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 NU-0129 in Treating Patients With Recurrent Glioblastoma or Gliosarcoma Undergoing Surgery (NCT03020017), a na early/phase 1 study. The primary outcome is Number of Patients With Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03020017 |
| Phase | EARLY/Phase 1 |
| Condition | Gliosarcoma, Recurrent Glioblastoma |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 8 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Northwestern University |
| Results posted | Yes |
2. Clinical Question
The registry describes a safety-focused study of intravenous NU-0129 in patients with recurrent glioblastoma or gliosarcoma who were undergoing tumor resection. NU-0129 is described as a spherical nucleic acid gold nanoparticle targeting BCL2L12. The study’s rationale is that the nucleic acid component can target that gene in tumor tissue; the registry’s stated purpose is to evaluate safety, not to establish comparative clinical benefit.
Population
Patients with recurrent glioblastoma or gliosarcoma undergoing surgery. The registry lists an enrollment of 8.
Intervention
NU-0129 administered intravenously over 20-50 minutes, followed by standard-of-care tumor resection within 8-48 hours.
Comparator
There is no concurrent comparator group. The registry identifies a single-group model and allocation as not applicable.
Primary question
How many participants experience adverse events after intravenous NU-0129, with events assessed up to 21 days after administration?
These elements define a descriptive, early-phase safety question. In a single-group study, recorded adverse events can be described among participants who received the study drug, but the design does not provide a randomized comparison for estimating what would have occurred without NU-0129. The timing of resection is part of the treatment sequence recorded for the study; it does not create a control condition.
The registry also lists outcomes concerning blood concentration, tumor-tissue biodistribution, feasibility, and blood half-life. Those outcomes address drug disposition and study procedures alongside safety. The record does not report results for those outcomes in the registry record for this analysis, so their findings cannot be characterized.
3. Trial Design
NCT03020017 is a completed early-phase study sponsored by Northwestern University. The registry identifies it as a phase 0, first-in-human study of NU-0129 in recurrent glioblastoma or gliosarcoma. The listed enrollment is 8. The study began on 2017-05-25, reached primary completion on 2018-09-06, and has a completed status in the registry record.
The design is single-group, with allocation recorded as not applicable and masking recorded as none. Participants received NU-0129 by intravenous infusion over 20-50 minutes and underwent standard-of-care tumor resection within 8-48 hours. As there is no concurrent control arm, the study can describe outcomes among those treated, but it cannot estimate a treatment-versus-control contrast from randomized groups. A before-and-after or external-control comparison is not reported in the registry record.
The primary outcome is the number of participants with adverse events, assessed up to 21 days after study drug administration. The registry says events are graded according to the NCI Common Terminology Criteria in Adverse Events version 4.03: Grade 1 is mild, Grade 2 moderate, Grade 3 severe, Grade 4 life-threatening, and Grade 5 fatal. The record also lists five outcome measures overall, including secondary outcomes related to drug concentration in blood, tumor-tissue biodistribution, and feasibility.
The record does not report a control group, a randomization procedure, or masking. It does not report a statistical analysis plan, a sample-size justification, or interim analyses. The absence of those details in the registry record should not be taken as evidence about what investigators may have planned outside the record. Because the study is early phase and single group, its design is primarily suited to documenting observed events and feasibility rather than comparing treatment effects.
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.
NU-0129
| Stage | NU-0129 Treatment |
|---|---|
| STARTED | 8 |
| COMPLETED | 8 |
| NOT COMPLETED | 0 |
Standard of Care Resection
| Stage | NU-0129 Treatment |
|---|---|
| STARTED | 8 |
| COMPLETED | 8 |
| NOT COMPLETED | 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 | NU-0129 Treatment |
|---|---|---|
| Participants analysed | 8 | |
| Age, Continuous | years | 55.5 (28 to 66) |
| Sex: Female, Male: Female | Participants | 4 |
| Sex: Female, Male: Male | Participants | 4 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 8 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 |
| Race (NIH/OMB): Asian | Participants | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 |
| Race (NIH/OMB): Black or African American | Participants | 1 |
| Race (NIH/OMB): White | Participants | 7 |
| Race (NIH/OMB): More than one race | Participants | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 |
| Region of Enrollment: United States | participants | 8 |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Adverse Events, measured over Up to 21 days after study drug administration.
| 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 adverse events, measured up to 21 days after NU-0129 administration. The registry describes safety assessment using event counts and grading under NCI CTCAE version 4.03. That grading distinguishes events by recorded severity, from mild through fatal, and gives a structured way to describe safety observations.
