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 Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay (NCT01872819), a na na study. The primary outcome is Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01872819 |
| Phase | NA |
| Condition | Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a) |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 16 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Washington |
| Results posted | Yes |
2. Clinical Question
Population
The study focused on poor-risk patients with relapsed or refractory acute myeloid leukemia (AML). The registry lists a broader set of conditions, including several AML subtypes, chronic myelomonocytic leukemia, myelodysplastic/myeloproliferative neoplasm, previously treated myelodysplastic syndromes, and refractory anemia with excess blasts. The primary outcome description says 14 patients were treated.
Intervention
A high-throughput drug sensitivity assay tested multiple drugs against a patient’s donated blood sample. The stated goal was to use the assay to help select treatment. Patients received 1 of 160 possible interventions based on the assay, with treatment described as chemotherapy or biological therapy.
Comparator
There was no separate control group. The registry identifies a single-group study, with allocation and masking listed as NA and NONE, respectively. The record therefore does not describe a randomized, masked comparison between assay-guided care and another treatment-selection approach.
Primary question
The main question was whether individualized drug screening could be performed and therapy initiated within the stated timeframe for poor-risk patients with relapsed or refractory AML. The outcome description also refers to the time from sample procurement to assay results. The timeframe is up to 21 days. The registry record does not report the measured median or full range, so the degree of feasibility cannot be assessed from the posted outcome information.
In statistical terms, this is primarily a feasibility question about a sequence of laboratory and treatment-planning steps. It is distinct from asking whether assay-guided treatment improves response or survival compared with an alternative strategy. The record lists a complete-response outcome as secondary, but does not report its result.
3. Trial Design
This was a completed, single-group study sponsored by the University of Washington. The registry lists enrollment of 16. The primary outcome description specifies that 14 patients were treated; the record does not explain the difference between enrollment and the treated population. The phase is listed as NA. Allocation is also listed as NA, and masking is listed as NONE.
The intervention combined laboratory drug screening with treatment selection. An assay tested multiple drugs against a patient’s donated blood sample, with the aim of matching a drug to the disease. The treatment description says that patients received 1 of 160 possible interventions based on the assay. The intervention categories include the antitumor drug screening assay, chemotherapy, and biological therapy. The registry does not give a control arm or a like-for-like comparison group.
The primary outcome concerns the achievability of performing individualized screening and initiating therapy within the specified period. Its timeframe is up to 21 days, and its description refers to the time from sample procurement to assay results. The intended summary measure is a median in days with a full range. The numerical median and range are not reported in the registry record.
The registry lists the study as completed, with a start date of 2013-08-02 and primary completion in 2014-11. These status and timing details do not substitute for outcome results. The record does not describe the recruitment flow, eligibility decisions for each participant, or the reasons any enrolled participant was not treated. It also does not report the protocol’s sample-size justification or a prespecified statistical analysis plan.
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 | Treatment (Chemotherapy, Biological Therapy) |
|---|---|
| STARTED | 14 |
| COMPLETED | 14 |
| NOT COMPLETED | 0 |
Analysis population for the primary outcome, as defined in the registry: 14 patients were treated.
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: relapsed, refractory AML patients. 16 patients were enrolled, 14 patients were treated.
| Characteristic | Unit | Treatment (Chemotherapy, Biological Therapy) |
|---|---|---|
| Participants analysed | 16 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 10 |
| Age, Categorical: >=65 years | Participants | 6 |
| Sex: Female, Male: Female | Participants | 6 |
| Sex: Female, Male: Male | Participants | 10 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 16 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 2 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 |
| Race (NIH/OMB): White | Participants | 14 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 16 |
6. Primary Endpoint
The registry lists the primary outcome as Achievability of Performing Individualized Drug Screening and Initiating Therapy Based on the Results of the Drug Screen for Poor Risk Patients With Relapsed or Refractory AML, measured over Up to 21 days.
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | Full Range |
| Unit | days |
| Endpoint classification | Time-to-Event Analysis |
The primary endpoint is intended to assess whether individualized drug screening could be carried out and therapy initiated within the study’s stated timeframe for poor-risk patients with relapsed or refractory AML. The registry gives a timeframe of up to 21 days. Its outcome description specifies time from sample procurement to assay results, while the endpoint title also refers to initiating therapy based on screening. These are related operational steps, but the record does not clarify how completion of the overall process was defined for analysis.
The registry identifies the measure as a median time in days, with a full range. A median describes the middle observation after times are ordered; a full range shows the lowest and highest observed times. In general, these summaries can describe both a typical turnaround and the spread of turnaround times without requiring a symmetric distribution. The registry record does not report the median, range, or any other numerical result for this endpoint.
