A Study With Tasquinimod Treating Patients With Hepatocellular, Ovarian, Renal Cell and Gastric Cancers: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Study With Tasquinimod Treating Patients With Hepatocellular, Ovarian, Renal Cell and Gastric Cancers (NCT01743469), a non-randomized phase 2 study. The primary outcome is Progression Free Survival (PFS) Rate, Defined as the Percentage of Patients Who Had Neither Progressed Nor Died as Measured by Centrally Analysed RECIST v1.1 (All Cohorts)..

2. Clinical Question

3. Trial Design

This was a Phase 2, multicentre, open-label, single-group proof-of-concept study of tasquinimod. It enrolled patients with advanced or metastatic hepatocellular, ovarian, renal cell, or gastric cancer who had progressed after standard therapies. The record lists enrollment of 201 and describes the study as completed. Its start and primary completion are recorded as 2012-12 and 2014-12.

Allocation is listed as non-randomized, masking as none, and the model as single-group. The four cancer cohorts were not randomized against each other, and the record does not describe a concurrent control arm. Each cohort’s regimen was one oral capsule of tasquinimod daily, at one of the listed doses: 0.25 mg, 0.5 mg, or 1 mg. Treatment was to continue until disease progression, loss to follow-up, withdrawal, or death. The record does not report how dose selection or dose changes were handled.

The primary outcome was a progression-free survival rate at a cohort-specific timepoint. It was based on central RECIST v1.1 assessment and counted patients who had neither progressed nor died by that point. The timepoint differed by cancer cohort: Week 12 for gastric carcinoma, Week 16 for hepatocellular and renal cell carcinoma, and Week 24 for ovarian carcinoma. The registry also lists response, clinical benefit, progression-free survival, and time-to-progression outcomes, using RECIST v1.1 across cohorts and Choi criteria for specified hepatocellular outcomes.

Although the registry describes an early-stopping design, it does not report the stopping rules or whether any cohort stopped early. The protocol’s planned sample size by cohort, the rationale for that size, and the details of any interim monitoring are not reported in the registry record. The primary endpoint estimates and confidence intervals are also not reported, so the design can be described but the primary findings cannot be assessed from this 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: In the hepatocellular carcinoma cohort 67 patients were screened, of whom 53 were treated with tasquinimod. In the ovarian carcinoma cohort 63 were screened, of whom 55 were treated. In the renal cell carcinoma cohort 44 were screened, of whom 38 were treated. In the gastric carcinoma cohort 27 were screened, of whom 21 were treated.

Active Treatment Phase

Reasons for not completing this period:

Follow-up Period - Post-Treatment

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: ITT population: All treated patients i.e. all patients who had received at least one dose of tasquinimod. An additional patient was included in the hepatocellular carcinoma cohort, since the 52nd (last patient planned in the protocol) and 53rd were screened at the same time. This 53rd was not included in the primary analysis.

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: Intent-to-treat (ITT) population: All treated patients i.e. all patients who had received at least one dose of tasquinimod.

6. Primary Endpoint

The registry lists the primary outcome as Progression Free Survival (PFS) Rate, Defined as the Percentage of Patients Who Had Neither Progressed Nor Died as Measured by Centrally Analysed RECIST v1.1 (All Cohorts)., measured over Week 12 (Gastric Carcinoma Cohort); Week 16 (Hepatocellular and Renal Cell Carcinoma Cohorts); Week 24 (Ovarian Carcinoma Cohort)..

The primary endpoint is the percentage of participants who had neither progressed nor died by a specified assessment time. This is a fixed-time summary of progression-free status: it asks whether a patient was known to remain alive without documented progression at the cohort’s assessment point. It is not the same as reporting the time until progression or death for each patient.

The assessment time varied by cohort: Week 12 for gastric carcinoma, Week 16 for hepatocellular and renal cell carcinoma, and Week 24 for ovarian carcinoma. The endpoint was based on centrally analysed RECIST v1.1. The record defines progression or death relative to the analysis timepoint with a three-day allowance. It describes a patient as having neither progressed nor died when central assessment confirmed no progression and the last qualifying examination or visit was at least seven days beyond the analysis timepoint. Patients without evidence that could establish either status, including some withdrawals without a tumor assessment proving progression, could be considered not assessable.

