This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis (NCT02473965), a randomized phase 2 study. The primary outcome is Percent of Subjects Achieving a 50% or Greater Reduction in CS Dose (Prednisone or Equivalent) From Baseline to Week 39.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02473965 |
| Phase | Phase 2 |
| Condition | Myasthenia Gravis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 60 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Grifols Therapeutics LLC |
| Results posted | Yes |
2. Clinical Question
Population
People with generalized myasthenia gravis who were dependent on corticosteroids. The primary endpoint description defines the modified intention-to-treat population as all randomized participants who received at least 1 dose of study medication, classified according to randomized treatment. The registry reports enrollment of 60 participants but does not report how many were included in this endpoint population.
Intervention
IGIV-C, or Immune Globulin (Human), 10% Caprylate/Chromatography Purified. The arm description specifies a loading dose of 2 g/kg and a maintenance dose of 1 g/kg. The registry record does not provide further details about the timing or duration of individual administrations.
Comparator
Placebo, described as 0.9% sodium chloride injection, USP or equivalent. The study is described as randomized, double-blind, and placebo-controlled. The record identifies quadruple masking but does not specify which participant, clinician, or study roles were masked.
Primary question
What percentage of participants achieved a reduction of at least 50% in daily corticosteroid dose, measured from baseline to Week 39? The endpoint concerns prednisone or an equivalent corticosteroid dose. The registry record does not report the arm-specific percentages or a treatment comparison for this endpoint.
The clinical aim was to evaluate IGIV-C as a corticosteroid-sparing agent in corticosteroid-dependent participants with generalized myasthenia gravis. This is a question about reducing the prescribed corticosteroid dose while participants remain under study, rather than a question about a single symptom score. The study also listed mean percent change in daily corticosteroid dose from baseline to Week 39 and median time to first episode of myasthenia gravis worsening as secondary outcomes. Results for those outcomes are not reported in the registry record.
The registry identifies Myasthenia Gravis as the condition, describes the study as multicenter, and lists Grifols Therapeutics LLC as sponsor. The phase is phase 2. Those design and context details frame the question, but do not establish what the endpoint results were or how precisely the groups differed. Without the outcome counts or percentages, the treatment effect cannot be assessed from the registry record.
3. Trial Design
NCT02473965 is a completed, randomized, parallel-group, phase 2 study described as multicenter, double-blind, and placebo-controlled. The registry lists enrollment of 60 participants. The study began in 2015-06, and primary completion was recorded as 2019-02. These dates describe the registry record and do not indicate when results were posted.
Participants were assigned to IGIV-C or placebo. The intervention arm description specifies an IGIV-C loading dose of 2 g/kg and a maintenance dose of 1 g/kg. The comparator was 0.9% sodium chloride injection, USP or equivalent. The registry classifies masking as quadruple, but does not explain which groups of people were masked or how masking was maintained. Details of the randomization procedure, including sequence generation and allocation concealment, are not reported in the registry record.
The primary endpoint was the percentage of participants who achieved a 50% or greater reduction in corticosteroid dose from baseline to Week 39. The dose was prednisone or an equivalent corticosteroid. The primary endpoint description says that the average daily dose was derived at scheduled visits from the prescribed dose and the time interval, taking prescribed dose changes between visits into account. Baseline was the last non-missing measurement before the first study dose.
The primary endpoint population was the modified intention-to-treat population: randomized participants who received at least 1 dose of study medication. Participants were analyzed according to their randomized treatment. The registry reports that the safety groups included 30 participants at risk in each arm. It does not report the endpoint population size by arm, or whether every enrolled participant was randomized and treated.
The record lists two secondary outcomes: mean percent change in daily corticosteroid dose from baseline to Week 39, and median time to the first episode of myasthenia gravis worsening. Outcome results, additional details of follow-up, and the full statistical analysis plan are not reported in the registry record. The description of a completed phase 2 trial does not by itself provide evidence about the magnitude or precision of the primary treatment comparison.
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: Subjects had symptoms at screening controlled by corticosteroids (CS), had been dependent on systemic CS for at least the preceding 3 months and had received a stable dose of CS for at least 1 month immediately prior to the screening visit.
