Testing JNJ-42756493 (Erdafitinib) as Potentially Targeting Treatment in Cancers With FGFR Amplifications (MATCH-Subprotocol K1): Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Testing JNJ-42756493 (Erdafitinib) as Potentially Targeting Treatment in Cancers With FGFR Amplifications (MATCH-Subprotocol K1) (NCT06308822), a na phase 2 study. The primary outcome is Objective Response Rate.

2. Clinical Question

3. Trial Design

This is a phase 2, single-group study of erdafitinib in the MATCH treatment subprotocol for tumors with FGFR amplifications. The registry record lists an enrollment of 35. Allocation is not applicable, and masking is listed as none. The listed conditions include advanced and refractory lymphomas, advanced and refractory malignant solid neoplasms, and refractory multiple myeloma.

Patients in the treatment group receive erdafitinib by mouth once daily on days 1-28. Cycles repeat every 28 days while disease has not progressed and toxicity remains acceptable. The treatment description also includes computed tomography and magnetic resonance imaging throughout the study; blood collection and tumor biopsy may also occur. These procedures provide clinical or biological information, but the registry record does not specify additional analysis plans for them.

The primary outcome is objective response rate, defined as the percentage of patients with a complete or partial response among eligible, treated patients with mutation status confirmed. Tumor assessments occur at baseline and then every 2 cycles for the first 26 cycles, followed by every 3 cycles until progression, up to 3 years post registration. Secondary outcomes are the 6-month progression-free survival rate and progression-free survival.

The study start is listed as 2018-08-20 and primary completion as 2022-07-27. Its status is active, not recruiting. The registry record does not report a randomized control group, a masking procedure, or the number included in the primary endpoint analysis.

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: The FGFR amplification status was determined by assay performed in a NCI-MATCH approved laboratory for all patients in this arm. Mutation status was confirmed by central MATCH assay for 18 patients.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Eligible, treated and mutation status confirmed

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: Patients who were eligible, started protocol therapy, and had mutation status confirmed at the analysis time were the analyzable patients

6. Primary Endpoint

The registry lists the primary outcome as Objective Response Rate, measured over Tumor assessments occurred at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until disease progression, up to 3 years post registration.

Objective response rate (ORR) is the percentage of patients whose tumors have a complete or partial response. Its denominator is defined as eligible, treated patients with mutation status confirmed. The response criteria are tailored to tumor type: RECIST version 1.1, the Cheson (2014) criteria for lymphoma, and the Response Assessment in Neuro-Oncology criteria for glioblastoma. The registry record refers to the master protocol for further details on complete and partial response definitions.

ORR is used as a summary of observed tumor shrinkage meeting prespecified response criteria. It differs from an outcome such as progression-free survival, which concerns time until progression or death. A response proportion can be informative in a single-group study because it describes how many patients meet the response definition, but it does not by itself establish how outcomes would compare under another treatment or without treatment.

The registry record specifies a two-sided binomial exact 90% confidence interval for ORR. A confidence interval conveys uncertainty around the estimated response proportion; its width depends in part on the number of evaluable patients and the observed response pattern. The record names the endpoint and method but does not report the ORR estimate, its interval, or the analyzed denominator. Consequently, the size and precision of the observed response proportion cannot be assessed from the registry record.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The primary outcome is a binary response status for each participant: a participant either meets the complete or partial response definition or does not. The study-level ORR is a proportion based on the eligible, treated, mutation-status-confirmed population. This kind of endpoint differs from a continuous measurement, such as a laboratory value, because its summary is the fraction of participants meeting a defined event criterion.

What does the confidence interval mean?

The registry record specifies a two-sided binomial exact 90% confidence interval for ORR. In general, a confidence interval gives a range of values compatible with the data under the statistical method used. It is not a guarantee that the true value lies in the interval, and it does not remove uncertainty. For a proportion, interval precision depends on the amount of information in the observed data, including the size of the analyzed population and the response pattern.

An exact binomial interval is a method for estimating uncertainty in a proportion using the binomial model. The word “exact” describes the interval procedure; it does not mean that the estimate is free of uncertainty. The record does not post the interval endpoints, so its width and the precision of the primary estimate cannot be assessed.

How is response defined?

Response classification is based on recognized criteria named in the registry record. RECIST version 1.1 is specified for tumors generally, the Cheson (2014) criteria for lymphoma, and the Response Assessment in Neuro-Oncology criteria for glioblastoma. Different tumor types may require different rules for determining whether disease has responded. The registry record refers to the master protocol for details of complete and partial response definitions; those details are not reported in this entry.

