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 A Study to Evaluate Long-term Safety of CC-486 (Oral Azacitidine) in Subjects With Hematological Disorders (NCT02494258), a na phase 2 study. The primary outcome is Number of Participants With Treatment Emergent Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02494258 |
| Phase | Phase 2 |
| Condition | Hematologic Neoplasm, Neoplasms, Hematologic Malignancies |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 5 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Celgene |
| Results posted | Yes |
2. Clinical Question
Population
Participants with hematological disorders who had taken part in other Celgene-sponsored CC-486 trials and were eligible to enter this rollover study. The primary outcome population is described as all treated participants.
Intervention
Oral azacitidine, also called CC-486. The study description says treatment was intended to continue at the same dose, schedule, and frequency as the last CC-486 dose in the parent study.
Comparator
There was no separate control group. The registry describes a single-arm study; the four dosing groups are not a randomized, like-for-like comparison.
Primary question
How many treated participants experienced treatment-emergent adverse events during the specified observation period? The endpoint includes events that begin or worsen after the first study dose, regardless of whether they are considered related to CC-486.
The study’s stated purpose was to evaluate long-term safety in people rolling over from eligible parent trials. It was an open-label phase 2 study, so participants and study personnel were not masked to treatment. The registry lists an enrollment of 5 and the primary outcome population as all treated participants; it does not report the number treated as a separate figure.
The observation period began when informed consent was signed on Day 1 and continued until 28 days after the last CC-486 dose or until the treatment discontinuation visit, whichever was later. The registry gives an upper duration of approximately 90 months. A secondary outcome was the number of participants who survived.
This question is descriptive rather than a test of comparative efficacy or safety. With no concurrent control group, the record cannot establish how the observed event experience compares with another treatment or with no study treatment. The intended contribution is a record of safety events during continued exposure among eligible rollover participants.
3. Trial Design
This was a phase 2, open-label, single-group rollover study of oral azacitidine (CC-486). The registry lists Celgene as the sponsor and describes enrollment of 5 participants. The study status is completed. The recorded study start was 2015-10-22, and the primary completion date was 2025-04-14.
Participants had taken part in other Celgene-sponsored CC-486 clinical trials for hematological disorders and needed to be eligible for rollover participation. The study description divides participation into screening, treatment, and follow-up periods. Treatment was intended to use the same dose, schedule, and frequency as the last CC-486 dose in the parent study. The registry’s arm description is truncated, so further details of those periods are not reported in the registry record.
The record lists four CC-486 dosing groups: 200 mg QD x 14 Days, 600 mg QD x 7 Days, 500 mg QD x 7 Days, and 200 mg QD x 7 Days. The group-specific safety counts are very small. These groups should not be interpreted as randomized arms or as a controlled dose comparison: the allocation is listed as not applicable, and the study model is single group. Differences in the posted counts cannot isolate dose effects from other differences between the participants or their parent studies.
There was no masking and no control group. No randomization procedure is reported in the registry record. The study’s stated focus was long-term safety, not a comparative efficacy question. The registry lists two outcome measures and no analyses. Details of the protocol’s statistical plan, including any planned comparisons or interim analyses, 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.
Overall Study
| Stage | CC-486 200 mg QD x 14 Days | CC-486 600 mg QD x 7 Days | CC-486 500 mg QD x 7 Days | CC-486 200 mg QD x 7 Days |
|---|---|---|---|---|
| STARTED | 2 | 1 | 1 | 1 |
| COMPLETED | 0 | 0 | 0 | 0 |
| NOT COMPLETED | 2 | 1 | 1 | 1 |
Reasons for not completing this period:
| Reason | CC-486 200 mg QD x 14 Days | CC-486 600 mg QD x 7 Days | CC-486 500 mg QD x 7 Days | CC-486 200 mg QD x 7 Days |
|---|---|---|---|---|
| Progressive disease | 0 | 0 | 0 | 1 |
| Physician Decision | 0 | 1 | 0 | 0 |
| Other reasons | 1 | 0 | 1 | 0 |
| Adverse Event | 1 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: All treated participants
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 | CC-486 200 mg QD x 14 Days | CC-486 600 mg QD x 7 Days | CC-486 500 mg QD x 7 Days | CC-486 200 mg QD x 7 Days | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 2 | 1 | 1 | 1 | 5 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 0 | 1 | 0 | 0 | 1 |
| Age, Categorical: >=65 years | Participants | 2 | 0 | 1 | 1 | 4 |
| Sex: Female, Male: Female | Participants | 1 | 0 | 0 | 0 | 1 |
| Sex: Female, Male: Male | Participants | 1 | 1 | 1 | 1 | 4 |
| Race/Ethnicity, Customized: Not Hispanic or Latino | Participants | 2 | 1 | 1 | 1 | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Participants With Treatment Emergent Adverse Events, measured over From informed consent signed (Day 1) and until 28 days after the last dose of CC-486, or until the treatment discontinuation visit, whichever was later (up to approximately 90 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 participants with treatment-emergent adverse events, reported as a participant count. The registry defines a treatment-emergent adverse event as an untoward medical occurrence that begins or worsens after the first dose of study treatment. Its definition includes unfavorable signs, symptoms, disease, or abnormal laboratory findings, whether or not they are considered related to the product. Worsening of a pre-existing condition may also qualify.
