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 12 -Month, Open-label, Multi-center Study to Explore the Health Outcomes of FTY720 (NCT01578330), a na phase 4 study. The primary outcome is Mean Patient-Reported Treatment Satisfaction Questionnaire for Medication Scores (TSQM-9).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01578330 |
| Phase | Phase 4 |
| Condition | Multiple Sclerosis, Relapsing-Remitting |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 42 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with relapsing-remitting multiple sclerosis who had previously been treated with other disease-modifying therapies. The primary endpoint population was the per-protocol population: patients with evaluable data for the primary variable from first to last visit. The record lists enrollment of 42, but does not report the number with evaluable primary endpoint data.
Intervention
Patients received fingolimod, also identified as FTY720, as oral capsules at 0.5 mg daily, with or without food. The study assessed outcomes after treatment over a 12-month period. Because the study was open-label, patients and study personnel were not masked to the treatment.
Comparator
There was no concurrent comparator group. The design is listed as single-group, with allocation recorded as NA and masking as none. Baseline measurements could be compared descriptively with month 12 measurements, but such a within-group comparison is not equivalent to a comparison against an untreated or alternative-treatment group.
Primary question
The primary question was how patient-reported satisfaction with medication, measured using the Treatment Satisfaction Questionnaire for Medication (TSQM-9), was described at baseline and month 12. The study also sought to assess tolerability in a small population. The registry record does not report TSQM-9 results, so the direction and precision of any change cannot be assessed from the record.
In statistical terms, the design can describe outcomes among the enrolled patients during follow-up, but it cannot separate treatment-related change from other influences using a concurrent control. Changes over time may reflect the treatment experience, natural variation, changes in health, or differences in who contributed data at each visit. The registry record does not report enough outcome data or analysis details to quantify those possibilities.
3. Trial Design
This was a phase 4, open-label, multicenter pilot study of fingolimod in patients with relapsing-remitting multiple sclerosis who had previously received other disease-modifying therapies. The study title describes a 12-month period, and the primary outcome timeframe is baseline and month 12. The registry record lists the study as completed, with enrollment of 42. The recorded start is 2012-10 and primary completion is 2015-04.
The study used a single-group model, with allocation recorded as NA and masking recorded as none. Patients received fingolimod 0.5 mg oral capsules daily, with or without food. Since there was no concurrent control group, the design does not support a like-for-like comparison between fingolimod and another treatment or no treatment. Baseline and follow-up observations can describe change within the group, but cannot establish what would have happened to comparable patients under a different condition.
The stated purpose was to assess patient satisfaction after 12 months of fingolimod treatment and to assess tolerability in a small population. The primary outcome was the mean TSQM-9 score, assessed at baseline and month 12. The record also lists patient-reported health-related quality of life measured using the SF-36 as secondary outcomes; the same wording appears in each of the three listed secondary entries.
The primary endpoint population was defined as the per-protocol population, including patients with evaluable data for the primary variable from first to last visit. The number meeting that definition is not reported in the registry record. The record lists four outcome measures and no analyses. It does not report the statistical analysis plan, the method for handling missing observations, or details of how the primary outcome was tested or summarized beyond the stated mean and standard deviation.
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.
Overall Study
| Stage | Fingolimod, FTY720 |
|---|---|
| STARTED | 42 |
| Per Protocol Population | 35 |
| COMPLETED | 35 |
| NOT COMPLETED | 7 |
Reasons for not completing this period:
| Reason | Fingolimod, FTY720 |
|---|---|
| Adverse Event | 2 |
| Protocol Violation | 5 |
Analysis population for the primary outcome, as defined in the registry: Per Protocol Population: included all patients who had evaluable data for the primary variable from first to last visit
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 | Fingolimod, FTY720 |
|---|---|---|
| Participants analysed | 42 | |
| Age, Continuous | Years | 35.7 (SD 9.4) |
| Sex: Female, Male: Female | Participants | 16 |
| Sex: Female, Male: Male | Participants | 26 |
6. Primary Endpoint
The registry lists the primary outcome as Mean Patient-Reported Treatment Satisfaction Questionnaire for Medication Scores (TSQM-9), measured over Baseline and month 12.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | Scores on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was the mean Patient-Reported Treatment Satisfaction Questionnaire for Medication score, or TSQM-9, at baseline and month 12. The questionnaire is described as a psychometric measure of satisfaction with medication, covering effectiveness, convenience, and global satisfaction. Each domain is constructed from its corresponding questionnaire items and transformed to a scale from 0 to 100. The record states that a positive change from baseline indicates improvement.
