This page provides an independent statistical analysis and educational interpretation of publicly reported results. ClinicalTrials.gov provides the official trial registry record. Numerical trial results on this page are restricted to the results posted in the ClinicalTrials.gov record; calculations added by Clinical Biostats are labelled as such.
1. Trial at a Glance
This page covers the registry results for Study of Talimogene Laherparepvec In Children With Advanced Non CNS Tumors (NCT02756845), a na phase 1 study. The primary outcome is Percentage of Participants Who Experienced a Dose-limiting Toxicity (DLT).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02756845 |
| Phase | Phase 1 |
| Condition | Advanced Non CNS Tumors |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 15 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Amgen |
| Results posted | Yes |
2. Clinical Question
Population
Children and adolescents with advanced non-CNS tumors that were amenable to direct injection. The registry record gives an enrollment of 15 and describes an older cohort aged 12 to ≤ 21 years and a younger cohort aged 2 to < 12 years. The primary endpoint population was defined separately as participants evaluable for dose-limiting toxicity, with eligibility based on follow-up opportunity and treatment exposure.
Intervention
Intralesional talimogene laherparepvec, also called TVEC in the cohort descriptions. The regimen began with a dose up to 4.0 mL at 10ᶺ6 PFU/mL, followed 21 days (+3 days) later by a dose up to 4.0 mL at 10ᶺ8 PFU/mL. Subsequent doses were scheduled every 14 days (± 3 days). These are the registry’s described dose limits and schedule, not a report of the amount each participant actually received.
Comparator
There was no separate control group. The registry identifies the study as single-group, open-label, and without masking. The age cohorts are described as treatment cohorts, not as randomized or like-for-like control groups. Comparisons between them cannot be interpreted as controlled estimates of an intervention effect.
Primary question
The primary endpoint asks what percentage of dose-limiting-toxicity-evaluable participants experienced a DLT from Day 1 to Day 35. The registry defines qualifying toxicities by severity, duration or clinical consequences, and attribution to talimogene laherparepvec. It does not report the observed percentage or confidence interval, so the result of that primary question cannot be determined from the registry record.
In practical statistical terms, this is a descriptive early-phase safety question in a pediatric population selected for tumors that could be injected directly. The study also listed tumor-response and time-to-event outcomes, including overall response rate, duration of response, time to response, time to progression, progression-free survival, and overall survival. Their presence as secondary outcomes does not make them controlled treatment comparisons. Without a control group, outcome patterns can be described, but differences attributable to treatment cannot be separated from other explanations using this design alone.
3. Trial Design
The registry describes NCT02756845 as a completed phase 1, multicenter, open-label, dose-de-escalation study of talimogene laherparepvec in pediatric participants with advanced non-CNS tumors amenable to direct injection. Enrollment was 15. The study began on 2017-08-16, and its primary completion date was 2022-01-17. The sponsor was Amgen.
Allocation is recorded as not applicable, masking as none, and the model as single-group. These features mean the record does not describe random assignment to intervention and control arms, nor blinded treatment assignment. The two age cohorts in the safety summaries do not constitute a randomized comparison. Differences in their counts or event listings should not be treated as estimates of what would have happened under a different treatment.
The intervention description gives a first dose up to 4.0 mL of 10ᶺ6 PFU/mL, followed 21 days (+3 days) later by a dose up to 4.0 mL of 10ᶺ8 PFU/mL. Subsequent doses were scheduled every 14 days (± 3 days). The description also refers to cohorts, but the full cohort dosing and dose-de-escalation rules are not reported in the registry record. The listed schedule should therefore be read as a regimen description, not evidence that every participant received every scheduled dose.
The listed primary outcome is the percentage of participants experiencing a dose-limiting toxicity over Day 1 to Day 35. Secondary outcomes include overall response rate, duration of response, time to response, time to progression, progression-free survival, and overall survival. Although the registry lists seven outcome measures, it reports zero statistical analyses and gives no numerical primary endpoint result. Details of the statistical analysis plan, including planned estimation methods and any decision rules, 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 | Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years |
|---|---|---|
| STARTED | 13 | 2 |
| Received Talimogene Laherparepvec | 13 | 2 |
| COMPLETED | 0 | 0 |
| NOT COMPLETED | 13 | 2 |
Reasons for not completing this period:
| Reason | Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years |
|---|---|---|
| Death | 12 | 2 |
| Withdrawal by Subject | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: DLT Analysis Set: all DLT evaluable participants defined as participants who had the opportunity to be followed for at least 35 days from the initial dosing of talimogene laherparepvec and received at least two treatments of talimogene laherparepvec (except participants who had a DLT after the first dose).