Overall Study
| Stage | IGIV-C | Placebo |
|---|---|---|
| STARTED | 30 | 30 |
| COMPLETED | 18 | 20 |
| NOT COMPLETED | 12 | 10 |
Reasons for not completing this period:
| Reason | IGIV-C | Placebo |
|---|---|---|
| Adverse Event | 6 | 4 |
| MG worsening | 4 | 1 |
| Withdrawal by Subject | 2 | 3 |
| Physician Decision | 0 | 2 |
Analysis population for the primary outcome, as defined in the registry: The mITT population included all randomized subjects who received at least 1 dose of study medication. Subjects were classified according to randomized 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.
Population: The modified intent-to-treat (mITT) population included all randomized subjects who received at least 1 dose of study medication. Subjects were classified according to randomized treatment.
| Characteristic | Unit | IGIV-C | Placebo | Total Title |
|---|---|---|---|---|
| Participants analysed | 30 | 30 | 60 | |
| Age, Continuous | years | 47.6 (SD 16.99) | 48.5 (SD 14.51) | 48.1 (SD 15.67) |
| Sex: Female, Male: Female | Participants | 16 | 18 | 34 |
| Sex: Female, Male: Male | Participants | 14 | 12 | 26 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 2 | 2 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 30 | 28 | 58 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 3 | 2 | 5 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 1 | 1 |
| Race (NIH/OMB): White | Participants | 27 | 27 | 54 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Baseline Daily Prednisone Equivalent Dose Level Stratification Categories: 15 mg to 40 mg/day | Participants | 29 | 28 | 57 |
| Baseline Daily Prednisone Equivalent Dose Level Stratification Categories: 41 to 60 mg/day | Participants | 1 | 2 | 3 |
| Baseline QMG Total Score | units on a scale | 12.1 (SD 6.98) | 11.2 (SD 6.48) | 11.6 (SD 6.69) |
6. Primary Endpoint
The registry lists the primary outcome as Percent of Subjects Achieving a 50% or Greater Reduction in CS Dose (Prednisone or Equivalent) From Baseline to Week 39, measured over Baseline/Week 0 (Visit 1) and Week 39 (Visit 14)..
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of subjects |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants who achieved a reduction of at least 50% in daily corticosteroid dose from baseline to Week 39. The dose is expressed as prednisone or an equivalent corticosteroid. It converts a change in prescribed dose into a binary outcome for each participant: either the reduction threshold was met or it was not. The group-level endpoint is the percentage meeting that definition.
This type of endpoint is used to make a dose-reduction target interpretable as a participant-level event. It can be clinically relevant to a corticosteroid-sparing study because it focuses on whether participants reached a specified reduction, rather than only describing an average change that could conceal variation across participants. However, categorizing a continuous dose change also discards information about how far above or below the threshold each participant was. The registry does not report the distribution of dose changes or the percentages achieving the threshold in either arm.
The endpoint description includes rules for dose derivation and missing observations. Average daily dose was derived at each scheduled visit using prescribed dose, time interval, and changes between visits. The worst observation carried forward method was used for missing Week 39 dose reductions; the last observation carried forward method was used when no Week 39 corticosteroid dose was prescribed for other reasons. Participants who discontinued early with adverse outcomes related to myasthenia gravis were considered not to have achieved the threshold.
These rules define how the endpoint was constructed, but do not reveal the observed results. The registry record does not report the number or percentage meeting the endpoint by treatment group, an estimated treatment contrast, or its uncertainty.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the clinical question, randomized comparison, endpoint definition, and selected safety counts. It does not report the primary endpoint values by arm. Consequently, no direction or size of the difference in the percentage achieving a 50% or greater corticosteroid-dose reduction can be described from the registry record. The record also does not provide a confidence interval or a p-value for this endpoint.
The record indicates that the primary outcome has several categories or time points and is not reduced to one comparison. That means a single treatment-effect estimate cannot be extracted from the available endpoint listing. Without the arm-specific results and analysis details, it is not possible to determine which endpoint categories or time points were compared, or how those comparisons were handled statistically. The secondary outcomes are named, but their results are not reported in the registry record.
