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 Phase l/II Study of Ruxolitinib for Acute Leukemia (NCT01251965), a non-randomized phase 1, phase 2 study. The primary outcome is Number of Participants With Dose Limiting Toxicities (DLTs).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01251965 |
| Phase | Phase 1, Phase 2 |
| Condition | Leukemia |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 27 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The official title identifies subjects with relapsed or refractory acute leukemia. The listed condition is leukemia. Further eligibility details, including disease subtypes and other participant characteristics, are not reported in the registry record.
Intervention
The study evaluated ruxolitinib, a JAK1/JAK2 inhibitor. The Phase I dose groups are described as 50 mg, 100 mg, and 200 mg by mouth twice a day for a 28 day cycle.
Comparator
There was no separate control group: the study is described as nonrandomized and single group. The dose groups are not a like-for-like control comparison, and assignment procedures beyond the stated design are not reported in the registry record.
Primary question
The primary endpoint counts participants with dose-limiting toxicities during the first 28 day cycle. The stated dose-finding rule defines the maximum tolerated dose as the highest dose at which no more than one out of six subjects experiences a dose-limiting toxicity during that cycle.
The study’s stated goals were to find the highest tolerable dose of ruxolitinib and learn whether it could help control acute leukemia, while studying safety. The registry lists participants with a response as a secondary outcome. However, response results and the primary dose-limiting toxicity counts are not reported in the registry record. The design therefore frames a safety and dose-finding question, but the posted results do not allow readers to assess the primary endpoint or the secondary response outcome.
3. Trial Design
This was a Phase I/II study sponsored by M.D. Anderson Cancer Center. The registry records an enrollment of 27 and describes the study as nonrandomized, unmasked, and single group. It lists ruxolitinib dose groups of 50 mg, 100 mg, and 200 mg, each described as an oral dose taken twice a day for a 28 day cycle. The groups represent dose levels in a dose-finding study, not randomized treatment and control arms.
The study start is recorded as 2010-12, and primary completion as 2012-07. Its status is terminated. The reason for termination, the timing of termination, and whether termination affected enrollment or follow-up are not reported in the registry record. The registry’s enrollment count alone does not establish how many participants were evaluable for the primary endpoint.
The primary outcome is a count of participants with dose-limiting toxicities by the end of the first 28 day cycle. The outcome description specifies that a participant needed to receive at least 80% of the originally assigned doses in the first cycle to be evaluable for dose-limiting toxicity assessment. The registry does not provide the number evaluable in each dose group or the primary endpoint counts.
No random allocation or masking is reported; the listed design explicitly states nonrandomized and unmasked. The detailed dose-escalation sequence, rules for moving between doses, cohort enrollment decisions, and any protocol changes are not reported in the registry record. The endpoint description does state the maximum tolerated dose criterion, but it does not supply the dose-level results needed to apply that criterion.
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 | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|
| STARTED | 4 | 5 | 18 |
| COMPLETED | 3 | 3 | 7 |
| NOT COMPLETED | 1 | 2 | 11 |
Reasons for not completing this period:
| Reason | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|
| Participant/physician decision | 1 | 2 | 0 |
| Death | 0 | 0 | 11 |
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 | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID | Total |
|---|---|---|---|---|---|
| Participants analysed | 4 | 5 | 18 | 27 | |
| Age, Continuous | years | 66 (51 to 69) | 70 (41 to 85) | 71 (55 to 83) | 69 (41 to 85) |
| Sex: Female, Male: Female | Participants | 3 | 1 | 9 | 13 |
| Sex: Female, Male: Male | Participants | 1 | 4 | 9 | 14 |
| Region of Enrollment: United States | Participants | 4 | 5 | 18 | 27 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Dose Limiting Toxicities (DLTs), measured over End of first 28 day cycle for toxicity.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of participants with dose-limiting toxicities during the first 28 day cycle. The registry describes the maximum tolerated dose as the highest dose level at which no more than one out of six subjects experiences a dose-limiting toxicity in that cycle. The outcome is therefore a participant count used within a dose-finding rule, rather than a measure of leukemia response.
The registry defines a non-hematologic dose-limiting toxicity as a clinically significant grade 3 or 4 adverse event or abnormal laboratory value, according to Common Toxicity Criteria for Adverse Effects criteria, assessed as related to study drug and not related to disease progression, intercurrent illness, or concomitant medications. The description specifies that this assessment concerns events during the first 28 days on study. It also states that participants who received at least 80% of their originally assigned doses in the first cycle were evaluable for dose-limiting toxicity assessment. A broader definition covering other toxicity categories is not reported in the registry record.