Assessments are scheduled at baseline, then every 2 cycles for the first 26 cycles and every 3 cycles thereafter until progression, up to 3 years post registration. Assessment timing matters because the opportunity to detect response can depend on when and how often tumors are evaluated. The entry gives the schedule but does not report the timing of individual responses or how assessments were handled when a participant stopped treatment or missed an assessment.

What can a single-group estimate establish?

General statistical interpretation: a single-group proportion describes the outcome observed in the analyzed group. Without a concurrent comparator, it does not directly estimate a difference attributable to treatment. The design listed for this study is single group; the record does not describe random assignment or a control arm. Any interpretation should therefore keep the descriptive nature of the endpoint distinct from a comparative treatment effect.

The record lists an enrollment of 35, but it does not say that this is the primary endpoint denominator. The endpoint definition requires eligibility, treatment, and confirmed mutation status, so the analysis population may be narrower than total enrollment. The number meeting those conditions is not reported in the registry record.

What do the secondary endpoints add?

The listed secondary outcomes are the 6-month PFS rate and PFS. A rate at a specified time summarizes the share of a population meeting a time-to-event status by that point, while a PFS analysis generally concerns time until progression or death. These are general descriptions; the registry entry does not report the analysis method, event counts, censoring details, or estimates for either secondary outcome. No additional statistical assumptions or model specifications are reported.

The record lists zero analyses. It does not provide analysis outputs for the primary or secondary endpoints, so no numerical estimate, hypothesis test, or uncertainty measure for those outcomes can be interpreted. General statistical principles can explain how such estimates would be read, but cannot substitute for the missing results.

How should planning calculations be read?

Any sample-size illustration based on the observed effect is planning information, not an observed study outcome and not observed power. The stated assumptions for such illustrations are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. Those assumptions do not describe the listed single-group design. The registry record does not report the trial's own sample-size justification or design assumptions. Consequently, a planning illustration should not be mistaken for evidence that the trial was designed or powered in that way.

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("Treatment (Erdafitinib)")
n      <- c(18)
events <- c(0)
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  Treatment (Erdafitinib)  18  0  0.0  0.0  18.5  -0.0  17.6  0.1  17.6

11. Multiplicity, Missing Data and Analysis Populations

The registry record lists one primary endpoint, ORR, and two secondary outcomes: the 6-month PFS rate and PFS. It does not report a multiplicity strategy, such as how interpretation would account for examining more than one outcome. The prespecified testing hierarchy, if any, is not reported in the registry record. Without that information and without posted outcome analyses, the record does not establish whether any inferential results were adjusted for multiple comparisons or were intended to be interpreted descriptively.

Missing-data handling is also not reported in the registry record. The primary endpoint population is defined as eligible, treated patients with mutation status confirmed, but the record does not describe how participants with missing or unevaluable tumor assessments were treated in the analysis, or whether additional analysis populations were used. It also does not give the number included in the primary endpoint denominator. These details matter because exclusion or classification of participants without an assessable response can affect a response proportion.

The assessment schedule is described, but rules for missed visits, discontinuation, or incomplete follow-up are not. For the secondary time-to-event outcomes, censoring rules and the handling of unavailable follow-up are not reported. No interim analyses or statistical analysis plan are reported in the registry record. These absences limit assessment of how analysis decisions were specified and applied.

The registry entry's description of the primary population includes confirmed mutation status, while the study title and condition concern FGFR amplification. The record does not clarify this wording or explain how mutation status relates to eligibility or analysis. That ambiguity should not be resolved by assumption.

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: mucositis oral (9.1%), vomiting (9.1%), hyponatremia (6.1%), anemia (3.0%), atrioventricular block complete (3.0%).

The safety summary lists one treatment group with 33 participants at risk for serious adverse events and 13 affected. It separately lists 33 at risk for other adverse events and 29 affected, and reports 32 deaths. These are registry counts; the entry does not provide a comparative control group or enough detail to interpret them as treatment-attributable effects. The timing and causes of deaths are not reported in the registry record.

The most frequently listed serious adverse event terms are mucositis oral, 9.1%; vomiting, 9.1%; hyponatremia, 6.1%; anemia, 3.0%; atrioventricular block complete, 3.0%; and Mobitz (type) II atrioventricular block, 3.0%. These percentages are reported as listed and should not be interpreted as comparative risks. The record does not explain whether participants could contribute more than one event term, provide event-level counts, or describe severity grades and exposure duration for these terms.

Counts of affected participants and percentages for selected event terms answer different descriptive questions and do not establish causation. The safety summaries should be interpreted alongside the limits of the reporting detail and the single-group design.