Counting participants with an event is a binary-outcome approach at the participant level: a participant is classified according to whether an adverse event was recorded during the stated assessment window. This differs from counting every event occurrence. One participant may experience more than one event, so participant counts and event counts answer different questions. The registry’s primary outcome title emphasizes the number of patients with adverse events, while its description says the number of adverse events will be assessed. The exact handling of multiple events per participant is not reported in the registry record.
A safety endpoint is relevant in a first-in-human study because it describes harms observed after exposure and helps characterize what was recorded during the specified follow-up. It is descriptive in this single-group design: without a concurrent comparator, an observed event count cannot by itself distinguish effects of the study drug from events associated with the underlying illness, surgery, or other circumstances. The registry record does not report a prespecified threshold for interpreting the count or a comparative safety analysis.
The result should therefore be read as a record of observed safety events during the defined period, not as a causal estimate or a definitive characterization of risk beyond the participants and observation window in this study.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry’s adverse-event figures describe one NU-0129 treatment group. The posted serious-event summary lists 8 at risk and 1 affected; the summary for other adverse events lists 8 at risk and 8 affected. The registry also lists 0 deaths. These are counts in a small, single-group study, and they should be interpreted as descriptive observations rather than comparative estimates.
The serious-event terms list cerebrospinal fluid leakage at 12.5%. This is a reported term and percentage in the registry record. It does not establish that NU-0129 caused the event. The record also does not provide details in the registry entries about event onset, duration, resolution, recurrence, attribution to treatment, or the clinical circumstances around surgery. Those details would be needed for a fuller assessment of individual events.
The distinction between serious and other adverse events matters. A serious adverse event is not simply another way to label a severe grade; seriousness and severity are different classifications. The registry’s listed endpoint uses CTCAE grades, while its safety summary separates serious events from other events. The record does not give a mapping between the serious-event classification and CTCAE grades for the listed cases. It would therefore be inappropriate to infer a grade from the serious-event label.
Because all participants were treated and there is no control group, the study cannot show whether the observed events occurred more often than they would have under another treatment or without NU-0129. The counts also do not establish how frequently similar events would occur in a broader population. In a small study, each observation has substantial influence on the overall descriptive picture, and uncommon events may not be observed even if they are possible.
No confidence interval, hypothesis test, or adjusted effect estimate is reported in the registry record. Without an estimate of uncertainty, the counts should not be read as precise estimates of the underlying event frequency. A confidence interval, in general, expresses the statistical uncertainty around an estimated quantity under a specified model; it does not resolve limitations caused by a single-group design, incomplete follow-up information, or limited enrollment.
The listed 0 deaths is a reported count for the adverse-event summary. It does not establish the absence of serious nonfatal events, nor does it answer questions about safety outside the reported assessment window. The registry identifies the primary safety timeframe as up to 21 days after administration. Longer-term safety findings are not reported in the registry record.
Overall, the posted results document adverse events in the treated group and include a serious-event term. They do not provide a controlled comparison, a causal attribution, or a precise estimate applicable beyond this study. The information is best interpreted as a limited descriptive safety record from an early-phase investigation.
9. Statistical Methods Explained
The registry describes a single-group study with a participant-level adverse-event outcome. The primary endpoint is the number of participants with adverse events during follow-up up to 21 days after study drug administration. This structure calls for descriptive summaries of counts and, when appropriate, proportions. The posted safety summary gives counts at risk and counts affected; no inferential analysis is listed in the registry record.
What does a participant-level count represent?
