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 Oral LBH589 in Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL) (NCT01090973), a na phase 2 study. The primary outcome is Number of Participants With Desired Response.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01090973 |
| Phase | Phase 2 |
| Condition | Non-Hodgkin's Lymphoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 1 |
| Arms in the primary outcome | n/a |
| Lead sponsor | H. Lee Moffitt Cancer Center and Research Institute |
| Results posted | Yes |
2. Clinical Question
Population
The intended population was people with relapsed or refractory chronic lymphocytic leukemia (CLL) or mantle cell lymphoma (MCL). The primary outcome description specifically refers to patients with relapsed or refractory aggressive MCL and CLL. The registry record does not provide further eligibility details, such as disease-stage criteria or requirements for prior treatment.
Intervention
The study evaluated oral LBH589, an investigational histone deacetylase inhibitor. The arm description specifies 40 mg once a day, 3 times weekly, on days 1, 3 & 5, then 8, 10 &12, then 15, 17 & 19, then 22, 24 & 26. The registry record describes a single treatment group rather than a comparison of different doses or regimens.
Comparator
There was no concurrent control group. Allocation is recorded as NA, masking as NONE, and the model as SINGLE GROUP. Consequently, the study was not designed to compare LBH589 with another treatment, placebo, or usual care. Changes in disease status cannot be attributed to treatment by a randomized between-group comparison.
Primary question
Investigators intended to assess the rate of overall and complete response according to World Health Organization (WHO) classification. The primary outcome is named Number of Participants With Desired Response, with an assessment timeframe of 8 weeks (2 cycles) unless treatment continues due to partial or complete response. The study was abandoned after only one patient because of low accrual and the sponsor losing interest in the single-agent. The patient had disease progression requiring more aggressive treatment and did not complete the study. The registry record does not report an analyzed response rate.
3. Trial Design
This was a Phase II, single-group study of oral LBH589 in people with relapsed or refractory CLL or MCL. The registry record identifies H. Lee Moffitt Cancer Center and Research Institute as the sponsor. It records the study start as 2010-03, primary completion as 2011-03, and status as TERMINATED. Enrollment was 1.
The treatment arm was described as oral LBH589 at 40 mg once a day, 3 times weekly. Its schedule listed treatment on days 1, 3 & 5, then 8, 10 &12, then 15, 17 & 19, then 22, 24 & 26. The outcome timeframe was 8 weeks (2 cycles), with continued treatment contemplated when a partial or complete response occurred. The record does not describe a second arm, dose comparison, randomization, or masking. Allocation is NA, masking is NONE, and the model is SINGLE GROUP.
The planned primary assessment was the rate of overall and complete response by WHO classification among patients with relapsed or refractory aggressive MCL and CLL. Listed secondary outcomes included response, response duration, progression-free survival, prolonged corrected QT interval, improved blood and lymphatic evaluation results, and adverse events. The registry record lists 7 outcome measures but no analyses. The study was abandoned after only one patient because of low accrual and the sponsor losing interest in the single-agent; the patient experienced disease progression requiring more aggressive treatment and did not complete the study. The registry record does not report a completed efficacy analysis or further detail about the conduct of the study.
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 | Oral Drug Treatment |
|---|---|
| STARTED | 1 |
| COMPLETED | 0 |
| NOT COMPLETED | 1 |
Reasons for not completing this period:
| Reason | Oral Drug Treatment |
|---|---|
| Disease progression | 1 |
Analysis population for the primary outcome, as defined in the registry: The study was abandoned after only one patient due to low accrual and the sponsor losing interest in the single-agent. The one patient had disease progression requiring more aggressive treatment and did not complete the study.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Oral Drug Treatment |
|---|---|---|
| Participants analysed | 1 | |
| Age, Categorical: <=18 years | Participants | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 |
| Age, Categorical: >=65 years | Participants | 1 |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 1 |
| Region of Enrollment: United States | participants | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Desired Response, measured over 8 weeks (2 cycles) unless treatment continues due to partial or complete response.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is named Number of Participants With Desired Response. Investigators intended to assess the rate of overall and complete response according to WHO classification in patients with relapsed or refractory aggressive MCL and CLL. The stated assessment timeframe was 8 weeks (2 cycles), unless treatment continued due to partial or complete response. The registry record does not report a response count, response rate, or analysis for this endpoint.
Response outcomes are used in clinical trials to describe whether a participant’s disease meets specified criteria for improvement. Overall response generally combines response categories defined by the study; complete response is a distinct category. The registry record identifies WHO classification as the framework, but does not provide additional response criteria or details on how an overall response would be calculated. The listed endpoint therefore describes the intended measurement, not an observed result.
