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 High Dose or High Dose Frequency Study of Alglucosidase Alfa (NCT00483379), a randomized phase 4 study. The primary outcome is Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00483379 |
| Phase | Phase 4 |
| Condition | Pompe Disease, Glycogen Storage Disease Type II (GSD-II), Glycogenesis 2 Acid Maltase Deficiency |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 13 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Genzyme, a Sanofi Company |
| Results posted | Yes |
2. Clinical Question
Population
Participants with Pompe disease who had not demonstrated an optimal response to the standard regimen of alglucosidase alfa. The primary endpoint population was narrower: participants enrolled because of decline in respiratory function while on standard treatment.
Intervention
Alglucosidase alfa 20 mg/kg every week for 52 weeks, described in the record as the frequent-dose arm.
Comparator
Alglucosidase alfa 40 mg/kg every other week for 52 weeks, described as the high-dose arm. Both groups received alglucosidase alfa, with different dose size and frequency.
Primary question
At Week 52, did participants enrolled for respiratory decline have an improvement, no change, or worsening in ventilator use compared with baseline? Each participant served as their own control. The registry record describes this outcome but does not report its results.
The study focused on people whose clinical response to standard treatment was considered suboptimal or who had experienced clinical decline in cardiac, respiratory, and/or motor function before study entry. The primary outcome description is specifically about respiratory decline, measured through change in ventilator use. It does not represent a comparison of all enrolled participants on a single continuous respiratory measure: the outcome categories are improvement, no change, and worsening. The registry record does not report how many participants in the primary endpoint population fell into each category.
The clinical question therefore has two parts. One is whether either alternative schedule was associated with a change in ventilator use relative to each participant’s baseline. The other is whether the pattern of those changes differed between the two randomized treatment groups. The registry record does not provide the endpoint counts needed to assess either part. It also does not report a treatment-effect estimate or uncertainty interval for this comparison.
3. Trial Design
This was a completed phase 4, randomized, open-label, parallel-group study of alglucosidase alfa in people with Pompe disease. Enrollment was 13 participants. The two regimens were alglucosidase alfa 20 mg/kg every week and alglucosidase alfa 40 mg/kg every other week; each was given for 52 weeks. The record describes the first as the frequent-dose arm and the second as the high-dose arm. Masking was none.
Participants had not demonstrated an optimal response to the standard regimen of 20 mg/kg every other week after a minimum of 6 months of treatment immediately before study entry. The record says enrollment was based on clinical decline or suboptimal response in cardiac, respiratory, and/or motor function parameters before the study. The primary endpoint population comprised participants enrolled due to decline in respiratory function while on standard treatment. The number of participants in that endpoint population is not reported in the registry record.
Random allocation means that the treatment regimen was assigned by chance rather than selected solely by the participant or treating clinician. In general, randomization can support a fairer comparison of groups, although with a small enrollment the groups may still differ in relevant characteristics by chance. Because masking was none, participants and study personnel were not blinded to the assigned schedule. In general, awareness of treatment can influence reporting, care, and assessment, particularly for outcomes requiring judgment. The registry record does not report additional safeguards for outcome assessment.
The study began in 2007-05 and primary completion was 2009-12; its status is completed. Listed secondary outcomes include left ventricular mass and its Z-score, left ventricular mass index, ventilator use at the last assessment, manual muscle testing total score, and gross motor function measure results. The record lists 15 outcome measures but reports 0 analyses. Detailed allocation procedures, an analysis population definition, and the statistical analysis plan are not reported in the registry record.
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: Fourteen participants were screened and enrolled; however, one withdrew before receiving any study infusions due to the burden of weekly trips to the medical center.