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.
Population: All enrolled participants.
| Characteristic | Unit | Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years | Total |
|---|---|---|---|---|
| Participants analysed | 13 | 2 | 15 | |
| Age, Continuous | years | 15.2 (SD 2.8) | 9.0 (SD 2.8) | 14.3 (SD 3.5) |
| Sex: Female, Male: Female | Participants | 5 | 0 | 5 |
| Sex: Female, Male: Male | Participants | 8 | 2 | 10 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 2 | 1 | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 10 | 1 | 11 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: White | Participants | 9 | 2 | 11 |
| Race/Ethnicity, Customized: Other | Participants | 3 | 0 | 3 |
| Race/Ethnicity, Customized: American Indian or Alaska Native | Participants | 1 | 0 | 1 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants Who Experienced a Dose-limiting Toxicity (DLT), measured over Day 1 to Day 35.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | 95% Confidence Interval |
| Unit | Percent of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants who experienced a dose-limiting toxicity (DLT) from Day 1 to Day 35. A DLT was not simply any unfavorable event: the registry states that the toxicity had to meet specified criteria and be judged related to talimogene laherparepvec. The criteria include selected severe non-hematologic toxicities, clinically consequential laboratory abnormalities, febrile neutropenia, serious herpetic events, grade 5 toxicity, and intolerable toxicity leading to permanent discontinuation. Toxicities were graded using Common Terminology Criteria for Adverse Events version 4.0.
The endpoint is expressed as a percentage, so its intended summary is the proportion of the relevant participants who met the DLT definition during the stated interval. In general, a defined early safety endpoint can help characterize treatment-limiting toxicity during a specific observation period. It is not a complete account of all possible harms over longer follow-up, and it does not by itself describe tumor response or survival.
The endpoint population is the DLT Analysis Set: participants who had the opportunity for at least 35 days of follow-up after initial dosing and received at least two treatments, except for a stated exception involving participants with a DLT after the first treatment. The remaining details of that exception and the observed endpoint value are not reported in the registry record. No numerator, denominator, percentage, or confidence interval for the primary endpoint is reported, so the frequency and precision of the observed DLT result cannot be assessed from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record establishes the study’s design and the definitions of its outcomes, but it does not report numerical results for the primary DLT endpoint. In particular, it gives no observed percentage of participants with a DLT and no associated 95% confidence interval. The listed primary question is therefore identifiable, but its answer cannot be read from the reported primary outcome information.
The study enrolled 15 participants and is described as single-group, open-label, and unmasked. The enrollment count is not necessarily the denominator for the DLT percentage: the primary analysis population required at least 35 days of follow-up opportunity and at least two treatments, with an exception described for a DLT after the first treatment. The number meeting that definition is not reported in the registry record. Without that denominator and the DLT count, an event rate cannot be recovered.
Safety information is listed separately for Cohort A1, aged 12 to ≤ 21 years, and Cohort B1, aged 2 to < 12 years. The record lists 5 participants with serious adverse events among 13 at risk in Cohort A1 and 1 among 2 at risk in Cohort B1. Other adverse events are listed for 13 among 13 at risk in Cohort A1 and 2 among 2 at risk in Cohort B1. These are descriptive counts in cohorts of different sizes, not a controlled comparison. The record does not establish that the reported events were caused by study treatment.
There is no comparator group against which to estimate an intervention effect. Consequently, the design cannot distinguish observed outcomes associated with treatment from the course of the underlying tumors, other care, or other influences. The secondary outcomes include response and survival measures, but the registry record does not provide their numerical results or analysis details. Their listing alone should not be interpreted as evidence of a particular level of response, disease control, or survival.
In general, a confidence interval communicates uncertainty around an estimated quantity; its width depends in part on the amount of information available. Here, the registry lists a 95% confidence interval as the primary endpoint’s dispersion measure, but does not report its limits. No inference about the precision of the primary estimate is therefore possible. Similarly, no p-values or model-based estimates are reported. The posted information supports a description of the study framework and selected safety counts, not a numerical conclusion about the primary endpoint or a causal treatment effect.
9. Statistical Methods Explained
The statistical methods that can be described are limited to the endpoint definitions and study structure reported in the registry. The record identifies the primary outcome as a percentage, specifies a 95% confidence interval as its dispersion measure, and defines a DLT-evaluable population. It does not report the estimator used for the percentage, the method used to construct the interval, or numerical results from a statistical analysis.
What does a percentage endpoint represent?