The outcome description also includes the WHO Performance Scale, which classifies levels of patient capability from normal activity to being unable to get out of bed. The record does not state how that scale was incorporated into the response endpoint. The primary question is difficult to answer from the posted results because the study stopped after enrollment of 1 and the participant did not complete the study. No efficacy estimate or uncertainty interval is reported.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a study intended to assess response to oral LBH589 in relapsed or refractory CLL and MCL, but its posted results do not establish an efficacy estimate. Enrollment was 1, and the study was abandoned after only one patient because of low accrual and the sponsor losing interest in the single-agent. The patient had disease progression requiring more aggressive treatment and did not complete the study. These details explain why the planned response assessment cannot be treated as a completed estimate of response in the intended population.
The primary outcome was the number of participants with a desired response, intended to capture overall and complete response by WHO classification. The registry record gives the planned assessment timeframe as 8 weeks (2 cycles), unless treatment continued due to partial or complete response. It does not report a response count, response proportion, confidence interval, or formal primary-outcome analysis. The single participant’s progression is a clinically relevant event described in the record, but it is not a basis for estimating response probability in a broader population.
The absence of a comparator is another important interpretive constraint. This was a single-group study with allocation recorded as NA and masking as NONE. Without a concurrent comparison group, the study could not separate treatment-related changes from the course of disease or other influences through a randomized contrast. Even if a response had been reported, a single-group result would require cautious interpretation and would not answer a comparative-effectiveness question.
The registry lists secondary outcomes for response, response duration, progression-free survival, prolonged corrected QT interval, improved blood and lymphatic evaluation results, and adverse events. It reports no analyses. Listing these outcomes does not establish that each was measured successfully or analyzed. The posted serious adverse event information identifies a serious-event category, but does not establish that LBH589 caused it.
Overall, the record supports a description of the study’s intent, design, early termination, and limited participant experience. It does not support a precise estimate of response, progression-free survival, or the frequency of harms in the target population. The record does not report a statistical analysis plan, analysis populations, or methods for handling incomplete outcome data.
9. Statistical Methods Explained
What does a single-group design estimate?
A single-group study observes outcomes among participants who receive the study treatment. It can describe what happened to those participants, but it does not provide a direct randomized contrast with an untreated or differently treated group. General statistical interpretation therefore distinguishes a within-group description from an estimate of the treatment’s effect relative to a comparator. In this study, the registry records a SINGLE GROUP model, allocation as NA, and masking as NONE. No concurrent control group is reported.
The primary outcome was intended to count participants with a desired response under WHO classification. The registry record does not report an analyzed count or rate. With enrollment of 1 and early termination, the observed experience cannot provide a stable estimate of response frequency for the broader population of people with relapsed or refractory CLL or MCL. This is a limitation of the available evidence, not a reason to infer an unreported statistical result.
What would a response rate mean?
In general, a response rate is a proportion: the number of participants meeting a response definition relative to the relevant analysis population. Its interpretation depends on clear response criteria, the timing of assessments, and how participants without an evaluable assessment are handled. The registry record states that the intended measure was overall and complete response by WHO classification and gives an assessment timeframe of 8 weeks (2 cycles), unless treatment continued due to partial or complete response. It does not report the detailed criteria, the response analysis population, or an estimate. Consequently, no response proportion can be interpreted from the registry record.
General statistical practice uses uncertainty intervals to show how much precision an estimated effect has. A confidence interval reflects the range of values compatible with the data under the chosen method and assumptions. It is not reported for the primary endpoint. With so little participant information and no reported response analysis, there is no posted interval to evaluate. It would be inappropriate to construct or imply one from the registry description alone.
Why does the absence of a comparator matter?
Randomized comparisons are designed to make treatment groups comparable, on average, with respect to factors that can affect outcomes. This study did not have a randomized comparison: the record identifies no allocation and a single group. General causal interpretation is therefore limited. A disease outcome observed during treatment does not, by itself, tell how that same participant would have fared under another treatment or without the study drug. The record describes progression requiring more aggressive treatment, but it does not provide a comparative estimate or establish causation.
Masking is recorded as NONE. In general, knowledge of treatment assignment can influence some assessments or decisions, particularly when outcomes involve judgment. The record does not describe safeguards for outcome assessment, and it does not report how response assessments were conducted. This should be treated as an information limitation, rather than as evidence that a particular assessment was biased.
What can secondary outcomes contribute?
The registry lists secondary outcomes concerning complete and partial response, response duration, progression-free survival, prolonged corrected QT interval, blood and lymphatic evaluation results, and adverse events. Such outcomes can add information about disease course and safety when they are defined, measured, and analyzed. The registry record does not report analyses for these outcomes. In general, a named outcome is not the same as a reported result: the record may identify a planned measurement without presenting an estimate or explaining whether the measure could be evaluated.