The definition is broad because safety monitoring aims to capture medical events occurring during treatment, rather than only events judged to have been caused by the study drug. A serious adverse event is a subset defined by outcomes such as death, life-threatening illness, hospitalization or prolonged hospitalization, persistent or significant disability, congenital anomaly or birth defect, or an important medical event requiring intervention to prevent such outcomes.
The endpoint observation window starts at informed consent signed on Day 1 and continues until 28 days after the last CC-486 dose or the treatment discontinuation visit, whichever is later; the registry states a duration of up to approximately 90 months. The outcome is useful for describing the events recorded during that window. A participant count does not, by itself, show event severity, duration, recurrence, or the degree to which an event was attributable to treatment. The posted record does not report a single overall result for this primary endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a small rollover study with an enrollment of 5, and the posted safety counts are sparse. The four dose-group entries report serious adverse events in the following terms: in the CC-486 200 mg QD x 14 Days group, 1 of 2 participants at risk was affected; in the 600 mg QD x 7 Days group, 0 of 1 was affected; in the 500 mg QD x 7 Days group, 1 of 1 was affected; and in the 200 mg QD x 7 Days group, 0 of 1 was affected. These are descriptive counts, not estimates from a controlled comparison.
For the other adverse-event entries, the registry lists 1 of 2 participants at risk in the 200 mg QD x 14 Days group, 1 of 1 in the 600 mg QD x 7 Days group, 1 of 1 in the 500 mg QD x 7 Days group, and 1 of 1 in the 200 mg QD x 7 Days group. The record also lists zero deaths in each dosing group. The registry’s primary endpoint is the number of participants with treatment-emergent adverse events, but a separate overall primary-endpoint count is not reported in the registry record. The group-level entries should not be substituted for a distinct overall result.
The serious-event terms listed include Hip fracture at 100.0% and Diarrhoea at 50.0%. The registry does not give the denominators for these term percentages in the available record, so they should not be read as stable estimates of event frequency. With very few participants, an event in one person can account for a large share of a group or a listed term category. Small counts also provide little precision about the range of safety experiences that might occur in a larger population.
Because the study was single-arm and nonrandomized, the observed counts cannot establish that CC-486 caused an event, nor can they quantify a difference from another treatment. The treatment-emergent definition expressly includes events whether or not related to the product. Attribution requires information beyond simple event occurrence, and a comparison would require an appropriate control and analysis. No such comparative analysis is reported in the registry record.
General statistical interpretation: counts are direct descriptions of the observed participants, while estimates meant to generalize beyond them are uncertain when the data are this sparse. A confidence interval, when reported, describes the precision of an estimate under its statistical assumptions; no confidence intervals or analyses are listed in the registry record. The posted safety figures are therefore best treated as a limited account of events recorded in this study, not as precise rates or comparative evidence.
9. Statistical Methods Explained
The registry reports participant counts rather than a modeled treatment effect for the primary safety outcome. This aligns with the stated endpoint, which asks how many participants experienced a treatment-emergent adverse event. The outcome is binary at the participant level: a participant either meets the event definition during the observation period or does not. Counts preserve the directly observed scale and avoid implying more precision than the small study can support.
Why a single-arm design matters
General statistical explanation: a randomized controlled comparison can help separate treatment differences from differences in participant characteristics, follow-up, and other influences. A single-arm study has no concurrent control group to provide that comparison. Here, the registry describes a rollover study in which participants came from eligible parent trials and continued CC-486. The posted results can describe events during participation, but they cannot by themselves estimate a treatment-versus-control contrast or establish that treatment caused an event.
