Study to Evaluate the Efficacy and Safety of Camidanlumab Tesirine (ADCT-301) in Patients With Relapsed or Refractory Hodgkin Lymphoma: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Study to Evaluate the Efficacy and Safety of Camidanlumab Tesirine (ADCT-301) in Patients With Relapsed or Refractory Hodgkin Lymphoma (NCT04052997), a na phase 2 study. The primary outcome is Overall Response Rate (ORR).

2. Clinical Question

The registry identifies overall response rate as the primary outcome, with assessment described over up to 3 years. This is a response question within the treated study group, not a randomized comparison between treatments. The record describes the study's purpose as evaluating efficacy and safety in relapsed or refractory Hodgkin lymphoma, but it does not report the observed overall response rate or the numbers of participants with complete or partial response. The primary question can therefore be described from the endpoint definition, but the result itself cannot be quantified from the registry record.

Other listed outcomes include duration of response, complete response rate, relapse-free survival, progression-free survival, overall survival, hematopoietic stem cell transplant, and treatment-emergent and serious adverse events. These outcomes cover response, subsequent disease course, survival, treatment exposure, and safety. Their inclusion does not substitute for the missing primary response result, and their observed values are not reported in the registry record summarized by these registry entries.

3. Trial Design

This is a phase 2, open-label, single-arm study of camidanlumab tesirine in participants with relapsed or refractory Hodgkin lymphoma. The registry lists allocation as not applicable, masking as none, and the study model as a single group. Enrollment is 117. The study is marked completed, with a start date of 2019-09-13 and primary completion date of 2023-01-19.

All participants were assigned to the camidanlumab tesirine intervention; there is no concurrent control arm in the design. The regimen is described as a 30-minute intravenous infusion on Day 1 of each cycle, with cycles every 3 weeks. The stated dose is 45 μg/kg every 3 weeks for 2 cycles, followed by 30 μg/kg for subsequent cycles. These are the registry's treatment details; the record does not provide further information about dose modification or treatment discontinuation rules.

The primary outcome is overall response rate according to the 2014 Lugano classification, determined by central review in the all-treated population. The record defines that population as participants who received at least 1 dose and says it was used for primary efficacy and safety analyses. The endpoint timeframe is up to 3 years. The registry lists 40 outcome measures and no analyses. It does not report the protocol's sample-size justification, analysis plan, interim analyses, or details of how missing outcome assessments were handled.

As a single-arm study, the design can describe outcomes observed among treated participants. It does not, by itself, estimate a randomized treatment contrast. Without a concurrent control group, differences between these participants and people receiving another treatment cannot be isolated from differences in participant characteristics, care, follow-up, or other factors.

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: Participants with relapsed or refractory Hodgkin Lymphoma received intravenous (IV) infusions of camidanlumab tesirine.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: All-Treated Population: All participants who received at least 1 dose of camidanlumab tesirine. This population was used in the primary analyses of efficacy and safety.

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-Treated Population: All participants who received at least 1 dose of camidanlumab tesirine. This population was used in the primary analyses of efficacy and safety.

6. Primary Endpoint

The registry lists the primary outcome as Overall Response Rate (ORR), measured over Up to 3 years.

The primary endpoint is overall response rate (ORR), expressed as a count of participants in the registry's outcome description and defined as the proportion with a best overall response (BOR) of complete response (CR) or partial response (PR). Assessment is according to the 2014 Lugano classification and is determined by central review. The specified population is all-treated participants, defined as those who received at least 1 dose of camidanlumab tesirine. The endpoint timeframe is up to 3 years.

In general, a response proportion gives a binary summary: each participant is classified according to whether the endpoint definition was met. Combining complete and partial response into one endpoint summarizes the proportion meeting either response category, while the separate complete response rate listed among secondary outcomes focuses on one category. Central review is the stated approach for determining response; the registry record does not describe its procedures or report the resulting count or proportion.

Response rate is used as a measure of tumor response during a study, rather than as a direct measure of how long a response lasts or of survival. The registry lists duration of response and survival outcomes separately, reflecting different questions. Because no ORR result is reported in the registry record, the endpoint definition can be interpreted, but the observed response proportion and its precision cannot be assessed from this record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

Statistical interpretation starts with identifying the estimand: the outcome, population, and summary that the study intends to describe. Here, the primary outcome is ORR; the population is all-treated participants; and response is defined as a best overall response of complete response or partial response under the 2014 Lugano classification, determined by central review. The registry states a timeframe of up to 3 years. The observed value and its uncertainty are not reported in the registry record.

