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 of ALT-801 in Patients With Bacillus Calmette-Guerin (BCG) Failure Non-Muscle Invasive Bladder Cancer (NCT01625260), a non-randomized phase 1, phase 2 study. The primary outcome is Safety Profile.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01625260 |
| Phase | Phase 1, Phase 2 |
| Condition | Non-muscle Invasive Bladder Cancer |
| Allocation | Non-Randomized |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 12 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Altor BioScience |
| Results posted | Yes |
2. Clinical Question
Population
Patients with BCG-failure non-muscle invasive bladder cancer who refused or were not medically fit to undergo radical cystectomy recommended by their participating urologist. BCG failure was defined as refractory, relapsing, or intolerant disease.
Intervention
ALT-801 combined with gemcitabine. The registry lists ALT-801 doses of 0.06 mg/kg and 0.08 mg/kg, each combined with gemcitabine at 1000 mg/m^2.
Comparator
There was no randomized control group. The record lists the two ALT-801 dose combinations, but does not describe them as a randomized, like-for-like comparison.
Primary question
What dose-limiting toxicities were observed with ALT-801 plus gemcitabine during the 12-week primary-outcome timeframe, as part of assessing safety and tolerability?
The study also aimed to determine a recommended dose and characterize the immunogenicity of the combination. Anti-tumor responses were to be assessed, and the registry lists duration of response, progression-free survival, event-free survival, and overall survival as secondary outcomes. These aims describe what the study set out to examine; they do not establish that results for those outcomes are available.
The record does not provide a concurrent comparator treatment or a randomized assignment process. Consequently, the central question is descriptive: what safety and other outcomes were recorded among patients receiving the combination at the dose levels studied? Any apparent difference between dose groups would need cautious interpretation because the groups were not randomized. The registry record does not report the primary dose-limiting toxicity result or results for the listed secondary outcomes.
3. Trial Design
This was a Phase Ib/II, open-label, multi-center study with competitive enrollment. The registry lists a single-group model, non-randomized allocation, and no masking. Enrollment is recorded as 12, and study status is terminated. The record lists a start date of 2012-03-30 and a primary completion date of 2018-03-08.
The study population was patients with BCG-failure non-muscle invasive bladder cancer. The brief description specifies refractory, relapsing, or intolerant BCG failure and says participants refused or were not medically fit to undergo radical cystectomy recommended by the participating urologist as the standard next therapy per urologic guidelines. The stated objectives included confirming safety and tolerability, determining a recommended dose, characterizing immunogenicity, and assessing anti-tumor responses.
The intervention was ALT-801 combined with gemcitabine. The registry lists an ALT-801 dose of 0.06 mg/kg with gemcitabine at 1000 mg/m^2 and an ALT-801 dose of 0.08 mg/kg with gemcitabine at 1000 mg/m^2. These are dose groups within a non-randomized study, not randomized treatment arms. The registry record does not describe a separate control regimen.
The primary outcome is titled Safety Profile and measures the number of dose-limiting toxicities over 12 weeks. Six outcome measures are listed, including the primary measure and secondary outcomes. However, the record does not report the primary outcome result, and it lists no outcome analyses. The record also does not report the protocol for assigning participants to the dose levels, the planned sample-size justification, or further details of the statistical analysis plan. The study's terminated status should be distinguished from whether any particular outcome was favorable or unfavorable: 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.
Before assignment: Only Phase 1B was enrolled, no subjects were enrolled in Phase 2.
Overall Study
| Stage | 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine |
|---|---|---|
| STARTED | 9 | 3 |
| COMPLETED | 2 | 2 |
| NOT COMPLETED | 7 | 1 |
Reasons for not completing this period:
| Reason | 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine |
|---|---|---|
| Adverse Event | 3 | 0 |
| Study Terminated by Investigator | 2 | 0 |
| Disease Progression | 2 | 0 |
| PI and subject decided to terminate treatment | 0 | 1 |
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 | 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | Total |
|---|---|---|---|---|
| Participants analysed | 9 | 3 | 12 | |
| Age, Continuous | years | 63.1 (SD 11.36) | 71.0 (SD 4.36) | 65.1 (SD 10.49) |
| Sex: Female, Male: Female | Participants | 2 | 1 | 3 |
| Sex: Female, Male: Male | Participants | 7 | 2 | 9 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): White | Participants | 8 | 3 | 11 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Patients with Bacillus Calmette-Guerin (BCG) Failure Non-Muscle Invasive Bladder Cancer | Participants | 9 | 3 | 12 |
6. Primary Endpoint
The registry lists the primary outcome as Safety Profile, measured over 12 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Number of DLTs |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint is a count: the number of dose-limiting toxicities associated with ALT-801 combined with gemcitabine during a 12-week timeframe. The registry describes it as a safety and tolerability measure. A toxicity count is intended to record events meeting the study's dose-limiting toxicity definition; that definition and the rules for attributing an event to treatment are not reported in the registry record.
