A Long-term, Continued Treatment and Follow-up Study in Participants With Hematologic Malignancies Treated With Duvelisib (IPI-145): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for A Long-term, Continued Treatment and Follow-up Study in Participants With Hematologic Malignancies Treated With Duvelisib (IPI-145) (NCT02711852), a na phase 2 study. The primary outcome is Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs).

2. Clinical Question

The central statistical question is descriptive: what adverse-event experience was recorded during continued duvelisib treatment in this selected group? Because participants entered after earlier duvelisib treatment, the findings concern people who reached this continued-treatment study, not everyone with a hematologic malignancy or everyone starting duvelisib. The record does not establish how participants in this study compare with people receiving another treatment. It also does not report results that would allow the listed response and survival outcomes to be evaluated.

3. Trial Design

This was a phase 2, single-group study of continued treatment and follow-up in participants with hematologic malignancies previously treated with duvelisib. The registry record identifies SecuraBio as the sponsor, reports enrollment of 19, and gives the status as completed. The recorded start is 2016-04, and the primary completion date is 2020-05-04.

There was no masking, and allocation is recorded as NA. The model is single group, with duvelisib as the only listed intervention. The study is not a randomized comparison, and the registry record does not describe a concurrent comparator. Consequently, outcomes are descriptive of the participants treated in this study; differences between treatment options cannot be estimated from this design.

Participants received the same dose they had received in their previous duvelisib study, with dosing twice daily. The protocol description allowed two dose reductions per participant and specified that doses were not less than 10 mg. Treatment was to continue until disease progression or unacceptable toxicity. The actual dose received by each participant and individual treatment duration are not reported in the registry record.

The primary outcome was the number of participants experiencing TEAEs, assessed over up to 45 months. The All Treated Analysis Set included all participants who received at least one dose. Best overall response, assessed by the investigator, and overall survival were listed as secondary outcomes. The registry record does not report the statistical analysis plan, detailed follow-up procedures, or results for these outcomes. It also does not report how participants were selected from the earlier studies or how many people were eligible but did not enter this study.

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

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All Treated Analysis Set included all participants who received at least one dose of duvelisib.

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: All Treated Analysis Set included all participants who received at least one dose of duvelisib.

6. Primary Endpoint

The registry lists the primary outcome as Number of Participants Experiencing Treatment-emergent Adverse Events (TEAEs), measured over Up to 45 months.

The primary endpoint counts participants who experienced treatment-emergent adverse events (TEAEs). Its unit is participants, and the recorded timeframe is up to 45 months. The registry record defines the All Treated Analysis Set as all participants who received at least one dose of duvelisib. It says that summaries of serious and all other non-serious adverse events, regardless of causality, are located in the Reported Adverse Events module.

A participant count is a direct way to describe how many people in the treated group had an event during the specified observation period. It can communicate the observed burden in this group, but it does not by itself describe the timing, duration, recurrence, or severity of every event. Nor does a count alone account for how long each participant was observed or exposed to treatment. Those details matter when participants have different observation or treatment durations.

The endpoint is safety-focused; it is not a measure of tumor response or survival. Although the registry record lists best overall response and overall survival as secondary outcomes, their numeric results are not reported in the registry record. The posted adverse-event counts do not establish the overall primary endpoint count: serious and other non-serious event categories are not identified as mutually exclusive, and the record does not give a combined participant total for TEAEs. That numeric endpoint result is not reported in the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

General statistical explanation: A single-group study describes outcomes within the treated group. Without a concurrent comparison group, there is no direct estimate of how outcomes would differ under another treatment. Random allocation is not reported; the registry record classifies allocation as NA and the study model as single group. The design therefore supports descriptive reporting, not a randomized treatment contrast.

General statistical explanation: A count records the number of participants with an event. It is different from an event count, which may include repeated events in the same participant, and different from a rate that accounts for time at risk. The primary endpoint is defined in participants, while the adverse-event summary also reports the number at risk and affected. The record does not provide individual follow-up durations, total time at risk, or exposure-adjusted rates. The number of participants experiencing any TEAE is not reported in the registry record.

General statistical explanation: Percentages can help express how common a reported event was in its analysis group, but interpretation depends on the denominator, event definition, and observation period. The listed serious terms are each reported as 5.3%. Those term-specific percentages should not be added: the record does not establish that terms occurred in distinct participants, and one participant can contribute more than one event term. The record does not give the underlying participant count separately for each listed term.

General statistical explanation: An uncertainty interval, such as a confidence interval, describes the range of estimates compatible with a statistical procedure and its assumptions. It is not a guarantee that the true value lies within a particular interval. No confidence intervals or other uncertainty estimates for the posted outcomes are reported in the registry record. With only descriptive counts available, the precision of the event estimates cannot be fully assessed from the posted information.

