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 Study to Allow Access to Single Agent Panobinostat for Patients Who Are on s.a. Panobinostat Treatment in a Novartis-sponsored Study and Continue to Benefit From the Treatment as Judged by the Investigator (NCT01802879), a na phase 2 study. The primary outcome is Overview of Adverse Events (Safety Set).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01802879 |
| Phase | Phase 2 |
| Condition | Hematologic Neoplasms |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 9 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novartis Pharmaceuticals |
| Results posted | Yes |
2. Clinical Question
Population
The study concerned patients with hematologic neoplasms who had completed a previous Novartis-sponsored panobinostat study. The registry describes eligibility in terms of being judged by the investigator to benefit from continued single-agent panobinostat. Nine participants were enrolled. The characteristics of the enrolled participants and the number who met each eligibility condition are not reported in the registry record.
Intervention
Participants received single-agent panobinostat. The listed regimen was 10 to 40 mg/day, 3 times per week every other week, as specified by the parent protocol design. The registry does not provide participant-level dose histories or describe changes in dose during follow-up.
Comparator
There was no comparator group. The study is classified as single-group, with no masking and no allocation. Its design therefore does not support a like-for-like comparison with another treatment or with a control group enrolled in this study.
Primary question
The primary outcome is an overview of adverse events in the safety set, collected from baseline through the stated follow-up period. The record also lists investigator-assessed clinical benefit as a secondary outcome, but numerical results for that outcome are not reported in the registry record. The study is therefore described by the posted safety counts, not by a reported estimate of comparative efficacy.
3. Trial Design
This is a phase 2, open-label, multicenter, single-group roll-over protocol. Its stated purpose was to allow continued single-agent panobinostat for patients who had completed a previous Novartis-sponsored panobinostat study and were judged by the investigator to benefit from ongoing treatment. The record describes nine enrolled participants and a completed study. The recorded start date is 2013-06-24, and the primary completion date is 2018-11-19.
The intervention was panobinostat, listed at 10 to 40 mg/day, 3 times per week every other week, according to the parent protocol design. Masking was none, and allocation was not applicable. There is no randomized control group, so the study does not provide a direct treatment comparison. Its single-group structure is consistent with the stated access purpose: continued treatment for a selected group already receiving panobinostat and considered by an investigator to benefit.
The primary outcome is an overview of adverse events in the safety set. The registry specifies collection from baseline up to 30 days after treatment at scheduled visits and gives a timeframe of baseline up to approximately 60 months. The registry also lists investigator-assessed clinical benefit as a secondary outcome. Numerical results for that secondary outcome are not reported in the registry record. The statistical analysis plan, eligibility criteria in full, and reasons for any difference between enrollment and the safety-set count are 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.
Before assignment: There was no screening period. Patients enrolled into trial directly from the parent protocol.
Overall Study
| Stage | Panobinostat - 10 to 40 mg/Day TIW QoW |
|---|---|
| STARTED | 8 |
| COMPLETED | 0 |
| NOT COMPLETED | 8 |
Reasons for not completing this period:
| Reason | Panobinostat - 10 to 40 mg/Day TIW QoW |
|---|---|
| Administrative problems | 2 |
| Disease progression | 6 |
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 | Panobinostat - 10 to 40 mg/Day TIW QoW |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 54 (SD 14.5) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 4 |
| Race and Ethnicity Not Collected | Participants | — |
| Parent protocol participants: CLBH589B2201 | participants | 2 |
| Parent protocol participants: CLBH589B2207 | participants | 3 |
| Parent protocol participants: CLBH589E2214 | participants | 1 |
| Parent protocol participants: CLBH589X2105 | participants | 2 |
6. Primary Endpoint
The registry lists the primary outcome as Overview of Adverse Events (Safety Set), measured over Baseline up to approximately 60 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is an overview of adverse events in the safety set, expressed as the number of participants with events. Adverse events were collected from baseline up to 30 days after treatment at scheduled visits. The stated outcome timeframe is baseline up to approximately 60 months. The registry says that severity was assessed using the current version of CTCAE; when CTCAE grading did not exist for an event, severity categories of mild, moderate, severe, and life-threatening, corresponding to Grades 1 - 4, were used.