This endpoint is used in dose-finding because it links observed first-cycle toxicity to a prespecified tolerability threshold. A count alone is interpretable only alongside the number assessed and the endpoint definition. The registry does not report the observed dose-limiting toxicity counts or evaluable participant counts, so the stated rule cannot be applied to identify a maximum tolerated dose from the posted results.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry records an enrollment of 27 and three ruxolitinib dose levels. The posted primary outcome is a count of participants with dose-limiting toxicities, but the outcome results are not reported in the registry record. Consequently, the available record does not show how many participants experienced a dose-limiting toxicity at any dose, how many were evaluable, or which dose level met the stated maximum tolerated dose criterion. The goal of determining a tolerable dose is described, but the record does not establish the result of that assessment.
The registry lists participants with a response as a secondary outcome, but response results are not reported in the registry record. The treatment’s effect on disease control therefore cannot be assessed from these posted results. A stated study aim is not itself evidence of an observed outcome, and no conclusion about efficacy follows from the study title or intervention description.
The safety information includes counts of participants at risk and affected by serious and other adverse events for each listed dose group. For 50 mg BID, 4 participants were at risk and 4 were affected; for 100 mg BID, 5 were at risk and 5 were affected; and for 200 mg BID, 18 were at risk and 18 were affected. Those are adverse-event summaries, not the missing primary dose-limiting toxicity results. The registry also lists serious adverse event terms with percentages, but the denominator and calculation basis for those percentages are not reported in the registry record. The terms should not be interpreted as dose-limiting toxicity rates or as evidence that the events were caused by ruxolitinib.
Because this was a nonrandomized, single-group study, differences across dose groups cannot be read as randomized estimates of a dose effect. Dose groups may differ in participant characteristics, exposure, or other factors, and the registry does not report enough information to assess those possibilities. The group sizes recorded for adverse events are also limited, particularly at the lower dose levels. Small counts can be compatible with substantial uncertainty; without endpoint counts and appropriate uncertainty estimates, the precision of a toxicity estimate cannot be judged.
No inferential analyses, confidence intervals, or statistical tests for the primary or secondary outcomes are reported in the registry record. A confidence interval, in general, describes the uncertainty around an estimated quantity; without a posted estimate and interval, readers cannot judge the range of values reasonably compatible with the data under the chosen method. The record supports a description of the planned question and the reported adverse-event summaries, but not a quantified estimate of the primary endpoint or a comparative treatment effect.
9. Statistical Methods Explained
What does the primary count represent?
The primary outcome is defined as a count of participants with dose-limiting toxicities by the end of the first 28 day cycle. In general, a count outcome records how many people meet a defined event criterion. To interpret such a count, readers need the number assessed as well as the number with the event. The denominator matters because the same event count can have different implications in groups of different sizes. The registry reports neither the primary event counts nor the evaluable counts by dose level, so the primary outcome cannot be summarized as an observed proportion or used to check the dose-finding rule.
The endpoint also depends on an attribution and severity definition. The record describes non-hematologic dose-limiting toxicities as clinically significant grade 3 or 4 adverse events or abnormal laboratory values assessed as related to study drug, and not related to disease progression, intercurrent illness, or concomitant medications. These judgments are part of the outcome definition: a reported adverse event is not automatically a dose-limiting toxicity. The registry does not report adjudication details or a separate definition for other toxicity categories.
How does the dose-finding rule work?
The registry states that the maximum tolerated dose is the highest dose at which no more than one out of six subjects experiences a dose-limiting toxicity during the first cycle. This is a rule for interpreting toxicity counts at dose levels. In general, a dose-finding rule maps observed outcomes to a dose decision; it is not the same as a randomized comparison of doses. The registry does not provide the primary endpoint counts needed to apply the stated rule, nor does it report how the study handled a dose level with fewer than six evaluable participants.
The record describes 50 mg BID as the starting dose and lists 100 mg BID and 200 mg BID as Phase I doses. It does not report the sequence in which participants entered those dose groups, the detailed escalation or stopping procedures, or whether the planned procedures changed. The maximum tolerated dose definition can be explained from the record, but the actual dose-selection process cannot be reconstructed from the information reported.
What can a single-group design estimate?