The four dosing groups are not a substitute for randomized comparison groups. The record lists allocation as not applicable and the model as single group. Even if counts differ across groups, the design does not establish that dose explains those differences. The registry record does not report a planned statistical comparison among the dose groups.
What counts and percentages convey
A participant count communicates how many people experienced an event in the recorded group. A percentage expresses a count relative to a denominator, but its precision depends strongly on how many participants contribute. General statistical explanation: with a small denominator, a single participant can materially change a percentage. Therefore, the listed serious-event term percentages should be interpreted alongside the sparse group counts, and not as precise estimates of what would happen in a broader population. Denominators for the term percentages are not reported in the registry record.
The posted primary outcome is defined as a count of participants, but the record does not include a separate overall count for that endpoint. Group-specific serious and other adverse-event entries offer related safety information, but they do not supply a complete overall primary-endpoint result. The distinction matters: the broad treatment-emergent definition covers events regardless of whether they are judged related, while the separate serious-event category uses seriousness criteria such as hospitalization, life-threatening illness, or death.
Observation time and event capture
Participants could have different lengths of observation, because the endpoint window runs from informed consent until 28 days after the last dose or the treatment discontinuation visit, whichever was later. The registry describes this period as up to approximately 90 months. A simple participant count does not adjust for differences in time under observation. General statistical explanation: when follow-up durations vary, event counts alone do not express how frequently events occurred per unit of time. The registry does not report a time-adjusted event-rate analysis.
For safety outcomes, the endpoint definition includes events beginning or worsening after the first study dose, including abnormal laboratory findings and worsening pre-existing conditions. This broad capture helps describe the medical events recorded during treatment, but it is not the same as a causal assessment. The definition explicitly includes events whether or not they are considered related to the product.
Precision and modeling
General statistical explanation: confidence intervals communicate uncertainty around an estimate, conditional on the method and assumptions used to calculate them. For sparse binary outcomes, intervals can be wide, and different methods can produce different interval limits. No confidence intervals, model-based effects, or inferential analyses are listed in the registry record. The registry lists no analyses, so no statistical test or adjusted estimate should be inferred from the descriptive counts.
General statistical explanation: over-dispersion is a concern when count data vary more than an assumed statistical model allows. It can make standard errors too small if a model does not account for that extra variation. The posted primary endpoint is a participant count, and the record does not describe a count regression model or an over-dispersion adjustment. Accordingly, over-dispersion should not be assumed to have been assessed.
The registry record does not report a sample-size justification, a statistical analysis plan, interim analyses, multiplicity adjustment, or methods for handling missing outcome data. These are important details for understanding planned inference, but their absence from the record does not show what may or may not have appeared in other study documents. The posted evidence supports descriptive reading of the listed counts, not reconstruction of an unreported analysis plan.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists two outcome measures: the primary measure is the number of participants with treatment-emergent adverse events, and a secondary measure is the number of participants who survived. It does not list analyses. A statistical method for controlling multiplicity across outcomes or across any subgroup or dose-group comparisons is not reported in the registry record. Since no inferential analyses are listed, the record does not show whether any multiplicity procedure was planned or applied.
Missing-data handling is also not reported in the registry record. The primary endpoint population is described as all treated participants, but the record does not explain how participants with incomplete follow-up, missing event information, or other unavailable outcome data were handled. It also does not provide a separate treated-participant count. The group-level at-risk counts identify the denominators used for those posted safety entries, but they do not explain whether any data were missing or excluded.
General statistical explanation: missing outcome information can affect interpretation if participants with unavailable data differ in their event experience from those whose outcomes are recorded. An analysis plan commonly specifies how such cases are handled, but the registry record does not report a plan or method. No assumptions about imputation, censoring, or exclusion should be inferred from the available record.
The record also does not report interim analyses or a sample-size justification. Their absence from the registry record means the basis and timing of any such procedures cannot be described. The posted counts should therefore be read as descriptive registry results without assuming unreported adjustments, missing-data methods, or planned inferential procedures.
