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 DS-3201b for Acute Myelogenous Leukemia (AML) or Acute Lymphocytic Leukemia (ALL) (NCT03110354), a non-randomized phase 1 study. The primary outcome is Number of Participants With Any Grade Treatment-emergent Adverse Event Classified as Dose-limiting Toxicities (Dose Escalation).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03110354 |
| Phase | Phase 1 |
| Condition | Leukemia, Myeloid, Acute, Leukemia, Lymphocytic, Acute |
| Allocation | Non-Randomized |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 28 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Daiichi Sankyo |
| Results posted | Yes |
2. Clinical Question
The study asked whether treatment-emergent adverse events meeting the registry’s dose-limiting toxicity definition occurred during the first cycle of DS-3201b treatment. It enrolled people with acute myelogenous leukemia (AML) or acute lymphocytic leukemia (ALL), described as having disease that did not respond or no longer responded to previous standard therapy. The registry describes dose escalation followed by additional enrollment at a dose selected after escalation; it does not report the result of that dose-selection process.
Population
Participants with AML or ALL who did not respond or no longer responded to previous standard therapy. The primary endpoint population is identified as the DLT Evaluable Set; its detailed membership criteria are not reported in the registry record.
Intervention
Oral DS-3201b, assigned in dose groups of 100 mg, 150 mg, 250 mg, 500 mg, or 700 mg.
Comparator
There was no randomized control group. The listed groups received different DS-3201b doses in a non-randomized, sequential study, so they are not a like-for-like control comparison.
Primary question
How many participants in the DLT Evaluable Set experienced at least one qualifying dose-limiting toxicity from baseline up to Day 28?
This is a dose-escalation safety question, not a randomized test of DS-3201b against another treatment. The registry names pharmacokinetic outcomes as secondary measures, including peak concentration, time to peak concentration, area under the plasma concentration-time curve, and trough concentration. Their results are not reported in the registry record.
3. Trial Design
The registry describes a phase 1 study with non-randomized allocation, no masking, and a sequential model. The brief summary divides the study into two parts. Part 1 was a dose-escalation stage intended to identify the highest dose participants could tolerate or a recommended dose. The summary then describes enrolling additional participants at that dose in Part 2. The registry record does not state which dose was selected or provide the detailed rules governing escalation.
The intervention was DS-3201b taken orally. The dose groups listed in the record are 100 mg, 150 mg, 250 mg, 500 mg, and 700 mg. Enrollment is recorded as 28. The study began on 2017-04-05, had a primary completion date of 2021-03-09, and its status is terminated. The registry record does not report why the study was terminated.
Because allocation was not randomized and there was no masking, the dose groups should be read as sequential dose cohorts, not as concurrent treatment and control groups. Participants at different doses may differ for reasons beyond dose, including the timing and sequence of enrollment; the record does not provide enough detail to assess those differences. Comparisons across dose groups therefore describe the registered cohorts and cannot isolate a dose effect in the way a randomized comparison is designed to do.
The study is registered for people with AML or ALL. Its brief summary says Part 1 would enroll people whose disease did not respond or no longer responded to prior standard therapy. Further eligibility details, participant characteristics by dose, treatment duration, and the operational details of dose escalation are not reported in the registry record. The record lists pharmacokinetic outcomes as secondary measures but does not give their estimates or analysis methods. Its design description is therefore sufficient to identify the broad safety and dose-escalation purpose, but not to reconstruct the full conduct of the trial.
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 | DS-3201b 100mg | DS-3201b 150 mg | DS-3201b 250 mg | DS-3201b 500 mg | DS-3201b 700 mg |
|---|---|---|---|---|---|
| STARTED | 4 | 4 | 3 | 10 | 7 |
| COMPLETED | 0 | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 4 | 4 | 3 | 10 | 7 |
Reasons for not completing this period:
| Reason | DS-3201b 100mg | DS-3201b 150 mg | DS-3201b 250 mg | DS-3201b 500 mg | DS-3201b 700 mg |
|---|---|---|---|---|---|
| Death | 1 | 0 | 0 | 1 | 0 |
| Start of new therapy | 0 | 0 | 0 | 0 | 1 |
| Failure to achieve response | 2 | 3 | 0 | 2 | 1 |
| Progressive disease | 1 | 1 | 3 | 4 | 2 |
| Adverse Event | 0 | 0 | 0 | 2 | 2 |
| Other | 0 | 0 | 0 | 1 | 1 |
Analysis population for the primary outcome, as defined in the registry: Dose-limiting toxicities were assessed in the DLT Evaluable Set.
