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 Allogeneic Stem Cell Transplant for CLL (NCT01629511), a registered phase 1, phase 2 study. The primary outcome is 100 Day Treatment Related Mortality (TRM).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01629511 |
| Phase | Phase 1, Phase 2 |
| Condition | Allogeneic Hematopoietic Stem Cell Transplantation Recipient, Chronic Lymphocytic Leukemia, Prolymphocytic Leukemia |
| Allocation | Not Stated |
| Design model | Sequential |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | M.D. Anderson Cancer Center |
| Results posted | Yes |
2. Clinical Question
Population
The registry record concerns people with chronic lymphocytic leukemia undergoing allogeneic hematopoietic stem cell transplantation. It also lists prolymphocytic leukemia, Richter syndrome, and allogeneic hematopoietic stem cell transplantation recipient among the conditions. Enrollment is 15. The available record does not describe participant eligibility criteria or baseline characteristics, so it cannot establish which clinical or disease features characterized the enrolled group.
Intervention
The treatment combines gemcitabine, clofarabine, and busulfan before donor stem cell transplantation. The study description says it examines gemcitabine dose and side effects as well as how the combination works with transplantation. The treatment listing also includes anti-thymocyte globulin, filgrastim, methotrexate, and tacrolimus. The arm description gives some treatment timing, but its text ends after “Starting day -2,” so the remainder of that regimen is not reported in the registry record.
Comparator
No separate control treatment or comparator group is described. The record identifies a sequential model and lists gemcitabine dose levels, but does not state the allocation method. The record also notes that no participants were treated at dose level 2. These dose-level groups should not be read as a randomized, like-for-like comparison. A treatment effect relative to another therapy or to no treatment cannot be estimated from the posted information.
Primary question
The primary endpoint is the number of deaths related to treatment by day 100 after allogeneic transplant. This is a count endpoint focused on an important early safety outcome. The registry record does not report the endpoint count, a rate, a confidence interval, or a statistical test. It therefore identifies the question the trial intended to address, but the posted results do not quantify the day-100 outcome or its precision.
3. Trial Design
This is a phase I/II study with a sequential model and no masking. The registry lists enrollment of 15 and identifies M.D. Anderson Cancer Center as sponsor. The study started on 2012-11-21, reached primary completion on 2018-04-25, and has a terminated status. The registry record does not state the allocation method. It does not describe a randomized control arm, and the listed dose levels are not a like-for-like control comparison.
The study description frames the work around gemcitabine dose and side effects, alongside treatment with clofarabine and busulfan followed by donor stem cell transplantation. The arm description includes treatment on specified days before transplantation and additional anti-thymocyte globulin for participants with matched unrelated donors. Its dosing description is incomplete in the registry record, so the full sequence and subsequent supportive-treatment details are not reported there. The intervention list also includes filgrastim, methotrexate, and tacrolimus.
A sequential study may address dose selection differently from a trial that compares treatment groups under random allocation. In general, sequential dose studies examine treatment at successive levels, with safety and tolerability informing which level is considered next. That general description should not be taken as a statement about the specific dose rules used in this trial: its detailed escalation or stopping procedures are not reported in the registry record. Nor does the record report a sample-size justification or an interim-analysis plan.
The primary endpoint is treatment-related mortality by day 100 after transplant. Overall survival is listed as a secondary outcome. The record lists six outcome measures but no analyses. Although adverse-event data are posted by gemcitabine dose level, they do not create a randomized comparison. The trial status is terminated; the reason for termination is not reported in the registry record.
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 | Gemcitabine Dose Level 1 | Gemcitabine Dose Level 2 | Gemcitabine Dose Level 3 | Gemcitabine Dose Level 4 |
|---|---|---|---|---|
| STARTED | 4 | 0 | 8 | 3 |
| COMPLETED | 4 | 0 | 8 | 3 |
| NOT COMPLETED | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: No participants were treated at dose level 2.