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 |
|---|---|---|---|
| CC-486 200 mg QD x 14 Days | 1 | 2 | 50.0% |
| CC-486 600 mg QD x 7 Days | 0 | 1 | 0.0% |
| CC-486 500 mg QD x 7 Days | 1 | 1 | 100.0% |
| CC-486 200 mg QD x 7 Days | 0 | 1 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| CC-486 200 mg QD x 14 Days | 0 of 2 |
| CC-486 600 mg QD x 7 Days | 0 of 1 |
| CC-486 500 mg QD x 7 Days | 0 of 1 |
| CC-486 200 mg QD x 7 Days | 0 of 1 |
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 |
|---|---|
| CC-486 200 mg QD x 14 Days | 1 of 2 |
| CC-486 600 mg QD x 7 Days | 1 of 1 |
| CC-486 500 mg QD x 7 Days | 1 of 1 |
| CC-486 200 mg QD x 7 Days | 1 of 1 |
Most frequent serious adverse event terms
| Event term | Organ system | CC-486 200 mg QD x 14 Days | CC-486 600 mg QD x 7 Days | CC-486 500 mg QD x 7 Days | CC-486 200 mg QD x 7 Days |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 1 of 2 | 0 of 1 | 0 of 1 | 0 of 1 |
| Hip fracture | Injury, poisoning and procedural complications | 0 of 2 | 0 of 1 | 1 of 1 | 0 of 1 |
Most frequent non-serious adverse event terms
| Event term | Organ system | CC-486 200 mg QD x 14 Days | CC-486 600 mg QD x 7 Days | CC-486 500 mg QD x 7 Days | CC-486 200 mg QD x 7 Days |
|---|---|---|---|---|---|
| Fatigue | General disorders and administration site conditions | 1 of 2 | 1 of 1 | 1 of 1 | 0 of 1 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 1 of 2 | 1 of 1 | 1 of 1 | 0 of 1 |
| Anaemia | Blood and lymphatic system disorders | 1 of 2 | 1 of 1 | 0 of 1 | 0 of 1 |
| Diarrhoea | Gastrointestinal disorders | 1 of 2 | 0 of 1 | 1 of 1 | 0 of 1 |
| Oedema peripheral | General disorders and administration site conditions | 1 of 2 | 0 of 1 | 1 of 1 | 0 of 1 |
| Myalgia | Musculoskeletal and connective tissue disorders | 0 of 2 | 1 of 1 | 1 of 1 | 0 of 1 |
| Neutropenia | Blood and lymphatic system disorders | 0 of 2 | 0 of 1 | 0 of 1 | 1 of 1 |
| Vertigo | Ear and labyrinth disorders | 0 of 2 | 1 of 1 | 0 of 1 | 0 of 1 |
| Dental caries | Gastrointestinal disorders | 0 of 2 | 1 of 1 | 0 of 1 | 0 of 1 |
| Dyspepsia | Gastrointestinal disorders | 0 of 2 | 0 of 1 | 1 of 1 | 0 of 1 |
Serious events with the highest arm-level rate: hip fracture (100.0%), diarrhoea (50.0%).
The serious adverse-event figures are participant counts within each listed dose group. For CC-486 200 mg QD x 14 Days, 1 of 2 participants at risk was affected; for 600 mg QD x 7 Days, 0 of 1; for 500 mg QD x 7 Days, 1 of 1; and for 200 mg QD x 7 Days, 0 of 1. The registry lists zero deaths in each group. These small denominators mean that the entries describe only the participants observed, rather than providing precise estimates for a wider population.
The registry also lists serious-event terms Hip fracture at 100.0% and Diarrhoea at 50.0%. The denominators behind these percentages are not reported in the registry record. The percentages should therefore be read as registry-listed proportions for those terms, not as population-level risks. They do not indicate how likely an event is for people outside this small study.
General statistical explanation: seriousness and treatment relatedness are distinct concepts. Seriousness is based on the event’s consequences or need for intervention, while treatment-emergent describes timing relative to study treatment. The registry’s treatment-emergent definition includes events whether or not related to the product. The counts alone do not establish causation or enable a comparison against another treatment.
12. What the Result Does, and Does Not, Mean
The result provides a limited descriptive account of adverse events recorded among participants in a small, open-label, single-arm rollover study. The posted entries show serious and other adverse events across four CC-486 dosing groups, along with zero deaths listed in each group. They do not provide a separate overall count for the primary endpoint, which is defined as the number of participants with treatment-emergent adverse events.
These findings do not establish whether CC-486 caused any particular event. The endpoint includes events regardless of whether they are considered related to the product, and the registry does not provide a controlled comparison. Nor do the counts demonstrate that one listed dose schedule is safer than another: the groups are not described as randomized like-for-like arms, and the small counts do not support a reliable dose comparison.
General statistical explanation: the precision of an estimate depends in part on the amount of information available. With very few participants, estimates can be highly uncertain and can change substantially when additional observations are collected. A percentage from a small group may look exact while still being an unstable estimate of a broader event frequency. The term percentages have no reported denominators in the registry record, which further limits their interpretation.