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: Demographic and baseline characteristics were assessed in the Safety Analysis Set.
| Characteristic | Unit | DS-3201b 100mg | DS-3201b 150 mg | DS-3201b 250 mg | DS-3201b 500 mg | DS-3201b 700 mg | Total |
|---|---|---|---|---|---|---|---|
| Participants analysed | 4 | 4 | 3 | 10 | 7 | 28 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 | 2 | 1 | 8 | 4 | 16 |
| Age, Categorical: >=65 years | Participants | 3 | 2 | 2 | 2 | 3 | 12 |
| Age, Continuous | years | 62.5 (SD 16.9) | 59.8 (SD 27.7) | 64.7 (SD 22.2) | 54.6 (SD 16.0) | 55.9 (SD 19.0) | 57.9 (SD 18.2) |
| Sex: Female, Male: Female | Participants | 2 | 2 | 2 | 9 | 3 | 18 |
| Sex: Female, Male: Male | Participants | 2 | 2 | 1 | 1 | 4 | 10 |
| Race/Ethnicity, Customized: White | Participants | 3 | 4 | 3 | 7 | 5 | 22 |
| Race/Ethnicity, Customized: Black or African American | Participants | 0 | 0 | 0 | 2 | 0 | 2 |
| Race/Ethnicity, Customized: Asian | Participants | 1 | 0 | 0 | 0 | 1 | 2 |
| Race/Ethnicity, Customized: American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race/Ethnicity, Customized: Other | Participants | 0 | 0 | 0 | 1 | 1 | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Any Grade Treatment-emergent Adverse Event Classified as Dose-limiting Toxicities (Dose Escalation), measured over Baseline up to Day 28.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts participants with any grade treatment-emergent adverse event classified as a dose-limiting toxicity. The stated timeframe is baseline up to Day 28, corresponding to the first 28-day cycle on study. The endpoint population is the DLT Evaluable Set. The registry record does not provide further detail on how participants qualified for that set.
The definition is based on clinically significant non-hematologic adverse events or abnormal clinical laboratory values judged clearly unrelated to disease progression or intercurrent illness. It includes specified severe liver enzyme or bilirubin abnormalities lasting at least 7 days, Grade 4 AST or ALT abnormalities of any duration, Grade 4 non-hematologic toxicities, qualifying Grade 5 toxicities, and other clinically significant non-hematologic Grade 3 or Grade 4 adverse events. The record states that adverse events were coded using MedDRA, Version 23.0.
In dose escalation, a DLT count is used to characterize acute tolerability within a defined early treatment period and to inform dose decisions. It is a count of participants meeting the endpoint definition, not a general measure of every possible adverse event or of longer-term safety. The registry record includes the endpoint definition but does not report the observed DLT counts, the denominator in the DLT Evaluable Set, or the dose-escalation decision based on those counts. Consequently, the endpoint’s intended role can be described, but its observed result cannot be interpreted from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The record identifies a primary safety endpoint and defines how a dose-limiting toxicity was to be classified, but it does not report the endpoint result. The registry lists 6 outcome measures and 0 analyses. No observed DLT count, DLT Evaluable Set denominator, effect estimate, confidence interval, or test result is reported in the registry record. The record therefore does not show how many participants met the primary endpoint at any dose.
The safety information is recorded by dose group. For serious adverse events, the registry lists 3 affected participants among 4 at risk in the 100mg group, 3 among 4 in the 150 mg group, 3 among 3 in the 250 mg group, 7 among 10 in the 500 mg group, and 6 among 7 in the 700 mg group. These are group-level counts in the safety record; they are not the primary DLT endpoint. A serious adverse event and a DLT are distinct classifications, and the registry’s primary endpoint definition includes criteria that cannot be inferred merely from whether an event was serious.
