Feasibility Clinical Study of Targeted and Genome-Wide Sequencing: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Feasibility Clinical Study of Targeted and Genome-Wide Sequencing (NCT01345513), a registered phase not stated study. The primary outcome is Time From Patient Recruitment to Final Results ≤ 21 Days in ≥ 90% of Patients.

2. Clinical Question

The study was titled “Feasibility Clinical Study of Targeted and Genome-Wide Sequencing” and lists solid tumors as its condition. Its stated purpose was to examine how cancer mutations could be identified using biopsy and blood samples and how genomic information might inform treatment decisions. Those aims describe the research context; they are not evidence that any treatment decision changed or that any clinical outcome improved.

The clinical question is therefore primarily about feasibility of returning genomic findings within a specified interval, rather than comparative treatment effectiveness. The record also lists secondary outcomes concerning actionable genomic results, adverse events due to tumor biopsies on study, and patient and physician experience. Results for these outcomes are not reported in the registry record.

3. Trial Design

This was a feasibility clinical study with an enrollment of 50 and a condition of solid tumors. The listed study intervention was sample collection for genome-wide sequencing. The registry record gives the study status as completed, with a start date of 2011-03 and a primary completion date of 2013-03-25. The listed follow-up period was up to 2 years from enrollment, or until death, whichever occurred first.

The record does not describe a randomized allocation or a control arm. It identifies one arm, “Solid Tumor Cancer,” and gives no arm description. No like-for-like treatment comparison can therefore be read from the design information. The record does not report the sequencing workflow in sufficient detail to determine which samples were obtained from each participant, how sequencing approaches were assigned, or whether any targeted-sequencing group was analyzed separately.

The primary outcome concerned the time from informed consent to reporting genomic results to the physician. Its title states a target of no more than 21 days in at least 90% of patients; the outcome description says average and range of the time in calendar days. These are related but not identical ways to express a turnaround objective: one is a threshold stated as a proportion, while the description specifies a mean and a full range. The registry record does not explain how that distinction was handled in the analysis.

Three secondary outcomes are listed: the number of participants with actionable genomic results, the number with adverse events due to tumor biopsies on study, and patient and physician experience. The record lists 4 outcome measures and 0 analyses. It does not report results for the primary or secondary outcomes. The sponsor is University Health Network, Toronto. The record does not report further design details such as the eligibility criteria, planned recruitment procedures, sample-size justification, or a statistical analysis plan.

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

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: Of the 56 participants approached, 50 agreed and consented to the study, and 49 had successful biopsies.

6. Primary Endpoint

The registry lists the primary outcome as Time From Patient Recruitment to Final Results ≤ 21 Days in ≥ 90% of Patients, measured over All patients will be followed for up to 2 years from study enrolment, or death, or whichever event occurs first..

The primary endpoint describes turnaround time for genomic results: the interval, in calendar days, between a participant providing informed consent and genomic results being reported to the physician. The title expresses a feasibility target of no more than 21 days in at least 90% of patients. The description says that the average and range of the interval were to be reported.

Turnaround time is relevant to a feasibility study because it quantifies the speed of a process, not whether a particular genomic finding leads to a specific treatment or outcome. A mean describes the average interval across the analyzed observations. A full range describes the lowest and highest observed intervals, so it can indicate how widely the individual timings varied. As a general statistical point, a mean alone can conceal unusually long or short observations, while a range depends strongly on the observations at the extremes. Neither measure, by itself, gives the full shape of the distribution.

The endpoint title and description emphasize different summaries. A mean and range do not, on their own, establish what proportion of participants met the stated turnaround threshold. To assess the title’s target directly, the number or proportion meeting that threshold would be needed. The registry record does not report that result, the observed mean, the range, or the number included in the endpoint analysis. It also does not state how timing was handled when results were not reported or when a participant did not complete the relevant process. The endpoint’s intended follow-up is described as up to 2 years from enrollment, or death, whichever occurred first, but this does not supply an observed turnaround-time result.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

The endpoint is a duration measured in calendar days, from informed consent to reporting genomic results to the physician. The record identifies the parameter as a mean and the dispersion measure as a full range. These choices describe different features of the observed times. The mean is an average; the range gives the minimum and maximum. Neither can be recovered from the stated target alone, and neither is reported as an observed result.