In general, an effect estimate describes the observed contrast between groups, while a confidence interval conveys the range of values compatible with the data under the model and assumptions used. A p-value addresses compatibility with a specified null hypothesis; it does not measure the size or clinical importance of an effect. None of these numerical summaries for the primary endpoint is reported in the registry record, so statistical significance and precision cannot be assessed.
The endpoint uses a threshold: a participant either achieved a reduction of at least 50% or did not. In general, threshold-based results are easier to summarize as event percentages, but they do not show how much the dose changed for participants on either side of the threshold. The secondary mean percent change outcome could provide a different view of dose change, and time to worsening could describe when an event occurred. Their results are also unavailable in the registry record.
The safety entries report serious adverse events in 4 of 30 participants at risk in the IGIV-C group and 6 of 30 at risk in the placebo group. Other adverse events affected 21 of 30 and 24 of 30 participants, respectively. Deaths were recorded for 1 participant in the IGIV-C group and 2 in the placebo group. These are descriptive counts as listed; the record does not provide enough information to infer that treatment caused or prevented any event.
Sample-size calculations elsewhere in the presentation are planning illustrations based on the observed effect, not evidence of the trial’s original design rationale and not observed power. The trial’s sample-size justification is not reported in the registry record. The reported enrollment alone cannot establish whether the study was adequately powered for its primary question. Interpretation therefore rests on the available design and safety information, while the principal efficacy result and its uncertainty remain unreported.
9. Statistical Methods Explained
The statistical details in a registry help define what was intended to be measured, which participants were included, and how missing observations were handled. They do not substitute for the outcome results. For this study, the endpoint description provides several important operational rules, but the primary treatment comparison and its uncertainty are not reported in the registry record.
What does a binary percentage represent?
The primary outcome is expressed as the percentage of participants achieving a 50% or greater reduction in daily corticosteroid dose by Week 39. In general, a binary endpoint assigns each participant to one of two outcome states according to a prespecified threshold. The group percentage then summarizes the proportion meeting that threshold. It is a participant-level event rate, not a measure of the average amount of dose reduction.
That distinction affects interpretation. Participants with dose reductions just below the threshold are grouped with those whose dose did not fall, while participants meeting the threshold are grouped together even if their reductions differ in size. A binary endpoint can therefore provide a clear target-based summary but cannot describe the full distribution of dose changes. The registry also lists mean percent change in daily corticosteroid dose as a secondary outcome, which would address a different feature of the data; its results are not reported in the registry record.
How was corticosteroid dose defined?
The endpoint description says that average daily corticosteroid dose was derived for each participant at scheduled visits. The derivation used the prescribed dose and the time interval, taking changes in prescribed dose between routinely scheduled visits into account. The primary endpoint compares the baseline measurement with Week 39. Baseline is defined as the last non-missing measurement before the first dose of study medication.
Doses are described as prednisone or equivalent, allowing the endpoint to refer to corticosteroid dosing in a common form. The registry does not report further details of conversion procedures or dose verification. Thus, the stated definition explains the intended measure, while some implementation details remain unreported in the registry record.
What do the missing-data rules do?
The description specifies two carried-forward approaches. For missing dose reduction at Week 39, the worst observation carried forward method was used. For a participant without a Week 39 corticosteroid prescription for other reasons, the last observation carried forward method was used to impute the prescribed dose. Participants who discontinued early with adverse outcomes related to myasthenia gravis were classified as not achieving the threshold.
In general, carried-forward methods replace a missing endpoint with a value based on an earlier observation or a specified rule. Such methods can make the analysis set more complete, but they rely on assumptions about what the unobserved later outcome would have been. The assumptions may not reflect the actual course after the last measurement. Different rules for different reasons for missingness can also affect the result, so the reasons for missing data and the number of participants affected are important for interpretation. Those counts are not reported in the registry record.
In general, sensitivity analyses can test whether conclusions change under alternative plausible assumptions about missing outcomes. Whether any sensitivity analyses were performed is not reported in the registry record. The stated imputation methods alone do not show whether the conclusions would be robust to other approaches.
