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 Safety and Efficacy Study of ALT-801 to Treat Progressive Metastatic Malignancies (NCT00496860), a na phase 1 study. The primary outcome is The Safety and Toxicity of ALT-801 in Patients With Progressive Metastatic Malignancies.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT00496860 |
| Phase | Phase 1 |
| Condition | Progressive Metastatic Malignancies |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 26 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Altor BioScience |
| Results posted | Yes |
2. Clinical Question
Population
The study enrolled patients with previously treated, progressive metastatic malignancies. The registry record reports enrollment of 26 participants. It does not describe the specific cancer types, eligibility criteria, baseline characteristics, or the number of participants included in each analysis population.
Intervention
The intervention was ALT-801, a recombinant fusion protein with an interleukin-2 component. The registry description says its targeting mechanism recognizes tumor cells with a specific tumor marker. The study evaluated ALT-801 through dose escalation, with the posted safety groups listed at 0.015 mg/kg/Dose, 0.040 mg/kg/Dose, and 0.080 mg/kg/Dose.
Comparator
There was no randomized or like-for-like control group. The study is recorded as single-group, open-label, and non-randomized. The dose groups represent exposure to ALT-801 at different doses; the registry record does not establish them as comparable treatment and control arms.
Primary question
The primary question was about the safety and toxicity of ALT-801, with the primary outcome described as the number of serious adverse events per cohort over 18 months. The study also sought to determine the maximum-tolerated dose and characterize ALT-801 pharmacokinetics. Clinical antitumor response, cell-mediated immune responses, and immunogenicity were listed as secondary outcomes. Their results are not reported in the registry record.
3. Trial Design
This was a Phase 1, open-label, non-randomized, multi-center, single-group dose-escalation study of ALT-801. The registry record lists enrollment of 26 and a completed status. The recorded start was 2007-05, and primary completion was 2009-10. The stated study purpose was to evaluate safety, determine the maximum-tolerated dose, and characterize the pharmacokinetic profile of ALT-801 in previously treated patients with progressive metastatic malignancies.
Allocation is listed as not applicable, and masking as none. Those design features matter when interpreting the dose groups: participants were not assigned through a randomized comparison with a control treatment, and treatment was not masked. The posted groups contain ALT-801 doses of 0.015 mg/kg/Dose, 0.040 mg/kg/Dose, and 0.080 mg/kg/Dose. Differences among these groups cannot be read as if they came from balanced randomized arms. The registry record does not report how participants were assigned to dose cohorts, the dose-escalation rules, or cohort-specific enrollment decisions.
The study is not a test of ALT-801 against another treatment or against placebo. Its primary focus was safety and toxicity, while antitumor response and immune outcomes were listed as secondary measures. The registry record does not report a statistical analysis plan, analysis populations, or results for those secondary outcomes. Although the study is marked completed, that status alone does not supply missing outcome details or explain how the study team reached dose-escalation decisions.
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 | ALT-801 0.015 mg/kg/Dose | ALT-801 0.040 mg/kg/ Dose | ALT-801 0.080 mg/kg/Dose |
|---|---|---|---|
| STARTED | 4 | 16 | 6 |
| COMPLETED | 3 | 15 | 6 |
| NOT COMPLETED | 1 | 1 | 0 |
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 | ALT-801 0.015 mg/kg/Dose | ALT-801 0.040 mg/kg/Dose | ALT-801 0.080 mg/kg/Dose | Total |
|---|---|---|---|---|---|
| Participants analysed | 4 | 16 | 6 | 26 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 2 | 15 | 5 | 22 |
| Age, Categorical: >=65 years | Participants | 2 | 1 | 1 | 4 |
| Age Continuous | years | 65 (50 to 79) | 51 (27 to 72) | 58 (53 to 65) | 54 (27 to 79) |
| Sex: Female, Male: Female | Participants | 2 | 6 | 1 | 9 |
| Sex: Female, Male: Male | Participants | 2 | 10 | 5 | 17 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 1 | 0 | 1 |
| Race (NIH/OMB): White | Participants | 4 | 15 | 6 | 25 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as The Safety and Toxicity of ALT-801 in Patients With Progressive Metastatic Malignancies, measured over 18 months.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Events |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is described as the number of serious adverse events per cohort, with events as the unit and an 18-month timeframe. A count endpoint records how many events occurred, rather than directly describing how many participants experienced at least one event. Those are different quantities: a participant can experience more than one event, so an event count should not automatically be interpreted as a count of affected participants.