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

The registry record documents a phase 2, single-group evaluation of erdafitinib in tumors with FGFR amplifications. It specifies an objective response endpoint, the population used for that endpoint, response criteria, assessment timing, and secondary PFS outcomes. It also lists adverse-event counts and selected serious event terms. These details describe the study's intended measurements and some of its safety reporting.

The primary ORR result itself is not reported in the registry record. There is no response estimate, confidence interval, numerator, or primary analysis denominator in the information available. Accordingly, the record does not show how often participants met the response criteria or how precise an estimate would be. The secondary PFS results are also not reported.

Even if a response estimate were available, a single-group result would describe outcomes in that group rather than establish how outcomes would differ under another treatment. No concurrent comparator is specified, and no randomized comparison is described. The study record therefore cannot by itself separate treatment effects from other influences on outcomes.

The event counts offer a descriptive account of reported safety categories but are not a comparison of risks between treatment groups. The registry record does not supply details such as event timing, causes of deaths, or exposure-adjusted analyses. Those gaps constrain how far the safety summaries can be interpreted.

Thus, the record is informative about the study framework and the endpoints selected, but incomplete as a report of efficacy results. It neither establishes a favorable nor an unfavorable primary response result because that estimate is absent. Interpretation should distinguish the planned measurement from a result and should not treat enrollment or safety counts as substitutes for the missing efficacy analysis.

14. Why This Trial Matters Statistically

This study raises several statistical issues common to trials that combine multiple tumor types under a biomarker-defined treatment strategy. The listed conditions span lymphomas, solid neoplasms, and multiple myeloma, while response classification uses different criteria for different disease settings. A single overall response proportion can be difficult to interpret without knowing the tumor-type mix, the number assessed in each group, and whether outcomes vary across those groups. Those breakdowns are not reported in the registry record.

The primary endpoint is a proportion, and its interpretation depends on a clearly defined denominator. The registry record specifies eligible, treated participants with mutation status confirmed, while enrollment is listed separately. Because the analyzed population size is not reported, the precision of the response estimate cannot be judged; the estimate itself is also absent.

The single-group structure is another important statistical feature. It allows description of response in the enrolled study population, but does not provide a randomized, concurrent comparison. This distinction matters whenever readers are tempted to interpret a response proportion as a comparative treatment effect. The registry record does not report a control group or comparative analysis.

Finally, the entry names secondary time-to-event outcomes but does not include their analysis methods or results. The absence of a reported multiplicity plan, missing-data rules, and analysis populations makes it difficult to assess the intended inferential framework. These are not reasons to presume a particular analysis; they identify details that are not reported in the registry record and that are relevant to statistical interpretation.

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 main limitation of the registry information is the absence of posted efficacy estimates. ORR is identified as the primary endpoint and its confidence-interval method is stated, but neither the estimate nor the interval is reported. The record also omits the primary analysis denominator and response count. This prevents assessment of both the observed result and its precision.

The study is listed as single group, with no concurrent comparator. The design can describe outcomes among participants who meet the endpoint population definition, but cannot support a direct randomized comparison based on the information in the registry record. The entry does not report tumor-type-specific results or the composition of the analyzed population, limiting interpretation across the varied conditions included.

Important analysis details are not reported: the handling of missing or unevaluable assessments, the analysis populations beyond the primary definition, the statistical approach for the secondary outcomes, and any multiplicity strategy. Interim analyses and the trial's own sample-size justification are not reported in the registry record. The registry entry also does not clarify the relationship between its mutation-status wording for the primary population and the FGFR amplification focus in the title and conditions.

Safety reporting is limited to aggregate counts and selected serious adverse event terms. Event timing, causes of deaths, exposure duration, and comparative safety information are not reported. These limitations are specific to the registry entries and should not be filled with assumptions about the protocol or analyses.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Testing JNJ-42756493 (Erdafitinib) as Potentially Targeting Treatment in Cancers With FGFR Amplifications (MATCH-Subprotocol K1) — NCT06308822.
• Registry record last updated 2026-09-15; status last verified 2026-09.
• Results first posted 2024-06-18.
• 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 phase 2, single-group study of erdafitinib for tumors with FGFR amplifications, with enrollment of 35 and status listed as active, not recruiting. Treatment is oral daily dosing on days 1-28 of repeating 28-day cycles. The primary outcome is ORR among eligible, treated participants with mutation status confirmed, assessed using criteria appropriate to tumor type and accompanied by a planned two-sided binomial exact 90% confidence interval.

The record lists the 6-month PFS rate and PFS as secondary outcomes, but does not report efficacy estimates or analysis outputs. It reports safety counts for serious and other adverse events and deaths, along with selected serious adverse event terms. Analysis populations beyond the primary definition, missing-data procedures, multiplicity handling, and the trial's own sample-size justification are not reported in the registry record.