Counts can be useful in early-phase dose evaluation because the objective includes characterizing tolerability and determining a recommended dose. In general, the count provides a direct summary of how many qualifying toxicities were recorded, rather than estimating a comparative treatment effect against a control group. Interpretation depends on how toxicity was defined, how long participants were observed, how missing observations were handled, and whether the count refers to events or participants. Those operational details are not reported in the registry record.
The endpoint's timeframe is 12 weeks, but a timeframe alone does not show whether every participant completed that period or whether follow-up differed by participant. The registry record does not report the dose-limiting toxicity count, a rate, an uncertainty interval, or an analysis comparing dose groups. Thus, the endpoint definition identifies what the trial intended to measure, while the posted record does not establish the observed primary result.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record identifies a safety-focused primary outcome, but does not report its observed dose-limiting toxicity count. It also lists no outcome analyses. That means the primary result cannot be interpreted from the registry record as posted: there is no reported estimate of the number of dose-limiting toxicities, no uncertainty measure, and no statistical comparison. Absence of a reported result is not evidence that the count was zero.
Some safety information is recorded by dose group. In the 0.06 mg/kg ALT-801 with 1000 mg/m^2 gemcitabine group, 9 participants were at risk for serious adverse events and 3 were affected; in the 0.08 mg/kg ALT-801 with 1000 mg/m^2 gemcitabine group, 3 were at risk and 1 was affected. These serious adverse event figures are not a substitute for the primary dose-limiting toxicity outcome. The registry does not state that the serious adverse events met the protocol definition of dose-limiting toxicity.
Other adverse events are also counted in the registry: 9 of 9 at-risk participants in the lower ALT-801 dose group and 3 of 3 in the higher dose group were affected. The record lists deaths as 2 and 1 in those respective groups. It does not say whether deaths were caused by study treatment, the disease, or other factors, nor does it provide timing or cause. These counts should therefore be read as reported safety information, not as evidence about causation.
The study was non-randomized and had no masking. The dose groups are not a like-for-like control comparison, and the record does not provide details about how participants entered each dose group. Even a difference in recorded events between these groups would not, by itself, establish that dose caused the difference. Small group counts also make estimates potentially imprecise; the registry does not provide confidence intervals or another quantified measure of uncertainty for these figures.
The listed secondary outcomes include response duration, progression-free survival, event-free survival, and overall survival. Their results are not reported in the registry record. The listed aims concerning recommended dose, immunogenicity, and anti-tumor response likewise should not be mistaken for findings. Overall, the registry provides design information and selected adverse-event counts, but not enough outcome results to assess the primary endpoint or draw comparative conclusions.
9. Statistical Methods Explained
In general, an early-phase study can focus on safety, tolerability, and dose selection rather than estimating a definitive comparative treatment effect. Here, the stated primary measure is a dose-limiting toxicity count over 12 weeks, and the study objectives include identifying a recommended dose. The record does not provide the statistical methods used to define, summarize, or compare dose-limiting toxicities. It does not report a planned hypothesis test or an estimate of uncertainty for the primary outcome.
What does a toxicity count represent?
In general, a count records how many events satisfy a specified definition during an observation period. Interpretation depends on whether the unit is an event or an affected participant: one participant might experience more than one event, depending on the endpoint rules. The primary outcome is described as a number of dose-limiting toxicities, but the registry record does not clarify the counting unit, the toxicity grading criteria, or whether repeat events in one participant could contribute more than once.