Patient-reported treatment satisfaction is useful because it captures a patient’s experience of medication rather than relying only on clinical or laboratory measures. The separate domains can distinguish perceived effectiveness from convenience and overall satisfaction. A mean offers a concise description of the group’s scores, while the standard deviation describes how dispersed individual scores are around that mean.
Those summaries do not, by themselves, show how many patients improved, whether change was consistent across domains, or how precisely the mean change was estimated. The registry record does not report the baseline or month 12 TSQM-9 means, standard deviations, changes, or confidence intervals. It therefore does not allow readers to assess the observed satisfaction scores or their uncertainty.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a completed single-group study with enrollment of 42 and a primary outcome measured at baseline and month 12. It does not report the TSQM-9 results or any analyses. Consequently, the record does not establish the average score at either visit, the direction or size of change, the variation among patients, or the precision of an estimated change. No treatment effect can be quantified from the posted outcome information.
The study’s open-label design is relevant to a patient-reported satisfaction endpoint. General statistical context: when participants know which medication they are taking, expectations and awareness of treatment may influence questionnaire responses. This does not show that responses were biased; it identifies a possible influence that cannot be separated from treatment experience in this design. Satisfaction is also not interchangeable with a clinical measure of disease activity or with a comparative estimate of treatment benefit.
Because there was no concurrent comparator, a baseline-to-month 12 change would describe the enrolled group over time, not the difference between treatment strategies. General statistical context: a within-group change may reflect several influences, including treatment, the course of the condition, other care, measurement variation, and which participants remain available for assessment. A control group can help address some competing explanations, but none is listed for this study.
The primary endpoint population was the per-protocol population, defined by evaluable primary-variable data from first to last visit. The record does not state how many participants met that definition or how missing observations were handled. If the patients contributing to a follow-up mean differed from those contributing to the baseline mean, a comparison of the reported means could reflect both score changes and differences in who was measured. The actual outcome data needed to evaluate this issue are not reported in the registry record.
The record lists serious adverse event counts and selected serious-event terms, but those figures are descriptive safety information, not a controlled estimate of risk attributable to fingolimod. A single treated group cannot show whether an event occurred more often than it would under another treatment or without treatment. The record also does not report deaths as a numeric result. Thus, the available information supports describing what safety counts are listed, but not drawing a comparative safety conclusion.
Overall, the registry entry identifies the study question, design, endpoint, and some safety counts, but not the primary outcome findings or analysis results. This absence should not be interpreted as evidence that the outcome did or did not change. It means that the direction, magnitude, and statistical uncertainty of the primary outcome are not reported in the registry record.
9. Statistical Methods Explained
The study is listed as a single-group, open-label study. General statistical context: a single-group longitudinal design observes a group at more than one time, while a comparative design also observes a separate group under a different treatment condition. Repeated observations can describe how measured outcomes vary over time, but a change within one group does not isolate the effect of treatment from other changes occurring over the same period. The absence of a concurrent comparator is therefore central to interpreting any baseline-to-follow-up result.
What does a mean describe?
The primary outcome is specified as a mean score, with standard deviation as the measure of dispersion. A mean is the arithmetic average of the observed scores in the analysis population. It gives a central summary, but can conceal differences between individuals: the same mean can occur when most patients have similar scores or when scores vary substantially. The standard deviation describes spread around the mean. It is not the same as uncertainty in the estimated mean; a confidence interval, if reported, would address the precision of an estimate under the assumptions of its analysis.