Which participants were analyzed?
The modified intention-to-treat population included all randomized participants who received at least 1 dose of study medication. Participants were classified according to their randomized treatment. In general, analyzing participants by randomized group helps preserve the comparison created by random assignment. Restricting the population to those who received a dose can differ from including every randomized participant, and the number excluded by that restriction matters when interpreting results.
The registry reports enrollment of 60 participants and gives safety group denominators of 30 at risk in each arm. It does not report the endpoint population counts by arm or the number with observed Week 39 measurements. The relationship between enrollment, randomization, treatment, and endpoint availability is therefore not fully described in the registry record.
How should estimates and planning calculations be read?
In general, a confidence interval communicates uncertainty around an estimated treatment contrast. Wider intervals indicate less precision, while narrower intervals indicate greater precision under the analysis assumptions. A p-value is a measure derived under a statistical model and null hypothesis; it is not a measure of effect magnitude. Neither a confidence interval nor a p-value for the primary endpoint is reported in the registry record, so the precision and statistical evidence for that comparison cannot be evaluated.
The sample-size figures associated with the presentation are planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation, and a normal approximation. They are not the trial’s own design justification. In general, a planning calculation estimates how many participants might be needed for a future study to detect a specified effect under stated assumptions. It is not observed power, and it cannot establish the strength of evidence in the completed study. The original sample-size rationale and the numerical planning results are not reported in the registry record.
Overall, the posted methods define a binary dose-reduction endpoint, a modified intention-to-treat population, and specific approaches to missing Week 39 data. They do not give the primary endpoint results, the treatment-effect estimate, its uncertainty, or enough analysis detail to reconstruct the intended comparison.
10. Multiplicity, Missing Data and Analysis Populations
The primary endpoint description refers to results by treatment group overall and by baseline daily prednisone-equivalent dose-level categories. The registry also lists two secondary outcomes: mean percent change in daily corticosteroid dose from baseline to Week 39 and median time to the first episode of myasthenia gravis worsening. The primary outcome is identified as having several categories or time points and is not reduced to a single comparison. These features raise questions about how multiple comparisons were defined and interpreted.
In general, multiplicity arises when several hypotheses, groups, categories, or time points are evaluated. If many comparisons are tested, the chance of at least one apparently unusual result can increase unless the analysis accounts for that structure. The registry does not report whether any multiplicity adjustment was planned or used, which outcomes or comparisons were considered primary for inference, or how subgroup analyses were to be interpreted. Those details are not reported in the registry record; no correction or unadjusted interpretation should be assumed.
The endpoint description specifies worst observation carried forward for missing Week 39 dose reductions and last observation carried forward for participants without a Week 39 prescription for other reasons. Early discontinuation with adverse outcomes related to myasthenia gravis was classified as failure to achieve the threshold. In general, these rules make missing-outcome handling part of the endpoint definition, but they also require assumptions about outcomes that were not observed. The number of missing observations, their reasons by group, and any sensitivity analyses are not reported in the registry record.