Time-to-event outcomes such as response duration and progression-free survival require defined event criteria, timing rules, and a plan for participants whose follow-up ends before an event is observed. The registry record does not describe these analysis details or provide estimates. No survival curve, event count for these outcomes, or uncertainty measure can be inferred from the outcome names alone.
What does statistical precision require?
General statistical precision depends on the amount and structure of the data as well as the analysis method. A small amount of participant information generally leaves substantial uncertainty around an estimate. The record reports enrollment of 1 and does not report an efficacy estimate or confidence interval. Therefore, the primary efficacy question remains unresolved by the posted analysis. The enrollment number is not a measure of observed power, and it does not establish a formal sample-size justification. That justification is not reported in the registry record.
The registry record also does not report a detailed statistical plan, planned interim analyses, covariate adjustment, missing-data methods, or analysis populations. These omissions mean readers cannot verify which participants or measurements would have contributed to each planned estimate, or how incomplete follow-up was to be handled. No such methods should be assumed. The most defensible statistical reading is descriptive: the study planned response and safety assessments, enrolled a single participant, and was terminated without reported outcome analyses.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists multiple outcomes, including the primary desired-response measure and secondary measures of complete and partial response, response duration, progression-free survival, corrected QT interval, blood and lymphatic evaluation results, and adverse events. When a study tests or estimates several outcomes, general statistical practice considers multiplicity: repeated testing can increase the chance of at least one apparently notable result by chance. A prespecified hierarchy, adjustment, or clear distinction between primary and exploratory outcomes can help readers interpret a group of results. The registry record does not report a multiplicity strategy or a detailed statistical analysis plan.
The absence of reported analyses makes multiplicity adjustments impossible to assess in this record. It also means there are no posted endpoint estimates to compare or interpret. The registry lists 7 outcome measures and reports no analyses; the existence of listed outcomes should not be read as evidence that all were completed or analyzed.
The participant did not complete the study, and the record states that disease progression required more aggressive treatment. This creates an incomplete observation of the planned study course. The registry record does not report which outcome assessments were completed, whether any data were missing, how an analysis population would be defined, or how incomplete data would be handled. It also does not report whether the participant was included in any efficacy or safety analysis. These details are important because different rules for incomplete observations can affect estimates, especially in a study with such limited enrollment.
Thus, missing-data handling, analysis populations, and multiplicity procedures are not reported in the registry record. No assumptions about these methods should be used to create results that are not posted.
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 |
|---|---|---|---|
| Oral Drug Treatment | 1 | 1 | 100.0% |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 5% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Oral Drug Treatment | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | Oral Drug Treatment |
|---|---|---|
| Death not associated with CTCAE term - Disease progression NOS | Blood and lymphatic system disorders | 1 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Oral Drug Treatment |
|---|---|---|
| Hemorrhage, GI - Upper GI NOS | Gastrointestinal disorders | 1 of 1 |
Serious events with the highest arm-level rate: death not associated with ctcae term - disease progression nos (100.0%).
The registry safety information lists an Oral Drug Treatment group with 1 participant at risk for serious adverse events and 1 participant affected. The serious-event term listed is “Death not associated with CTCAE term - Disease progression NOS,” at 100.0%. The record categorizes this event as not associated with a CTCAE term and identifies disease progression NOS in the term.
These figures describe the registry’s reported safety classification; they do not establish that LBH589 caused the event. The registry record also states that the participant experienced disease progression requiring more aggressive treatment and did not complete the study. The safety entry should be read alongside that description, without inferring a causal relationship between the drug and the event. The record does not provide further detail about event timing, clinical circumstances, or an investigator’s causality assessment.
Because the group includes only 1 participant at risk, the listed percentage is highly dependent on that individual’s experience and cannot characterize the frequency of serious harms in a wider patient population. The serious adverse event figures should therefore be understood as a report from this small study, not as a reliable estimate of risk. The registry record does not report a broader comparative safety analysis.
12. What the Result Does, and Does Not, Mean
The record shows that investigators planned a Phase II assessment of oral LBH589 for relapsed or refractory CLL and MCL, with response as the primary outcome and a set of disease and safety outcomes also listed. It also shows that the study ended after enrollment of 1. The stated reasons were low accrual and the sponsor losing interest in the single-agent. The participant experienced disease progression requiring more aggressive treatment and did not complete the study.