Treatment Period
| Stage | Alglucosidase Alfa 20 mg/kg Every Week | Alglucosidase Alfa 40 mg/kg Every Other Week |
|---|---|---|
| STARTED | 6 | 7 |
| COMPLETED | 4 | 7 |
| NOT COMPLETED | 2 | 0 |
Reasons for not completing this period:
| Reason | Alglucosidase Alfa 20 mg/kg Every Week | Alglucosidase Alfa 40 mg/kg Every Other Week |
|---|---|---|
| Adverse Event | 1 | 0 |
| Withdrawal by Subject | 1 | 0 |
Extension Period
| Stage | Alglucosidase Alfa 20 mg/kg Every Week | Alglucosidase Alfa 40 mg/kg Every Other Week |
|---|---|---|
| STARTED | 1 | 2 |
| COMPLETED | 1 | 2 |
| NOT COMPLETED | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: All participants who enrolled due to decline in respiratory function while on standard treatment.
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 20 mg/kg Every Week | Alglucosidase Alfa 40 mg/kg Every Other Week | Total |
|---|---|---|---|---|
| Participants analysed | 6 | 7 | 13 | |
| Age, Continuous | years | 23.3 (SD 27.75) | 16.8 (SD 15.56) | 19.8 (SD 21.29) |
| Age, Customized: <18 years | participants | 4 | 5 | 9 |
| Age, Customized: >= 18 and <=65 years | participants | 2 | 2 | 4 |
| Age, Customized: >65 years | participants | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 2 | 3 | 5 |
| Sex: Female, Male: Male | Participants | 4 | 4 | 8 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 6 | 6 | 12 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Life-stage of Disease Onset: Infantile-onset Pompe Disease | participants | 4 | 5 | 9 |
| Life-stage of Disease Onset: Late-onset Pompe Disease | participants | 2 | 2 | 4 |
| Parameter in Clinical Decline: Cardiac | participants | 0 | 0 | 0 |
| Parameter in Clinical Decline: Respiratory | participants | 1 | 1 | 2 |
| Parameter in Clinical Decline: Motor Skills | participants | 5 | 6 | 11 |
| Cross-Reactive Immunologic Material (CRIM) Assay Result: Positive | participants | 0 | 3 | 3 |
| Cross-Reactive Immunologic Material (CRIM) Assay Result: Negative | participants | 1 | 0 | 1 |
| Cross-Reactive Immunologic Material (CRIM) Assay Result: Unknown | participants | 5 | 4 | 9 |
6. Primary Endpoint
The registry lists the primary outcome as Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment, measured over Baseline, Week 52.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is participants’ efficacy response during treatment compared with baseline among those who entered the study because of respiratory decline while receiving standard treatment. Respiratory change is assessed through ventilator use. The endpoint categories are improvement, no change, and worsening: improvement means less ventilator support, while worsening means more ventilator support. Each participant served as their own control, so the endpoint asks how ventilator use at Week 52 compared with that participant’s baseline.
Ventilator use is used in this endpoint as a measure of change in respiratory support. Categorizing the change makes the clinical direction of the response easy to distinguish, but it does not show the size of a change within a category. For example, the category labels alone cannot indicate how much ventilator use changed, or whether changes varied substantially among participants assigned to the same category. The registry record does not report the category counts or the exact rule used to classify a participant’s change.
The endpoint’s baseline comparison is not, by itself, a randomized comparison between regimens. To assess whether the response differed by assigned regimen, the category distribution would need to be reported for the relevant randomized groups, along with an appropriate comparative analysis. Those results are not reported in the registry record. The record also does not report a treatment-effect estimate or confidence interval for this endpoint.
As general statistical context, an endpoint should be interpreted in light of its definition, the population in which it was assessed, and the way missing observations were handled. Those details matter for understanding what the outcome represents and how representative it is of all randomized participants. The registry record does not report the missing-data handling for this outcome.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question, treatment schedules, and the categories used for the primary respiratory outcome, but it does not report the primary endpoint results. It therefore does not show how many participants improved, had no change, or worsened in ventilator use at Week 52. Nor does it report the size or precision of a comparison between the two regimens. No conclusion about a difference in respiratory response can be drawn from the outcome description alone.
The enrollment was 13 participants across a randomized, parallel-group study. This is a small study population, so any group comparison would need to be read with attention to uncertainty. In general, estimates from small samples can be imprecise, and a few participants’ outcomes may materially affect the apparent distribution. The record does not provide outcome counts or confidence intervals that would allow the precision of the primary result to be evaluated.