Safety outcomes are used to describe events observed during treatment and follow-up, including their seriousness and severity. They are descriptive: a participant count can indicate how many people in the safety set had an event, but does not by itself establish that treatment caused the event. Interpretation also depends on the population assessed, observation period, event definitions, and completeness of collection.
The posted primary outcome is not a measure of clinical benefit. Investigator-assessed clinical benefit appears as a secondary outcome, but its numerical results are not reported in the registry record. The endpoint description does not specify a numerical summary for each adverse-event category beyond participant counts.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry reports nine enrolled participants, while the adverse-event results identify eight participants at risk in the safety set. In that set, serious adverse events affected 2 participants, and other adverse events affected 4 participants. The record lists 0 deaths. These are descriptive counts from a small, single-group study; they should not be read as comparative estimates against another treatment or a control group.
The registry lists non-cardiac chest pain and cerebrovascular accident among the top serious-event terms, each with a recorded value of 12.5%. The record does not provide further detail in the posted results about event timing, recurrence, severity, relatedness to treatment, or whether a participant experienced more than one event. The listed percentages should therefore be read as the registry presents them, without inferring additional counts or participant-level patterns.
The study enrolled participants who had already completed a prior panobinostat study and were judged by an investigator to benefit from continued treatment. This selection is important when interpreting the results: the participants do not represent an unselected population, and there is no concurrent comparison group. The observed counts describe events in this selected safety set over the recorded follow-up, but cannot isolate the effect of panobinostat from other possible explanations.
The registry lists investigator-assessed clinical benefit as a secondary outcome, but numerical results are not reported in the registry record. Consequently, the posted information does not provide a numerical estimate of clinical benefit to interpret alongside the safety counts. No inferential analyses are listed. Confidence intervals, adjusted estimates, and hypothesis-test results are not reported in the registry record.
Small participant counts make descriptive summaries especially sensitive to the experience of each individual participant. General statistical context: with a small denominator, a count can change noticeably when one participant's status differs, and estimates of event frequency can be imprecise. The registry does not provide confidence intervals to quantify that uncertainty. The counts are useful as a description of the recorded safety experience, but they do not establish comparative risk or a causal relationship.
9. Statistical Methods Explained
The registry classifies the primary outcome as a count of participants with an event. This is a binary outcome at the participant level: for a particular event definition, each participant is classified as having or not having the event during the specified observation period. General statistical context: a participant count differs from an event count, because one participant may experience more than one event. The posted figures should be interpreted according to their stated units and categories, rather than assumed to be counts of every event occurrence.
What does a single-group design allow?
The study is listed as single-group, with no allocation and no masking. The safety results therefore describe participants receiving panobinostat, without a concurrent control group. General statistical context: a control group can help distinguish outcomes occurring during treatment from outcomes that might also occur without that treatment or under another treatment. Without such a comparison, an observed adverse event cannot be attributed to the study drug solely from its occurrence in the study. Nor can the registry results establish whether event frequency differs from another treatment.
The roll-over setting also shapes the population being described. Participants had completed a previous Novartis-sponsored panobinostat study, and continued treatment was for patients judged by the investigator to benefit. This design was intended to permit access to continued treatment, rather than to enroll a new randomized comparison. The registry does not report enough detail to characterize how this selection affected the safety results.
How should participant counts be read?
The safety results identify eight participants at risk. Serious adverse events are reported for 2 participants, and other adverse events for 4 participants. These counts are not adjusted estimates and are not accompanied by confidence intervals or hypothesis tests in the registry record. General statistical context: a confidence interval expresses uncertainty around an estimate under a statistical model; it is not a guarantee that the true value lies within the interval. No such intervals are reported for these counts, so the precision of a population-level event-frequency estimate cannot be assessed from the posted results alone.
The record gives an adverse-event collection period from baseline up to 30 days post treatment at scheduled visits, and a timeframe of baseline up to approximately 60 months. These descriptions convey the intended observation span but do not give individual follow-up durations or participant-level exposure. General statistical context: when follow-up time differs across participants, simple participant counts do not account for those differences. A time-to-event analysis may address timing and exposure differently, but no such analysis is listed in the registry record.
What does the severity description mean?
