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 Dose Optimization Trial of CD19 Redirected Autologous T Cells (NCT01747486), a randomized phase 2 study. The primary outcome is Number of Patients Achieving Complete Response Within 3 Months.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01747486 |
| Phase | Phase 2 |
| Condition | Adult Patients Who Have Relapsed or Refractory CLL (3rd Line) or SLL |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | None (open label) |
| Enrollment | 42 |
| Arms in the primary outcome | n/a |
| Lead sponsor | University of Pennsylvania |
| Results posted | Yes |
2. Clinical Question
Population
Adults with relapsed or refractory CLL, described as 3rd Line, or SLL. The primary outcome’s evaluable set included patients who received their randomized dose and either completed at least 3-month follow-up after infusion or discontinued because of disease progression, new cancer therapy or death.
Intervention
Autologous T cells engineered to express CD19 chimeric antigen receptors, identified as CART-19. The higher target dose was 1-5x10e8 cells.
Comparator
The lower target dose was 1-5x10e7 CART-19 cells. The study randomized participants between these two dose levels in parallel groups.
Primary question
How many evaluable patients in each randomized dose group achieved complete response, including complete response with incomplete marrow recovery, within 3 months? The registry record does not report the arm-specific primary outcome counts or a comparison estimate.
This is a dose-optimization question, not a comparison of CART-19 with no treatment or with a different therapy. The intended contrast is between two target dose levels of the same intervention. The outcome is a participant count, and the registry definition restricts that count to an evaluable set rather than simply describing everyone enrolled.
The record describes the study as randomized, open-label and parallel group. Randomization is intended to allocate participants to the dose groups by chance, while open-label means masking was not used. The record does not report how randomization was generated or whether allocation was concealed. It also does not report the primary outcome values needed to assess how the response counts differed between groups.
3. Trial Design
The registry describes a phase 2, randomized, open-label, parallel-group study sponsored by the University of Pennsylvania. Enrollment was 42. The trial assessed two target doses of autologous CART-19 cells: 1-5x10e8 and 1-5x10e7. The brief summary says the trial was conducted in two stages, but the details of those stages are not reported in the registry record.
The intervention is described as autologous T cells engineered to express CD19 chimeric antigen receptors with tandem TCR Zeta and 4-1 BB co-stimulatory domains. The higher-dose arm description relates the target dose to 10-50% transduced cells in 1 x10e9 total cells; the lower-dose description relates it to 10-50% transduced cells in 1 x10e8 total cells. The record does not describe further treatment procedures or explain how the dose was selected for an individual participant.
Parallel-group means participants were assigned to a dose group and the groups were followed as separate arms, rather than each participant receiving both doses in sequence. Randomization supports a planned between-group comparison, but the record does not report the randomization method, allocation concealment, stratification, or the number randomized to each arm for the primary outcome. The arms’ safety denominators are reported separately, but they should not be assumed to be the primary-outcome denominators.
The primary outcome timeframe is 3 months. The analysis population is described as evaluable patients who received the randomized intended dose and either completed at least 3-month follow-up after infusion or discontinued for disease progression, new cancer therapy or death. The record’s status is completed. It does not report a statistical analysis plan, interim analyses, a sample-size justification, or details of the two trial stages beyond the brief summary.
4. Participant Flow and Accounting
The registry reports how participants moved through each stage of the study. All counts below are exactly as posted; missing counts are not inferred from other stages.
Before assignment: Study was conducted in 2 stages. Stage 1: subjects were randomized into one of the two arms (Arm 1: Higher dose \& Arm 2 : Lower dose) Stage 2: the selected dose cohort was expanded to enroll additional subjects. Based on the Stage 1 analysis performed in Nov 2014, Arm 1 was chosen for expansion in Stage 2 and enrolled additional 12 subjects.