There are 19 enrolled participants, and the primary endpoint population is defined as all participants who received at least one dose. The adverse-event record lists 19 at risk for serious events and 19 at risk for other events. These denominators describe the reported safety groups; they do not supply the missing duration or participant-level event history. The registry record does not give a separate count of participants excluded from the primary analysis, or explain whether any enrolled participant did not receive treatment.

The registry record lists the primary endpoint as the number of participants experiencing TEAEs over up to 45 months. The available adverse-event summary separates serious from other non-serious events and states that causality is not required for inclusion. General interpretation should therefore distinguish event occurrence from attribution: a reported event during treatment is not by itself proof that treatment caused it. The record does not provide the attribution assessments or a treatment-related subset of these figures.

The sample-size illustrations use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a stated fraction of the observed effect. Those are planning assumptions, not the design of this single-group study. A sample-size illustration based on an observed effect is not observed power and does not show that this study had a particular chance of detecting an effect. No numeric sample-size justification for the trial's own design is reported in the registry record.

For binary outcomes, general power calculations depend on the assumed event frequencies, effect to detect, allocation, significance level, and statistical method. For time-to-event outcomes, calculations also depend on event accrual and assumptions about the event process. The registry record does not report the protocol's sample-size rationale, power target, or design assumptions for the listed outcomes. No such assumptions should be inferred from the planning illustration.

The registry record lists three outcome measures and zero analyses. It names TEAEs as the primary outcome and best overall response and overall survival as secondary outcomes, but provides no analysis estimates for response or survival. Statistical methods for handling missing observations, censoring, repeated events, or competing events are not reported in the registry record. The record also does not report interim analyses, prespecified subgroup analyses, or a detailed statistical analysis plan. Without those details, the precision and analytical handling of the outcomes cannot be reconstructed.

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("Duvelisib")
n      <- c(19)
events <- c(17)
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  Duvelisib  19  17  89.5  66.9  98.7  68.6  97.1  68.3  96.8

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists three outcome measures: the primary endpoint is the number of participants experiencing TEAEs, and the secondary outcomes are best overall response as assessed by the investigator and overall survival. It lists zero analyses. Numeric results for best overall response and overall survival are not reported in the registry record, and a numeric result for the primary endpoint is also not reported as a combined endpoint count.

When a study evaluates several outcomes, general statistical practice may address multiplicity, since repeated testing can affect the interpretation of nominal significance levels. The registry record does not report whether any multiplicity adjustment or testing hierarchy was planned. It does not report a prespecified statistical analysis plan or hypothesis-testing procedure. No adjustment method or sequence of outcome testing should therefore be inferred.

The primary endpoint population is defined as all participants who received at least one dose of duvelisib. This definition identifies the analysis population for that endpoint, but the registry record does not describe missing-data rules, participant withdrawals, or how incomplete outcome information was handled. It does not report the number of participants with missing response assessments or survival follow-up, nor does it specify censoring rules for overall survival. Further analysis-population definitions for the secondary outcomes are not reported in the registry record.

These omissions limit assessment of how outcomes were selected, analyzed, and made comparable across participants. They also prevent a reader from determining whether missingness or multiple outcome testing changed the interpretation of the posted results. The descriptive adverse-event counts remain interpretable as recorded counts, but do not fill in those unreported analysis details.

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 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: diarrhoea (5.3%), enteritis (5.3%), fatigue (5.3%), pyrexia (5.3%), pneumonia cytomegaloviral (5.3%).

The adverse-event summary lists 19 participants at risk for serious events, with 5 affected, and 19 at risk for other events, with 16 affected. It also lists 5 deaths. The registry record describes the event summaries as including serious and all other non-serious adverse events regardless of causality. The deaths are reported without a stated relationship to treatment; attribution and timing are not reported in the registry record.

The serious adverse-event terms listed are Diarrhoea, Enteritis, Fatigue, Pyrexia, Pneumonia cytomegaloviral, and Pneumonia viral. Each term has a reported percentage of 5.3%. The figures are event-term summaries, not evidence that the terms represent separate groups of participants. A participant may have more than one event, so the term percentages should not be summed to infer a total number of affected participants.

Counts describe how many participants were recorded as affected; they do not describe event duration, recurrence, severity beyond the serious classification, or the time each person was observed. The record gives a primary endpoint timeframe of up to 45 months, but does not provide participant-level exposure or follow-up time. The safety figures are descriptive and do not establish a causal relationship between treatment and an event.

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

The posted safety information documents that adverse events and deaths were recorded among participants in a single-group study of continued duvelisib treatment. The serious and other non-serious event summaries give affected-participant counts, and the registry lists several serious event terms with percentages. These observations describe the experience recorded for this study group; they do not by themselves establish that duvelisib caused the events or deaths.

The study population had previously received duvelisib in a sponsor-approved study. It is therefore a selected group entering continued treatment, rather than an unselected population beginning therapy. The design has no concurrent control group, so the record cannot show how the same participants would have fared under a different treatment or without continued treatment. The counts should not be interpreted as comparative treatment effects.