Serious adverse events are relevant to a Phase 1 dose-escalation study because safety and tolerability are central study questions. Recording events by cohort can help describe the safety experience at the studied doses and inform the study’s stated aim of determining a maximum-tolerated dose. That purpose does not establish that any particular dose was tolerated or that a maximum-tolerated dose was identified; those findings are not reported in the registry record.
The posted group-level safety information includes counts of participants at risk and affected, but the registry record does not provide a cohort-level event count for the primary endpoint as described. Participant counts affected by serious adverse events should therefore not be substituted for the primary endpoint’s number of events. The endpoint’s detailed event definitions and rules for assigning events to cohorts are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted information describes a small Phase 1 dose-escalation study without a randomized control group. The safety groups are listed at 0.015 mg/kg/Dose, 0.040 mg/kg/Dose, and 0.080 mg/kg/Dose, with 4, 16, and 6 participants at risk for serious adverse events, respectively. The corresponding serious-event affected counts are 0, 2, and 4. These are descriptive group figures, not estimates from a randomized treatment comparison. The registry record does not report uncertainty intervals or a formal statistical comparison across doses.
The primary outcome is defined as the number of serious adverse events per cohort over 18 months. The group-level affected counts do not necessarily represent event counts: the registry record does not clarify whether each affected participant had one event or multiple events. The endpoint’s event-count result is not reported in the registry record in a form that establishes how many serious adverse events occurred per cohort. This distinction limits interpretation of the primary endpoint.
The record also lists serious adverse-event terms with percentages: Thrombocytopenia and Cardiac Ischemia/Infarction at 16.7% each; THROMBOSIS and Pulmonary Infiltrates at 16.7% each; and Pleural Effusion and Death-NOS at 6.2% each. The denominator and calculation details for these percentages are not reported in the registry record. The terms should not be taken as dose-specific estimates, nor can their percentages be combined into an overall event rate from the information available.
Clinical antitumor response, ALT-801-induced cell-mediated immune responses, and immunogenicity are listed as secondary outcomes, but their results are not reported in the registry record. No efficacy estimate, treatment-effect comparison, confidence interval, or p-value is reported. The posted information therefore supports a descriptive account of selected safety data, but not a conclusion about comparative efficacy, the precision of a treatment effect, or whether ALT-801 changes clinical outcomes.
The absence of a control group also means that events cannot be distinguished from events that might have occurred without ALT-801 or under another treatment. The dose groups are not a like-for-like control comparison. Cohort sizes differ, and the registry record does not explain the assignment process or provide adjustments for differences among cohorts. These limitations are especially important when reading across dose levels: the group counts alone do not establish a dose-response relationship or a causal effect of dose.
9. Statistical Methods Explained
Statistical methods should be read in light of the study’s purpose and design. The registry record describes an open-label, non-randomized, single-group dose-escalation study. Its primary focus is safety and toxicity, with the primary endpoint described as a number of serious adverse events per cohort over 18 months. The record does not report a statistical analysis plan, inferential analyses, or a formal comparison among the dose cohorts.
What does a count endpoint describe?
A count endpoint records the number of occurrences during a defined period or within a defined group. For an adverse-event endpoint, an event count and a participant count are not interchangeable. One participant may contribute more than one event, depending on the endpoint definition and counting rules. The registry record describes the primary outcome as an event count but posts group information in terms of participants at risk and affected. The event-count results and the rules for counting repeated events are not reported in the registry record.
Counts can be analyzed with statistical methods designed for count data. The choice depends on features such as the distribution of counts, the amount of variation, whether repeated events are possible, and whether participants have different observation times. Over-dispersion is a general statistical issue in which observed count variability exceeds what a simpler count model assumes; ignoring it can make uncertainty estimates too optimistic. The registry record does not report whether count models were used, whether over-dispersion was assessed, or how exposure time was handled.