Stage 1
| Stage | Target Dose of 1-5x10e8 | Target Dose of 1-5x10e7 |
|---|---|---|
| STARTED | 15 | 15 |
| COMPLETED | 3 | 2 |
| NOT COMPLETED | 12 | 13 |
Reasons for not completing this period:
| Reason | Target Dose of 1-5x10e8 | Target Dose of 1-5x10e7 |
|---|---|---|
| Physician Decision | 3 | 0 |
| Withdrawal by Subject | 4 | 3 |
| Disease progression | 4 | 9 |
| Death | 1 | 1 |
Stage 2
| Stage | Target Dose of 1-5x10e8 | Target Dose of 1-5x10e7 |
|---|---|---|
| STARTED | 12 | 0 |
| COMPLETED | 3 | 0 |
| NOT COMPLETED | 9 | 0 |
Reasons for not completing this period:
| Reason | Target Dose of 1-5x10e8 | Target Dose of 1-5x10e7 |
|---|---|---|
| Physician Decision | 2 | 0 |
| Withdrawal by Subject | 1 | 0 |
| New Cancer Therapy | 3 | 0 |
| Disease Progression | 3 | 0 |
Analysis population for the primary outcome, as defined in the registry: The Evaluable Set comprised all patients who received high dose (1-5×10\^8) or low dose (1-5×10\^7) CTL019 transduced cells as randomized, and completed at least 3-month follow-up after the infusion or discontinued due to disease progression, new cancer therapy or death.
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: In high dose cohort, 15 patients were enrolled in Stage 1 and 12 were in stage 2.
| Characteristic | Unit | Target Dose of 1-5x10e8 | Target Dose of 1-5x10e7 | Total |
|---|---|---|---|---|
| Participants analysed | 27 | 15 | 42 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 19 | 12 | 31 |
| Age, Categorical: >=65 years | Participants | 8 | 3 | 11 |
| Sex: Female, Male: Female | Participants | 6 | 4 | 10 |
| Sex: Female, Male: Male | Participants | 21 | 11 | 32 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 0 | 1 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 26 | 15 | 41 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 26 | 15 | 41 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 27 | 15 | 42 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients Achieving Complete Response Within 3 Months, measured over 3 months.
| 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 patients achieving complete response within 3 months. The definition includes complete response with incomplete marrow recovery. The registry describes the measure as a count of participants, not as a time-to-response outcome or a continuous measurement. It specifies that the count is assessed in evaluable patients.
A response endpoint gives a clinically interpretable description of how many people met a stated response definition during a specified follow-up period. In a randomized dose comparison, response counts can be compared between the assigned dose groups to assess whether the observed response experience differs. The endpoint’s interpretation depends on the definition of response, the timeframe, and the population included in the analysis. Here, the record names the response category and timeframe, and describes the evaluable population, but does not report the outcome counts or a between-group estimate.
Because the outcome is a participant count, its analysis would ordinarily account for the number of evaluable participants in each group. A count alone does not establish how common the outcome was unless its denominator is also clear. Nor does a difference in counts by itself express the precision of a comparison: an estimate and an uncertainty measure, such as a confidence interval, would be needed to describe that precision. Those primary-outcome results are not reported in the registry record.
The evaluable-set rule excludes participants who do not meet the stated follow-up or discontinuation conditions. That makes the endpoint’s analysis population narrower than the full enrolled group unless everyone enrolled meets the rule. The record does not report how many participants were evaluable for this endpoint in each arm.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the planned question and the endpoint definition, but it does not report the primary outcome results. In particular, it gives no arm-specific number of evaluable participants achieving complete response within 3 months, no corresponding denominators, and no estimated contrast between dose groups. Without those results, the direction, magnitude and precision of the primary comparison cannot be assessed from the registry record.
Enrollment was 42, but that total does not by itself show how many participants contributed to the primary endpoint. The outcome is defined for an evaluable set, and the record’s eligibility rule for that set permits inclusion after either completing at least 3-month follow-up or discontinuing for disease progression, new cancer therapy or death. Arm-specific evaluable counts are not reported in the registry record. The safety denominators should not be substituted for those missing primary-outcome denominators.