The primary endpoint is the number of participants experiencing TEAEs over up to 45 months, but that combined endpoint result is not reported in the registry record. The posted serious and other event summaries do not establish a total number of distinct participants with any TEAE. Results for the listed secondary outcomes, best overall response and overall survival, are not reported in the registry record either. No estimate of response or survival can be drawn from their presence in the outcome list alone.

The record also does not provide uncertainty intervals, individual follow-up durations, or exposure-adjusted rates. These omissions limit how precisely the observed counts can be interpreted, especially if participants differed in their time on treatment or under observation. The reported death count has no stated attribution, and should not be interpreted as a treatment-related death count.

In short, the registry record provides descriptive safety counts for a small continued-treatment group but not the comparative evidence or outcome analyses needed to determine relative effects. The findings do not establish benefit, lack of benefit, or causation. They are limited to the events and population described in the record.

14. Why This Trial Matters Statistically

This study illustrates a common statistical distinction: describing events in a treated group is not the same as estimating a treatment effect. The design is single group, with no randomized comparator. Its adverse-event counts can document what was recorded among participants during continued treatment, but they cannot separate treatment effects from disease course, prior treatment, or other influences.

The population definition also matters. Participants had previously received duvelisib and entered a continued-treatment study. Findings from this group may not represent people who have not previously received the drug. The registry record does not report how entry into the study was determined, so the degree of selection cannot be quantified from the available information.

Statistically, the endpoint is defined as a participant count, while interpretation also depends on observation time, event overlap, and attribution. The record reports up to 45 months for the endpoint timeframe, but not participant-level exposure or a combined count for any TEAE. The listed serious event terms each have a percentage, yet those percentages do not indicate mutually exclusive participant groups. These details show why event counts should be read with their definitions and denominators rather than treated as complete summaries of risk.

The registry record lists overall survival and best overall response as secondary outcomes, but provides no numeric results for them. It also does not report the analysis plan, missing-data approach, or multiplicity procedures. Those omissions prevent evaluation of how outcome estimates would have been constructed or how precisely they were measured.

The statistical value of the record is therefore in its description of a continued-treatment cohort and its reporting of safety events, alongside clear limits on comparison and inference. Recognizing those limits helps distinguish recorded observations from causal conclusions or estimates of relative treatment effects.

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 main limitation is the single-group design. Allocation is recorded as NA, masking as none, and no concurrent comparator is described. The study cannot provide a randomized comparison, and the observed event counts cannot establish what would have happened under another treatment or without continued treatment.

Participants had previously been treated with duvelisib in a sponsor-approved study. This continued-treatment population may differ from people who did not enter the earlier studies or did not continue into this study. The registry record does not report the selection process, eligibility flow, or reasons participants entered or did not enter the study. How representative the enrolled group was cannot be assessed from the record.

Enrollment was 19. The primary endpoint timeframe was up to 45 months, but individual follow-up and treatment durations are not reported in the registry record. The adverse-event summaries give participant counts but no exposure-adjusted rates. The combined number of participants experiencing any TEAE is not reported, and the serious and other event categories are not stated to be mutually exclusive. Event timing, recurrence, severity detail, and treatment attribution are also not reported.

Five deaths are listed, but cause, timing, and relationship to treatment are not reported in the registry record. Numeric results for best overall response and overall survival are not reported, and the primary endpoint's combined count is not reported. The registry record also lacks a detailed statistical analysis plan, missing-data methods, uncertainty estimates, and multiplicity procedures. These gaps limit assessment of precision, analysis choices, and the scope of conclusions that can be drawn from the posted results.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: A Long-term, Continued Treatment and Follow-up Study in Participants With Hematologic Malignancies Treated With Duvelisib (IPI-145) — NCT02711852.
• Registry record last updated 2023-09-11; status last verified 2023-08.
• Results first posted 2023-02-21.
• 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 record describes a completed phase 2, single-group study of continued duvelisib treatment in participants with hematologic malignancies previously treated with duvelisib. It reports enrollment of 19, identifies SecuraBio as sponsor, gives a start of 2016-04 and primary completion date of 2020-05-04, and records no masking.

Participants continued their previous duvelisib dose twice daily, with two dose reductions allowed per participant and doses not less than 10 mg. Treatment continued until disease progression or unacceptable toxicity. The primary endpoint was the number of participants experiencing TEAEs over up to 45 months. Best overall response and overall survival were listed as secondary outcomes.

The safety summary lists 5 participants affected by serious adverse events, 16 affected by other adverse events, and 5 deaths; event summaries were reported regardless of causality. The numeric combined TEAE endpoint result and secondary outcome results are not reported in the registry record. With no comparator, the posted safety information is descriptive and does not establish causal or comparative treatment effects.