The primary outcome uses each participant’s baseline as a reference, but the study’s treatment question is comparative: it concerns two alternative schedules. A within-participant change can describe what happened after treatment began, but it does not alone separate treatment-related change from other influences over time. Randomization supports comparison of assigned groups in general; the result still depends on the reported group data, analysis, and handling of participants without endpoint observations. These details and the primary analysis are not reported in the registry record.
The listed secondary outcomes cover cardiac, respiratory, and motor-function measures. Their inclusion indicates that the study considered multiple dimensions of clinical response, but the registry record does not report their results or analyses. The number of outcome measures is 15, while the record lists 0 analyses. Without outcome estimates, uncertainty measures, and an account of which analyses were planned, the magnitude, consistency, and precision of any observed changes cannot be assessed.
The safety record reports serious adverse event counts for the treatment groups and separate extension groups. Those figures describe recorded events within the registry categories; they do not establish comparative risk without further detail about follow-up, event definitions, and the populations contributing data. Death counts are not reported in the registry record. The listed serious adverse event terms and percentages likewise should not be treated as evidence of a causal relationship to a particular regimen.
In general, statistical interpretation distinguishes the direction of an estimate from its precision and from whether the analysis supports a causal comparison. Here, the outcome results and inferential statistics needed for that assessment are not reported in the registry record. The study design can be described, but the registry information does not support a quantitative conclusion about efficacy or a comparative conclusion about safety.
9. Statistical Methods Explained
What does randomization contribute?
Randomization assigns participants to treatment groups by chance. In general, this can reduce systematic differences between groups at the start of a study and makes the randomized comparison a useful way to assess differences associated with assigned treatment. It does not guarantee that groups are identical, particularly in a small study. The record identifies the study as randomized but does not report the random sequence method, concealment procedures, or group-specific baseline characteristics.
This study used a parallel-group design: participants were assigned to one of two alglucosidase alfa schedules, rather than moving between schedules as part of a crossover design. The comparison is between 20 mg/kg every week and 40 mg/kg every other week, both continued for 52 weeks. Because both the dose size and dosing frequency differ, the study compares the regimens as packages. The registry record does not describe a design that isolates the effect of dose size from the effect of frequency.
What does open-label mean?
Open-label means the masking field is none: treatment assignment was not concealed from participants and study personnel through blinding. In general, knowledge of assignment can influence subjective reporting, treatment decisions, or assessment. The extent of such influence depends on the outcome and procedures used. The registry record does not report whether outcome assessors were otherwise independent or whether any specific steps were used to limit assessment bias.
How should the baseline comparison be understood?
The primary endpoint describes change from baseline in ventilator use and says that each participant served as their own control. In general, a within-participant comparison can describe how a measure changed over time for the same person. It does not by itself provide the randomized treatment contrast, because changes over time may reflect influences other than assigned treatment. A comparison between randomized groups is needed to assess whether outcomes differed by regimen, and the registry record does not report that analysis.
The outcome is categorized as improvement, no change, or worsening. In general, categorization can make the direction of change straightforward to communicate, but it can discard information about the amount of change. Interpretation also depends on the definitions or thresholds used to assign categories. The registry record does not report those classification rules or the counts in each category.
What do effect estimates and confidence intervals tell readers?
In general, an effect estimate describes the observed difference or association under a specified analysis. A confidence interval describes the range of values compatible with the data and the method used, and conveys the estimate’s precision. A confidence interval does not remove uncertainty or show that every value within it is equally plausible. The registry record does not report an effect estimate, confidence interval, or other inferential statistic for the primary endpoint. It is therefore not possible to assess the direction or precision of the randomized comparison from the registry entry.
Why does a small enrollment matter?
In general, a small sample can produce unstable estimates: the outcomes of a small number of participants can have a substantial influence on group summaries. It can also leave broad uncertainty around comparisons. This is a reason to interpret estimates cautiously, not a basis for assuming what the results would have been. Enrollment was 13, but group-level primary outcome counts and precision estimates are not reported.