In general, a percentage for a binary outcome summarizes how many participants in a defined population experienced the event of interest. The definition of that population matters as much as the event definition: changing who is counted can change the interpretation of the estimate. For this study, the primary population was limited to participants who had the opportunity for at least 35 days of follow-up after initial dosing and received at least two treatments, subject to an exception for an early DLT. The number of participants who met these rules is not reported in the registry record.
The DLT definition is clinically structured rather than a count of every adverse event. The record specifies that toxicities had to be judged related to talimogene laherparepvec and lists qualifying grades and clinical circumstances. These include grade 4 non-hematologic toxicity; grade 3 non-hematologic toxicity lasting more than 3 days despite optimal supportive care; selected grade 3 or higher laboratory abnormalities with specified consequences; grade 3 or 4 febrile neutropenia; thrombocytopenia below 25 x 10^9/L with bleeding requiring intervention; serious herpetic events; grade 5 toxicity; and intolerable toxicity leading to permanent discontinuation. The exact endpoint depends on applying these rules consistently.
What does the confidence interval add?
In general, a confidence interval gives a range of estimates compatible with the observed data under the interval’s statistical assumptions. It conveys uncertainty that a point estimate alone cannot express. It is not a guarantee that the true value lies inside the interval, and it does not address every source of uncertainty, such as selection, measurement, or lack of a control group. Although the registry names a 95% confidence interval for the DLT percentage, it does not give the interval limits or say how they were calculated. The estimate’s precision therefore cannot be judged from the registry record.
For a binary outcome, the choice of interval method can matter, particularly when the analyzed group is small or the event is uncommon or frequent. This is a general statistical consideration, not a claim about the method used in this study. The registry record does not report whether the analysis used an exact method, a large-sample approximation, or another approach. No method should be attributed to the study without that information.
How should the design shape interpretation?
In general, random assignment to a concurrent control group supports a direct comparison by helping balance other influences across groups. This study is listed as single-group, with allocation not applicable and no masking. Its age cohorts are not described as randomized controls. Safety counts in those cohorts can be read descriptively, but a difference between cohort counts would not isolate a treatment effect. The record also does not provide adjusted estimates or a model that could address differences between participants.
The study is labeled dose-de-escalation, but the complete dose-de-escalation rules and statistical decision process are not reported in the registry record. The intervention description gives a scheduled dose sequence, including the initial and later concentration levels and timing. It does not state the actual received dose for each participant. A planned dose schedule and the doses delivered are different kinds of information.
What can be inferred from the reported analysis information?
The registry lists seven outcome measures and zero statistical analyses. It gives no numerical primary outcome result, secondary outcome estimates, p-values, or model specifications. Thus, the design and endpoints are described, but a statistical finding cannot be reconstructed. General methods such as confidence-interval estimation or comparison of event proportions explain how such results might be interpreted if reported; they should not be mistaken for methods documented as having been used.
The record includes a planning illustration framework based on an observed effect, with assumptions involving a two-sided significance level of 0.05, equal allocation, and a normal approximation. It also distinguishes planning quantities by outcome type. These are described as planning illustrations, not observed power. No numerical planning results are reported in the record, and the single-group design does not itself provide the two-arm comparison described by those assumptions. Such illustrations cannot fill in the missing primary result or establish what the trial was designed to detect.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists a primary safety endpoint and secondary outcomes covering overall response rate, duration of response, time to response, time to progression, progression-free survival, and overall survival. When a study examines several outcomes, the chance of at least one apparently notable result can increase if each is tested without accounting for the others. In general, multiplicity procedures can control or describe that issue, but the appropriate approach depends on the analysis plan and the intended claims.
The registry record does not report a multiplicity strategy, a hierarchy of secondary endpoints, or inferential results for those outcomes. It also lists zero statistical analyses. It is therefore not possible to determine whether secondary outcomes were formally tested, how any testing was handled, or which results, if any, were intended as confirmatory. The endpoint list alone does not establish that every measure was analyzed or that any one result should be treated as definitive.
Missing-data methods are not reported in the registry record. The primary endpoint definition does require a specified opportunity for follow-up and a minimum treatment exposure, with an exception for a DLT after the first treatment. This definition does not provide the number excluded, the reasons for exclusion, or the handling of participants whose follow-up or treatment exposure was incomplete. Those details matter because the evaluable population may differ from all enrolled participants.
The record does not report the number included in each outcome analysis, the handling of missing outcome assessments, or any sensitivity analysis. It also does not give the full statistical analysis plan or clarify whether any analysis populations beyond the DLT Analysis Set were prespecified. These omissions limit assessment of how closely reported results, if any, would represent all enrolled participants.