The TSQM-9 is described as having effectiveness, convenience, and global satisfaction subscales. Item scores are combined within those domains, adjusted to the stated domain range, and transformed to a 0-to-100 scale. The record says a positive change from baseline indicates improvement. This direction convention helps interpret a reported change, but no actual baseline or month 12 values or changes are reported. A scale definition alone cannot establish the observed level of satisfaction or its change.
What would a baseline comparison mean?
Comparing baseline and month 12 scores in the same group addresses whether the group’s measured scores differ across those visits. General statistical context: when measurements are paired within individuals, an analysis can use each person’s change and account for the relationship between that person’s measurements. A comparison of two group means may instead treat the observations as separate summaries. The registry record does not report which approach, if any, was used, nor does it report a test, estimate, or confidence interval for change. No specific analysis method should therefore be attributed to the investigators.
A confidence interval is a statistical range that expresses the uncertainty around an estimated quantity under a specified method and its assumptions. It is useful because a point estimate alone does not show how precisely the data locate the quantity of interest. For a within-group change, a confidence interval would describe uncertainty in that change estimate; it would not remove the limitations of having no concurrent comparator. The registry record does not report such an interval for the primary endpoint.
Why does the analysis population matter?
The primary endpoint population is defined as the per-protocol population: patients with evaluable primary-variable data from first to last visit. This definition ties the result to data availability across the stated visits. The number included in that population is not reported. General statistical context: an analysis limited to patients with evaluable follow-up can be affected if availability of data is related to health, experience, or the outcome itself. The size and direction of any such influence cannot be assessed without information about missingness and the analysis population.
How should safety counts be read?
Safety counts describe observed events or affected participants within the treated group, according to the registry’s categories. They do not automatically provide a causal comparison. General statistical context: an event occurring after treatment does not, on timing alone, establish that treatment caused it. A comparator and consistent event ascertainment can aid comparative interpretation, but this study is listed as single-group and open-label. The listed serious and other adverse event counts should therefore be read descriptively.
What do planning illustrations represent?
The record describes sample-size planning illustrations based on the observed effect, using a two-sided significance level of 0.05, equal allocation between two hypothetical arms, and a normal approximation; the effect to detect is a stated fraction of the observed effect. These are planning illustrations, not observed power and not evidence that this single-group study randomized participants between arms. No calculated planning values or observed primary endpoint effect are reported in the registry record. General statistical context: sample-size planning asks how many participants a proposed design would require under specified assumptions; it does not show how likely a completed study was to achieve a result, nor does it replace reporting an estimate and its uncertainty.
General statistical context: over-dispersion occurs when outcome variability is greater than a model assumes. Accounting for it can matter because underestimated variability may make uncertainty appear too small. The primary endpoint is continuous and is summarized as a mean and standard deviation; the registry record does not report a model, a variance assumption, or an over-dispersion assessment. No conclusion about whether such an adjustment was needed can be drawn.
10. Multiplicity, Missing Data and Analysis Populations
The record lists one primary endpoint and three secondary outcome entries. Each secondary entry uses the same wording for mean patient-reported health-related quality of life measured with the SF-36. The record lists four outcome measures overall and no analyses. It does not explain whether the repeated secondary descriptions represent distinct assessments, nor does it report a multiplicity strategy for interpreting results across outcomes or visits.
General statistical context: when several outcomes or comparisons are examined, the chance of at least one apparently notable result can increase if each is considered separately. A prespecified approach to multiplicity can help clarify how evidence across multiple analyses should be interpreted. The registry record does not report whether any adjustment, hierarchy, or other strategy was planned. Since analyses and results are not reported, there is no basis for reconstructing such a strategy after the fact.