In general, a comparison of response counts needs both the number meeting the response definition and the number assessed in each group. An effect estimate describes the contrast; a confidence interval describes the range of estimates compatible with the observed data under the method’s assumptions. A p-value, if reported, would address compatibility with a specified null hypothesis under an analysis model, not the clinical importance of an effect. These are general statistical concepts. No primary-outcome estimate, confidence interval or p-value is reported in the registry record.
The record identifies a randomized design, which is relevant because random assignment is intended to make groups comparable on average. It does not report details of the randomization process, nor an analysis adjusting for any baseline characteristics. No adjusted or unadjusted primary comparison is reported. Therefore, the design description alone cannot establish the observed effect or its precision.
The posted safety figures describe serious adverse events and deaths by dose group, but they do not fill the gap in the primary outcome. They address different outcomes and have their own denominators. The registry record does not provide a causal assessment of deaths or adverse events, and the reported counts should be read as descriptive figures rather than as proof that one dose caused a particular outcome.
The trial’s stated aim was to determine the optimal dose from the two assessed dose levels. The registry record does not report a dose-selection rule, a statistical decision criterion, or the primary results. As a result, the record supports describing the comparison that was planned and the outcome that was defined, but not identifying an optimal dose from the posted primary outcome information.
Any sample-size planning illustration is distinct from the trial’s observed results. The record describes an illustration based on a fraction of the observed effect, a two-sided significance level of 0.05, equal allocation, and a normal approximation. Such an illustration is not observed power and is not a substitute for the actual outcome counts or an estimate with uncertainty. The numerical planning results are not reported in the registry record.
9. Statistical Methods Explained
What does a randomized parallel-group comparison mean?
In general, randomization assigns participants to groups by chance. Its purpose is to reduce systematic differences in assignment and support a fair comparison of outcomes across groups. It does not guarantee that groups will be identical, and it does not remove the need to describe the groups, the analysis population and the uncertainty around an effect estimate. The registry identifies this study as randomized and parallel group, but the randomization procedure and allocation concealment are not reported in the registry record.
In a parallel-group design, the assigned groups are followed separately. For this trial, the intended contrast is between the 1-5x10e8 and 1-5x10e7 CART-19 target doses. Because the study was open-label, masking was not used. In general, knowledge of assignment can affect care, reporting or outcome assessment, depending on the outcome and how it is measured. The registry record does not describe steps taken to limit such influence or whether response assessment was independently reviewed.
How should a participant count be understood?
The primary outcome is a count of participants meeting a response definition within 3 months. For a count to be interpretable as a group outcome, the relevant population at risk of being counted must also be clear. The record defines an evaluable set, including patients who received the intended randomized dose and completed follow-up or discontinued for specified reasons. It does not report the number evaluable in each arm or the number achieving the endpoint in either arm.
In general, binary outcomes can be reported as counts and proportions, with a comparison expressed as a difference, ratio or another effect measure. Which measure is appropriate depends on the analysis plan and the scientific question. Each estimate has uncertainty, commonly conveyed with a confidence interval. A confidence interval is a range of values compatible with the observed data and the method’s assumptions; it is not a guarantee that the true effect lies in that range. These are general explanations, not descriptions of analyses reported for this trial. The registry record does not report a primary-outcome effect measure or confidence interval.
What do p-values and significance levels convey?
In general, a p-value describes how unusual data at least as incompatible with a specified null hypothesis would be, if that hypothesis and the model assumptions held. It does not measure the probability that the null hypothesis is true, the size of an effect, or the clinical importance of an effect. A significance level is a threshold chosen for a testing procedure; it is not itself an outcome result.
The record’s description of a planning illustration refers to a two-sided significance level of 0.05. That value pertains to the stated planning assumptions and should not be mistaken for a reported trial p-value. The registry record reports no primary-outcome p-value or inferential analysis. It also does not report whether a formal testing hierarchy was used.
Why do analysis populations matter?
Different analysis populations answer different questions. An analysis of all randomized participants, an analysis based on treatment received, and an analysis limited to evaluable participants can yield different estimates because they include different people and make different assumptions. The primary endpoint is explicitly defined for an evaluable set. Its stated rule requires receipt of the intended dose and either completion of at least 3-month follow-up or discontinuation for disease progression, new cancer therapy or death.