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 |
|---|---|---|---|
| Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | 5 | 13 | 38.5% |
| Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years | 1 | 2 | 50.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | 12 of 13 |
| Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years | 2 of 2 |
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 |
|---|---|
| Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | 13 of 13 |
| Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years | 2 of 2 |
Most frequent serious adverse event terms
| Event term | Organ system | Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years |
|---|---|---|---|
| Nausea | Gastrointestinal disorders | 1 of 13 | 0 of 2 |
| Vascular device infection | Infections and infestations | 1 of 13 | 0 of 2 |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | 1 of 13 | 0 of 2 |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | 1 of 13 | 0 of 2 |
| Skin ulcer | Skin and subcutaneous tissue disorders | 1 of 13 | 0 of 2 |
| Metastatic malignant melanoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 0 of 13 | 1 of 2 |
| Osteosarcoma metastatic | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 13 | 0 of 2 |
| Tumour haemorrhage | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | 1 of 13 | 0 of 2 |
| Cranial nerve disorder | Nervous system disorders | 0 of 13 | 1 of 2 |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | 1 of 13 | 0 of 2 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Cohort A1: Talimogene Laherparepvec (TVEC) - Aged 12 to ≤ 21 Years | Cohort B1: Talimogene Laherparepvec (TVEC) - Aged 2 to < 12 Years |
|---|---|---|---|
| Pyrexia | General disorders | 9 of 13 | 2 of 2 |
| Vomiting | Gastrointestinal disorders | 6 of 13 | 1 of 2 |
| Headache | Nervous system disorders | 5 of 13 | 1 of 2 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 4 of 13 | 1 of 2 |
| Fatigue | General disorders | 4 of 13 | 0 of 2 |
| Anaemia | Blood and lymphatic system disorders | 3 of 13 | 0 of 2 |
| Constipation | Gastrointestinal disorders | 3 of 13 | 0 of 2 |
| Nausea | Gastrointestinal disorders | 2 of 13 | 1 of 2 |
| Influenza like illness | General disorders | 3 of 13 | 0 of 2 |
| Gamma-glutamyltransferase increased | Investigations | 2 of 13 | 1 of 2 |
Serious events with the highest arm-level rate: metastatic malignant melanoma (50.0%), cranial nerve disorder (50.0%), nausea (7.7%), vascular device infection (7.7%), musculoskeletal chest pain (7.7%).
The serious adverse event summaries are grouped by age cohort. Cohort A1, aged 12 to ≤ 21 years, lists 13 participants at risk and 5 affected by serious adverse events. Cohort B1, aged 2 to < 12 years, lists 2 at risk and 1 affected. The registry also lists 12 deaths in Cohort A1 and 2 in Cohort B1. These are cohort-level safety counts; the registry record does not establish that deaths or adverse events were caused by talimogene laherparepvec.
For other adverse events, the record lists 13 affected among 13 at risk in Cohort A1 and 2 affected among 2 at risk in Cohort B1. The listed top serious event terms include metastatic malignant melanoma and cranial nerve disorder, each at 50.0%, followed by nausea, vascular device infection, musculoskeletal chest pain, and pulmonary embolism, each at 7.7%. The registry does not provide further context in these summaries about event timing, recurrence, severity for each term, or causal attribution.
In general, adverse-event counts describe what was recorded in the stated population; they do not by themselves establish causation. The cohorts are small and differ in size, so the counts should not be read as a comparative safety estimate. They are also separate from the primary DLT endpoint, which has its own criteria, time window, and evaluable population. The primary DLT result is not reported in the registry record.
12. What the Result Does, and Does Not, Mean
The registry record describes an early-phase, single-group study of intralesional talimogene laherparepvec in pediatric participants with advanced non-CNS tumors amenable to direct injection. It specifies a primary DLT endpoint, the observation interval, toxicity grading framework, and a DLT-evaluable population. It also provides safety counts by age cohort and lists response and time-to-event outcomes as secondary measures.
Those details explain what the study set out to assess and how certain outcomes were defined. They do not show how many evaluable participants experienced a DLT, because the primary percentage and its confidence interval are not reported. Nor does the registry give numerical results for the secondary outcomes. The listed enrollment of 15 is not a substitute for the primary analysis denominator, which is defined by follow-up and treatment-exposure criteria.
In general, a single-group study can document events observed during treatment and follow-up, but it cannot provide the same treatment-effect comparison as a controlled study. Without a concurrent comparator, observed tumor outcomes cannot be attributed to the intervention by design alone. The open-label, unmasked structure also means that participants and investigators were not blinded to treatment assignment, as no alternative assignment is described.