For a binary endpoint, each participant is classified as having or not having the event of interest within the defined observation period. This can make the result straightforward to describe, but it collapses several possible experiences into one status. A participant with one event and a participant with multiple events can both contribute to the participant count as affected. An event-count analysis would instead count event occurrences and needs rules for repeated events. The registry’s title refers to the number of patients with adverse events, and its description refers to the number of adverse events. How multiple events are counted or grouped is not reported in the registry record.
The distinction between a count and a proportion is also important. A count describes how many participants were recorded as affected. A proportion relates that count to a defined denominator, such as those at risk or assessed. The denominator must be appropriate to the outcome and observation window. The registry provides at-risk and affected counts in the adverse-event summary, but details about participant-level missingness, evaluability rules, or changes to denominators are not reported in the registry record. No new proportions or derived quantities are calculated in this interpretation.
How should uncertainty be considered?
Statistical uncertainty is relevant even when a result is reported as a simple count. In general, an estimate based on a limited group can vary substantially from the value that would be observed in another group of similar patients. A confidence interval is a way to express uncertainty around an estimate under stated assumptions. It does not correct for bias, omitted participants, unclear event definitions, or the lack of a control group. The registry record does not list confidence intervals or another uncertainty analysis for the primary outcome.
For binary outcomes, models may be used to estimate event probabilities or compare groups. Such methods require choices about the outcome definition, analysis population, and handling of incomplete observations. With a single group and no randomized comparator, a model cannot create the missing counterfactual comparison. The record does not report a model, a hypothesis test, or an adjusted estimate for the primary outcome.
Why does the design affect interpretation?
Randomized comparisons, in general, use allocation by chance to create groups intended to be comparable in expectation. This study has no randomized comparator: allocation is listed as not applicable, and the model is single group. Thus, the observed event counts describe the treated group only. They cannot separate events related to treatment from events associated with the underlying disease, tumor resection, or other factors. Nor can they establish how the same participants would have fared under an alternative condition.
Masking is recorded as none. In general, masking can reduce the influence of knowledge about treatment assignment on some assessments, but its role depends on the outcome and study procedures. Here, the record identifies no masking, and it does not describe additional steps to reduce assessment or reporting bias. Adverse events can be influenced by how events are elicited, documented, classified, and attributed; those procedures beyond the stated CTCAE grading are not reported in the registry record.
What does the safety classification add?
The registry names CTCAE version 4.03 and gives the severity scale from Grade 1 through Grade 5. A standardized grading scheme provides shared categories for describing severity. It does not, on its own, establish causality or seriousness. In general, severity describes intensity, whereas seriousness is a regulatory classification based on consequences or criteria. Since the registry’s safety summary separates serious events from other events and does not show a grade-to-seriousness crosswalk for each case, the categories should not be treated as interchangeable.
Over-dispersion is a general issue for event-count outcomes when observed variability exceeds what a simple count model expects. It matters especially when participants can experience repeated events and the analysis counts events rather than people. A participant-level binary outcome avoids treating multiple events as independent observations, but it also loses information about frequency and recurrence. The registry does not report an event-count model or a repeated-event analysis, so over-dispersion is a general methodological consideration, not a finding about this trial.
How should planning illustrations be read?
Sample-size calculations are planning tools that describe the number of participants a future study might require under assumptions about an effect, significance level, allocation, and statistical model. They are not observed results and cannot establish the probability that this completed study detected an effect. The registry record does not report the trial’s own sample-size justification or design assumptions. Any planning illustration based on the observed effect is a retrospective illustration, not the study’s prospective rationale and not observed power. The sponsor’s analysis takes precedence if a recalculated value differs from a sponsor-reported analysis; no sponsor analysis is reported in the registry record for this endpoint.
The record lists no analysis entries for the outcomes in the registry entries. Consequently, the statistical procedures used by the investigators, the handling of missing data, and any prespecified analysis populations are not reported in the registry record. General explanations of methods describe possible statistical considerations; they should not be mistaken for methods documented as having been used in this study.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists one primary safety outcome and several secondary outcomes concerning blood concentration, tumor-tissue biodistribution, feasibility, and blood half-life. When multiple outcomes are analyzed, multiplicity can arise because several statistical questions are examined. In general, testing many hypotheses can increase the chance of at least one apparently notable result under a global null hypothesis. Researchers may address this through a prespecified hierarchy, adjustment, or by treating secondary findings as exploratory. The registry record does not report a multiplicity strategy, a hierarchy for interpreting outcomes, or inferential analyses for the secondary outcomes.