The primary population is described as per protocol and includes patients with evaluable primary-variable data from first to last visit. The number included and the amount or pattern of missing data are not reported in the registry record. The record does not describe imputation, exclusion rules beyond the population definition, or sensitivity analyses. General statistical context: missing data can affect a mean or change estimate if the observed participants differ systematically from those without follow-up. Without counts and methods, the extent and direction of any such effect cannot be judged.
The absence of these details does not establish that the study used an inappropriate approach. It means that the handling of multiple outcomes and incomplete observations is not reported in the registry record, limiting independent assessment of the analysis.
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 |
|---|---|---|---|
| Fingolimod, FTY720 | 4 | 42 | 9.5% |
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 |
|---|---|
| Fingolimod, FTY720 | 24 of 42 |
Most frequent serious adverse event terms
| Event term | Organ system | Fingolimod, FTY720 |
|---|---|---|
| Multiple Sclerosis aggravated | Immune system disorders | 1 of 42 |
| Fracture | Injury, poisoning and procedural complications | 1 of 42 |
| Medication taken before pregnancy | Injury, poisoning and procedural complications | 1 of 42 |
| Middle ear infection surgery | Surgical and medical procedures | 1 of 42 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Fingolimod, FTY720 |
|---|---|---|
| Urinary infection | Renal and urinary disorders | 9 of 42 |
| Upper respiratory infection | Respiratory, thoracic and mediastinal disorders | 8 of 42 |
| Lymphopenia | Blood and lymphatic system disorders | 7 of 42 |
| Increase of hepatic enzymes | Hepatobiliary disorders | 5 of 42 |
| Multiple Sclerosis Aggravated | Immune system disorders | 4 of 42 |
| Headache | Nervous system disorders | 4 of 42 |
| Hyperlipidemia | Metabolism and nutrition disorders | 4 of 42 |
| Tooth abscess | Infections and infestations | 3 of 42 |
| Nausea | Gastrointestinal disorders | 3 of 42 |
| Incontinence | Renal and urinary disorders | 3 of 42 |
Serious events with the highest arm-level rate: multiple sclerosis aggravated (2.4%), fracture (2.4%), medication taken before pregnancy (2.4%), middle ear infection surgery (2.4%).
The registry record lists 42 patients at risk for serious adverse events and 4 affected in the fingolimod group. For other adverse events, it lists 42 at risk and 24 affected. These are descriptive counts for the study group; they are not estimates of how event frequency compares with another treatment or with no treatment. The record does not report a numeric death count.
The selected serious-event terms are Multiple Sclerosis aggravated, Fracture, Medication taken before pregnancy, and Middle ear infection surgery, each listed as 2.4%. These percentages should be read as the registry’s reported entries for those terms, not as evidence that the events were caused by fingolimod. The record does not provide further event-level detail in the listed information, such as timing, severity beyond the serious classification, or individual clinical context.
General statistical context: safety counts are influenced by the size and characteristics of the group observed, the duration and completeness of follow-up, and how events are defined and collected. A small single-group study can describe events observed among its participants, but cannot by itself determine comparative risk or establish causality. The absence of a reported death count should be read as not reported in the registry record, rather than interpreted as confirmation that no deaths occurred.
12. What the Result Does, and Does Not, Mean
The registry record identifies a study of patient-reported treatment satisfaction and tolerability among patients with relapsing-remitting multiple sclerosis who had previously received other disease-modifying therapies. It documents an open-label, single-group design, a treatment period described as 12 months, enrollment of 42, and a primary endpoint assessed at baseline and month 12. It also lists adverse event counts for the fingolimod group.
What the record does not provide is the primary endpoint result. The TSQM-9 means, standard deviations, changes, and confidence intervals are not reported, and the record lists no analyses. It is therefore not possible from this record to state whether satisfaction scores changed, how large any change was, how variable scores were, or how precise an estimate might be. Missing results are not evidence of no change, and they are not evidence of improvement.
The study design also limits what a change over time could mean. Without a concurrent comparator, an observed within-group change would not establish how patients would have fared under a different treatment or without treatment. The open-label design is particularly relevant when the outcome is patient-reported: awareness of treatment may influence responses, although the record does not establish that such influence occurred.