As a general statistical explanation, a fixed-time rate can make an outcome easier to communicate than a full time-to-event curve, but it depends on whether participants have adequate assessment near the chosen timepoint and on how unassessable cases are handled. A confidence interval expresses uncertainty around the estimated rate; it does not remove limitations from incomplete follow-up or the lack of a concurrent control. The registry gives the endpoint definition and its stated 95% confidence interval measure, but the numerical estimates and intervals are not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a binary status at a fixed assessment time: a participant is classified as having neither progressed nor died, or not meeting that definition. The registry reports the outcome as a percentage of participants and specifies a 95% confidence interval. The numerical estimates are not reported in the registry record.

What does a fixed-time percentage represent?

As a general statistical explanation, a fixed-time percentage answers a question about the proportion meeting a defined condition at a particular point in follow-up. It differs from a time-to-event analysis, which uses information about when progression or death occurred and can account for differing follow-up durations through methods designed for censored observations. The registry lists both a fixed-time primary rate and secondary time-to-event outcomes, but it does not report their numerical results.

For the primary endpoint, the point of assessment depended on cancer cohort: Week 12 for gastric carcinoma, Week 16 for hepatocellular and renal cell carcinoma, and Week 24 for ovarian carcinoma. Thus, the rates would refer to different follow-up times across cohorts. Even if numerical cohort estimates were available, they would not be directly comparable as if they were measured at one common timepoint.

How should the confidence interval be understood?

In general, a confidence interval describes uncertainty in an estimate under the statistical method used. A narrower interval indicates greater precision under that method; a wider interval indicates less precision. It is not a range that guarantees the true value lies within it, and it does not address every source of uncertainty. For example, it does not correct for bias from missing assessments, differences in patient characteristics, or the lack of randomization. The registry names a 95% confidence interval for the primary endpoint but does not report the interval values or the calculation method.

What does the RECIST assessment contribute?

The primary outcome is described as centrally analysed RECIST v1.1. Central assessment provides a common review approach for determining tumor status, while RECIST supplies criteria for classifying response or progression. The registry’s endpoint description also states a progression definition using RECIST v1.0 wording, while the primary outcome title and event rules refer to RECIST v1.1. This difference in the record’s wording should be noted rather than resolved by assumption. The record does not provide further clarification.

The endpoint rules also specify how the timing of an event relates to the analysis point, and how a participant could be classified as having neither progressed nor died. It describes a three-day allowance for progression or death relative to the analysis timepoint, and requires the last qualifying assessment to be at least seven days beyond that point for the no-progression status. A patient without an adequate tumor assessment could be considered not assessable. These rules make assessment timing and the treatment of incomplete follow-up relevant to interpretation.

What is the role of the secondary time-to-event outcomes?

As a general explanation, progression-free survival and time to progression use event times rather than simply classifying each patient at one fixed time. A time-to-event analysis commonly has to distinguish an observed event from censoring, when follow-up ends before the event is observed. The registry lists progression-free survival from first study treatment to progression or death, and time to progression, with RECIST v1.1 for all cohorts and Choi criteria for specified hepatocellular outcomes. However, its record does not state the analysis methods, censoring rules, or numerical findings for those outcomes. Those details should not be inferred from the outcome names alone.

What do the planning illustrations mean?

Any sample-size values associated with planning are illustrations based on the observed effect, not the trial’s own design or observed power. The stated illustration assumptions are a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect to detect defined as a stated fraction of the observed effect. For time-to-event outcomes, the illustration uses a total number of events; for other outcomes, it uses participants per arm. These are generic planning assumptions and do not describe the actual study design, which is listed as single-group and non-randomized.

As a general statistical principle, power is a design quantity calculated under assumptions about an effect, variability, allocation, and analysis. It is not a measure of whether a completed study has found a real effect. A planning illustration based on an observed effect cannot be interpreted as observed power, and its two-arm assumptions do not create a control group in this trial. The registry does not report the trial’s own sample-size justification or statistical analysis plan.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Hepatocellular Carcinoma Cohort", "Ovarian Carcinoma Cohort", "Renal Cell Carcinoma Cohort", "Gastric Carcinoma Cohort")
n      <- c(52, 55, 38, 21)
events <- c(14, 4, 5, 2)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Hepatocellular Carcinoma Cohort  52  14  26.9  15.6  41.0  16.8  40.3  16.8  40.3
ARM  Ovarian Carcinoma Cohort  55  4  7.3  2.0  17.6  2.9  17.3  3.0  17.3
ARM  Renal Cell Carcinoma Cohort  38  5  13.2  4.4  28.1  5.8  27.3  5.9  27.4
ARM  Gastric Carcinoma Cohort  21  2  9.5  1.2  30.4  2.7  28.9  2.9  29.2