Missing data can affect both denominators and interpretation. For example, an outcome may be unavailable because an assessment was not completed or an event was not recorded. The registry record does not describe missingness, reasons for missing observations, imputation, or sensitivity analyses. It also does not report a defined analysis population, such as an intention-to-treat or safety population, beyond the adverse-event summary’s at-risk counts. Therefore, the posted counts should be read using their stated denominators without assuming how unrecorded observations were handled.
The record lists no analysis entries in the registry entries. The statistical analysis plan, including any methods for multiplicity or missing data, is not reported in the registry record. These omissions limit reconstruction of the analysis and make it impossible to determine whether any planned procedures differed from the descriptive 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 |
|---|---|---|---|
| NU-0129 Treatment | 1 | 8 | 12.5% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| NU-0129 Treatment | 0 of 8 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| NU-0129 Treatment | 8 of 8 |
Most frequent serious adverse event terms
| Event term | Organ system | NU-0129 Treatment |
|---|---|---|
| Cerebrospinal fluid leakage | Nervous system disorders | 1 of 8 |
Most frequent non-serious adverse event terms
| Event term | Organ system | NU-0129 Treatment |
|---|---|---|
| Lymphocyte count decreased | Investigations | 2 of 8 |
| Hypophosphatemia | Metabolism and nutrition disorders | 2 of 8 |
| Alkaline phosphatase increased | Investigations | 1 of 8 |
| Platelet count decreased | Investigations | 1 of 8 |
| White blood cell decreased | Investigations | 1 of 8 |
| Hypertension | Vascular disorders | 1 of 8 |
Serious events with the highest arm-level rate: cerebrospinal fluid leakage (12.5%).
The registry’s adverse-event summary lists 8 at risk and 1 affected for serious adverse events, and 8 at risk and 8 affected for other adverse events. It lists 0 deaths. The serious-event terms include cerebrospinal fluid leakage at 12.5%. These are descriptive safety figures for the NU-0129 treatment group.
The term “serious” should not be read as a severity grade. The registry separately describes CTCAE grades, from Grade 1 (mild) through Grade 5 (fatal), but does not report the grade associated with each serious event in the registry entries. The figures also do not establish that an event was caused by NU-0129. Event attribution, onset, resolution, and relation to tumor resection are not reported in the registry record.
The safety summary’s counts refer to participants at risk and affected, while the term percentage is presented as a reported registry value. No further calculations are needed to interpret these entries, and they should not be extended to populations or follow-up periods beyond those described. The primary outcome’s assessment window is up to 21 days after administration; longer-term safety information is not reported in the registry record.
With no concurrent comparator, these observations cannot establish whether events were more or less frequent than under another condition. They are a record of what was reported for this treated group, not a comparative estimate or a definitive causal assessment.
12. What the Result Does, and Does Not, Mean
The results provide a descriptive account of adverse events reported for participants who received NU-0129 in this early-phase, single-group study. The registry identifies a serious-event count and a count for other adverse events, as well as a serious-event term and a death count. This information is relevant to understanding what was recorded during the study’s stated safety follow-up.
The study does not show what would have happened to a comparable group without NU-0129 or under another intervention. There is no randomized comparator, and the registry does not report a controlled comparison. Consequently, observed events cannot be attributed to the study drug solely on the basis of their occurrence after administration. The study’s surgical context also matters for interpretation, but the registry entries do not specify how individual events were related to surgery or other clinical circumstances.
The results are not a precise estimate of safety in a broader population. The listed enrollment is 8, and a small study provides limited information about uncommon or delayed events. The primary follow-up window is up to 21 days after administration; safety beyond that period is not reported in the registry record. A lack of a reported event in this study would not prove that it cannot occur elsewhere.