A count can also depend on how much observation each participant contributes. In general, if follow-up differs, raw counts may not be directly comparable without considering observation time or a prespecified approach to incomplete follow-up. The 12-week timeframe is stated, but completion of that observation period and the handling of early discontinuation or missing assessments are not reported in the registry record.
Why is the study structure important?
In general, random assignment helps balance measured and unmeasured participant characteristics across treatment groups. This study is recorded as non-randomized, with no masking and a single-group model. The two listed dose combinations therefore do not provide a randomized control comparison. Without reported details on dose assignment, differences between the groups could reflect participant selection, timing, follow-up, or other factors as well as dose. The registry does not report an adjustment strategy to address such differences.
In general, masking can reduce the potential for expectations to affect some assessments or reporting. Since masking is listed as none, the record does not describe a masked assessment process. This does not establish that bias occurred, but it is relevant context for interpreting outcomes, especially those involving clinical judgment. The registry record does not provide further information about outcome adjudication or blinded review.
What does a confidence interval add?
In general, a confidence interval expresses the statistical uncertainty around an estimate under a specified analysis. It helps readers distinguish a precise estimate from one that is compatible with a broad range of values. No confidence interval for the primary endpoint or the adverse-event counts is reported in the registry record. Without such an interval, and without the primary count itself, the precision of the primary result cannot be assessed from this record.
In general, a p-value addresses how compatible data are with a specified null model under the assumptions of a statistical test. A p-value is not a measure of clinical importance, and it does not repair limitations in design or missing outcome reporting. The registry record lists no outcome analyses, so no trial p-value or inferential comparison should be inferred from the descriptive counts.
How should planning calculations be understood?
In general, a sample-size illustration based on an observed effect describes a hypothetical future study under chosen assumptions. It is not the trial's observed power, and it does not establish that this study was designed around that effect. The registry record does not report the trial's sample-size justification or an outcome-based planning rationale. Any planning illustration should be kept separate from observed outcomes and interpreted only under its stated assumptions; the assumptions do not supply missing primary results.
In general, sparse counts can yield substantial uncertainty, and standard approximations may be less dependable when event information is limited. A planning calculation using a normal approximation is an assumption for that calculation, not evidence that the underlying outcome distribution or the trial's analysis followed that method. The registry does not report a statistical model for the primary count or explain whether over-dispersion was considered. Over-dispersion, in general, occurs when variability in counts exceeds what a simple count model assumes; if present, it can make conventional standard errors too small unless the analysis accounts for it. There is no basis in the record to say whether it was present or how it was handled.
The registry lists four time-to-event secondary outcomes, but does not report their estimates, event counts, censoring rules, or analyses. In general, time-to-event methods account for the time until an event and can include participants whose event has not been observed by the end of follow-up. Their interpretation depends on definitions of the event, follow-up, censoring, and analysis populations. Those details are not reported in the registry record. Accordingly, no survival estimate, hazard comparison, or precision claim can be recovered from the listed outcome names alone.
Overall, the study's statistical design and outcome definitions are only partly described in the registry record. The available information supports describing the study structure and the adverse-event counts, but not reconstructing a full analysis. The primary endpoint result, analysis populations, handling of missing data, model choices, and inferential results are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists six outcome measures, including a primary safety endpoint and secondary outcomes of duration of response, progression-free survival, event-free survival, and overall survival. When a study examines multiple outcomes, the chance of finding an apparently notable result among them can increase. In general, multiplicity procedures may specify a hierarchy, adjust testing thresholds, or identify outcomes as exploratory. The registry record does not report a multiplicity strategy, a testing hierarchy, or whether any secondary analyses were exploratory.
The primary endpoint is identified as the number of dose-limiting toxicities over 12 weeks, but no primary result or analysis is listed. The record does not state how participants with incomplete observation were included, whether missing values were imputed, or whether participants who discontinued treatment remained in an analysis. The rules used to define the safety analysis population and any other analysis populations are not reported in the registry record.
In general, missingness can affect interpretation when the reason data are unavailable is related to health status, treatment experience, or the outcome itself. Missing observations are not necessarily evidence of an event or of its absence. Because the registry does not describe missing-data methods or follow-up completeness, the effect of missing data on the safety summary and secondary outcomes cannot be assessed.