In general, a single-group study can describe outcomes observed among participants who received the study intervention. Without a concurrent control group and random allocation, it does not isolate the intervention’s effect from changes over time, participant differences, disease course, or other care. The dose groups in this study do not supply a randomized control comparison. Any difference in observed event counts between doses would need cautious interpretation, especially when group sizes and follow-up may differ. The registry does not report enough detail to assess exposure duration or follow-up by dose level.
Randomization is useful in comparative studies because it can balance measured and unmeasured factors on average across groups. That general benefit does not apply as a design feature in this nonrandomized study. The lack of masking also means that the registry does not describe blinded assessment. The record does not state whether outcome assessors were otherwise independent or whether toxicity assessments used procedures intended to limit assessment bias.
How should uncertainty be understood?
In general, estimates based on small groups are imprecise: a small change in the observed event count can substantially change the estimated event frequency. Confidence intervals can communicate this uncertainty by giving a range around an estimate under specified assumptions. They do not remove limitations in study design or account automatically for missing outcomes, inconsistent assessment, or confounding. No confidence intervals or other uncertainty estimates for the primary outcome are reported in the registry record.
The adverse-event summaries report participants at risk and affected for serious and other adverse events. Those counts describe the listed groups, but they do not replace endpoint-specific counts. Serious adverse events and dose-limiting toxicities are distinct classifications in the record: the former are presented as safety summaries, while the latter are defined through severity, timing, attribution, and evaluability criteria. A serious adverse event should not be assumed to meet the primary endpoint definition.
What is not available for statistical review?
The registry does not report the primary endpoint results, response results, confidence intervals, hypothesis tests, or a detailed statistical analysis plan. It also does not report the approach to interim analyses, missing outcome data, or analysis populations. In general, a statistical analysis plan specifies how outcomes, populations, and uncertainty will be handled; without such details, readers cannot determine which methods were intended or whether posted outcomes follow prespecified procedures. No method should be inferred from the study phase or from the endpoint definition alone.
The sample-size justification and any target power are not reported in the registry record. Planning calculations, in general, depend on assumptions about an effect, variability or event frequency, allocation, and analysis method. Such calculations are distinct from the number enrolled and from observed statistical power. The registry’s enrollment figure is not a justification for precision or power, and the record does not provide the information needed to evaluate a planned sample size.
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.
| Arm | Participants | Events | Observed % | Clopper-Pearson 95% CI | Wilson 95% CI | Bayesian 95% credible interval |
|---|---|---|---|---|---|---|
| Ruxolitinib 50 mg BID | 3 | 0 | 0.0% | 0.0% to 70.8% | 0.0% to 56.1% | 0.6% to 60.2% |
| Ruxolitinib 100 mg BID | 3 | 0 | 0.0% | 0.0% to 70.8% | 0.0% to 56.1% | 0.6% to 60.2% |
| Ruxolitinib 200 mg BID | 7 | 0 | 0.0% | 0.0% to 41.0% | 0.0% to 35.4% | 0.3% to 36.9% |
R code and output
arm <- c("Ruxolitinib 50 mg BID", "Ruxolitinib 100 mg BID", "Ruxolitinib 200 mg BID")
n <- c(3, 3, 7)
events <- c(0, 0, 0)
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 Ruxolitinib 50 mg BID 3 0 0.0 0.0 70.8 0.0 56.1 0.6 60.2
ARM Ruxolitinib 100 mg BID 3 0 0.0 0.0 70.8 0.0 56.1 0.6 60.2
ARM Ruxolitinib 200 mg BID 7 0 0.0 0.0 41.0 0.0 35.4 0.3 36.911. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary safety endpoint and a secondary outcome of participants with a response. It does not report results for either outcome. The number and structure of planned statistical tests, any hierarchy among outcomes, and any adjustment for multiple testing are not reported in the registry record. It is therefore not possible to determine whether multiplicity was addressed or how any reported inferential results should be interpreted; no inferential results are posted.
Missing-data methods and the handling of participants who did not complete the first 28 day cycle are not reported in the registry record. The endpoint description says participants needed to receive at least 80% of assigned doses in that cycle to be evaluable for dose-limiting toxicity assessment, but it does not give the number meeting that requirement or explain how participants who did not meet it were handled. The distinction between enrolled participants and endpoint-evaluable participants is important for interpreting toxicity counts.
Analysis populations are not reported in the registry record. The record does not say whether any analyses used all enrolled participants, only participants who received treatment, or a defined evaluable population. It also does not describe interim analyses, stopping boundaries beyond the stated maximum tolerated dose rule, or sensitivity analyses for missing outcomes. These omissions limit assessment of the intended analysis and the extent to which reported results, if available elsewhere, would represent all enrolled participants.