The record includes a general description of assumptions for planning illustrations based on an observed effect. Such illustrations are not the trial’s own design justification and are not observed power. No planning-illustration values are reported in the registry record. The sample-size justification and the assumptions used to design the trial are not reported in the registry record.
What can be inferred from the reported safety counts?
In general, an event count describes how many participants in a stated group had an event under the registry’s reporting convention. It is not, on its own, a causal estimate of the risk attributable to treatment. Interpreting group counts requires clarity about the event definition, time at risk, follow-up completeness, and whether participants could contribute to more than one event category. The registry lists serious and other adverse event counts for treatment and extension groups, but does not report all of these contextual details. A comparison of counts should not be treated as a definitive comparison of safety.
What statistical analysis details are missing?
The record lists 0 analyses. It does not report the statistical test or model for the primary outcome, an adjustment strategy, the treatment-effect measure, confidence intervals, missing-data procedures, or a prespecified analysis population. It also does not report interim analyses or a statistical analysis plan. Without those details and the outcome results, readers cannot verify how the endpoint was analyzed or assess whether the reported analysis followed a prespecified approach. These are limits on interpretation, not evidence that a particular method was or was not used.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 15 outcome measures, including multiple cardiac, respiratory, and motor-function measures. When a study assesses multiple outcomes or makes multiple comparisons, there is a general possibility that some results may appear notable by chance. Statistical methods can address this issue, but the appropriate approach depends on which outcomes were primary, which analyses were planned, and the intended interpretation of secondary findings. The registry record does not report a multiplicity strategy or a statistical analysis plan. It lists 0 analyses, so the record does not support an assessment of how multiplicity was handled.
The primary endpoint description gives the time points as baseline and Week 52, while a listed secondary outcome refers to ventilator use at the last assessment, approximately Week 52. The registry record does not report how those time points were reconciled in the analysis or how the endpoint categories were defined. It also does not report whether any interim analyses were performed.
Missing-data handling is important because participants without an endpoint measurement may differ from those with one. In general, an analysis should state how such observations were addressed and which participants were included. The registry record does not report the number of participants with missing primary outcome data, the reasons for missingness, or the method used to handle it.
The primary endpoint population is described as participants enrolled due to respiratory decline on standard treatment, whereas overall enrollment was 13. The number included in the primary endpoint analysis and the rules for inclusion in that analysis are not reported in the registry record. The analysis populations for secondary outcomes are also not reported. These omissions prevent readers from determining the denominator for endpoint results or how the analysis relates to all randomized participants.
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 |
|---|---|---|---|
| Treatment: Alglucosidase Alfa 20 mg/kg Every Week | 2 | 6 | 33.3% |
| Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week | 1 | 7 | 14.3% |
| Extension: Alglucosidase Alfa 20 mg/kg Every Week | 1 | 1 | 100.0% |
| Extension: Alglucosidase Alfa 40 mg/kg Every Other Week | 0 | 2 | 0.0% |
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 |
|---|---|
| Treatment: Alglucosidase Alfa 20 mg/kg Every Week | 6 of 6 |
| Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week | 7 of 7 |
| Extension: Alglucosidase Alfa 20 mg/kg Every Week | 1 of 1 |
| Extension: Alglucosidase Alfa 40 mg/kg Every Other Week | 1 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment: Alglucosidase Alfa 20 mg/kg Every Week | Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week | Extension: Alglucosidase Alfa 20 mg/kg Every Week | Extension: Alglucosidase Alfa 40 mg/kg Every Other Week |