A timing endpoint is relevant to a feasibility question because a laboratory result is useful for treatment planning only if it is available within the clinical process being studied. However, this endpoint alone would not establish that the assay-guided treatment was more effective than another approach. The record does not report a comparative estimate for treatment benefit, and the study has no separate control group described.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a single-group feasibility study, not a randomized comparison. Its primary question concerns the ability to perform individualized drug screening and initiate therapy within the stated timeframe. That question is clinically and operationally different from whether assay-guided treatment changes response or survival relative to another strategy.
The primary outcome is defined as a median time in days with a full range, over a timeframe of up to 21 days. However, the numerical median and range are not reported in the registry record. Without those values, readers cannot determine how long the process took for the treated participants, how variable the timing was, or whether the observed times met the intended timeframe. The registry’s outcome description refers specifically to time from sample procurement to assay results, while the outcome title also mentions initiating therapy. The record does not clarify whether the reported timing was meant to cover only assay turnaround or the full path through treatment initiation.
The primary outcome population is described as 14 patients treated, while overall enrollment is listed as 16. No participant flow or explanation of this difference is reported in the registry record. This matters when interpreting feasibility: a result among treated participants may not describe the experience of everyone enrolled or considered for the process.
The record lists complete response, defined by criteria of Cheson et al., as a secondary outcome. It does not report a response result. The registry also indicates 0 analyses, which means no analyses are posted there; it does not establish what analyses may have been conducted elsewhere. No effect estimate, confidence interval, or p-value is reported for the primary outcome in the record. Thus, the posted information does not allow an assessment of statistical precision or a formal test of a prespecified hypothesis.
The safety section records serious adverse events for 11 of 14 participants at risk. The listed serious-event terms include febrile neutropenia and several other events, with registry percentages shown for each. These are descriptive safety figures in a single group, not a comparison of risks between treatments. Deaths are not reported in the registry record. These safety details do not fill the gap in the missing primary timing result or establish a comparative safety effect.
Overall, the record identifies the study’s intended feasibility question and the broad assay-guided treatment approach, but does not post the primary numerical outcome. The posted information therefore supports a description of the design and reported safety events, not a conclusion about how often the process met its timeframe or whether assay-guided treatment changed clinical outcomes.
9. Statistical Methods Explained
The registry describes a median-time endpoint with a full range. It does not report a numerical result for that measure, a confidence interval, or an inferential analysis. The methods below explain how such summaries are generally interpreted; they should not be read as a description of analyses confirmed to have been performed in this study.
What does a median time summarize?
In general, a median is the central value after observations have been ordered from shortest to longest. It can be useful for timing data because a small number of unusually long or short times may pull an arithmetic mean more strongly than they shift the median. A full range gives the lowest and highest observed values and shows the total span of the recorded observations. The range is sensitive to extreme observations, so it does not by itself describe how values are distributed between the endpoints.
For this study, the planned measure is a median in days and a full range, with the outcome timeframe described as up to 21 days. The registry record does not report the median or range. It therefore does not show the observed central time or the spread of times. Nor does the record state how incomplete timing observations, if any, were handled.
What is the timing event?
The outcome title refers to performing individualized drug screening and initiating therapy based on the screen. The outcome description says the time is from sample procurement to assay results. Those statements do not fully define the event being timed: assay completion and treatment initiation are separate operational milestones. The registry record does not report whether the primary measure ended when the assay result became available, when therapy began, or at another point. A clear operational definition is important because different endpoints can yield different timing summaries even when they refer to the same care pathway.
The stated timeframe is up to 21 days. In general, a threshold can make a feasibility endpoint easier to interpret if the record reports how many participants met it and how that status was defined. No such count or proportion is reported. The registry also does not report whether participants who did not reach the assay or treatment milestone were included in the time summary, treated as failures to meet the timeframe, or excluded.
What can a single-group design establish?
A single-group study can describe what happened to participants who underwent the assay-guided process. Without a comparator, it cannot separate the effect of the assay-guided strategy from other influences or show what would have happened under another treatment-selection strategy. General statistical interpretation of a between-group treatment effect requires a comparison; this record describes no separate control group.
The design is listed as single group, with allocation NA and masking NONE. These details are consistent with the absence of a randomized, masked comparison, but the registry does not provide further information about how each treatment option was selected or how decisions were made. It states that patients received 1 of 160 possible interventions based on the assay. The record does not report treatment-specific outcome estimates or comparative analyses across those interventions.
How should secondary outcomes be read?
The registry lists complete response, defined by criteria of Cheson et al., as a secondary outcome. A defined outcome can make the intended clinical measure clearer, but a definition is not itself a result. The registry record does not report the number of complete responses, a response rate, or an analysis of that outcome. The record reports 2 outcome measures and 0 analyses; the latter describes what is posted in the registry and does not establish that no analysis was conducted outside it.
What do precision measures tell us?