The modified intention-to-treat population consisted of randomized participants who received at least 1 dose, analyzed by randomized assignment. The record does not give the endpoint analysis population size by arm, the number excluded from the randomized population, or the number contributing observed Week 39 data. The original statistical analysis plan, including prespecified analysis hierarchy and handling of multiplicity, is 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 |
|---|---|---|---|
| IGIV-C | 4 | 30 | 13.3% |
| Placebo | 6 | 30 | 20.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| IGIV-C | 1 of 30 |
| Placebo | 2 of 30 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| IGIV-C | 21 of 30 |
| Placebo | 24 of 30 |
Most frequent serious adverse event terms
| Event term | Organ system | IGIV-C | Placebo |
|---|---|---|---|
| Myasthenia gravis | Nervous system disorders | 4 of 30 | 1 of 30 |
| Myasthenia gravis crisis | Nervous system disorders | 1 of 30 | 2 of 30 |
| Renal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 30 | 1 of 30 |
| Pneumonia | Infections and infestations | 0 of 30 | 1 of 30 |
| Sepsis | Infections and infestations | 0 of 30 | 1 of 30 |
| Subcutaneous abscess | Infections and infestations | 0 of 30 | 1 of 30 |
| Pneumonia staphylococcal | Infections and infestations | 0 of 30 | 1 of 30 |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | 0 of 30 | 1 of 30 |
| Cardiac arrest | Cardiac disorders | 0 of 30 | 1 of 30 |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | 0 of 30 | 1 of 30 |
Most frequent non-serious adverse event terms
| Event term | Organ system | IGIV-C | Placebo |
|---|---|---|---|
| Headache | Nervous system disorders | 10 of 30 | 3 of 30 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 4 of 30 | 6 of 30 |
| Myasthenia gravis | Nervous system disorders | 7 of 30 | 2 of 30 |
| Upper respiratory tract infection | Infections and infestations | 6 of 30 | 3 of 30 |
| Nasopharyngitis | Infections and infestations | 2 of 30 | 5 of 30 |
| Nausea | Gastrointestinal disorders | 5 of 30 | 1 of 30 |
| Cough | Respiratory, thoracic and mediastinal disorders | 3 of 30 | 2 of 30 |
| Oedema peripheral | General disorders | 2 of 30 | 3 of 30 |
| Back pain | Musculoskeletal and connective tissue disorders | 0 of 30 | 5 of 30 |
| Fatigue | General disorders | 2 of 30 | 2 of 30 |
Serious events with the highest arm-level rate: myasthenia gravis (13.3%), myasthenia gravis crisis (6.7%), renal cell carcinoma (3.3%), pneumonia (3.3%), sepsis (3.3%).
The safety entries report participant counts at risk, counts affected by serious adverse events, counts affected by other adverse events, and deaths. In the IGIV-C group, 30 participants were at risk; 4 were affected by serious adverse events, 21 by other adverse events, and 1 death was recorded. In the placebo group, 30 participants were at risk; 6 were affected by serious adverse events, 24 by other adverse events, and 2 deaths were recorded.
The registry also lists serious adverse event terms with percentages: Myasthenia gravis, 13.3%; myasthenia gravis crisis, 6.7%; renal cell carcinoma, 3.3%; pneumonia, 3.3%; sepsis, 3.3%; and subcutaneous abscess, 3.3%. These are the listed terms and percentages; the registry excerpt does not specify the denominator or clarify whether participants could contribute to more than one term. Counts of participants affected and counts of events are not interchangeable.
These figures are descriptive. They do not establish that a treatment caused an event, nor do they determine comparative safety on their own. The record does not provide event-level timing, severity details beyond the serious-event classification, exposure-adjusted rates, or a statistical comparison of safety groups. The listed deaths should not be attributed to treatment on the basis of these counts.
12. What the Result Does, and Does Not, Mean
The study addresses a focused statistical question: whether participants with corticosteroid-dependent generalized myasthenia gravis achieved a reduction of at least 50% in daily corticosteroid dose by Week 39 under IGIV-C or placebo. The registry provides the endpoint definition, study design, population rule, missing-data conventions, and selected safety counts. It does not report the arm-specific primary endpoint percentages or the treatment contrast.
As a result, the posted registry information does not show whether the groups differed on the primary endpoint, how large any difference was, or how precisely it was estimated. It also does not provide a confidence interval or a p-value for the primary comparison. No conclusion about the primary endpoint can be drawn from the study design or from the trial’s completion status alone.
In general, a randomized comparison is designed to help distinguish treatment-group differences from differences that arise because of participant characteristics. But randomization does not guarantee that a result is precise, and a trial’s design description is not an outcome. Interpretation requires the outcome values, analysis population, and uncertainty estimates, alongside the endpoint and missing-data rules.
The safety counts show that serious adverse events and deaths were recorded in both groups, and that other adverse events affected participants in both groups. Those counts are descriptive and do not establish causation or a treatment-related difference. The registry does not report enough detail to assess event timing, exposure, or the uncertainty of comparative safety estimates.