11. Multiplicity, Missing Data and Analysis Populations

The registry lists multiple outcomes: the primary fixed-time progression-free survival rate, response and response-rate measures, clinical benefit, progression-free survival, and time to progression. Some secondary outcomes apply across all cohorts, while Choi-criteria outcomes are listed for the hepatocellular carcinoma cohort. The record also lists 11 outcome measures and 5 analyses. It does not report how the statistical analyses were organized across those outcomes or whether any adjustment for multiple testing was planned.

As a general statistical explanation, testing or estimating many outcomes can increase the chance of apparently notable findings arising by chance, particularly when analyses are selected after results are known. Prespecified outcome hierarchies or multiplicity adjustments can help address that issue, but the registry record does not describe such procedures. No conclusion about whether multiplicity was controlled is warranted from the listed outcomes alone.

The record defines the intention-to-treat population as all treated patients who received at least one dose of tasquinimod. It also describes an additional patient in the hepatocellular carcinoma cohort because the last patient planned and another patient were screened at the same time. The sentence explaining the status of that additional patient is incomplete in the registry record. The actual denominators for the primary endpoint by cohort, the analysis population used for each reported outcome, and the handling of missing endpoint data are not reported in the registry record.

The endpoint definition allows some patients without a qualifying assessment to be considered not assessable. How such patients were handled in calculating the primary rates is not reported. The record also does not state whether missing follow-up was treated as failure, excluded from the denominator, or addressed by another method. Those choices can affect a fixed-time percentage, so the results cannot be fully interpreted without them. The protocol’s sample-size rationale, interim-analysis procedures, stopping rules, and detailed statistical plan are not reported in the registry record.

12. Safety Results

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

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: nausea (9.5%), vomiting (9.5%), pleural effusion (7.3%), asthenia (4.8%), oedema peripheral (4.8%).

The registry lists serious adverse event counts separately for each cancer cohort. The hepatocellular carcinoma cohort lists 14 participants with serious adverse events among 53 at risk; the ovarian carcinoma cohort lists 19 among 55; the renal cell carcinoma cohort lists 11 among 38; and the gastric carcinoma cohort lists 7 among 21. The record also lists deaths of 38, 22, 22, and 16 in those respective cohorts. These are descriptive figures, not estimates from a randomized comparison.

The most frequently listed serious-event terms are nausea and vomiting, each reported as 9.5%; pleural effusion at 7.3%; and asthenia, oedema peripheral, and cellulitis, each at 4.8%. The registry record does not provide the event counts for each term alongside these percentages, or enough detail to establish whether participants could be represented in more than one term. The percentages should therefore be read as listed, not added together.

The record does not establish that the listed events or deaths were caused by tasquinimod. It also does not provide a control group, event timing, exposure-adjusted rates, or a detailed account of how adverse events were collected. The counts describe what the registry reports for these cohorts; they do not by themselves determine comparative risk or causality.

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

This study addresses an exploratory proof-of-concept question: whether progression-free status at a specified time could be assessed in patients with four types of advanced or metastatic cancer treated with tasquinimod after progression on standard therapies. The registry gives the planned endpoint definition and cohort-specific timing. It does not report the numerical primary endpoint rates or confidence intervals, so the magnitude and precision of the observed primary findings cannot be interpreted from the registry record.

Even if those rates were available, the design would support description of outcomes in the treated cohorts rather than a randomized estimate of treatment effect. The study is recorded as single-group, non-randomized, and open-label, without a concurrent comparator. Consequently, outcomes could not be separated from other influences using a randomized between-group contrast. The four cohorts also differ by cancer type and by the timepoint used for the primary outcome, limiting direct comparisons across them.

The record’s status as completed and its enrollment figure of 201 describe the study, not its efficacy findings. Likewise, the secondary outcome list identifies additional questions measured but does not disclose their results. The listed serious adverse event and death counts provide descriptive safety information, but they do not establish cause, comparative risk, or a balance of benefits and harms.