The registry also does not report the statistical analysis plan, any interim analyses, or stopping rules. These details should not be inferred from the phase, the study's terminated status, or the listed objectives. The reason for termination is not reported in the registry record. Without those methods and outcome data, readers cannot determine whether outcomes were analyzed according to a prespecified plan or how multiple comparisons and incomplete observations were handled.
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 |
|---|---|---|---|
| 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 3 | 9 | 33.3% |
| 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 1 | 3 | 33.3% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 2 of 9 |
| 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 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 |
|---|---|
| 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 9 of 9 |
| 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 3 of 3 |
Most frequent serious adverse event terms
| Event term | Organ system | 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine |
|---|---|---|---|
| Hypotension | Vascular disorders | 1 of 9 | 1 of 3 |
| Skin Infection | Infections and infestations | 1 of 9 | 0 of 3 |
| Soft Tissue Infection | Infections and infestations | 0 of 9 | 1 of 3 |
| Anaphylaxis | Immune system disorders | 1 of 9 | 0 of 3 |
Most frequent non-serious adverse event terms
| Event term | Organ system | 0.06 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine | 0.08 mg/kg ALT-801 With 1000 mg/m^2 Gemcitabine |
|---|---|---|---|
| Chills | General disorders | 7 of 9 | 2 of 3 |
| Fatigue | General disorders | 6 of 9 | 2 of 3 |
| Edema limbs | General disorders | 4 of 9 | 2 of 3 |
| Pruritus | Skin and subcutaneous tissue disorders | 3 of 9 | 3 of 3 |
| Fever | General disorders | 3 of 9 | 2 of 3 |
| Nausea | Gastrointestinal disorders | 4 of 9 | 1 of 3 |
| Alanine aminotransferase increased | Investigations | 4 of 9 | 1 of 3 |
| Anorexia | Metabolism and nutrition disorders | 2 of 9 | 3 of 3 |
| Hypotension | Vascular disorders | 3 of 9 | 2 of 3 |
| Aspartate aminotransferase increased | Investigations | 3 of 9 | 1 of 3 |
Serious events with the highest arm-level rate: soft tissue infection (33.3%), hypotension (33.3%), skin infection (11.1%), anaphylaxis (11.1%).
The registry reports serious adverse events by dose group. For 0.06 mg/kg ALT-801 with 1000 mg/m^2 gemcitabine, 9 participants were at risk and 3 were affected. For 0.08 mg/kg ALT-801 with 1000 mg/m^2 gemcitabine, 3 were at risk and 1 was affected. These are group-level counts; they do not establish that an event was caused by ALT-801 or gemcitabine.
The record lists soft tissue infection and hypotension at 33.3% each, and skin infection and anaphylaxis at 11.1% each, among its top serious terms. The registry does not clarify the denominator or whether participants could contribute to more than one term. The percentages therefore should not be interpreted as mutually exclusive categories or used to infer an overall serious-event rate.
The listed deaths are 2 in the lower-dose group and 1 in the higher-dose group. The registry record does not report their causes, timing, relationship to treatment, or whether they were included in the serious-event counts. The number of deaths does not by itself establish treatment-related mortality. These summaries also do not replace the primary dose-limiting toxicity result, which is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The record establishes the study's aims and design: an open-label, non-randomized Phase Ib/II evaluation of ALT-801 with gemcitabine in patients with BCG-failure non-muscle invasive bladder cancer. It identifies two ALT-801 dose combinations and a primary safety endpoint defined as a dose-limiting toxicity count over 12 weeks. It also reports selected adverse-event counts by dose group.
It does not report the result for the primary endpoint. The absence of that result means the registry record cannot establish how many dose-limiting toxicities occurred under the study's definition or how the result related to dose selection. Nor does the record report results for the listed secondary outcomes. Study objectives, such as assessing anti-tumor responses or determining a recommended dose, are not themselves evidence that a particular response or dose recommendation was observed.
The serious adverse-event and death counts are descriptive. They cannot show that the combination caused an event or death, and the registry does not give the event-level details needed to assess attribution, timing, or overlap. Likewise, these counts do not establish a difference between dose levels. The groups were not randomized and are not a like-for-like control comparison.