Why does the design limit comparisons?
Randomization can help balance known and unknown participant characteristics between treatment groups. This study is recorded as non-randomized and single-group, with dose cohorts rather than a randomized control arm. As a general principle, differences between non-randomized cohorts may reflect differences in participants, treatment history, timing, or other factors as well as dose. The registry record does not report methods for adjusting for such differences. The posted cohort counts are therefore descriptive and do not establish that dose caused any difference in safety outcomes.
Masking is also absent. In general, open-label knowledge of treatment can affect reporting, assessment, or management of outcomes, depending on the endpoint and study procedures. The registry record does not describe procedures intended to reduce these possibilities. It also does not report how adverse events were collected, classified, or adjudicated.
What would uncertainty estimates add?
A confidence interval is a general statistical way to express the uncertainty around an estimate. A narrow interval indicates greater precision under the method’s assumptions; a wide interval indicates less precision. No confidence intervals are reported for the safety groups or for a treatment effect in the registry record. Without them, the precision of the posted estimates cannot be evaluated from the registry information.
A p-value is a general measure used in some statistical tests to assess how compatible observed data are with a specified null model. It does not by itself measure the size or clinical importance of an effect. The registry record reports no p-values and no formal hypothesis tests for the posted dose groups. It would be inappropriate to infer statistical significance from the group counts or adverse-event percentages alone.
How should planning calculations be understood?
The registry’s planning assumptions describe an illustration based on an observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. The stated effect to detect is a fraction of the observed effect. For time-to-event calculations, the assumption specifies a total number of events using the Schoenfeld approach; other calculations are expressed as participants per arm. These are planning assumptions, not evidence that the study used that design or analysis. In particular, this trial has no randomized two-arm comparison, and an observed treatment-effect estimate is not reported in the registry record. Any resulting planning illustration should not be interpreted as observed power or as a result of the trial.
Sample-size planning is generally a prospective exercise: it asks how much information might be needed to detect an effect under stated assumptions. Observed power, by contrast, is not a substitute for reporting an estimate and its uncertainty. The registry record does not report the original sample-size justification, a planned power target, or a protocol-based sample-size calculation. No conclusion about the adequacy of enrollment for a particular statistical objective follows from enrollment alone.
What can be concluded from the posted analyses?
The record lists zero analyses. It does not report analytic methods for the primary endpoint, handling of repeated events, missing outcomes, analysis populations, or any adjustments for multiple outcomes. The absence of those details means readers cannot reconstruct an inferential analysis from the registry entry. The most defensible reading is descriptive: the record lists cohort sizes, affected-participant counts, and selected adverse-event terms, while leaving important definitions and analysis details unreported.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists a primary safety outcome and secondary outcomes covering clinical antitumor response, ALT-801-induced cell-mediated immune responses, and immunogenicity. It also records five outcome measures, but does not report an analysis plan explaining which outcomes were tested, in what order, or whether any adjustment for multiple testing was planned. Multiplicity matters in general because examining many outcomes or contrasts can increase the chance of at least one apparently unusual result. Without a stated testing strategy and results, no multiplicity-adjusted interpretation can be made.
Missing-data methods are not reported in the registry record. It does not state whether outcome data were incomplete, how incomplete observation was handled, or whether participants who discontinued treatment remained in any analysis. The analysis populations are also not reported. The enrollment count alone does not establish how many participants contributed to each outcome or how follow-up was handled.
The record lists zero analyses, and it does not provide a statistical analysis plan, confidence intervals, or p-values. It is therefore not possible to determine whether analyses were prespecified, how endpoint definitions were applied, or how any missing observations affected estimates. These omissions are relevant to interpretation; they do not establish that data were missing or that a particular method was used.