The registry record does not report a full accounting of participants from enrollment through endpoint assessment, including arm-specific evaluable totals or reasons for any other missing outcome information. It also does not describe imputation, sensitivity analyses, or an intention-to-treat analysis. These omissions limit assessment of how missing observations or exclusions might affect a primary comparison.
How should planning illustrations be read?
In general, sample-size planning asks how many participants might be needed under specified assumptions to detect an effect with a chosen statistical procedure. The registry’s description of its planning illustration specifies equal allocation, a normal approximation, a two-sided significance level of 0.05, and an effect defined as a stated fraction of the observed effect. The record also distinguishes participant counts per arm from total events for time-to-event settings; the primary endpoint in this trial is a participant count.
A planning illustration based on an observed effect is conditional on that effect and the stated assumptions. It is not an observed trial result and does not establish the probability that this completed trial detected an effect. “Observed power” is not a replacement for an effect estimate and its uncertainty. The record does not report the numerical sample-size illustrations or a prospective sample-size justification for the trial. The planning assumptions therefore should not be read as evidence that the study was designed around a particular target effect.
What does over-dispersion mean in general?
For count data, over-dispersion means that variability exceeds what a chosen statistical model expects. When present, it can make standard errors too small if the model does not account for the extra variation. This is a general concept, not a claim about the primary endpoint in this trial: the endpoint is a count of participants meeting a binary response definition, and the registry record does not report a model or any assessment of over-dispersion.
Overall, the design and endpoint description are enough to identify the intended dose comparison and the outcome timeframe. They are not enough to reconstruct an analysis. The statistical model, effect measure, adjustment strategy, testing plan, uncertainty estimates and handling of missing primary outcomes are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record does not report a multiplicity plan, such as a hierarchy for testing outcomes, adjustment for multiple comparisons, or a rule for choosing among analyses. The record describes one primary outcome measure, but it also indicates that the primary outcome has several categories or time points and was not reduced to one comparison. The specific categories or time points and their role in the analysis are not reported in the registry record. Without a stated testing plan and reported results, the risk of multiplicity affecting interpretation cannot be assessed.
The endpoint is defined in an evaluable population. Eligible participants had to receive the intended randomized dose and either complete at least 3-month follow-up after infusion or discontinue because of disease progression, new cancer therapy or death. This definition is important: it specifies who could contribute to the primary endpoint, but does not report the evaluable denominator in either arm or the number excluded from that population.
The registry record does not report how other missing outcome data were handled, whether participants with incomplete assessment were excluded, or whether any imputation or sensitivity analysis was used. It also does not report an intention-to-treat analysis. In general, missingness can affect interpretation when the likelihood of having an observed outcome is related to participant characteristics or outcome status. That is a general concern, not evidence that missingness affected this trial.
The record gives total enrollment and separate safety denominators, but neither identifies the primary-outcome analysis population. The primary results and analysis-population flow are not reported in the registry record, so the extent to which the endpoint represents all randomized participants cannot be determined.