In general, a confidence interval describes a range of values compatible with the data and the method used to calculate it. A narrower interval indicates greater statistical precision under that method, while a wider interval indicates more uncertainty. A p-value, when calculated under a stated test, summarizes how compatible the observed data are with a specified null hypothesis; it does not measure the size or practical importance of an effect. Neither a confidence interval nor a p-value for the primary timing outcome is reported in the registry record. Without the endpoint result and a stated inferential analysis, precision and hypothesis testing cannot be evaluated from the posted information.
How should the planning illustrations be read?
The page includes a sample-size section based on planning assumptions, not a reported design calculation for this trial. Such illustrations are not observed power and do not show what this study was powered to detect. The stated assumptions use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and effects expressed as fractions of an observed effect. Those hypothetical two-arm assumptions do not describe the actual single-group design listed in the registry. The registry record does not report the protocol’s sample-size justification or a prespecified power calculation. The planning illustrations should therefore be treated as general scenarios, not as evidence about the adequacy of the enrolled group for the study’s actual feasibility question.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary feasibility outcome and a secondary complete-response outcome. It reports 2 outcome measures and 0 analyses. No multiplicity procedure, such as an adjustment for testing several outcomes, is reported in the registry record. The record also does not report a prespecified statistical analysis plan, so it is not possible to determine whether an adjustment was planned or whether the analyses were intended to be descriptive.
Multiplicity is a general concern when several outcomes or comparisons are tested: the chance of at least one apparently notable finding can increase as the number of tests increases. That general issue does not establish that this study made multiple inferential tests. The registry does not post p-values or analyses for the primary or secondary outcome, and no results are given for comparisons among treatment options.
The registry lists enrollment of 16 and says 14 patients were treated in the primary outcome population. It does not report why these counts differ, the number with usable assay results, or the number included in any timing analysis. Missing outcome data, reasons for missingness, and methods for handling missing values are not reported in the registry record. The record likewise does not define analysis populations beyond the statement that 14 patients were treated for the primary outcome.
These omissions limit assessment of selection and missing-data bias. For example, if timing summaries were based only on participants who completed the assay or began treatment, they might not describe the feasibility of the full process for everyone entering it. That is a general consideration, not a claim about what occurred in this study. The registry does not report enough information to determine how participants without a complete timing observation were handled.
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 |
|---|---|---|---|
| Treatment (Chemotherapy, Biological Therapy) | 11 | 14 | 78.6% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Treatment (Chemotherapy, Biological Therapy) | 0 of 0 |
Most frequent serious adverse event terms
| Event term | Organ system | Treatment (Chemotherapy, Biological Therapy) |
|---|---|---|
| febrile neutropenia | Infections and infestations | 5 of 14 |
| seizure | Nervous system disorders | 2 of 14 |
| hypokalemia | Investigations | 2 of 14 |
| hyperbilirubinemia | Investigations | 2 of 14 |
| hypotension | Vascular disorders | 2 of 14 |
| tumor lysis syndrome | Metabolism and nutrition disorders | 2 of 14 |
| pneumonia | Infections and infestations | 1 of 14 |
| Hyperglycemia | Metabolism and nutrition disorders | 1 of 14 |
| headache | Nervous system disorders | 1 of 14 |
| malignant neoplasm progression | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 14 |
Serious events with the highest arm-level rate: febrile neutropenia (35.7%), seizure (14.3%), hypokalemia (14.3%), hyperbilirubinemia (14.3%), hypotension (14.3%).
The registry’s safety group lists 14 participants at risk and 11 affected by serious adverse events. It lists no participants at risk or affected for other adverse events in that group. The serious-event terms shown include febrile neutropenia at 35.7%, and seizure, hypokalemia, hyperbilirubinemia, hypotension, and tumor lysis syndrome at 14.3% each. These are the percentages as listed in the registry record.
These figures are descriptive. They do not provide a comparison with another treatment group, and they should not be interpreted as estimates of relative risk. A participant may have experienced more than one event term; the registry information does not establish whether the listed terms are mutually exclusive. The percentages therefore should not be added together to infer a total proportion.
The deaths entry is null, so deaths are not reported in the registry record; this is not evidence that no deaths occurred. The safety summary also does not establish how long participants were followed for adverse events or how events were attributed. Those details are not reported in the registry record. As with other outcomes, the posted information gives a limited descriptive account and does not support a comparative safety conclusion.
12. What the Result Does, and Does Not, Mean
The study addresses a specific operational question: whether drug sensitivity screening could be performed and used in planning treatment for patients with poor-risk, relapsed or refractory AML. The registry describes a process in which multiple drugs were tested against a patient’s donated blood sample and treatment was selected based on the assay. The primary outcome was intended to summarize timing, using a median and full range in days.