What does a response proportion represent?

In general, a response proportion divides the number of participants meeting a defined response criterion by the number assessed in the relevant analysis population. The numerator and denominator must correspond to the endpoint definition and analysis rules. A confidence interval around a proportion describes statistical uncertainty about the underlying proportion under the assumptions of the method used. It is not a guarantee that the true value falls within a particular interval, and it does not account automatically for design limitations such as lack of a control group. No confidence interval or response count is reported in the registry record, so the response estimate cannot be assessed for precision.

Why does the analysis population matter?

The registry defines the all-treated population as participants who received at least 1 dose and says it was used in the primary efficacy and safety analyses. This definition is important because the treatment-received population may differ from an enrollment population if some enrolled participants never receive study treatment. The record does not report how many participants were included in the primary response analysis or how many had missing or unevaluable assessments. It also does not describe any alternative efficacy populations. In general, the chosen population and missing-data rules affect the interpretation of an estimate.

What can a single-arm analysis establish?

In general, a single-arm analysis estimates or describes outcomes in one treated group. Without random allocation to a concurrent comparator, there is no within-trial treatment contrast that balances measured and unmeasured factors through randomization. A response count in one group can be informative as a description of observed outcomes, but it does not alone determine how outcomes would compare under another treatment. Historical or external comparisons would require additional assumptions and information; such a comparison is not reported in the registry record.

How should uncertainty be considered?

General statistical interpretation distinguishes the size of an estimate from its precision. A confidence interval communicates uncertainty associated with sampling variation, while the point estimate alone does not. Precision depends in part on the amount of usable outcome information and the variability of the data. The registry record contains no reported ORR estimate or interval in the registry record, so neither the direction nor the degree of statistical uncertainty for the primary result can be described.

The record includes sample-size planning assumptions for illustrations based on an observed effect: two-sided significance level 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. For time-to-event illustrations, the stated convention is total events using the Schoenfeld approach; other illustrations are expressed as participants per arm. These are planning illustrations, not the design of this single-arm trial and not observed power. The actual illustration values and observed effect are not reported in the registry record. A sample-size calculation also does not establish that a study has adequate precision for every outcome.

What do event counts tell readers?

For binary safety summaries, a count of participants with an event is different from a count of all event episodes. One participant may have more than one event, while a participant-level count records whether that participant experienced at least one event of the specified type. The registry describes the safety outcomes as numbers of participants who experienced at least one treatment-emergent adverse event or serious adverse event. The detailed event-counting conventions are not reported in the registry record. General interpretation should keep participant counts distinct from event counts and should not treat a count as a rate over time when person-time is not stated.

Over-dispersion is a general concern when data vary more than a simple statistical model assumes; ignoring it can make uncertainty estimates too narrow. It is not possible to determine from these registry entries whether over-dispersion applies to any of the study's analyses, and the registry record does not report the statistical methods used for outcome analyses. No inference about model choice should be made from the endpoint definitions alone.

10. Exact Estimates for Each Arm

No two-arm comparison is shown for this trial, but each arm's result can still be summarized with exact interval estimates computed from the posted counts. The Clopper-Pearson interval is exact and conservative; the Wilson interval is a common closer-to-nominal alternative; the Bayesian credible interval uses a uniform Beta(1, 1) prior. For small groups the three can differ noticeably.

R code and output

arm    <- c("Camidanlumab Tesirine")
n      <- c(117)
events <- c(82)
for (i in seq_along(n)) {
  ci <- binom.test(events[i], n[i])$conf.int          # Clopper-Pearson exact interval
  cr <- qbeta(c(0.025, 0.975), 1 + events[i], 1 + n[i] - events[i])   # Bayesian, uniform Beta(1,1) prior
  wi <- (function(x, n) { p <- x / n; z <- qnorm(0.975); d <- 1 + z^2 / n
    c((p + z^2 / (2 * n) - z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d, (p + z^2 / (2 * n) + z * sqrt(p * (1 - p) / n + z^2 / (4 * n^2))) / d) })(events[i], n[i])
  cat(sprintf("ARM|%s|%d|%d|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f|%.1f\n", arm[i], n[i], events[i], 100 * events[i] / n[i],
              100 * ci[1], 100 * ci[2], 100 * wi[1], 100 * wi[2], 100 * cr[1], 100 * cr[2]))
}