The registry also records other adverse events: 3 affected participants among 4 at risk at 100mg, 4 among 4 at 150 mg, 3 among 3 at 250 mg, 9 among 10 at 500 mg, and 7 among 7 at 700 mg. The record does not provide enough information in these counts to establish event severity, timing, recurrence, or relationship to treatment. They should not be interpreted as evidence that one dose is safer or less safe than another.
Deaths are listed as counts by group: 4, 4, 3, 10, and 6 for the 100mg, 150 mg, 250 mg, 500 mg, and 700 mg groups, respectively. The registry information presented does not specify the cause, timing, or treatment relationship for these deaths. Those counts do not establish that DS-3201b caused the deaths.
The listed serious-event terms include pneumonia (75.0%), febrile neutropenia (50.0%), disease progression (50.0%), gastrointestinal hemorrhage (33.3%), cholecystitis infective (33.3%), and Clostridium difficile infection (33.3%). The percentage denominators and the full context for these term-level figures are not reported in the registry record. These values are descriptive event information, not comparative treatment effects. With non-randomized dose groups, small group-level counts, and no reported primary endpoint results, conclusions about dose response or comparative safety are not supported by the registry record.
9. Statistical Methods Explained
The registry describes a non-randomized, unmasked, sequential phase 1 study. Its primary measure is a count of participants who experienced any qualifying dose-limiting toxicity during the first cycle. No primary endpoint analysis or inferential result is reported in the registry record. The statistical ideas below explain how such data are generally interpreted; they do not describe unreported procedures from this study.
What does a participant count represent?
For a binary endpoint such as whether a participant did or did not experience a DLT, a count records the number meeting the definition in a specified analysis population and timeframe. To interpret a count as a rate, the relevant denominator is also needed. The registry identifies the DLT Evaluable Set as the population but does not report its size or the observed number meeting the endpoint. Without both values, the DLT frequency cannot be read from the record.
A participant-level count is also different from an event count. One participant could experience more than one event, but the primary endpoint is worded as the number of participants with any qualifying event. Counting participants avoids treating multiple events in the same participant as if they came from separate people. The registry’s adverse-event summaries also distinguish participants affected from participants at risk, but those safety summaries are not interchangeable with the defined DLT endpoint.
Why does the endpoint definition matter?
Safety endpoints depend on prespecified rules about event type, severity, duration, timing, and attribution. Here, the record defines DLTs through specified non-hematologic events or laboratory abnormalities during the first cycle, with exceptions for events clearly related to disease progression or intercurrent illness. The definition determines which observations count toward the endpoint. A broad adverse-event count cannot substitute for it: events may be reported as adverse events without satisfying the endpoint criteria, while a qualifying event must meet the DLT definition.
In general, clear endpoint definitions improve consistency in classification. They do not remove judgment from decisions such as whether an event is clinically significant or clearly related to another cause. The registry says adverse events were coded using MedDRA, Version 23.0, but does not report further details about review procedures, adjudication, or missing-event handling.
How should dose groups be compared?
In general, random assignment helps balance participant characteristics across treatment groups, supporting a causal comparison under the trial’s assumptions. This study is recorded as non-randomized and sequential, so dose groups are not exchangeable by design. Group differences could reflect dose, but could also reflect cohort composition, enrollment timing, or other factors. The registry record does not describe adjustments or other methods that would separate those explanations.
Sequential dose escalation is generally structured to use emerging safety information when deciding whether to proceed to another dose. That general purpose does not establish the specific rules used in this study. The registry record does not report escalation boundaries, stopping rules, an interim-analysis plan, or the dose-selection algorithm. It is not possible to infer those details from the sequence of listed dose groups alone.
What do uncertainty measures add?
General explanation: a confidence interval describes the range of values reasonably compatible with the observed data under a statistical model and its assumptions. In small groups, uncertainty around a proportion or count-based estimate can be substantial. A point estimate alone can hide that uncertainty. The registry record does not report confidence intervals for the primary endpoint or the listed safety counts, so their precision cannot be assessed through an interval estimate.
General explanation: a significance test addresses compatibility between data and a specified statistical null model; it does not measure clinical importance, prove causation, or show that groups are equivalent. Since the registry lists 0 analyses, no primary endpoint test or inferential comparison is reported. The absence of a reported test is not evidence that an effect is absent.