What does the mean describe?

As a general statistical explanation, the mean combines all measured durations into one average. It is useful for describing the overall level of a set of observations, but it does not indicate how many observations were near that average. If a small number of durations are much longer than the rest, they can pull the mean upward. That possibility matters for turnaround times because a process may have a modest average while still including some substantially delayed results. The registry record does not report the individual durations or their distribution, so it is not possible to assess whether that issue arose in this study.

What does the full range add?

As a general concept, the full range reports the smallest and largest observed values. It can make the extremes visible, but it does not show how observations are distributed between those extremes. It is also sensitive to the number of observations: a range may widen when more observations are included because there are more opportunities to observe an extreme value. The registry record identifies a range as the dispersion measure but does not report its values. It does not report a median, standard deviation, or confidence interval for the endpoint.

How does the stated target differ from the summaries?

The primary endpoint title states that the goal is a duration of no more than 21 days in at least 90% of patients. That target is naturally evaluated by counting how many observations meet the cutoff and relating that count to the number assessed. A mean and range alone do not answer that question. For example, the mean could describe the average duration without indicating how many observations were within the cutoff; the range could reveal an extreme value without describing the proportion that met the target. The registry record does not explain whether the title’s threshold was the formal analysis criterion, whether the mean and range were the planned summaries, or how they were reconciled.

What would uncertainty measures tell a reader?

In general, a confidence interval expresses uncertainty around an estimated quantity under a statistical model and its assumptions. It is not a guarantee that the true value lies in the interval, and it does not replace the estimate itself. For a proportion meeting a target, an interval would help convey how precisely that proportion was estimated. For a mean duration, an interval would convey uncertainty about the average. No endpoint estimate or confidence interval is reported in the registry record, so precision cannot be assessed.

How should the design shape interpretation?

The record does not describe randomization or a comparator. General statistical interpretation of a single-arm feasibility study is therefore different from interpretation of a randomized comparison: process measurements can describe what happened among those assessed, but they cannot by themselves establish that one strategy caused a better result than another. The record does not state whether the sample collection or sequencing procedures were uniform across participants, whether multiple approaches were compared, or how any differences were handled.

The sample-size assumptions described for planning illustrations use a two-sided significance level of 0.05, equal allocation between two arms, a normal approximation, and an effect expressed as a fraction of an observed effect. Time-to-event planning is described in terms of total events using the Schoenfeld method, while other planning outputs are described as participants per arm. These are planning assumptions, not evidence that the registered feasibility study had two randomized arms. The study record lists no comparator and does not report a study-specific sample-size justification. Any planning illustration based on an observed effect is distinct from an observed trial result; it cannot supply an outcome that the registry does not report.

Over-dispersion is a general statistical issue for count outcomes: it occurs when variability exceeds what a simple count model assumes. Accounting for it can be important when modeling counts, because an overly restrictive model may understate uncertainty. The registry record does not report a count-model analysis or describe an over-dispersion adjustment. That general concept should not be mistaken for a method known to have been used in this study.

The record’s outcome descriptions and adverse-event entries do not provide enough detail to reconstruct an analysis. There are no reported endpoint estimates, analysis populations, missing-data rules, or model specifications. Consequently, the methods information supports explaining what the intended summaries mean, but not reproducing or validating an observed statistical result.