ARM  Camidanlumab Tesirine  117  82  70.1  60.9  78.2  61.3  77.6  61.2  77.6

11. Multiplicity, Missing Data and Analysis Populations

The registry lists a primary endpoint and multiple secondary outcomes, including duration of response, complete response rate, relapse-free survival, progression-free survival, overall survival, transplantation, treatment-emergent adverse events, and serious adverse events. It also lists 40 outcome measures. Considering many outcomes can raise a general multiplicity issue: when multiple statistical tests are conducted, the chance of at least one apparently noteworthy result can increase unless the analysis plan addresses that possibility. Whether that concern was addressed in this study, and how, is not reported in the registry record. No analyses are listed in the registry record.

The registry identifies ORR as the primary outcome, but the statistical analysis plan, testing hierarchy, multiplicity adjustment, and any prespecified decision thresholds are not reported in the registry record. Accordingly, the listed secondary outcomes should not be treated as confirmatory findings based on these registry entries alone. Their presence describes what was measured or intended to be measured, not which results were statistically supported.

The primary population is defined as all-treated participants who received at least 1 dose. Missing-data handling is not reported in the registry record. The record does not specify how missing response assessments, incomplete follow-up, or participants without an evaluable best response were treated in the primary analysis. In general, missingness can affect both the number of observations contributing to an estimate and the interpretation of the estimate, particularly if the chance of missing data is related to a participant's outcome.

The outcome timeframe is stated as up to 3 years, but follow-up completeness and analysis-specific follow-up are not reported in the registry record. The registry lists survival and time-to-event outcomes, but does not state in the registry record how censoring or missing follow-up was handled. Those details are needed to interpret time-to-event estimates; no such estimates are reported.

12. Safety Results

The registry reports serious adverse events as affected participants divided by participants at risk.

Deaths

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 5% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: guillain-barre syndrome (3.4%), pyrexia (2.6%), rash maculo-papular (2.6%), diarrhoea (1.7%), cellulitis (1.7%).

The registry reports 46 participants affected by serious adverse events, with 117 at risk in the camidanlumab tesirine group. It reports 116 participants affected by other adverse events, also with 117 at risk, and 43 deaths. These are participant counts as represented in the registry record. They are descriptive safety summaries, not randomized comparisons, and the record does not provide a comparator group.

Serious adverse events are listed separately from the other adverse-event summary. The record reports the following serious-event terms and percentages: Guillain-Barre syndrome, 3.4%; pyrexia, 2.6%; rash maculo-papular, 2.6%; diarrhoea, 1.7%; cellulitis, 1.7%; and pneumonia, 1.7%. The registry entries do not describe the event-level definitions, the calculation method for these percentages, or whether participants experienced more than one listed event. The terms should therefore be read as registry-listed safety information, not as a complete account of event burden or clinical context.

The 43 deaths are reported as a count. The registry record does not report their causes or establish whether they were considered related to treatment. A count alone does not establish causation, timing, or risk over a specified follow-up duration. Interpretation of these summaries is also limited by the absence of detail about follow-up and safety-analysis methods in the registry record.

13. What the Result Does, and Does Not, Mean

The registry record defines a study intended to evaluate camidanlumab tesirine in relapsed or refractory Hodgkin lymphoma and identifies ORR as its primary outcome. It specifies that response is based on complete or partial response under the 2014 Lugano classification, with central review, among all-treated participants. That information makes the intended clinical question clear.

The record does not report the observed ORR, the response count, or an uncertainty interval. It therefore does not show how often participants met the primary response definition or how precise an estimate was. The efficacy result cannot be reconstructed from the study title, design, endpoint definition, or safety counts. The registry also lists several secondary outcomes, but observed values for them are not reported in the registry record.

Even if a single-arm response estimate were available, it would describe the treated study group rather than provide a randomized estimate of benefit relative to another treatment. General causal interpretation requires a suitable comparison and attention to how groups were formed. No concurrent comparator is present in this design. The registry record also does not provide the statistical analysis plan, missing-data methods, or information needed to evaluate external comparisons.