The record also does not provide statistical uncertainty intervals, a detailed analysis plan, or information about missing observations. These limitations constrain how much can be inferred from the counts. The secondary outcomes listed in the registry concern blood concentration, tumor-tissue biodistribution, feasibility, and half-life, but their results are not reported in the registry record for this interpretation.
Thus, the study contributes a limited safety description for a first-in-human investigation and documents the outcomes recorded in the treated group. It does not establish a causal treatment effect, comparative safety, efficacy, or long-term risk. Those questions require evidence beyond the descriptive results recorded here, and such evidence is not reported in the registry record.
13. Why This Trial Matters Statistically
Early-phase studies pose distinct statistical challenges because their purpose and design may differ from confirmatory comparative trials. This study is identified as phase 0 and first-in-human, with a single treatment group and a safety-focused primary outcome. The statistical task is therefore chiefly to describe observed events clearly, preserve distinctions between participants and event occurrences, and avoid interpreting counts as comparative effects.
Its outcome definitions illustrate why denominators and classification rules matter. The primary endpoint is framed around participants with adverse events, while the description refers to the number of adverse events. Those quantities can differ when an individual experiences more than one event. The registry gives serious and other adverse-event counts, but the detailed rules for repeated events and analysis populations are not reported in the registry record. Clear definitions are essential for reproducibility and for comparing safety summaries across studies.
The design also demonstrates an important boundary of statistical inference: a model cannot replace an absent control group. Counts among treated participants can describe what was observed, but they do not identify what would have occurred under an alternative condition. This distinction remains important even when results are presented as percentages or subjected to statistical modeling.
Finally, the registry record leaves several analysis details unspecified, including the statistical plan, missing-data handling, multiplicity strategy, and uncertainty estimates. Explicit reporting of these choices helps readers judge how results were produced and how much confidence to place in them. The study is statistically informative as a compact example of descriptive safety reporting, while its design and record limitations prevent broader causal conclusions.
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.
Several limitations are specific to the available record for this study. It is single group, with allocation listed as not applicable and masking listed as none. There is no concurrent control group against which to compare adverse-event counts. The observed safety figures therefore cannot distinguish effects associated with NU-0129 from events associated with recurrent disease, surgery, or other circumstances.
The listed enrollment is 8, limiting the amount of information available about less frequent events and the precision of any underlying event frequency. The registry’s primary outcome has a follow-up period of up to 21 days after administration. Longer-term follow-up and safety findings are not reported in the registry record.
The record gives counts for serious and other adverse events, a serious-event term, and a death count, but the registry entries do not include event-level descriptions, onset or resolution, attribution, or CTCAE grade for each reported event. The exact approach to repeated events and the relationship between the primary outcome’s participant count and the event-count wording are not reported. Missing observations and the rules for defining the analysis population are also not reported.
The registry lists secondary outcomes, but their results are not reported in the registry record for this interpretation. It also does not report the statistical analysis plan, confidence intervals, hypothesis tests, sample-size justification, interim analyses, multiplicity procedures, or missing-data methods. These omissions limit independent assessment of analytic choices and uncertainty. They should be understood as limits of what the registry record reports, not as evidence that the investigators did or did not undertake particular procedures outside the record.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: NU-0129 in Treating Patients With Recurrent Glioblastoma or Gliosarcoma Undergoing Surgery — NCT03020017.
- Registry record last updated 2022-08-26; status last verified 2022-08.
- Results first posted 2022-08-26.
- 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
NCT03020017 is a completed phase 0, first-in-human, single-group study of NU-0129 in recurrent glioblastoma or gliosarcoma. The registry lists an enrollment of 8 and describes intravenous treatment followed by standard-of-care tumor resection. The primary outcome is the number of participants with adverse events up to 21 days after administration, graded using NCI CTCAE version 4.03.
The safety summary lists serious and other adverse-event counts, cerebrospinal fluid leakage at 12.5% among the serious-event terms, and 0 deaths. These figures describe the treated group and do not provide a randomized comparison. The registry record does not report detailed event attribution, inferential analyses, uncertainty estimates, or the statistical plan. Results for the listed secondary outcomes are not reported in the registry record for this analysis. The findings are best read as a limited descriptive safety record, not as evidence of comparative effects or longer-term safety.