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: Patients age 18-70 with CLL, PLL, or Richter's Transformation who were eligible for allogeneic transplantation
| Characteristic | Unit | Gemcitabine Dose Level 1 | Gemcitabine Dose Level 2 | Gemcitabine Dose Level 3 | Gemcitabine Dose Level 4 | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 4 | 0 | 8 | 3 | 15 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 3 | 0 | 7 | 3 | 13 |
| Age, Categorical: >=65 years | Participants | 1 | 0 | 1 | 0 | 2 |
| Age, Continuous | years | 56 (39 to 67) | — | 57.5 (36 to 67) | 60 (59 to 61) | 59 (39 to 67) |
| Sex: Female, Male: Female | Participants | 3 | 0 | 2 | 0 | 5 |
| Sex: Female, Male: Male | Participants | 1 | 0 | 6 | 3 | 10 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 1 | 0 | 2 | 2 | 5 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 3 | 0 | 6 | 1 | 10 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 4 | 0 | 8 | 3 | 15 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | Participants | 4 | 0 | 8 | 3 | 15 |
| Disease Category: Diseases : Chronic Lymphocytic Leukemia (CLL) | Participants | 3 | 0 | 5 | 1 | 9 |
| Disease Category: Diseases : Prolymphocytic Leukemia (PLL) | Participants | 1 | 0 | 2 | 2 | 5 |
| Disease Category: Diseases: Richter's | Participants | 0 | 0 | 1 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as 100 Day Treatment Related Mortality (TRM), measured over 100 days post transplant.
| Element | Registry entry |
|---|---|
| Result type | Count Of Participants |
| Measure of spread | not applicable |
| Unit | Participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint counts deaths judged related to treatment by day 100 after allogeneic transplant. It is a count of participants, not a continuous measurement. The timeframe makes the endpoint specific to an early period after transplantation, and the treatment-related attribution distinguishes it conceptually from a count of deaths from any cause. The registry record does not describe how treatment relationship was adjudicated, what events qualified, or how incomplete follow-up was handled.
Early treatment-related mortality is used in clinical research as a safety outcome because it focuses attention on deaths associated with the treatment course during a defined period. In general, a fixed-time endpoint gives a clear time window for describing events. It does not, by itself, convey the timing of each event within that window, the reasons for deaths, or outcomes beyond the specified period. Overall survival is separately listed as a secondary outcome, but no outcome value or analysis for it is reported in the registry record.
The posted primary outcome information does not include the number of treatment-related deaths by day 100, a denominator for an analyzed population, or an uncertainty interval. The note that no participants were treated at dose level 2 describes exposure at that dose level; it is not the endpoint result. Without the endpoint count and analysis population, the primary question cannot be quantified from the registry record. The intended measure is clear, but its observed magnitude and precision are not reported.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study question and lists several safety counts, but it does not report a numerical result for the primary endpoint. The endpoint is defined as treatment-related deaths by day 100 post transplant. No count for that outcome, estimate of risk, confidence interval, or statistical test is reported in the registry record. As a result, the posted information does not allow a quantitative interpretation of day-100 treatment-related mortality.
The adverse-event listings are organized by gemcitabine dose level. At dose level 1, the record lists 4 participants at risk for serious adverse events and 0 affected; it lists 4 at risk for other adverse events and 4 affected, with 1 death. At dose level 2, the listed at-risk and affected counts are 0 for both serious and other adverse events, with 0 deaths. At dose level 3, the record lists 8 at risk and 0 affected for serious adverse events, 8 at risk and 8 affected for other adverse events, and 1 death. At dose level 4, it lists 3 at risk and 0 affected for serious adverse events, 3 at risk and 3 affected for other adverse events, and 1 death.
These figures are descriptive counts. They should not be treated as estimates from a randomized comparison: the registry record identifies a sequential model, does not state the allocation method, and explicitly notes that the arms are not a like-for-like control comparison. The dose-level groups may also differ in who was treated and when. The record does not provide enough detail to assess comparability or adjust for differences.
The listed deaths in the adverse-event groups are not identified as treatment-related deaths by day 100. They must not be substituted for the primary endpoint count. The record does not explain their timing, cause, or relationship to treatment. Likewise, zero serious adverse events in the listed groups is a descriptive entry, not proof that serious events could not occur; the number of participants at risk and the way events were collected matter to its interpretation.
Overall survival is listed among the secondary outcomes, but its results are not reported in the registry record. The record lists six outcome measures and zero analyses. No effect estimate or inferential result should be inferred from the study title, treatment rationale, or adverse-event counts. The available record supports a description of what was planned and which safety counts were posted, not a conclusion about comparative efficacy or treatment effect.
9. Statistical Methods Explained
The registry record identifies a count endpoint, lists adverse-event counts, and describes a sequential phase I/II study. It reports no analyses. The concepts below explain how such information is generally interpreted; they do not describe an unreported analysis or protocol procedure.
What a count endpoint says
The primary endpoint is the number of participants who died in relation to treatment by day 100 after transplant. A count is an absolute number of events. To interpret it as a proportion or risk, a reader also needs the relevant analysis population: who was included in the denominator, whether all enrolled participants were followed for the full period, and how incomplete observation was addressed. Those details and the endpoint count are not reported in the registry record. The note that no participants were treated at dose level 2 concerns that dose group's exposure, not a calculated primary endpoint.