The adverse-event summaries are descriptive. They report serious and other adverse events in the two age cohorts, along with selected serious-event terms. They do not, by themselves, establish that the intervention caused an event or explain the clinical course of each participant. The death counts should likewise be read as recorded cohort summaries, not as proof of a treatment-related effect.
Accordingly, the registry information supports understanding of the population, regimen description, endpoint criteria, and selected safety reporting. It does not support a numerical assessment of the primary DLT rate, precision of that rate, efficacy, or comparative benefit and harm. These limits reflect what is reported in the registry record, not a conclusion that an unreported result was favorable or unfavorable.
13. Why This Trial Matters Statistically
This trial is statistically informative as an example of how early-phase pediatric studies define safety questions in a population with advanced tumors and a treatment requiring direct injection. The primary endpoint is not an unrestricted count of adverse events: it applies a specified DLT definition, attribution requirement, observation window, and evaluability rule. Each of these choices determines what the endpoint represents.
The design also illustrates why study structure governs the strength of conclusions. A single-group, open-label study can describe safety observations, but it does not create a concurrent control comparison. The two age cohorts provide descriptive strata, not randomized arms. Their unequal sizes and limited counts make it especially important not to treat differences between them as treatment-effect estimates.
Another statistical issue is the distinction between enrollment and analysis population. Although enrollment is 15, the primary endpoint uses a DLT-evaluable definition based on follow-up opportunity and treatment exposure. The number qualifying under that definition is not reported. Without a clear analysis denominator and event count, neither the primary percentage nor its precision can be interpreted.
The registry lists several secondary outcomes, including response and survival measures, but does not report numerical analysis results. This highlights the difference between registering an outcome and reporting evidence about it. The absence of reported estimates, confidence intervals, and analysis details means that the scope for statistical interpretation is limited even though the endpoint definitions are detailed.
More broadly, this record shows why safety summaries, endpoint rules, and study design should be read together. Event counts can describe what was recorded; a defined endpoint specifies what qualifies; and the study design determines whether outcomes can be compared causally. Here, the available record is useful for understanding those components, while leaving the primary numerical result and its uncertainty unavailable.
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 principal limitation for interpreting this trial’s statistical results is that the registry record does not report the numerical primary endpoint result. The DLT percentage, event count, evaluable denominator, and 95% confidence interval are all unavailable. Although the DLT Analysis Set is defined in part, the final analysis population and the details of the exception for an early DLT are not reported.
The study is single-group, open-label, and unmasked, with no separate control group. The age cohorts are not presented as like-for-like controls. Consequently, the design does not support a controlled estimate of treatment effect, and descriptive outcomes cannot separate treatment-associated observations from other influences. The record does not report the actual dose received by each participant or the full dose-de-escalation rules.
The safety summaries give counts for serious and other adverse events and selected serious event terms, but do not report detailed timing, event-level follow-up, or causal attribution in those summaries. Cohort B1 contains 2 participants at risk, so its count is particularly limited as a basis for characterizing the range of possible event patterns. No comparative inference should be drawn from the cohort summaries.
Although response and time-to-event outcomes are listed, their numerical results, analysis populations, censoring rules, missing-data handling, and statistical methods are not reported in the registry record. The multiplicity approach and full statistical analysis plan are also not reported. These omissions restrict evaluation of precision, bias, and the status of any secondary findings. The registry record identifies the study as completed, but completion status does not supply missing outcome estimates.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study of Talimogene Laherparepvec In Children With Advanced Non CNS Tumors — NCT02756845.
- Registry record last updated 2024-02-20; status last verified 2024-02.
- Results first posted 2024-02-05.
- 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
NCT02756845 is a completed phase 1, multicenter, open-label, single-group, dose-de-escalation study sponsored by Amgen. It enrolled 15 pediatric participants with advanced non-CNS tumors amenable to direct injection. The intervention was intralesional talimogene laherparepvec, with age-specific cohorts listed in the safety summaries.
The primary endpoint was the percentage of DLT-evaluable participants experiencing a dose-limiting toxicity from Day 1 to Day 35. The record defines qualifying toxicities and identifies a 95% confidence interval as the dispersion measure, but does not report the primary percentage, its denominator, or interval limits. Secondary outcomes include response and time-to-event measures, but their numerical results and analysis details are not reported in the registry record.
Safety counts are provided for serious and other adverse events by cohort, alongside selected serious-event terms. Because the design has no control group, these summaries are descriptive rather than comparative. Overall, the registry record documents the study framework and endpoint definitions but does not provide the numerical results needed to assess the primary endpoint or its precision.