These registry entries do not amount to an estimate of response or a comparison of LBH589 with another treatment. The registry record does not report a response count, response rate, progression-free survival estimate, response-duration result, or formal analysis. The participant’s progression is an individual outcome described in the record; it cannot establish what would happen to other people with these diseases or what would have happened to this participant under another treatment.
The record also does not establish that the study drug caused the serious adverse event category listed in the safety information. The entry identifies “Death not associated with CTCAE term - Disease progression NOS” and the record describes progression requiring more aggressive treatment, but does not report further causal assessment or clinical detail. The small enrollment prevents a meaningful estimate of event frequency in a broader population.
Statistically, the central issue is the difference between a planned study question and an analyzable result. The protocol description identifies what investigators intended to measure, but the posted results do not supply an efficacy analysis. No confidence interval or other measure of precision is reported. A single-group design also lacks a concurrent comparator, limiting causal interpretation even when outcomes are observed.
Accordingly, the record is informative about the study’s design and why it stopped, but it does not answer whether LBH589 changes response or safety outcomes in the intended population. It should not be interpreted as evidence of benefit or lack of benefit.
13. Why This Trial Matters Statistically
This trial illustrates why registry records need to be read as records of both study intent and study execution. A clearly stated endpoint can describe the question investigators planned to ask, but it does not guarantee that an estimate will be available. Here, the planned primary measure was overall and complete response by WHO classification, yet the study stopped after enrollment of 1 and the registry record reports no analysis.
The case also highlights how accrual and study discontinuation affect statistical information. A trial can be designed to answer a clinically relevant question but still yield too little participant information for an interpretable estimate. The reason for termination matters to the context: the record attributes abandonment to low accrual and the sponsor losing interest in the single-agent. The participant’s progression and non-completion further limit what can be learned about the planned response timeframe.
The single-group design makes the distinction between observation and causal evidence especially important. Outcomes during treatment can be described, but, without a comparator, they do not provide a randomized estimate of how treatment changes outcomes relative to an alternative. The absence of reported analyses and statistical methods means readers also cannot assess precision, handling of incomplete data, or planned multiplicity procedures.
For biostatisticians and clinical researchers, the record is a useful example of how limited enrollment can leave planned endpoints unanswered, and how safety entries should be interpreted in light of their denominator and context. Registry outcome listings, treatment schedules, and event labels are valuable information, but they cannot substitute for analyzed results. The appropriate conclusion is about the limits of the available evidence, not about treatment efficacy.
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 trial-specific limitation is enrollment of 1, followed by termination before the participant completed the study. The registry states that the study was abandoned because of low accrual and the sponsor losing interest in the single-agent. This leaves little information for assessing the intended response endpoint, and the registry record does not report an efficacy analysis.
The intended population included relapsed or refractory CLL and MCL, while the primary outcome description refers to relapsed or refractory aggressive MCL and CLL. The record does not provide further eligibility criteria or explain how the two disease groups would have been represented in analyses. With only the single enrolled participant, no reliable disease-specific estimate is available.
The design was single-group, with allocation recorded as NA and masking as NONE. There was no concurrent control group for a direct comparison. The registry record does not report details of response assessment procedures, a statistical analysis plan, analysis populations, handling of incomplete outcomes, or an interim-analysis plan. The participant’s non-completion is particularly relevant because the primary outcome timeframe was 8 weeks (2 cycles), unless treatment continued due to partial or complete response, and the record does not describe what assessments were completed.
The listed secondary outcomes extend across response, time-to-event outcomes, laboratory or evaluation results, and adverse events, but the registry reports no analyses. The serious-event listing is based on the study’s very limited enrollment and does not establish causation. Overall, the record documents an early-terminated study with sparse outcome information; it cannot support precise efficacy or safety estimates.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Oral LBH589 in Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) and Mantle Cell Lymphoma (MCL) — NCT01090973.
- Registry record last updated 2013-12-16; status last verified 2012-01.
- Results first posted 2012-03-28.
- 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
NCT01090973 is a Phase II, single-group study of oral LBH589 in relapsed or refractory CLL and MCL. The registry identifies H. Lee Moffitt Cancer Center and Research Institute as sponsor, records enrollment of 1, and lists status as TERMINATED. The intended primary outcome was the number of participants with a desired response, assessing overall and complete response by WHO classification at 8 weeks (2 cycles), unless treatment continued due to partial or complete response.
The study was abandoned after only one patient because of low accrual and the sponsor losing interest in the single-agent. The participant had disease progression requiring more aggressive treatment and did not complete the study. The registry lists 7 outcome measures but reports no analyses. It does not provide an efficacy estimate, a detailed statistical analysis plan, or methods for handling incomplete data. The posted record therefore describes the study’s intent and termination, but does not provide an analyzable estimate of response or comparative treatment effect.