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 |
|---|---|---|---|
| ALT-801 0.015 mg/kg/Dose | 0 | 4 | 0.0% |
| ALT-801 0.040 mg/kg/Dose | 2 | 16 | 12.5% |
| ALT-801 0.080 mg/kg/Dose | 4 | 6 | 66.7% |
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 |
|---|---|
| ALT-801 0.015 mg/kg/Dose | 4 of 4 |
| ALT-801 0.040 mg/kg/Dose | 16 of 16 |
| ALT-801 0.080 mg/kg/Dose | 6 of 6 |
Most frequent serious adverse event terms
| Event term | Organ system | ALT-801 0.015 mg/kg/Dose | ALT-801 0.040 mg/kg/Dose | ALT-801 0.080 mg/kg/Dose |
|---|---|---|---|---|
| Pleural Effusion | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 1 of 16 | 0 of 6 |
| Death-NOS | General disorders | 0 of 4 | 1 of 16 | 0 of 6 |
| Thrombocytopenia | Blood and lymphatic system disorders | 0 of 4 | 0 of 16 | 1 of 6 |
| Cardiac Ischemia/Infarction | Cardiac disorders | 0 of 4 | 0 of 16 | 1 of 6 |
| THROMBOSIS | Vascular disorders | 0 of 4 | 0 of 16 | 1 of 6 |
| Pulmonary Infiltrates | Respiratory, thoracic and mediastinal disorders | 0 of 4 | 0 of 16 | 1 of 6 |
Most frequent non-serious adverse event terms
| Event term | Organ system | ALT-801 0.015 mg/kg/Dose | ALT-801 0.040 mg/kg/Dose | ALT-801 0.080 mg/kg/Dose |
|---|---|---|---|---|
| Fever | General disorders | 4 of 4 | 16 of 16 | 6 of 6 |
| Hypoalbuminemia | Investigations | 3 of 4 | 16 of 16 | 6 of 6 |
| Rigors/chills | General disorders | 2 of 4 | 14 of 16 | 6 of 6 |
| Hypophosphatemia | Investigations | 2 of 4 | 15 of 16 | 4 of 6 |
| Hypocalcemia | Investigations | 3 of 4 | 12 of 16 | 5 of 6 |
| Hyponatremia | Investigations | 2 of 4 | 13 of 16 | 4 of 6 |
| Hemoglobin | Blood and lymphatic system disorders | 2 of 4 | 12 of 16 | 3 of 6 |
| Platelets | Blood and lymphatic system disorders | 3 of 4 | 10 of 16 | 4 of 6 |
| Hypotension | Cardiac disorders | 2 of 4 | 9 of 16 | 5 of 6 |
| Nausea | Gastrointestinal disorders | 1 of 4 | 10 of 16 | 3 of 6 |
Serious events with the highest arm-level rate: thrombocytopenia (16.7%), cardiac ischemia/infarction (16.7%), thrombosis (16.7%), pulmonary infiltrates (16.7%), pleural effusion (6.2%).
The serious adverse-event group information lists 4 participants at risk and 0 affected at 0.015 mg/kg/Dose; 16 at risk and 2 affected at 0.040 mg/kg/Dose; and 6 at risk and 4 affected at 0.080 mg/kg/Dose. These figures describe the posted group counts. They should not be read as numbers of serious adverse events, because the registry record does not clarify whether affected participants experienced more than one event.
The separate list of serious adverse-event terms includes Thrombocytopenia and Cardiac Ischemia/Infarction at 16.7% each, THROMBOSIS and Pulmonary Infiltrates at 16.7% each, and Pleural Effusion and Death-NOS at 6.2% each. The denominators and relationship between these term percentages and the dose groups are not reported in the registry record. The percentages cannot be used to infer dose-specific incidence or an overall serious-event rate.
The group-level death field is not populated, so death counts by dose are not reported in the registry record. The Death-NOS term in the adverse-event list should not be treated as a complete account of deaths or reconciled with group-level counts without further information. The record also does not provide event timing, duration, outcome, or attribution to ALT-801.
12. What the Result Does, and Does Not, Mean
The registry record describes a completed Phase 1 study that evaluated ALT-801 in patients with progressive metastatic malignancies. The posted safety information gives participant counts at risk and affected in three dose groups, along with selected serious adverse-event terms and percentages. Those data provide a limited descriptive view of safety observations recorded in the registry.