The study therefore contributes information about how a corticosteroid-sparing question was structured and measured, but the registry record alone does not permit assessment of the efficacy result. The secondary outcome results, sample-size justification, full statistical analysis plan, and multiplicity procedures are also not reported in the registry record. The distinction is important: endpoint definitions describe what would count as an outcome, whereas results show what was observed and how uncertain the comparison is.
13. Why This Trial Matters Statistically
This trial is statistically informative in part because its primary outcome translates a dose-management aim into a defined participant-level threshold: a reduction of at least 50% in daily corticosteroid dose by Week 39. Stating the threshold, baseline definition, and dose-derivation approach makes the intended measure more interpretable than a general reference to dose reduction alone.
The endpoint also illustrates a familiar trade-off in clinical measurement. A binary threshold creates a concise event percentage that can be compared between randomized groups, but it compresses a range of dose changes into two categories. The listed mean percent change outcome could offer a complementary summary of dose change, while time to first worsening addresses a different clinical dimension. The registry names these outcomes but does not report their results.
The missing-data rules matter because the endpoint depends on a dose measurement at Week 39. The record distinguishes missing dose reductions from the absence of a prescribed dose for other reasons and specifies different carried-forward approaches. This makes the analysis definition more explicit, while also highlighting the need to know how many observations were missing and whether conclusions were sensitive to the assumptions. Those details are not reported in the registry record.
The registry also demonstrates why design information and numerical results must be read separately. Randomization, masking, a placebo comparator, and a defined analysis population describe the trial structure. They do not reveal the effect estimate, its precision, or the role of multiple endpoint categories and time points. The absence of those results limits what can be learned about the primary comparison from this record.
Finally, the listed safety counts offer a descriptive view of events in both arms, but should not be treated as causal evidence. Together, the study’s endpoint definition and safety reporting illustrate how careful statistical interpretation depends on both operational detail and complete reporting of results.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The main limitation of the registry record for statistical interpretation is that it does not report the primary endpoint results by treatment group. There is no reported treatment contrast, confidence interval, or p-value for the percentage achieving a 50% or greater dose reduction. The primary outcome is also described as having several categories or time points and is not reduced to a single comparison, so a unique primary estimate cannot be identified from the available record.
The endpoint population is defined, but its size by arm is not reported. Although enrollment is listed as 60 and the safety groups each include 30 participants at risk, the record does not establish the number contributing to the primary analysis or the number with observed Week 39 doses. The extent and distribution of missing data are not reported. This limits assessment of the carried-forward rules and their possible influence on the endpoint.
Further analysis details are not reported in the registry record, including the randomization procedure, sample-size justification, full statistical analysis plan, and procedures for multiplicity. The record also does not provide results for the listed secondary outcomes. The safety information is limited to selected counts and listed serious adverse event terms; details such as event timing and exposure-adjusted comparisons are not reported.
These limitations concern the information available in the registry record, not necessarily the full trial documentation. The observed primary effect and its precision cannot be inferred from enrollment, design labels, endpoint definitions, or safety counts.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Efficacy and Safety of IGIV-C in Corticosteroid Dependent Patients With Generalized Myasthenia Gravis — NCT02473965.
- Registry record last updated 2020-03-30; status last verified 2020-03.
- Results first posted 2020-02-26.
- 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
NCT02473965 is a completed phase 2, multicenter, randomized, parallel-group, double-blind, placebo-controlled study of IGIV-C as a corticosteroid-sparing agent in corticosteroid-dependent participants with generalized myasthenia gravis. The registry lists enrollment of 60, a start date of 2015-06, and primary completion in 2019-02. Grifols Therapeutics LLC is the listed sponsor.
The primary endpoint was the percentage achieving a 50% or greater reduction in daily corticosteroid dose from baseline to Week 39. The modified intention-to-treat population included randomized participants who received at least 1 dose, analyzed according to randomized group. The endpoint description specifies dose derivation and carried-forward methods for missing Week 39 data.
The registry lists serious and other adverse-event counts and deaths by treatment group. It does not report the primary endpoint results, effect estimate, or uncertainty, and does not report secondary outcome results, the sample-size justification, or the full statistical analysis plan.