The record states that severity was assessed according to the current version of CTCAE. For an adverse event without a CTCAE grade, the described categories were mild, moderate, severe, and life-threatening, corresponding to Grades 1 - 4. Seriousness and severity are distinct concepts in safety reporting: seriousness concerns regulatory or clinical consequences as defined by reporting criteria, while severity describes intensity. The posted information does not provide a complete event-by-event severity breakdown, so the serious-event counts should not be treated as a severity distribution.
What analyses are available?
No analyses are listed in the registry record. The statistical analysis plan, any prespecified methods for summarizing the safety outcome, and any approach to missing observations are not reported in the registry record. General statistical context: an analysis plan can clarify which participants are included, how repeated events are handled, and how uncertainty is quantified. Without those details, interpretation should remain close to the reported participant counts and outcome definitions.
Planning calculations are distinct from observed trial results. General statistical context: a sample-size planning illustration describes assumptions for a hypothetical design; it does not describe the number of participants actually observed or establish the power of a completed study. The registry record does not report a trial-specific sample-size justification, so no such rationale can be inferred from the enrollment count.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists an overview of adverse events as the primary outcome and investigator-assessed clinical benefit as a secondary outcome. It does not report a statistical analysis plan, a hierarchy of outcome tests, or a strategy for controlling multiplicity. The number of analyses listed is 0. Thus, the posted record does not provide a set of inferential tests from which to assess how multiple comparisons were handled.
Numerical results for the secondary clinical-benefit outcome are not reported in the registry record. No reasons for the absence of those results are stated. It is not possible to infer whether the outcome was assessed but not posted, whether data were unavailable, or whether another explanation applies.
The enrollment count is nine, while the safety results identify eight participants at risk. The registry does not explain the difference or report the characteristics of participants who were not included in the safety set. The primary outcome describes the safety set, but the method used to define that set is not reported in the registry record.
The record does not report how missing safety observations were handled, whether every participant completed follow-up, or how discontinuation affected observation time. The stated event-collection period and approximate timeframe do not establish that all participants were observed for the same duration. These omissions limit assessment of the completeness and comparability of the safety summaries.
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 |
|---|---|---|---|
| Panobinostat - 10 to 40 mg/Day TIW QoW | 2 | 8 | 25.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Panobinostat - 10 to 40 mg/Day TIW QoW | 0 of 8 |
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 |
|---|---|
| Panobinostat - 10 to 40 mg/Day TIW QoW | 4 of 8 |
Most frequent serious adverse event terms
| Event term | Organ system | Panobinostat - 10 to 40 mg/Day TIW QoW |
|---|---|---|
| Non-cardiac chest pain | General disorders | 1 of 8 |
| Cerebrovascular accident | Nervous system disorders | 1 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Panobinostat - 10 to 40 mg/Day TIW QoW |
|---|---|---|
| Neuropathy peripheral | Nervous system disorders | 2 of 8 |
| Neutropenia | Blood and lymphatic system disorders | 1 of 8 |
| Diarrhoea | Gastrointestinal disorders | 1 of 8 |
| Gastric disorder | Gastrointestinal disorders | 1 of 8 |
| Asthenia | General disorders | 1 of 8 |
| Nasopharyngitis | Infections and infestations | 1 of 8 |
| Blood creatinine increased | Investigations | 1 of 8 |
| Platelet count decreased | Investigations | 1 of 8 |
| Bone pain | Musculoskeletal and connective tissue disorders | 1 of 8 |
| Osteoarthritis | Musculoskeletal and connective tissue disorders | 1 of 8 |
Serious events with the highest arm-level rate: non-cardiac chest pain (12.5%), cerebrovascular accident (12.5%).
The safety results identify eight participants at risk. Serious adverse events were reported for 2 participants, and other adverse events for 4 participants; the record lists 0 deaths. The registry also identifies non-cardiac chest pain and cerebrovascular accident as top serious-event terms, each with a recorded value of 12.5%. Those percentages are reported as event-term values; the record does not provide additional participant-level detail about event counts or overlap.
These figures describe the safety set and do not provide a comparison with an untreated group or another treatment. The registry specifies that adverse events were collected from baseline up to 30 days after treatment at scheduled visits, with a stated timeframe of baseline up to approximately 60 months. Individual follow-up durations and event-level timing are not reported in the registry record.
Seriousness should not be confused with severity. The record describes CTCAE severity grading, but the figures presented do not give a full severity distribution for all events. The counts alone do not establish causation, comparative risk, or the experience of people outside this selected study population.