The record also cannot establish how the observed event experience compares with people who did not receive CC-486 or with a different treatment. Such conclusions require an appropriate comparison and analysis, neither of which is reported for this single-arm study. The posted information is useful for understanding what safety events were recorded in this rollover cohort, but it should not be treated as a definitive estimate of risk or a causal treatment effect.
Important context remains unreported in the registry record, including the overall primary-endpoint result, methods for handling missing data, and a statistical analysis plan. These omissions limit how far the results can be interpreted. The appropriate reading is descriptive: the entries summarize observed safety counts in the recorded groups, with substantial limits on precision and comparison.
13. Why This Trial Matters Statistically
Rollover studies address a distinct descriptive question: what safety events are recorded when eligible participants continue treatment beyond their parent trials? This study’s stated purpose was long-term safety evaluation among participants from other Celgene-sponsored CC-486 trials. Its observation window extended from informed consent through a period tied to the last dose or treatment discontinuation visit, with a registry duration of up to approximately 90 months.
Statistically, the study illustrates the value and limits of small safety datasets. Participant counts can document that events occurred and can preserve information about serious outcomes in a defined cohort. They are often easier to interpret than a complex model when the dataset is sparse. At the same time, small counts provide little precision, and percentages can be especially sensitive to the experience of an individual participant.
The single-arm structure also clarifies what this evidence can contribute. It records events during CC-486 exposure, but it does not provide a randomized control comparison. The dosing groups do not overcome that limitation. The counts are therefore descriptive and cannot isolate treatment effects from other influences or support a controlled comparison among dose schedules.
The registry’s definitions are important to interpretation. Treatment-emergent events include occurrences that begin or worsen after the first dose, whether or not they are considered related to the product; serious events are identified by clinical consequence criteria. Keeping those categories distinct helps prevent a timing-based event count from being mistaken for proof of causation.
Finally, the record’s missing analysis details matter. No overall primary-endpoint count, statistical analysis plan, missing-data method, or multiplicity approach is reported in the registry record. Clear reporting of these details helps readers understand what was measured, which participants contributed, and how uncertainty was handled. Without them, the most defensible interpretation remains a cautious description of the posted counts.
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 enrollment of 5 limits the amount of safety information available. The group-specific counts are sparse, and the registry does not report confidence intervals or other measures of precision. The serious-event term percentages have no reported denominators, further restricting interpretation. The figures should not be treated as stable estimates of event frequencies beyond this study.
The study was open-label and single-arm, with no randomized comparator. As a result, the posted data do not support a causal comparison with another treatment or with no treatment. The four dosing groups are not a like-for-like control comparison, and any apparent difference between their counts cannot be attributed to dose on the basis of this design.
The registry lists the primary endpoint but does not report a distinct overall result for it. Although group-level entries describe serious and other adverse events, they do not replace the overall treatment-emergent adverse-event participant count. The record also does not report the number treated separately from enrollment or explain the handling of missing outcome information.
Other relevant details are not reported in the registry record, including a sample-size justification, statistical analysis plan, interim analyses, multiplicity methods, and time-adjusted event analyses. The length of observation could vary because the endpoint window was tied to each participant’s last dose or discontinuation visit, but no adjustment for differing follow-up is reported. These limitations constrain both precision and the scope of conclusions that can be drawn from the registry results.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study to Evaluate Long-term Safety of CC-486 (Oral Azacitidine) in Subjects With Hematological Disorders — NCT02494258.
- Registry record last updated 2026-04-02; status last verified 2026-03.
- Results first posted 2026-04-02.
- 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
NCT02494258 is a completed phase 2, open-label, single-arm rollover study sponsored by Celgene. It evaluated long-term safety of oral azacitidine (CC-486) in eligible participants from other Celgene-sponsored CC-486 trials for hematological disorders. The registry lists enrollment of 5 and four dosing groups: 200 mg QD x 14 Days, 600 mg QD x 7 Days, 500 mg QD x 7 Days, and 200 mg QD x 7 Days.
The primary outcome was the number of participants with treatment-emergent adverse events among all treated participants. A secondary outcome was the number of participants who survived. Group-level serious and other adverse-event counts and zero deaths in each group are listed, but a separate overall primary-endpoint count is not reported in the registry record. No analyses are listed. The results are descriptive and do not provide a controlled comparison or establish causation.