12. 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 |
|---|---|---|---|
| Ruxolitinib 50 mg BID | 4 | 4 | 100.0% |
| Ruxolitinib 100 mg BID | 5 | 5 | 100.0% |
| Ruxolitinib 200 mg BID | 18 | 18 | 100.0% |
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 |
|---|---|
| Ruxolitinib 50 mg BID | 4 of 4 |
| Ruxolitinib 100 mg BID | 5 of 5 |
| Ruxolitinib 200 mg BID | 18 of 18 |
Most frequent serious adverse event terms
| Event term | Organ system | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|---|
| Death | General disorders | 0 of 4 | 0 of 5 | 11 of 18 |
| Pneumonia | Respiratory, thoracic and mediastinal disorders | 2 of 4 | 3 of 5 | 1 of 18 |
| Hemoptysis | Respiratory, thoracic and mediastinal disorders | 3 of 4 | 1 of 5 | 0 of 18 |
| Fever | General disorders | 1 of 4 | 1 of 5 | 1 of 18 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 4 | 0 of 5 | 2 of 18 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 0 of 5 | 2 of 18 |
| Stroke | Nervous system disorders | 0 of 4 | 0 of 5 | 2 of 18 |
| Abdominal pain | Gastrointestinal disorders | 0 of 4 | 0 of 5 | 1 of 18 |
| Alveolar hemorrhage | Respiratory, thoracic and mediastinal disorders | 1 of 4 | 0 of 5 | 0 of 18 |
| Cerebral edema and mass | Nervous system disorders | 1 of 4 | 0 of 5 | 0 of 18 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Ruxolitinib 50 mg BID | Ruxolitinib 100 mg BID | Ruxolitinib 200 mg BID |
|---|---|---|---|---|
| Infections | Infections and infestations | 4 of 4 | 2 of 5 | 18 of 18 |
| Platelets decreased | Investigations | 2 of 4 | 3 of 5 | 6 of 18 |
| Neutrophils count decreased | Investigations | 2 of 4 | 2 of 5 | 6 of 18 |
| Alanine aminotransferase increased (ALT) | Investigations | 1 of 4 | 2 of 5 | 4 of 18 |
| Creatinine Increased | Investigations | 1 of 4 | 2 of 5 | 4 of 18 |
| Aspartate aminotransferase increased (AST) | Investigations | 1 of 4 | 1 of 5 | 2 of 18 |
| Fatigue | General disorders | 1 of 4 | 1 of 5 | 1 of 18 |
| Mucositis | Gastrointestinal disorders | 0 of 4 | 0 of 5 | 3 of 18 |
| Edema: head and neck | General disorders | 1 of 4 | 0 of 5 | 2 of 18 |
| Blood bilirubin increased | Investigations | 0 of 4 | 2 of 5 | 1 of 18 |
Serious events with the highest arm-level rate: hemoptysis (75.0%), death (61.1%), pneumonia (60.0%), alveolar hemorrhage (25.0%), cerebral edema and mass (25.0%).
For 50 mg BID, the registry reports 4 participants at risk and 4 affected for serious adverse events, and 4 at risk and 4 affected for other adverse events. For 100 mg BID, it reports 5 at risk and 5 affected for each category. For 200 mg BID, it reports 18 at risk and 18 affected for each category. These are participant counts within the listed groups; they do not state how many events each participant experienced.
The registry lists serious adverse event terms with percentages: Hemoptysis, 75.0%; Death, 61.1%; Pneumonia, 60.0%; Alveolar hemorrhage, 25.0%; Cerebral edema and mass, 25.0%; and Fever, 25.0%. The denominator, time period, and calculation basis for these term percentages are not reported in the registry record. They should not be treated as incidence estimates for a dose group or as evidence of causation. Serious adverse event summaries also do not directly supply the primary endpoint counts, which require the specific dose-limiting toxicity definition and first-cycle evaluation.
13. What the Result Does, and Does Not, Mean
The record establishes that a Phase I/II, nonrandomized, single-group study of ruxolitinib in relapsed or refractory acute leukemia was conducted, with an enrollment of 27 and three listed dose levels. It also specifies a first-cycle dose-limiting toxicity endpoint and a maximum tolerated dose rule. Those details clarify the question the study was designed to address.