10. Multiplicity, Missing Data and Analysis Populations

The registry record lists 4 outcome measures, including the primary turnaround-time outcome and secondary outcomes concerning actionable genomic results, biopsy-related adverse events, and patient and physician experience. It lists 0 analyses. No multiplicity plan is reported in the registry record. In general, multiplicity arises when several outcomes or comparisons are evaluated: interpreting each test separately without accounting for the number of tests can increase the chance of a chance finding. The record does not say whether any adjustment or hierarchy was planned, and no inferential outcome results are reported.

Missing-data handling is not reported in the registry record. The record does not explain how participants with incomplete timing information, unavailable genomic results, or incomplete experience assessments were treated. It does not state whether any imputation was planned or used, or whether analyses were restricted to participants with complete observations. The primary outcome’s follow-up description says all patients would be followed for up to 2 years from enrollment, or death, whichever occurred first; it does not specify how those events affected the endpoint analysis.

Analysis populations are also not reported in the registry record. There is no stated definition of who was included in the primary endpoint summary or secondary outcome counts, and no account of exclusions from analysis. Although the record gives an enrollment of 50 and adverse-event reporting identifies 49 at risk in each of two categories, it does not explain that relationship. No inference about why the counts differ is warranted.

These omissions limit assessment of completeness, precision, and susceptibility to bias. Without outcome results and analysis-population definitions, it is not possible to determine whether the reported or intended summaries include all enrolled participants or how incomplete observations may have affected them.

11. Safety Results

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

Other (non-serious) adverse events

Participants with at least one non-serious event at or above the registry's 0% reporting threshold.

Most frequent serious adverse event terms

Most frequent non-serious adverse event terms

Serious events with the highest arm-level rate: grade 2 pneumothorax (2.0%), grade 3 cellulitis (2.0%).

The adverse-event information reports 49 participants at risk for serious adverse events, with 2 affected, and 49 at risk for other adverse events, with 6 affected. The record separately lists Grade 2 pneumothorax at 2.0% and Grade 3 cellulitis at 2.0% among the top serious terms. These are registry entries, not a complete narrative of event timing, duration, management, or outcome. The death count is not reported in the registry record.

The adverse-event outcome of interest is described as adverse events due to tumor biopsies on study, but the posted adverse-event figures do not establish that the listed events were caused by biopsies. The event attribution is not reported in the registry record. The record also does not explain whether the serious and other-event categories overlap, or provide event-level details that would allow the affected-participant counts to be reconciled with the listed terms. The 2.0% figures should be read as reported percentages, not converted into counts or used to infer an unreported denominator.

These figures describe reported safety events in this study; they do not, on their own, establish comparative safety. No control group is listed, and the registry does not provide enough information about surveillance or causal assessment to compare event rates with another approach. The numbers should not be interpreted as proof that the sequencing process caused or did not cause the events.

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

The registry record describes a completed feasibility study focused on sequencing-related genomic results for people with solid tumors. Its primary objective was framed as a turnaround-time target: results reported to a physician within no more than 21 days for at least 90% of patients. This framing identifies what the study sought to assess, but the record does not report whether the target was met.

The posted record does not give the observed average or range of turnaround times, the proportion meeting the target, or an analysis of uncertainty. It also does not report results for the secondary outcomes of actionable genomic findings, biopsy-related adverse events, or patient and physician experience. Completion status is a status designation, not evidence of a particular endpoint result.

More broadly, the study’s stated rationale concerns finding cancer mutations and exploring how genomic information might help researchers and doctors make treatment decisions. The record does not report whether results changed decisions, whether a particular treatment was selected, or whether patient outcomes improved. It therefore cannot establish that genome-wide sequencing is clinically effective, that it is superior to another strategy, or that it benefits patients.

The absence of a listed comparator is important. This study is not described as a randomized comparison, and no control group is provided. Even if a process result had been reported, a single-group feasibility result would describe that study experience rather than establish a causal advantage over another method. The sample-size planning assumptions do not change that interpretation: planning illustrations are not observed outcomes, and they do not supply missing analyses.