|---|---|---|---|---|---|
| Pneumonia | Infections and infestations | 2 of 6 | 0 of 7 | 0 of 1 | 0 of 2 |
| Supraventricular tachycardia | Cardiac disorders | 0 of 6 | 1 of 7 | 0 of 1 | 0 of 2 |
| Dysphagia | Gastrointestinal disorders | 0 of 6 | 1 of 7 | 0 of 1 | 0 of 2 |
| Device related infection | Infections and infestations | 0 of 6 | 1 of 7 | 0 of 1 | 0 of 2 |
| Fibula fracture | Injury, poisoning and procedural complications | 0 of 6 | 0 of 7 | 1 of 1 | 0 of 2 |
| Weight decreased | Investigations | 0 of 6 | 1 of 7 | 0 of 1 | 0 of 2 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 6 | 0 of 7 | 0 of 1 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Treatment: Alglucosidase Alfa 20 mg/kg Every Week | Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week | Extension: Alglucosidase Alfa 20 mg/kg Every Week | Extension: Alglucosidase Alfa 40 mg/kg Every Other Week |
|---|---|---|---|---|---|
| Upper respiratory tract infection | Infections and infestations | 2 of 6 | 3 of 7 | 0 of 1 | 0 of 2 |
| Diarrhoea | Gastrointestinal disorders | 2 of 6 | 2 of 7 | 0 of 1 | 0 of 2 |
| Pyrexia | General disorders | 2 of 6 | 2 of 7 | 0 of 1 | 0 of 2 |
| Fall | Injury, poisoning and procedural complications | 1 of 6 | 1 of 7 | 1 of 1 | 1 of 2 |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | 1 of 6 | 1 of 7 | 0 of 1 | 1 of 2 |
| Dermatitis diaper | Skin and subcutaneous tissue disorders | 1 of 6 | 2 of 7 | 0 of 1 | 0 of 2 |
| Tachycardia | Cardiac disorders | 1 of 6 | 1 of 7 | 0 of 1 | 0 of 2 |
| Abdominal pain upper | Gastrointestinal disorders | 2 of 6 | 0 of 7 | 0 of 1 | 0 of 2 |
| Nausea | Gastrointestinal disorders | 1 of 6 | 0 of 7 | 1 of 1 | 0 of 2 |
| Vomiting | Gastrointestinal disorders | 2 of 6 | 0 of 7 | 0 of 1 | 0 of 2 |
Serious events with the highest arm-level rate: fibula fracture (100.0%), pneumonia (33.3%), respiratory failure (16.7%), supraventricular tachycardia (14.3%), dysphagia (14.3%).
For the treatment group receiving alglucosidase alfa 20 mg/kg every week, the registry reports 2 participants affected by serious adverse events among 6 at risk. For the group receiving 40 mg/kg every other week, it reports 1 affected among 7 at risk. In the extension groups, the corresponding serious adverse event counts are 1 among 1 at risk for the weekly regimen and 0 among 2 at risk for the every-other-week regimen.
The record also reports other adverse events affecting 6 of 6 participants in the weekly treatment group and 7 of 7 in the every-other-week treatment group. In the extension groups, the other adverse event counts are 1 of 1 and 1 of 2, respectively. These are counts within the registry’s adverse-event categories; the record does not give enough detail to interpret them as comparative risks over equivalent follow-up.
The listed serious adverse event terms and percentages are Fibula fracture, 100.0%; Pneumonia, 33.3%; Respiratory failure, 16.7%; Supraventricular tachycardia, 14.3%; Dysphagia, 14.3%; and Device related infection, 14.3%. The registry record does not explain the denominators or event-specific counting conventions for these percentages. Death counts are not reported in the registry record. These safety entries describe reported events; they do not establish that treatment caused an event or that one regimen is safer.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a small randomized, open-label phase 4 study compared two alternative alglucosidase alfa schedules in people with Pompe disease who had not responded optimally to standard treatment. It also defines a primary respiratory outcome based on change in ventilator use from baseline to Week 52. These design details clarify what the study set out to examine.
The record does not report the primary endpoint results. It therefore cannot establish the number of participants who improved, remained unchanged, or worsened, nor whether those categories differed between randomized groups. It also does not provide an effect estimate or confidence interval. Without these results, the direction, size, and precision of any treatment comparison are not reported in the registry record.
The listed secondary outcomes indicate that the study considered cardiac, respiratory, and motor-function measures, but their results are not reported in the registry record. The adverse event counts provide descriptive safety information, but do not by themselves establish a causal relationship or support a reliable comparison between regimens. Follow-up and analysis details that would aid interpretation are not reported.