The study's terminated status is a registry status, not an outcome finding. The reason for termination is not reported in the registry record. The record does not provide enough information about dose assignment, follow-up, missing observations, endpoint definitions, or analysis methods to resolve these uncertainties.
In general, a trial can contribute useful descriptive safety information even when it cannot support a controlled treatment-effect estimate. For this record, however, the available information is limited: selected adverse-event counts are posted, while the defined primary result and secondary outcome analyses are not. Conclusions should remain confined to those recorded details rather than extending to efficacy, comparative safety, or clinical use.
13. Why This Trial Matters Statistically
This trial is statistically notable for the gap between a clearly stated safety objective and the absence of a reported primary outcome value. The registry identifies dose-limiting toxicity count over 12 weeks as the primary measure, but gives no count or analysis. That distinction matters: a well-specified endpoint tells readers what investigators intended to measure, while a result is needed to assess what was observed.
The design also illustrates why dose groups should not automatically be read as a controlled comparison. The record lists two dose combinations, but allocation was non-randomized and the study had no masking. In general, treatment-group comparisons are strongest when assignment supports comparability and outcome assessment is protected from expectations. Without those safeguards, group differences may have explanations other than dose. The registry does not report methods that would address these concerns.
Another statistical issue is the interpretation of sparse safety data. Counts of participants affected by serious adverse events and deaths are useful descriptive information, but they do not alone quantify uncertainty, establish causation, or answer the primary dose-limiting toxicity question. The lack of reported precision measures and event details limits what can be concluded from the counts.
Finally, the record lists multiple secondary outcomes but provides no analyses for them. In general, interpreting several outcomes requires attention to prespecification, multiplicity, missingness, and analysis populations. Those details are not reported. The trial therefore offers a useful example of how registry design fields and posted safety summaries can inform statistical reading, while also showing why endpoint results and analysis details are essential to interpreting a study.
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 does not report the primary dose-limiting toxicity count, despite identifying that measure as the primary outcome. It also provides no estimates or uncertainty intervals for the listed secondary outcomes. This prevents assessment of the primary safety result and of the stated anti-tumor and time-to-event objectives.
The study is non-randomized, open-label, and recorded as a single-group model. Although two dose combinations are listed, the record does not provide a randomized comparison or explain how participants were assigned to those dose levels. Consequently, the groups should not be treated as exchangeable controls. The registry does not report adjustment methods that might address differences between them.
The registry does not report the operational definition of dose-limiting toxicity, event attribution rules, assessment procedures, or completeness of follow-up over the 12-week timeframe. Analysis populations, missing-data methods, and procedures for handling multiple outcomes are also not reported. These omissions limit reproducibility and make it difficult to assess possible biases in the observed summaries.
Safety information is limited to group counts and selected serious-event terms. The record does not clarify the denominator for the listed term percentages, whether event categories overlap, or the causes and treatment relationships of deaths. The reason for the terminated status is not reported. The registry also does not report the sample-size justification, interim analyses, or a full statistical plan. Together, these limitations restrict interpretation to the design description and the posted adverse-event summaries, without supporting firm comparative or efficacy conclusions.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of ALT-801 in Patients With Bacillus Calmette-Guerin (BCG) Failure Non-Muscle Invasive Bladder Cancer — NCT01625260.
- Registry record last updated 2024-07-19; status last verified 2024-07.
- Results first posted 2024-07-19.
- 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
The registry describes NCT01625260 as a Phase Ib/II, open-label, multi-center, non-randomized study of ALT-801 combined with gemcitabine for BCG-failure non-muscle invasive bladder cancer. The study is listed as terminated, with enrollment of 12. The two recorded dose combinations used ALT-801 at 0.06 mg/kg or 0.08 mg/kg with gemcitabine at 1000 mg/m^2.
The primary outcome was the number of dose-limiting toxicities over 12 weeks. The record lists six outcome measures but no outcome analyses, and the primary result is not reported in the registry record. Selected serious adverse-event and death counts are reported by dose group; they do not establish causation or a randomized dose comparison.
The registry does not report the reason for termination, the primary result, secondary outcome results, the statistical analysis plan, or methods for missing data and multiplicity. Interpretation is therefore limited to the stated design, objectives, and posted safety summaries.