For a count endpoint, it is important to distinguish an event count from a rate. A rate needs a defined amount of observation time, whereas a fixed-period risk is based on the number of people who experienced the event within the specified period and the number at risk. Neither a risk estimate nor a rate can be recovered from a bare count without the relevant denominator and follow-up information. No such primary-endpoint estimate is reported in the registry record.
What uncertainty measures would add
General statistical explanation: a confidence interval describes the range of values reasonably compatible with the observed data under the statistical model and repeated-sampling procedure used. It helps readers assess precision; a point estimate alone does not show how much uncertainty surrounds it. A p-value addresses a different question, relating observed data to a specified null hypothesis under model assumptions. Neither replaces an effect estimate or provides a measure of clinical importance. The registry record reports no confidence interval, p-value, or primary endpoint estimate, so these aspects of uncertainty cannot be evaluated for this outcome.
General statistical explanation: sparse event data can yield substantial uncertainty, even when an observed count is small. A count of zero in a group does not establish that the underlying event probability is zero. Interpretation depends on the group size, observation period, event definition, and follow-up completeness. The serious adverse-event listings contain zero affected participants in the groups with participants at risk, but those descriptive counts are not accompanied by uncertainty intervals or inferential analyses in the registry record.
How the dose-level listings should be read
The study has a sequential model, and adverse-event counts are listed separately for gemcitabine dose levels. This organization describes safety observations within those groups. It does not establish that groups were assigned comparably or that a difference between groups would be caused by dose. The allocation method is not stated, and the registry record warns that the arms are not a like-for-like control comparison. In general, nonrandomized groups may differ in participant characteristics, treatment timing, eligibility, or other factors; the record does not provide details that would establish whether any of these differences occurred.
General statistical explanation: over-dispersion means that variability in count data exceeds what a simple model assumes. If present, it can make model-based uncertainty estimates too narrow unless the analysis accounts for it. The registry record does not report a count model, a dispersion assessment, or any inferential analysis. It is therefore not possible to say whether over-dispersion was assessed or whether a particular model would have been appropriate.
Planning illustrations and trial design
The posted planning assumptions describe 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 description says the effect to detect is a stated fraction of the observed effect. For time-to-event illustrations, the stated quantity is a total number of events; for other illustrations, it is participants per arm. These are planning assumptions, not evidence that the trial itself used those choices. The trial record describes a sequential study and does not state equal allocation or a randomized two-arm design. No trial sample-size justification is reported in the registry record.
Planning sample sizes are hypothetical design calculations, not observed evidence and not “observed power.” They depend on assumptions about the effect, allocation, outcome, significance level, and statistical approximation. If those assumptions differ from the study design or the available data, the illustration may not describe the trial's actual operating characteristics. No specific planning result should be interpreted as an observed trial outcome.
Multiplicity and analysis populations
The record lists six outcome measures, identifies overall survival as a secondary outcome, and reports zero analyses. Multiple outcomes can create multiplicity concerns when several hypotheses are tested: the chance of at least one apparently unusual result may increase if no adjustment or prespecified testing strategy is used. A multiplicity plan, hierarchy among outcomes, and primary analysis strategy are not reported in the registry record. It is not possible to determine whether any adjustment was planned or conducted.
Analysis populations also matter. In general, an analysis should state which enrolled, treated, or followed participants contribute to each result. The registry record does not describe the population used for the primary endpoint or the adverse-event counts, nor does it report methods for missing outcomes or incomplete follow-up. These omissions limit assessment of both the numerical results and the methods that would be needed to interpret them.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists six outcome measures and identifies overall survival as a secondary outcome. It does not report an analysis for any outcome. A plan for handling multiplicity is not reported in the registry record. General statistical explanation: when several outcomes or hypotheses are examined, repeated testing can increase the chance of at least one result appearing statistically notable by chance. Prespecified outcome hierarchies or adjustment procedures can help define how such findings should be interpreted. No such hierarchy or procedure is described in the registry record, so it is not possible to determine how multiplicity was addressed.
The analysis population for the primary endpoint is not reported in the registry record. The record states that no participants were treated at dose level 2, but does not specify which population was used for other outcome summaries or whether participants were analyzed according to treatment received. A sample-size justification, interim-analysis plan, and statistical analysis plan are also not reported. Their absence from the registry record does not establish whether they existed elsewhere; it means they cannot be evaluated from the posted record.