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 |
|---|---|---|---|
| Target Dose of 1-5x10e8 | 23 | 27 | 85.2% |
| Target Dose of 1-5x10e7 | 9 | 15 | 60.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Target Dose of 1-5x10e8 | 6 of 27 |
| Target Dose of 1-5x10e7 | 6 of 15 |
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 |
|---|---|
| Target Dose of 1-5x10e8 | 24 of 27 |
| Target Dose of 1-5x10e7 | 11 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Target Dose of 1-5x10e8 | Target Dose of 1-5x10e7 |
|---|---|---|---|
| Cytokine Release Syndrome | Immune system disorders | 12 of 27 | 6 of 15 |
| Pyrexia | General disorders | 5 of 27 | 5 of 15 |
| Febrile Neutropenia | Blood and lymphatic system disorders | 4 of 27 | 4 of 15 |
| Pneumonia | Infections and infestations | 1 of 27 | 1 of 15 |
| Sinusitis | Infections and infestations | 1 of 27 | 1 of 15 |
| Upper respiratory tract infection | Infections and infestations | 1 of 27 | 1 of 15 |
| Acute kideny injury | Renal and urinary disorders | 1 of 27 | 1 of 15 |
| Influenza | Infections and infestations | 0 of 27 | 2 of 15 |
| Abdominal Pain | Gastrointestinal disorders | 1 of 27 | 0 of 15 |
| Hypercalcaemia | Metabolism and nutrition disorders | 1 of 27 | 0 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Target Dose of 1-5x10e8 | Target Dose of 1-5x10e7 |
|---|---|---|---|
| CD4 lymphocytes decreased | Investigations | 10 of 27 | 11 of 15 |
| Anaemia | Blood and lymphatic system disorders | 12 of 27 | 7 of 15 |
| Cytokine release syndrome | Immune system disorders | 12 of 27 | 6 of 15 |
| White blood cell count decreased | Investigations | 8 of 27 | 9 of 15 |
| Fatigue | General disorders | 10 of 27 | 6 of 15 |
| Pyrexia | General disorders | 4 of 27 | 8 of 15 |
| Lymphocyte count decreased | Investigations | 8 of 27 | 4 of 15 |
| Hypocalcemia | Metabolism and nutrition disorders | 8 of 27 | 4 of 15 |
| Neutrophil count decreased | Investigations | 6 of 27 | 6 of 15 |
| Nausea | Gastrointestinal disorders | 3 of 27 | 7 of 15 |
Serious events with the highest arm-level rate: cytokine release syndrome (44.4%), pyrexia (33.3%), febrile neutropenia (26.7%), influenza (13.3%), pneumonia (6.7%).
The registry reports serious adverse event figures separately for the target dose of 1-5x10e8 and the target dose of 1-5x10e7. In the higher-dose group, 23 of 27 participants at risk were affected by a serious adverse event; in the lower-dose group, 9 of 15 were affected. These are participant counts, not a comparison adjusted for follow-up or other factors. The registry also reports 24 of 27 and 11 of 15 participants at risk with other adverse events, respectively. Those figures are distinct from the serious adverse event counts.
The listed serious adverse event terms include Cytokine Release Syndrome at 44.4%, Pyrexia at 33.3%, Febrile Neutropenia at 26.7%, Influenza at 13.3%, Pneumonia at 6.7%, and Sinusitis at 6.7%. The registry record does not explain the denominator for each listed percentage or whether participants could be counted under more than one term. The terms therefore should not be added together or treated as mutually exclusive categories.
Six deaths are reported in each dose group. The registry figures do not establish the cause of death or whether a death was attributed to the intervention. Serious adverse event counts are descriptive safety information; without fuller definitions, follow-up details and an analysis, they do not by themselves establish a causal difference between doses.
12. What the Result Does, and Does Not, Mean
The registry record describes a randomized comparison of two CART-19 target doses and defines complete response within 3 months as the primary outcome. It also identifies the evaluable population used for that endpoint. These details clarify what the study set out to compare and which participants were intended to contribute to the primary outcome.
What the record does not provide is the primary response count for either group, the evaluable denominators, or an estimate of the difference between groups. It therefore does not show whether the response counts were higher in one group, how large any difference was, or how precisely a difference was estimated. No conclusion about which dose had the better primary outcome can be drawn from the information reported in the registry record.
Even when a numerical difference is available, interpretation depends on uncertainty, outcome definitions, analysis population and the conduct of the trial. A confidence interval can show how much uncertainty surrounds an estimate, while the choice of analysis population can determine which participants are represented. These are general considerations. The registry record does not report the primary estimate, confidence interval, statistical test or analysis details needed for that assessment.
The safety counts provide a separate description of serious and other adverse events and deaths by dose group. They do not replace the primary outcome, and the record does not provide enough detail to attribute those events to the intervention or to interpret the differences causally. The record also does not report a formal dose-selection criterion or the results needed to apply one.