As a general statistical point, a confidence interval would help communicate the precision of a cohort rate, while the endpoint’s missing-data rules would clarify how incomplete assessments contributed. Neither can be evaluated fully from the available registry information: the confidence interval values, cohort-specific primary denominators, and missing-data handling are not reported in the registry record. The findings should therefore be understood as not numerically interpretable from this record, rather than as evidence for or against an effect.

14. Why This Trial Matters Statistically

The trial is statistically notable for asking a common proof-of-concept question across four distinct cancer cohorts while using cohort-specific fixed-time progression-free survival endpoints. This structure makes the endpoint definition and assessment schedule central to interpretation. The timepoints differ across cohorts, so a percentage in one cohort would describe a different follow-up horizon from a percentage in another.

It also illustrates the distinction between a descriptive single-group result and a controlled treatment comparison. The study is listed as non-randomized, open-label, and single-group. An outcome rate in such a cohort can describe what was observed among treated patients, but it cannot by itself estimate what would have happened under a comparator treatment. This matters especially when readers might otherwise compare outcome rates across the cancer cohorts as if their differences reflected treatment response alone.

The registry’s endpoint rules highlight practical issues in binary outcome analysis: assessment timing, central review, the definition of progression or death, and classification of participants without sufficient follow-up. These details determine who is counted as having neither progressed nor died and how an unassessable status is handled. Yet the record does not report the numerical primary estimates, confidence intervals, cohort denominators for those estimates, or missing-data methods.

Finally, the study lists a range of secondary outcomes and serious adverse event information. That breadth makes it important to know the planned statistical approach and how multiple outcomes were handled. Those methods are not reported in the registry record. The study therefore offers a useful example of why design, endpoint definitions, analysis populations, and outcome reporting must be read together before drawing statistical conclusions.

15. 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 primary limitation for interpreting efficacy is that the registry record does not report the numerical primary endpoint rates or their 95% confidence intervals. It also does not report cohort-specific endpoint denominators, so the precision and completeness of each fixed-time estimate cannot be evaluated. Although the endpoint definition identifies patients who may be considered not assessable, the record does not explain how those patients were handled in the rate calculation.

The study’s non-randomized, single-group design leaves no concurrent control comparison. The open-label status and absence of masking are also relevant features of the design, although the record does not describe how these factors may have affected assessment or conduct. The four cohorts involve different cancer types and use different primary assessment times, so any numerical rates would need to be interpreted within their own cohort and endpoint context rather than as directly comparable measures.

The registry’s primary endpoint wording refers to central RECIST v1.1 assessment, while part of its progression definition refers to RECIST v1.0. The record does not clarify this difference. Its description of the intention-to-treat population also includes an additional hepatocellular carcinoma patient and ends with an incomplete sentence about that patient’s inclusion status. These record details leave aspects of the endpoint and population description unresolved.

The registry lists secondary outcomes and adverse event counts, but does not report the statistical analysis plan, multiplicity procedures, interim analyses, stopping rules, or the study’s own sample-size justification. It does not provide detailed adverse-event timing, attribution, or comparative data. These omissions restrict both efficacy and safety interpretation. The completed study status does not resolve the absence of those results and methods.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Study With Tasquinimod Treating Patients With Hepatocellular, Ovarian, Renal Cell and Gastric Cancers — NCT01743469.
• Registry record last updated 2019-01-08; status last verified 2019-01.
• Results first posted 2018-05-07.
• 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.

19. Record Summary

The registry describes a completed Phase 2, multicentre, open-label, non-randomized, single-group proof-of-concept study of tasquinimod in advanced or metastatic hepatocellular, ovarian, renal cell, and gastric cancers. Enrollment is listed as 201. Tasquinimod was taken orally each day at one of the listed doses, with treatment continuing until progression, loss to follow-up, withdrawal, or death.

The primary outcome was a centrally assessed RECIST v1.1 progression-free survival rate at cohort-specific timepoints: Week 12 for gastric carcinoma, Week 16 for hepatocellular and renal cell carcinoma, and Week 24 for ovarian carcinoma. The registry does not report the numerical primary rates or confidence intervals. Serious adverse event counts and selected event-term percentages are listed, but the record does not establish causality or provide a randomized comparison. The detailed statistical plan, missing-data handling, and multiplicity procedures are not reported in the registry record.