Missing-data methods and completeness of follow-up are not reported in the registry record. For a fixed-time endpoint, readers need to know whether participants had outcome ascertainment through the stated period and how deaths with uncertain treatment attribution or unavailable follow-up were handled. No such details are reported for the day-100 endpoint. The registry record also does not describe how missing adverse-event information was handled. Consequently, the posted counts cannot establish whether all eligible observations were captured or whether missingness could affect interpretation.
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 |
|---|---|---|---|
| Gemcitabine Dose Level 1 | 0 | 4 | 0.0% |
| Gemcitabine Dose Level 3 | 0 | 8 | 0.0% |
| Gemcitabine Dose Level 4 | 0 | 3 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Gemcitabine Dose Level 1 | 1 of 4 |
| Gemcitabine Dose Level 2 | 0 of 0 |
| Gemcitabine Dose Level 3 | 1 of 8 |
| Gemcitabine Dose Level 4 | 1 of 3 |
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 |
|---|---|
| Gemcitabine Dose Level 1 | 4 of 4 |
| Gemcitabine Dose Level 2 | 0 of 0 |
| Gemcitabine Dose Level 3 | 8 of 8 |
| Gemcitabine Dose Level 4 | 3 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Gemcitabine Dose Level 1 | Gemcitabine Dose Level 2 | Gemcitabine Dose Level 3 | Gemcitabine Dose Level 4 |
|---|---|---|---|---|---|
| Infection | Infections and infestations | 4 of 4 | 0 of 0 | 8 of 8 | 3 of 3 |
| Nausea | Gastrointestinal disorders | 3 of 4 | 0 of 0 | 8 of 8 | 3 of 3 |
| Fluid Overload | General disorders | 4 of 4 | 0 of 0 | 5 of 8 | 3 of 3 |
| Mucositis | Gastrointestinal disorders | 4 of 4 | 0 of 0 | 6 of 8 | 2 of 3 |
| Transaminitis | Hepatobiliary disorders | 3 of 4 | 0 of 0 | 6 of 8 | 3 of 3 |
| Neutropenic Fever | General disorders | 3 of 4 | 0 of 0 | 7 of 8 | 2 of 3 |
| Elevated Bilirubin | Hepatobiliary disorders | 3 of 4 | 0 of 0 | 5 of 8 | 1 of 3 |
| Diarrhea | Gastrointestinal disorders | 2 of 4 | 0 of 0 | 4 of 8 | 2 of 3 |
| Skin Rash | Skin and subcutaneous tissue disorders | 3 of 4 | 0 of 0 | 4 of 8 | 1 of 3 |
| Acute Graft versus Host Disease | General disorders | 3 of 4 | 0 of 0 | 3 of 8 | 2 of 3 |
The safety listings separate serious adverse events, other adverse events, and deaths by gemcitabine dose level. For dose level 1, serious adverse events are listed for 4 participants at risk and 0 affected; other adverse events are listed for 4 at risk and 4 affected, with 1 death. Dose level 2 has 0 at risk and 0 affected for both event groupings, with 0 deaths. For dose level 3, the listings are 8 at risk and 0 affected for serious adverse events, 8 at risk and 8 affected for other adverse events, and 1 death. For dose level 4, they are 3 at risk and 0 affected for serious adverse events, 3 at risk and 3 affected for other adverse events, and 1 death.
These are counts, not comparative effect estimates. The record does not define the “other” event category, describe event severity within that category, or provide timing and follow-up details. It also does not say whether the listed deaths were treatment-related or occurred by day 100. They should not be interpreted as the primary treatment-related mortality outcome. Dose level 2 has no participants at risk, so its zero event counts do not represent evidence from treated participants. The sequential model and the absence of a like-for-like control comparison further limit comparisons across levels.
12. What the Result Does, and Does Not, Mean
The record describes a clinical study intended to examine gemcitabine dose and side effects in a regimen involving clofarabine, busulfan, and allogeneic stem cell transplantation for chronic lymphocytic leukemia. It specifies a primary safety question: treatment-related deaths by day 100 after transplant. It also posts adverse-event counts by dose level and lists overall survival as a secondary outcome. These details clarify the study's focus, but the primary endpoint result and secondary outcome results are not reported in the registry record.
The adverse-event listings show the counts recorded for each dose-level group. In groups with participants at risk, the registry lists zero affected by serious adverse events and nonzero counts for other adverse events. The listings also include deaths, but do not identify their timing or treatment relationship. The data therefore cannot be used to infer that the deaths met the primary endpoint definition, nor can absence of listed serious events be taken as proof of no risk.
The posted information does not support a comparative estimate of benefit or harm. The record does not describe a randomized control arm, states that allocation is not stated, and cautions that the arms are not a like-for-like control comparison. Group counts alone cannot separate dose effects from differences in participants or other aspects of treatment. No inferential analyses, confidence intervals, or p-values are reported.