12. What the Result Does, and Does Not, Mean
The posted results describe adverse events recorded among eight participants in the safety set of a completed, open-label, single-group panobinostat roll-over study. Serious adverse events affected 2 participants, other adverse events affected 4 participants, and the record lists 0 deaths. The serious-event terms identified include non-cardiac chest pain and cerebrovascular accident, each recorded at 12.5%.
These results do not establish that panobinostat caused any particular event. Events occurring during treatment may have multiple explanations, and this study has no concurrent control group for comparison. The study population was also selected: participants had completed an earlier panobinostat study and were judged by an investigator to benefit from continued single-agent treatment. The findings therefore describe this study group, not an unselected patient population.
The posted results do not provide numerical findings for the secondary outcome of investigator-assessed clinical benefit. They also do not report confidence intervals or inferential analyses for the safety counts. As a result, the precision of estimates beyond the descriptive counts cannot be assessed from the registry record.
The record is informative about the reported safety experience under the study's observation and reporting framework. It does not, on its own, determine comparative safety, treatment effect, or how outcomes would differ under another treatment. Those questions require evidence and analyses beyond the single-group counts.
13. Why This Trial Matters Statistically
This study illustrates how a trial's purpose and design shape the questions its results can answer. The roll-over protocol was intended to allow continued access to single-agent panobinostat for patients who had completed a prior sponsored study and were judged to benefit. A single-group design can describe safety events among participants receiving treatment, but it does not create a direct comparison with another treatment or a control group.
The difference between nine enrolled participants and eight participants at risk in the safety results also highlights why analysis populations matter. The registry does not explain the difference, so the safety counts must be understood as applying to the stated safety set rather than automatically to everyone enrolled.
Finally, a listed secondary outcome is not the same as a reported result. Investigator-assessed clinical benefit appears in the registry, but its numerical results are not reported in the registry record. Distinguishing outcome definitions from available numerical findings helps prevent readers from treating a study's stated aims as evidence of a particular result.
General statistical context: descriptive counts can be useful for documenting what was observed, while uncertainty and comparisons require suitable methods and enough information about participants, follow-up, and analysis. The registry record gives a limited descriptive safety summary, not a comparative estimate of treatment effects.
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 study enrolled nine participants, and the safety results identify eight participants at risk. The registry does not explain this difference or describe the participants excluded from the safety set. The small study population also means the findings describe a limited group; the record does not provide enough participant characteristics to assess how closely that group resembles other populations.
The single-group design has no concurrent comparator, and the study was open-label with no masking. The safety counts cannot establish whether the events were more or less frequent than they would have been under another treatment or without treatment. The selected roll-over population had completed a previous Novartis-sponsored panobinostat study and was judged by an investigator to benefit from continuation, which further limits generalization to other groups.
The registry gives a timeframe of baseline up to approximately 60 months and says adverse events were collected up to 30 days post treatment at scheduled visits. Individual follow-up durations, exposure histories, and event timing are not reported in the registry record. Neither the handling of missing observations nor the definition of the safety set is reported.
Numerical clinical-benefit results, confidence intervals, inferential analyses, and a trial-specific sample-size justification are not reported in the registry record. The posted safety information is therefore descriptive and does not establish causation, comparative risk, or the magnitude and precision of a clinical-benefit effect.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study to Allow Access to Single Agent Panobinostat for Patients Who Are on s.a. Panobinostat Treatment in a Novartis-sponsored Study and Continue to Benefit From the Treatment as Judged by the Investigator — NCT01802879.
- Registry record last updated 2019-12-06; status last verified 2019-11.
- Results first posted 2019-12-06.
- 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
This completed phase 2 study was an open-label, multicenter, single-group roll-over protocol of single-agent panobinostat. It enrolled nine participants and was intended to allow continued treatment for patients who had completed a previous Novartis-sponsored panobinostat study and were judged by the investigator to benefit. The listed regimen was 10 to 40 mg/day, 3 times per week every other week, according to the parent protocol design.
The primary outcome was an overview of adverse events in the safety set. The registry reports eight participants at risk, serious adverse events in 2 participants, other adverse events in 4 participants, and 0 deaths. Investigator-assessed clinical benefit is listed as a secondary outcome, but numerical results are not reported in the registry record. There is no comparator group, and no analyses are listed.