The primary numerical result is not reported in the registry record. As a consequence, readers cannot tell from the posted information how long screening took, how much timing varied, or whether treatment was initiated within the stated timeframe. The endpoint description also leaves unclear whether the timing measure ended at assay results or at therapy initiation. The outcome title includes both screening and treatment initiation, while the description specifically says time from sample procurement to assay results.
The trial’s single-group design means it does not provide a direct comparison with treatment selection without the assay or with another strategy. The listed complete-response outcome is secondary, but its result is not reported. The record therefore does not establish whether the assay-guided approach changed response or any other clinical outcome.
The safety summary reports serious adverse events among treated participants, but without a control group it cannot show whether the event frequency was higher or lower than under another approach. These data also do not establish causation for individual events.
In short, the registry record describes the intended workflow, the study design, and a limited safety summary. It does not post the primary timing estimate or the secondary response result. The conclusions that can be drawn are therefore about what the study set out to assess and what the record reports—not about the effectiveness of the assay-guided treatment strategy.
13. Why This Trial Matters Statistically
This study is statistically interesting because it links a laboratory process to a clinical decision pathway. The relevant question is not only whether an assay can produce drug sensitivity information, but also whether the information is available within the timeframe intended for treatment planning. A timing endpoint can capture an operational feature that a response endpoint alone would miss.
The design also illustrates why the question being asked should be distinguished from the design used. A single-group feasibility study can describe the experience of participants receiving an assay-guided process. It cannot, by itself, estimate how outcomes would differ under another treatment-selection strategy. That distinction matters when interpreting clinical outcomes or safety events: a descriptive observation in one group is not a comparative treatment effect.
The registry’s stated treatment pathway includes 1 of 160 possible interventions, emphasizing that the intervention was not one uniform drug regimen. Treatment selection varied according to the assay, so interpreting outcomes would require attention to what was actually given and how results were summarized. The registry does not post treatment-specific analyses or comparative estimates.
The record also shows the importance of complete outcome reporting. It identifies a median-time measure and a stated timeframe, but does not report the median or full range. Without those values, the central feasibility question cannot be answered from the posted results. The record’s different enrollment and treated counts also highlight why participant flow and analysis populations matter for interpreting a feasibility outcome.
Finally, this example separates a useful research aim from evidence of a measured result. The registry describes an approach designed to match treatment to disease characteristics, but the posted information does not establish whether the process met its timing objective or affected response. Clear endpoint definitions, participant accounting, and numerical outcome reporting are essential for readers to evaluate such a study.
14. Limitations
- The posted summary statistics are the only data these results rest on; patient-level data, full protocols and statistical analysis plans are not available.
- Recalculated results are simple, unadjusted calculations from posted summaries. They do not replace the sponsor's prespecified analysis.
The most direct limitation is that the registry does not report the primary endpoint value. Although the intended summary is a median time in days with a full range, neither statistic is posted. Readers therefore cannot assess the observed timing or its variability.
The endpoint description is also not fully aligned with its title. The title refers to performing individualized screening and initiating therapy, while the description says time from sample procurement to assay results. The registry record does not clarify which milestone defined the timing endpoint or how meeting the timeframe was determined.
The record lists enrollment of 16 and states that 14 patients were treated, but does not describe participant flow or explain the difference. It does not report how many participants had assay results, whether all treated participants had complete timing observations, or how missing data were handled. The analysis population is therefore not fully characterized.
The single-group design has no separate comparator. This limits interpretation to a description of the assay-guided treatment pathway and does not permit a like-for-like estimate against another strategy. The registry also lists 1 of 160 possible interventions, but does not report treatment-specific results or how outcomes varied across interventions.
Complete response is listed as a secondary outcome, but no result is posted. The registry does not report a statistical analysis plan, multiplicity handling, confidence intervals, or primary-outcome analyses. The safety record gives serious-event figures but does not report deaths, follow-up duration, or event attribution. These are limitations of the registry information and do not establish that such details were absent from other study materials.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay — NCT01872819.
- Registry record last updated 2018-07-11; status last verified 2018-06.
- Results first posted 2017-04-17.
- 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
NCT01872819 is a completed, single-group study sponsored by the University of Washington. It evaluated the use of a high-throughput drug sensitivity assay to guide treatment for poor-risk patients with relapsed or refractory AML. Enrollment was listed as 16; the primary outcome description says 14 patients were treated. Patients received 1 of 160 possible interventions based on the assay.
The primary outcome concerned the achievability of screening and initiating therapy within a timeframe of up to 21 days. The intended measure was a median time in days with a full range, but those numerical results are not reported in the registry record. Complete response was listed as a secondary outcome, with no result posted. The safety summary records serious adverse events for 11 of 14 participants at risk. The single-group design does not provide a control comparison.