General statistical explanation: a study's planned question and its observed result are distinct. A registry entry can document an endpoint without reporting the numerical outcome needed to answer it. Likewise, enrollment and adverse-event counts do not establish precision, causality, or the outcomes of people who were not represented in a particular group. The status of terminated records can signal that study conduct ended, but the reason and circumstances are not reported. Overall, the record supports a limited descriptive account of the design and posted safety counts, not a conclusion that the regimen is effective, ineffective, safe, or unsafe.
13. Why This Trial Matters Statistically
This trial is statistically notable because it links a dose-focused phase I/II question with a transplantation regimen and a fixed-time treatment-related mortality endpoint. These are distinct analytical tasks. Dose selection is concerned with safety and tolerability across levels, while a day-100 mortality count describes a defined clinical outcome after transplant. The registry record provides some dose-level safety counts but does not report the primary endpoint result or analyses that would connect the observations to a formal estimate.
The sequential model also highlights why study design matters when reading group-level counts. Dose-level groups in a sequential study are not necessarily exchangeable. Without randomized allocation or detailed methods for accounting for differences, a contrast between groups should not be treated as a causal dose comparison. The record specifically cautions that the arms are not a like-for-like control comparison. That caveat is central to interpreting any apparent pattern across dose levels.
The record also illustrates the difference between an endpoint definition and a complete statistical result. Naming treatment-related mortality by day 100 identifies what investigators intended to measure, but interpretation also requires the observed count, the analysis population, follow-up completeness, event attribution procedures, and an uncertainty assessment. These details are not reported in the registry record. The adverse-event counts offer descriptive information, but are not substitutes for the missing primary endpoint analysis.
Finally, six outcome measures are listed while zero analyses are reported. That makes reporting of outcome definitions and analysis methods especially relevant: without results and a multiplicity strategy, readers cannot assess how outcomes were evaluated or whether findings were selected. The statistical importance of the record lies as much in these interpretive limits as in the regimen and endpoint it describes.
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 registry record is incomplete for evaluating the primary endpoint. It names treatment-related deaths by day 100 after transplant but does not report the outcome count, an analyzed denominator, an effect estimate, or an uncertainty interval. The outcome population field notes that no participants were treated at dose level 2, but does not provide a day-100 result for the other levels. The method used to determine whether a death was treatment-related is not reported.
Enrollment is 15, and the posted adverse-event counts are divided among gemcitabine dose levels, with no participants listed at dose level 2. The record describes the study as sequential and does not state allocation. It also explicitly cautions that the arms are not a like-for-like control comparison. The small and unequal-looking group listings are descriptive; they cannot support a controlled dose-effect interpretation. Baseline features and dose-level assignment details are not reported in the registry record.
The safety summaries lack event definitions and follow-up detail. The distinction between serious and other adverse events is reflected in the categories, but the record does not give the criteria for those categories, the observation period for adverse events, or the timing and attribution of deaths. The deaths in these listings cannot be assumed to match the primary endpoint. The handling of missing safety information and incomplete follow-up is not reported.
The record lists six outcome measures but zero analyses. It does not report a statistical analysis plan, multiplicity handling, analysis populations, sample-size justification, interim-analysis procedures, or the reason for termination. These gaps prevent assessment of whether the posted counts represent the complete intended analysis and how uncertainty was handled. The available registry information therefore supports only a cautious descriptive reading, not strong conclusions about comparative outcomes.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Allogeneic Stem Cell Transplant for CLL — NCT01629511.
- Registry record last updated 2020-02-24; status last verified 2020-02.
- Results first posted 2020-02-10.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
NCT01629511 is titled “Allogeneic Stem Cell Transplant for CLL.” Its official title describes clofarabine, gemcitabine, and busulfan followed by allogeneic stem cell transplantation for chronic lymphocytic leukemia. The study is listed as phase I and phase II, with a sequential model and no masking. M.D. Anderson Cancer Center is the sponsor, and enrollment is 15.
The study started on 2012-11-21, had primary completion on 2018-04-25, and is listed as terminated. The primary outcome is treatment-related mortality by day 100 post transplant, measured as a participant count. Overall survival is listed as a secondary outcome. The record lists six outcome measures and zero analyses.
Adverse-event counts are listed for gemcitabine dose levels 1 through 4. The record states that no participants were treated at dose level 2 and cautions that the arms are not a like-for-like control comparison. The primary endpoint result, analysis methods, and reason for termination are not reported in the registry record.