In general, a study’s design and its results answer different questions. Randomization describes how treatment assignment was made; it does not substitute for outcome data. Likewise, an endpoint description explains what was measured but does not reveal what happened. The record supports describing the trial and the outcomes it intended to assess, but not a conclusion about whether either regimen produced a particular clinical result.
The study should therefore be read as an incomplete account of the evidence needed for a quantitative efficacy or comparative safety interpretation. The absence of reported results in the registry record is not evidence that no change occurred, that the regimens were equivalent, or that a treatment effect was absent.
13. Why This Trial Matters Statistically
This trial raises a useful statistical distinction between a within-participant change and a randomized comparison. The primary outcome asks how ventilator use at Week 52 compared with baseline for participants enrolled because of respiratory decline. That change is clinically descriptive, but the treatment question is whether outcomes differed between the two assigned schedules. The registry record does not report the group outcome counts or a comparative estimate needed to answer that question.
The study also illustrates why the definition of an endpoint matters. Improvement, no change, and worsening convey direction, but without classification rules and counts readers cannot assess how participants were categorized or how responses were distributed. In general, a categorical outcome may be easier to interpret than a complex measurement, while concealing differences in the size of changes within categories.
Both treatment arms used the same biological intervention, but differed in dose and frequency. Consequently, any randomized contrast would concern the complete schedules rather than dose or frequency in isolation. The open-label design and small enrollment are also relevant when considering the uncertainty and possible influences on outcome assessment. These design features do not determine the result, but they shape how a result should be interpreted.
Finally, the record lists multiple outcome measures and descriptive safety counts without reporting analyses or primary results. This underscores that a registry entry can document study aims and design while leaving the statistical evidence needed for interpretation unavailable. Careful readers distinguish the planned question, the observed data, and the conclusions those data can support.
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 interpreting this trial from the registry record is that primary and secondary outcome results are not reported. The primary respiratory endpoint has no reported category counts, treatment comparison, effect estimate, or uncertainty interval. The registry record therefore does not permit an assessment of the direction or precision of the primary result.
Enrollment was 13, and the number of participants in the primary endpoint population is not reported. The record does not identify the analysis population or explain how participants without endpoint measurements were handled. This makes it impossible to determine the denominator for the primary outcome or whether the reported population, if any, corresponded to all randomized participants.
The study was open-label, and the record does not describe assessor masking or other measures to limit bias in outcome assessment. It also does not report the rules used to classify ventilator-use changes as improvement, no change, or worsening. The relationship between the primary endpoint timing and the secondary measure at the last assessment, approximately Week 52, is not explained.
Although 15 outcome measures are listed, the record lists 0 analyses and does not report a statistical analysis plan, multiplicity approach, or interim analyses. The sample-size justification is not reported in the registry record. Safety counts are available, but details on follow-up, event definitions, and event-specific denominators are not reported. These limitations restrict interpretation of efficacy and comparative safety; they do not establish what the unreported results would have shown.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: High Dose or High Dose Frequency Study of Alglucosidase Alfa — NCT00483379.
- Registry record last updated 2014-03-07; status last verified 2014-02.
- Results first posted 2011-04-22.
- 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
This completed phase 4 trial was randomized, open-label, and parallel-group, with enrollment of 13 participants. It compared alglucosidase alfa 20 mg/kg every week with 40 mg/kg every other week, each administered for 52 weeks. Participants had not demonstrated an optimal response to standard treatment. The study began in 2007-05, and primary completion was 2009-12.
The primary outcome assessed change in ventilator use from baseline to Week 52 among participants enrolled because of respiratory decline on standard treatment. The outcome categories were improvement, no change, and worsening, with each participant serving as their own control. The registry record lists 15 outcome measures and 0 analyses. It does not report primary or secondary outcome results, the statistical analysis plan, missing-data procedures, or the analysis population. Serious and other adverse event counts are reported by treatment and extension group, but the context needed for a comparative safety interpretation is limited.