The posted results do not report the primary dose-limiting toxicity counts, the number evaluable at each dose, or a selected maximum tolerated dose. They also do not report the secondary response results. Accordingly, the registry record does not allow a reader to determine from the posted outcomes how the study’s dose-finding objective was resolved or what response was observed. The listed serious adverse event summaries provide safety information but do not fill those gaps.
Even if dose-specific event counts were available, the design would not support treating differences between dose groups as randomized causal comparisons. In general, nonrandomized groups can differ for reasons other than the assigned dose. The record does not supply participant-level characteristics or sufficient methods to evaluate confounding, exposure, or the precision of any dose-specific estimate.
The result does not establish whether ruxolitinib controls disease, whether one listed dose is preferable, or whether the adverse events were caused by treatment. These conclusions require outcome information and an appropriate analysis; they should not be inferred from study aims, dose descriptions, or adverse-event term listings alone. The registry record’s terminated status is reported, but the reason for termination and its implications for the study results are not reported.
14. Why This Trial Matters Statistically
This study is statistically notable for the distinction between a dose-finding objective and an efficacy comparison. Its primary endpoint is a first-cycle toxicity count interpreted through a stated maximum tolerated dose rule. That is a different question from whether treatment improves disease outcomes, and the registry lists response as secondary rather than as the primary endpoint.
The nonrandomized, single-group structure also shapes what the results could establish. Dose levels are not randomized treatment arms with a control group. Even with complete outcome reporting, cross-dose differences would need to be interpreted as observational dose-group patterns rather than as unbiased causal contrasts. The design is therefore central to interpreting any apparent differences in outcomes.
Another important statistical issue is the distinction between enrolled and evaluable participants. The record specifies a minimum level of first-cycle dose receipt for dose-limiting toxicity evaluation, but does not state the number who met that criterion. Without those denominators and endpoint counts, readers cannot assess the observed toxicity frequency or apply the maximum tolerated dose rule.
Finally, completeness of results reporting matters. The registry identifies the outcomes of interest and gives some serious adverse event summaries, but does not report primary or response outcome results, statistical analyses, or uncertainty estimates. This leaves the central dose-finding and disease-response questions unresolved in the registry record. Clear outcome counts, participant flow, analysis populations, and uncertainty estimates would be necessary for a fuller statistical interpretation.
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 interpretation is that the registry does not report the primary endpoint results. The number of dose-limiting toxicities and the number evaluable at each dose are absent, so the stated maximum tolerated dose rule cannot be checked. The secondary response outcome is listed, but its results are also not reported in the registry record.
The study is nonrandomized, unmasked, and single group, with no separate control group. The dose cohorts therefore cannot be read as randomized comparisons. The registry does not report detailed allocation procedures, participant characteristics by dose, exposure duration, or follow-up sufficient to evaluate potential differences between cohorts.
The enrollment figure is 27, while the adverse-event summaries list 4 participants at risk for the 50 mg BID group, 5 for the 100 mg BID group, and 18 for the 200 mg BID group. The registry does not explain the relationship between these group counts and the overall enrollment or report the number evaluable for the primary endpoint. The reason for termination is also not reported.
Statistical methods beyond the endpoint definition are not reported in the registry record. This includes the sample-size justification, analysis populations, missing-data procedures, uncertainty estimates, and any interim analyses or multiplicity approach. The serious adverse event term percentages lack a reported denominator and calculation basis. These limitations prevent a complete assessment of outcome precision, potential bias, and how safety summaries relate to the primary dose-finding question.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Phase l/II Study of Ruxolitinib for Acute Leukemia — NCT01251965.
- Registry record last updated 2025-06-08; status last verified 2025-05.
- Results first posted 2019-09-06.
- 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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19. Record Summary
NCT01251965 is a Phase I/II study of ruxolitinib for acute leukemia, sponsored by M.D. Anderson Cancer Center. The official title describes subjects with relapsed or refractory acute leukemia. The registry records an enrollment of 27, a nonrandomized, unmasked, single-group design, and a terminated status. Study start is recorded as 2010-12, with primary completion recorded as 2012-07.
The listed dose groups are 50 mg, 100 mg, and 200 mg by mouth twice a day for a 28 day cycle. The primary outcome is the count of participants with dose-limiting toxicities during the first cycle, and the registry states a maximum tolerated dose criterion. Primary endpoint results and secondary response results are not reported in the registry record. Serious adverse event summaries are listed, but the denominator for the reported term percentages is not reported.