They do not establish that ALT-801 caused a particular event, that a dose caused a difference between cohorts, or that any dose was safe or unsafe. A randomized control group is absent, and the dose cohorts are not a like-for-like comparison. The record does not report event attribution, detailed event definitions, or cohort-level counts of serious adverse events for the primary outcome as described. Nor does it report uncertainty estimates that would show the precision of the posted counts or percentages.
The study’s stated aims also included determining the maximum-tolerated dose and characterizing pharmacokinetics. Whether those aims were achieved is not reported in the registry record. The listed secondary outcomes include antitumor response and immune-response measures, but their results are not reported. The record therefore does not support conclusions about an antitumor effect, immune response, or comparative benefit.
These limits are not evidence that an outcome did or did not occur. They mark what can be established from the registry entry: a study design, dose groups, selected safety figures, and a set of outcomes, with substantial analytic and outcome details not reported. A reader should distinguish those descriptive observations from causal or efficacy claims, which require information not present in the registry record.
13. Why This Trial Matters Statistically
This study illustrates why design and endpoint definitions matter when reading early-phase clinical research. Its stated aims center on safety, dose escalation, maximum-tolerated dose, and pharmacokinetics. A safety-focused study asks different questions from a randomized comparative trial designed to estimate a treatment effect. The registry record identifies the study as open-label, non-randomized, and single-group, so its dose cohorts should not be interpreted as a controlled comparison.
It also highlights a basic measurement distinction: the primary outcome is described as a number of serious adverse events, while the posted group data include participants affected. Event counts and affected-participant counts answer different questions. The record does not report enough detail to connect those group figures to the primary event-count endpoint. Recognizing that distinction prevents a descriptive participant count from being mistaken for the endpoint’s event count.
Finally, the entry shows how missing analysis details affect statistical interpretation. Without reported analysis methods, uncertainty estimates, analysis populations, missing-data procedures, or results for secondary outcomes, the extent and precision of the evidence cannot be assessed from the registry record. The study remains informative as a record of its stated safety objectives and selected posted observations, but those observations do not substitute for complete outcome reporting or a controlled estimate of treatment effect.
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 detailed eligibility criteria, participant characteristics, cancer types, prior treatments, or baseline safety status. Without those details, it is difficult to understand how representative the enrolled group was or to assess whether the dose cohorts differed in important ways. The dose-escalation rules, cohort assignment process, and reasons for cohort sizes are also not reported.
Although the primary endpoint is described as the number of serious adverse events per cohort over 18 months, cohort-level event counts and detailed counting rules are not reported. The posted affected-participant counts cannot clarify whether participants experienced repeated events. The record does not report event dates, duration, severity grading, outcome, or investigator attribution. It also does not specify the denominator used for the listed serious-event percentages or connect those terms to specific dose groups.
The record does not report participant flow, follow-up completeness, discontinuations, analysis populations, or missing-data handling. Results for pharmacokinetics, maximum-tolerated dose, clinical antitumor response, cell-mediated immune responses, and immunogenicity are not reported in the registry record. There are no reported confidence intervals, p-values, or formal dose comparisons. The group-level death field is not populated, and the registry entry does not explain the Death-NOS term in relation to death counts.
These limitations prevent a detailed evaluation of outcome precision, dose-related patterns, and the completeness of safety reporting. They do not establish that unreported events or analyses were absent; they indicate that the registry record does not document them.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety and Efficacy Study of ALT-801 to Treat Progressive Metastatic Malignancies — NCT00496860.
- Registry record last updated 2013-07-22; status last verified 2013-07.
- Results first posted 2013-07-15.
- 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 record describes a completed Phase 1, open-label, non-randomized, multi-center, dose-escalation study of ALT-801 in previously treated patients with progressive metastatic malignancies. Enrollment was 26. The primary outcome concerned the number of serious adverse events per cohort over 18 months. The record lists three dose groups and selected serious adverse-event counts and terms.
There was no randomized control group, and the dose cohorts are not a like-for-like comparison. The record does not report the primary endpoint’s cohort-level event counts, analysis methods, uncertainty estimates, analysis populations, missing-data procedures, or results for the listed secondary outcomes. Consequently, the posted data support only a limited descriptive account and do not establish a causal dose effect or an efficacy result.