease. Enrollment was 90. The registry lists the start as 2005-09 and primary completion as 2007-09. Alglucosidase alfa was infused intravenously at 20 milligrams per kilogram every other week for 78 weeks; placebo was infused intravenously every other week for 78 weeks.
The primary outcome summarizes treatment-emergent adverse events during weeks 0-78 in the safety population. Serious adverse events were reported for 13 patients among 60 at risk in the alglucosidase alfa group and 6 among 30 at risk in the placebo group; the registry’s overall figure is 19 among 90. The registry entries do not include a single inferential comparison for the primary outcome, efficacy results, or mortality figures. The statistical plan and original sample-size justification are not reported in the registry record.