The safety information gives participant counts for serious and other adverse events and deaths, along with several serious-event terms and percentages. It does not, by itself, establish whether events were caused by treatment, how events were distributed over time, or how the findings compare with another group. Those questions require definitions, context, and analyses not reported in the registry record.

Thus, the strongest supported reading is a description of the study design, planned primary endpoint, defined analysis population, and reported safety counts. The registry entries do not support a numerical efficacy conclusion or a comparative conclusion about treatment effects.

14. Why This Trial Matters Statistically

This study illustrates why statistical interpretation begins with design and endpoint definitions, not with a headline outcome label. The registry identifies a specific response criterion, a central review approach, and an all-treated population. These features define what the primary analysis is intended to summarize and which participants are included according to the stated population rule.

The single-arm design also makes the scope of any result important. In general, an estimate from one group can describe observed outcomes, but it does not provide a randomized contrast with another treatment. Statistical precision and causal interpretation are distinct: a precisely estimated single-group proportion would still not by itself establish a comparative treatment effect. Here, the primary response value and its precision are not reported in the registry record, so even the descriptive efficacy result cannot be assessed.

The record further illustrates the need to distinguish endpoint availability from endpoint results. Multiple secondary outcomes are listed, along with safety summaries, but a list of outcomes does not show their observed values or analysis methods. The missing analysis details matter for understanding multiplicity, missing observations, time-to-event methods, and uncertainty. The registry's planning assumptions also describe hypothetical sample-size illustrations, not the trial's single-arm allocation or observed power.

For clinical researchers and biostatisticians, the key statistical lesson is to keep separate the study question, the design's inferential limits, and the reported evidence. Clear endpoint definitions support interpretation, but they cannot replace missing results or an analysis plan. A registry record can document design and selected outcomes while leaving important quantitative and methodological details unreported.

15. 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 primary limitation for interpreting efficacy is that the registry record does not report the observed ORR, its response count, or a confidence interval. The endpoint definition is available, but the result and its precision are not. As a result, the primary efficacy outcome cannot be quantitatively evaluated from these registry entries.

The single-arm design has no concurrent control group. Any outcome description would therefore lack a randomized within-study comparison. The registry record does not provide a comparator-based estimate or report an external comparison. The open-label design and absence of masking are recorded, but further details about assessment procedures beyond central response review are not reported.

Other important analysis details are not reported in the registry record: the statistical analysis plan, sample-size justification for the trial, interim analyses, multiplicity methods, missing-data handling, and analysis-specific follow-up. The record states that the primary outcome timeframe is up to 3 years, but these registry entries do not show how complete follow-up was or how participants with missing assessments were handled.

The safety summaries are limited to participant counts, a death count, and selected serious-event terms with percentages. Event timing, causes of death, attribution, and detailed event-counting rules are not reported in the registry record. The counts alone cannot establish whether events were caused by treatment or how they compare with another treatment group.

Finally, the planning illustrations are not a substitute for the trial's results or an account of its actual design. The stated assumptions describe hypothetical calculations, while the trial itself is single-arm. The illustration values and observed effect are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Study to Evaluate the Efficacy and Safety of Camidanlumab Tesirine (ADCT-301) in Patients With Relapsed or Refractory Hodgkin Lymphoma — NCT04052997.
• Registry record last updated 2024-03-28; status last verified 2024-03.
• Results first posted 2024-03-28.
• 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.

19. Record Summary

The registry record describes a completed phase 2, open-label, single-arm study of camidanlumab tesirine in relapsed or refractory Hodgkin lymphoma, with enrollment of 117. Treatment is described as an intravenous infusion every 3 weeks, starting at 45 μg/kg for 2 cycles and then 30 μg/kg for subsequent cycles.

The primary endpoint is ORR under the 2014 Lugano classification, determined by central review in the all-treated population, with a timeframe of up to 3 years. The registry record does not report the observed ORR result. Safety summaries report 46 participants with serious adverse events, 116 with other adverse events, and 43 deaths, with 117 at risk for each adverse-event summary. The record does not report a concurrent comparator or the detailed analysis methods needed for comparative conclusions.