How should planning calculations be understood?
The registry’s sample-size assumptions describe planning illustrations based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. The assumptions also distinguish participants per arm for non-time-to-event outcomes from total events for time-to-event outcomes. These are general planning concepts, not observed trial results or evidence of achieved power.
The record does not state that these assumptions were the study’s original design justification, and no sample-size illustration values or observed primary effect are reported in the registry record. A planning calculation cannot establish the actual precision of this sequential dose-escalation study, particularly when its design is not a two-arm randomized comparison. Sample-size figures, when used for planning, describe a hypothetical design under stated assumptions rather than the certainty of a completed result.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 6 outcome measures and 0 analyses. It names a primary DLT measure and several secondary pharmacokinetic measures, but does not report outcome estimates, hypothesis tests, confidence intervals, or a statistical analysis plan. A multiplicity strategy—how the chance of false-positive findings across multiple tests would be handled—is not reported in the registry record. With no inferential analyses reported, it is not possible to determine whether any adjustment was planned or used.
Missing-data methods are not reported in the registry record. The record does not explain how participants with incomplete Day 28 follow-up, unavailable laboratory measurements, or incomplete pharmacokinetic sampling would be handled. It also does not report the number of participants with missing measurements or reasons for missingness. These omissions matter because the interpretation of a count or measurement can depend on which participants are included and how incomplete observations are treated.
The primary endpoint population is named as the DLT Evaluable Set, but its detailed inclusion criteria and size are not reported. The record does not say whether the analysis included all enrolled participants, only participants meeting evaluability requirements, or how those requirements were applied. Consequently, the registered enrollment of 28 should not be treated as the denominator for the primary endpoint.
The record also does not report interim analyses, a stopping boundary, a prespecified statistical plan, or the rationale for the recorded termination status. These elements should not be inferred from the study’s phase, sequential model, or dose groups. The available record supports a description of the planned endpoint and registered safety summaries, but not a reconstruction of the analysis decisions or handling of incomplete data.
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 |
|---|---|---|---|
| DS-3201b 100mg | 3 | 4 | 75.0% |
| DS-3201b 150 mg | 3 | 4 | 75.0% |
| DS-3201b 250 mg | 3 | 3 | 100.0% |
| DS-3201b 500 mg | 7 | 10 | 70.0% |
| DS-3201b 700 mg | 6 | 7 | 85.7% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| DS-3201b 100mg | 4 of 4 |
| DS-3201b 150 mg | 4 of 4 |
| DS-3201b 250 mg | 3 of 3 |
| DS-3201b 500 mg | 10 of 10 |
| DS-3201b 700 mg | 6 of 7 |
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 |
|---|---|
| DS-3201b 100mg | 3 of 4 |
| DS-3201b 150 mg | 4 of 4 |
| DS-3201b 250 mg | 3 of 3 |
| DS-3201b 500 mg | 9 of 10 |
| DS-3201b 700 mg | 7 of 7 |
Most frequent serious adverse event terms
| Event term | Organ system | DS-3201b 100mg | DS-3201b 150 mg | DS-3201b 250 mg | DS-3201b 500 mg | DS-3201b 700 mg |
|---|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | 1 of 4 | 2 of 4 | 1 of 3 | 2 of 10 | 2 of 7 |
| Disease progression | General disorders | 1 of 4 | 2 of 4 | 0 of 3 | 3 of 10 | 2 of 7 |
| Pneumonia | Infections and infestations | 3 of 4 | 2 of 4 | 0 of 3 | 1 of 10 | 0 of 7 |
| Sepsis | Infections and infestations | 1 of 4 | 1 of 4 | 0 of 3 | 1 of 10 | 1 of 7 |