The safety information can describe the counts and terms recorded in the treated group, but it cannot determine whether events were caused by treatment or whether their frequency differs from another condition. The trial’s stated focus on satisfaction and tolerability does not replace reporting the outcome data needed to evaluate either. Accordingly, the registry record supports a description of the study’s purpose, design, and listed safety observations, but not a conclusion about treatment effect, comparative safety, or the statistical strength of the primary endpoint findings.
13. Why This Trial Matters Statistically
This study is statistically informative as an example of the distinction between describing a treatment experience and estimating a comparative treatment effect. Its single-group design can characterize observations among participants who received fingolimod, including patient-reported satisfaction and recorded adverse events. Without a concurrent comparator, however, changes during follow-up cannot be attributed uniquely to treatment.
The choice of a patient-reported endpoint also makes the measurement strategy important. The TSQM-9 separates effectiveness, convenience, and global satisfaction into distinct domains and transforms scores to a 0-to-100 scale. This can make the outcome relevant to patients’ experience, while also requiring clear reporting of domain-specific results, changes, variability, and uncertainty. The registry record describes the scoring approach but does not report the resulting scores or analyses.
The stated per-protocol population definition illustrates why analysis populations and data availability matter. A result based on patients with evaluable observations across visits may not represent all enrolled participants if follow-up availability is related to outcomes. Because the number in that population and missing-data handling are not reported, readers cannot assess how much the analysis population differs from enrollment.
Finally, the registry record separates what can be documented from what remains uncertain. It gives design and enrollment information and lists safety counts, yet no primary outcome analyses are reported. That distinction is central to responsible statistical interpretation: a clearly described endpoint and study design do not substitute for the estimate, its uncertainty, and a transparent account of the analysis. The record therefore offers context for the study question, but not the numerical evidence needed to assess the primary outcome.
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 most direct limitation for interpreting the primary outcome is that the registry record does not report TSQM-9 values or analyses. Without means, standard deviations, changes, or precision estimates, the primary question cannot be answered from the posted results. The record also lists no analyses, so the method used to compare baseline with month 12 is not reported.
The single-group design has no concurrent control, and the study is open-label. A change over time cannot distinguish treatment-associated change from other influences, while awareness of treatment may be relevant to self-reported satisfaction. These are design considerations, not evidence that a particular bias occurred.
The primary endpoint was defined for a per-protocol population with evaluable data from first to last visit, but the number in that population is not reported. The record also does not report missing-data amounts, reasons for missingness, or how incomplete observations were handled. This limits assessment of whether the analyzed group represents all enrolled patients.
The record lists repeated wording for the three SF-36 secondary outcome entries, but does not clarify whether they correspond to distinct assessments. It does not report how multiple outcomes or comparisons were handled. Safety information is also limited to listed counts and terms; the record does not give a comparative group or establish causality. These limitations constrain interpretation of both outcome effectiveness and safety, rather than showing that any particular result was favorable or unfavorable.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A 12 -Month, Open-label, Multi-center Study to Explore the Health Outcomes of FTY720 — NCT01578330.
- Registry record last updated 2016-06-07; status last verified 2016-04.
- Results first posted 2016-06-07.
- 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 phase 4 study was a completed, open-label, multicenter, single-group pilot of fingolimod in patients with relapsing-remitting multiple sclerosis previously treated with other disease-modifying therapies. Enrollment was 42. Patients received fingolimod 0.5 mg oral capsules daily, with or without food, and the study assessed outcomes over a 12-month period.
The primary outcome was mean TSQM-9 treatment satisfaction at baseline and month 12, in a per-protocol population defined by evaluable data from first to last visit. The record lists three secondary SF-36 outcome entries and four outcome measures overall. Primary outcome results and analyses are not reported in the registry record.
The safety listing records 4 affected for serious adverse events and 24 affected for other adverse events, with 42 at risk in each category. A numeric death count is not reported.