Accordingly, the posted information supports a description of the trial design, dose groups, endpoint definition and reported safety counts. It does not support a claim that one dose is optimal, that either dose has a particular response advantage, or that a treatment does or does not work.
13. Why This Trial Matters Statistically
Dose comparisons raise a statistical question that differs from asking whether an intervention has an effect relative to no intervention. The groups in this study were assigned different target doses of the same CART-19 intervention. The relevant primary question is therefore about the comparative outcome experience under those dose assignments, with the result interpreted in light of the analysis population and uncertainty.
The study’s randomized, parallel-group design is relevant to that comparison because allocation by chance is intended to reduce systematic differences between groups. Yet the design label alone is not a result. To evaluate a dose contrast, readers need arm-specific outcome counts, denominators and an estimate of the contrast, together with a measure of its precision. The primary results needed for that evaluation are not reported in the registry record.
The evaluable-set definition also matters statistically. The primary outcome is not simply described for everyone enrolled; it is defined for participants who received their intended randomized dose and met a follow-up or discontinuation condition. Knowing how many participants entered that set in each group would help readers understand the basis of the comparison. Those counts are not reported for the primary endpoint.
The record’s safety data illustrate why outcomes must be kept distinct. Serious adverse event counts, other adverse event counts, deaths and complete response measure different things. A count in one outcome category cannot be used to infer another, and descriptive safety figures do not establish a primary efficacy comparison.
Finally, the stated planning illustration assumptions are not evidence of achieved precision or observed power. Statistical interpretation depends on the actual endpoint results and the method used to analyze them. Since the record does not report the primary estimates or analysis, its statistical importance lies in defining a randomized dose question whose result cannot be evaluated from the posted primary outcome information alone.
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 main limitation for interpreting the primary endpoint is that the registry record does not report its arm-specific results. It gives neither the number of evaluable participants who achieved complete response within 3 months nor the corresponding evaluable denominators. No effect estimate, confidence interval or p-value is reported. Consequently, the magnitude and precision of the dose comparison cannot be assessed.
The outcome is restricted to an evaluable set, but the record does not report how many participants in each arm met that definition or how many did not. Although the eligibility rule identifies completion of follow-up or specified reasons for discontinuation, the record does not explain the handling of other missing assessments, provide a participant flow, or report sensitivity analyses. The extent to which the evaluable population differs from all randomized participants is therefore unclear.
The record describes a randomized, open-label design but does not report the randomization procedure, allocation concealment or details of outcome assessment. It also does not report an analysis plan, adjustment strategy, multiplicity approach, interim analyses, or sample-size justification. The mention of a two-stage trial does not include enough detail to characterize the stages or their influence on analysis.
The safety information is also limited for comparative interpretation. The record provides participant counts and selected serious adverse event terms, but does not explain the denominators for the listed percentages or provide event-level timing, duration, severity grading or causal attribution. Death counts are reported without a cause or attribution. These limitations do not change the reported figures, but they constrain what can be concluded from them.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Dose Optimization Trial of CD19 Redirected Autologous T Cells — NCT01747486.
- Registry record last updated 2023-06-22; status last verified 2019-08.
- Results first posted 2019-02-26.
- 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
NCT01747486 is a completed phase 2 randomized, open-label, parallel-group study sponsored by the University of Pennsylvania. Enrollment was 42. It compared target doses of 1-5x10e8 and 1-5x10e7 autologous CART-19 cells in adults with relapsed or refractory CLL or SLL.
The primary outcome was the number of evaluable patients achieving complete response, including complete response with incomplete marrow recovery, within 3 months. The evaluable-set definition required receipt of the intended randomized dose and completion of at least 3-month follow-up or discontinuation for disease progression, new cancer therapy or death. Arm-specific primary outcome results and analysis estimates are not reported in the registry record.
The registry reports serious and other adverse event counts and deaths by dose group, as well as selected serious adverse event terms. It does not report the statistical analysis plan, multiplicity approach, handling of other missing primary outcome data, or the details of the trial’s two stages.