| Cellulitis | Infections and infestations | 0 of 4 | 1 of 4 | 0 of 3 | 0 of 10 | 2 of 7 |
| Perirectal abscess | Infections and infestations | 0 of 4 | 1 of 4 | 0 of 3 | 0 of 10 | 1 of 7 |
| Clostridium difficile infection | Infections and infestations | 0 of 4 | 0 of 4 | 1 of 3 | 1 of 10 | 0 of 7 |
| Hemorrhage intracranial | Nervous system disorders | 1 of 4 | 0 of 4 | 0 of 3 | 0 of 10 | 0 of 7 |
| Acute kidney injury | Renal and urinary disorders | 1 of 4 | 0 of 4 | 0 of 3 | 0 of 10 | 0 of 7 |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | 1 of 4 | 0 of 4 | 0 of 3 | 0 of 10 | 0 of 7 |
Most frequent non-serious adverse event terms
| Event term | Organ system | DS-3201b 100mg | DS-3201b 150 mg | DS-3201b 250 mg | DS-3201b 500 mg | DS-3201b 700 mg |
|---|---|---|---|---|---|---|
| Diarrhea | Gastrointestinal disorders | 0 of 4 | 2 of 4 | 2 of 3 | 4 of 10 | 4 of 7 |
| Nausea | Gastrointestinal disorders | 1 of 4 | 2 of 4 | 1 of 3 | 4 of 10 | 4 of 7 |
| Vomiting | Gastrointestinal disorders | 0 of 4 | 1 of 4 | 0 of 3 | 6 of 10 | 3 of 7 |
| Decreased appetite | Metabolism and nutrition disorders | 2 of 4 | 0 of 4 | 1 of 3 | 2 of 10 | 5 of 7 |
| Hypomagnesaemia | Metabolism and nutrition disorders | 0 of 4 | 1 of 4 | 1 of 3 | 4 of 10 | 2 of 7 |
| Dysgeusia | Musculoskeletal and connective tissue disorders | 0 of 4 | 0 of 4 | 1 of 3 | 4 of 10 | 2 of 7 |
| Headache | Musculoskeletal and connective tissue disorders | 2 of 4 | 1 of 4 | 0 of 3 | 1 of 10 | 3 of 7 |
| Hypokalaemia | Metabolism and nutrition disorders | 0 of 4 | 2 of 4 | 0 of 3 | 2 of 10 | 2 of 7 |
| Hypotension | Vascular disorders | 0 of 4 | 0 of 4 | 1 of 3 | 2 of 10 | 3 of 7 |
| Chills | General disorders | 0 of 4 | 0 of 4 | 1 of 3 | 2 of 10 | 2 of 7 |
Serious events with the highest arm-level rate: pneumonia (75.0%), febrile neutropenia (50.0%), disease progression (50.0%), gastrointestinal hemorrhage (33.3%), cholecystitis infective (33.3%).
The serious adverse-event summaries give the number of participants at risk and the number affected in each dose group. These are descriptive counts. They do not indicate whether an event was judged related to DS-3201b, whether it met the primary DLT definition, or when it occurred. Serious adverse events and DLTs are not synonymous: the primary endpoint uses its own criteria, including event type, grade, timing, and specified attribution rules.
The record lists deaths by group but does not report their causes, timing, or relationship to treatment. The counts alone cannot be attributed to DS-3201b. The listed serious-event terms and percentages are also descriptive; their percentage denominators are not reported in the registry record. A term appearing in this list does not establish a comparative risk or a treatment effect.
Group sizes are small and differ across dose levels, and allocation was non-randomized and sequential. Therefore, apparent differences between groups should not be treated as reliable dose comparisons. The safety summaries offer a record of reported events, but the registry does not provide enough detail to establish causality or quantify comparative risk.
12. What the Result Does, and Does Not, Mean
The registry record documents a phase 1 investigation of oral DS-3201b in people with AML or ALL, with dose escalation as a central purpose and a DLT count defined for the first cycle. It also records adverse-event summaries for the listed dose groups. These details establish the broad safety question addressed by the study and the categories of information recorded.
They do not establish the observed result for the primary endpoint. The record does not report the number of participants with DLTs, the DLT Evaluable Set denominator, or an estimate with an uncertainty interval. It also does not report pharmacokinetic outcome results. Therefore, the registry record cannot show whether a particular dose met the intended dose-selection criteria or how precisely any safety or pharmacokinetic quantity was estimated.
The adverse-event counts and term-level percentages are not evidence of causation. Some listed serious-event terms include disease progression, and the registry does not provide the context needed to distinguish illness-related events from treatment-related events. The deaths recorded by group likewise cannot be attributed to treatment from these counts alone. No randomized control group was used, so the dose groups do not provide a controlled comparison of DS-3201b against another intervention.
More generally, a registered endpoint definition is not itself an endpoint result, and a descriptive safety summary is not a causal estimate. The non-randomized, sequential design is appropriate to describe as dose escalation, but comparisons among its groups can be affected by cohort differences and timing. The record does not report adjustment methods that would address those concerns.
The study status is terminated, but the record does not state why. That status should not be interpreted as evidence of benefit, harm, success, or failure. The posted information supports a careful account of the design, endpoint definition, and registered safety counts, while leaving the primary outcome and several key analysis details unavailable in the registry record.
13. Why This Trial Matters Statistically
This study illustrates a distinction important in early-phase research: a trial may register a detailed safety endpoint and dose groups without reporting the outcome values needed to evaluate that endpoint. Here, the DLT definition is specific about the first-cycle timeframe and qualifying events, but the primary DLT count and analysis population size are not reported. Without those results, the central dose-escalation safety question cannot be answered from the registry record.
It also shows why design labels shape interpretation. Sequential, non-randomized dose groups are not randomized comparisons. Their safety counts describe observed cohorts, but differences across dose levels cannot be attributed to dose alone on the basis of the registry information. This matters especially when group sizes are small and the record does not provide uncertainty intervals or adjusted analyses.
Finally, the record separates several concepts that can otherwise be conflated: dose-limiting toxicity, serious adverse events, other adverse events, deaths, and pharmacokinetic outcomes. They address different aspects of safety and drug exposure. A serious adverse event does not automatically satisfy the DLT criteria, and a group-level death count does not establish treatment causation. Interpreting early-phase records carefully requires preserving these distinctions and acknowledging when outcome results and analysis details are absent.
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.
Several limitations specific to this trial follow from the registry information. The study was non-randomized, unmasked, and sequential, with no randomized control group. Dose groups therefore should not be treated as equivalent groups differing only in dose. Differences in who entered each cohort or when they entered could affect observed safety summaries, and the registry record does not describe methods for addressing such differences.
The registry records enrollment of 28, but does not provide the DLT Evaluable Set size or the primary endpoint count. It also does not report participant characteristics by dose, detailed eligibility criteria, or the rules used to determine evaluability. This limits assessment of the analysis population and of how representative each cohort was of the enrolled population.
The safety record does not supply detailed event-level timing, duration, severity, or treatment attribution for the summaries. The percentage denominators for the listed serious-event terms are not reported in the registry record. The deaths are listed as counts without causes or relationship to treatment. These limitations prevent causal interpretation and make dose-to-dose safety comparisons particularly uncertain.
Further, the registry lists 0 analyses and does not report a statistical analysis plan, missing-data methods, interim analyses, dose-escalation rules, or the reason for termination. The primary completion date and terminated status provide study-record context, but do not explain why the study ended or what decision followed. These are limitations of what the registry record reports; they should not be filled in by speculation.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: DS-3201b for Acute Myelogenous Leukemia (AML) or Acute Lymphocytic Leukemia (ALL) — NCT03110354.
- Registry record last updated 2024-01-31; status last verified 2024-01.
- Results first posted 2024-01-30.
- 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
NCT03110354 is a phase 1, non-randomized, unmasked, sequential study of oral DS-3201b in people with AML or ALL. The registry lists dose groups of 100 mg, 150 mg, 250 mg, 500 mg, and 700 mg, and enrollment of 28. The study began on 2017-04-05, its primary completion date is 2021-03-09, and its status is terminated.
The primary outcome is the number of participants with any qualifying DLT from baseline up to Day 28, assessed in the DLT Evaluable Set. The registry defines qualifying events but does not report the primary outcome count, its denominator, or an analysis. It lists 6 outcome measures and 0 analyses. Dose-group safety counts and serious-event terms are recorded, but the registry does not provide enough context to establish causation or make controlled dose comparisons. The reason for termination and detailed statistical, missing-data, and dose-escalation procedures are not reported in the registry record.