The safety entries provide a limited account of adverse events, including the reported serious and other-event counts and two listed serious-event terms. They do not establish event causation or comparative risk. The record does not report whether those events were related to study biopsies.

The appropriate reading is narrow: the registry records an intended feasibility objective and some safety reporting, but does not post the primary endpoint result or secondary outcome findings. Any conclusion about actual turnaround performance, actionability, decision-making effects, clinical benefit, or comparative safety would require information not reported in the registry record.

13. Why This Trial Matters Statistically

Feasibility studies address whether a research process can be carried out and whether its operational targets are met. In this study, the primary question was not framed as whether a treatment improved a clinical outcome. It focused on the interval between consent and reporting genomic results to a physician, with a target stated in terms of a time cutoff and a proportion of patients. That makes the distinction between an operational objective and a clinical-effectiveness claim especially important.

Statistically, the endpoint also illustrates why a target needs a matching summary. An average and range describe timing from different angles, while the target asks how many observations fell within a defined interval. Reporting the summary that directly corresponds to the question makes it easier to judge whether the operational goal was achieved. The registry record names both the target and the average-and-range description but does not provide results or explain their relationship.

The study is not a randomized comparison and does not list a comparator. That means its design information is suited to describing a process, not estimating a comparative treatment effect. This distinction prevents a common overreading: a feasibility rationale about how genomic results might inform decisions is not itself evidence that decisions changed or outcomes improved.

The record also shows why outcome reporting and analysis details matter. Without observed endpoint values, analysis populations, missing-data rules, or a multiplicity plan, readers cannot judge precision, completeness, or the robustness of any outcome finding. No such finding is reported in the registry record. The safety entries are likewise limited and do not establish causation or comparative risk.

For biostatisticians and clinical researchers, the key lesson is to align the endpoint definition, its stated target, the planned summary, and the reported analysis. A feasibility objective can be clinically relevant as a measure of research workflow, while still requiring careful separation from claims about clinical utility or treatment benefit.

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 most important trial-specific limitation of the registry record is that it does not report the observed primary endpoint. Although the title specifies a target of no more than 21 days in at least 90% of patients and the outcome description specifies an average and full range, neither the observed timing values nor the proportion meeting the target is reported. This prevents assessment of the study’s central feasibility question.

The record lists 0 analyses and does not provide results for the secondary outcomes. It does not report the number of participants with actionable genomic results, findings from patient and physician experience, or the number of participants with adverse events due to tumor biopsies on study. The adverse-event information does not state whether events were attributed to biopsies, and the death count is not reported in the registry record.

Design and analysis information is also limited. No comparator or randomized allocation is described, the listed arm has no description, and the record does not report eligibility criteria, analysis populations, missing-data procedures, or a multiplicity plan. It does not explain the relation between the enrollment of 50 and the adverse-event risk counts of 49. That difference should not be interpreted as evidence about withdrawals or missingness because the reason is not reported in the registry record.

The registry title refers to targeted and genome-wide sequencing, while the listed intervention is sample collection for genome-wide sequencing. The record does not explain how targeted sequencing was used, how approaches were assigned, or whether they were compared. The study’s completed status and stated rationale do not resolve these gaps. As a result, the record supports understanding the intended question but not estimating turnaround performance, clinical utility, or comparative safety.

15. Related Tutorials

16. Related Statistical Calculators

17. Sources

• ClinicalTrials.gov: Feasibility Clinical Study of Targeted and Genome-Wide Sequencing — NCT01345513.
• Registry record last updated 2020-03-23; status last verified 2020-03.
• Results first posted 2020-03-23.
• 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.

18. Record Summary

NCT01345513 is listed as a completed feasibility clinical study of targeted and genome-wide sequencing for solid tumors. The record gives an enrollment of 50, identifies sample collection for genome-wide sequencing as the intervention, and names University Health Network, Toronto as the sponsor. The listed study start is 2011-03, and the primary completion date is 2013-03-25.

The primary outcome concerns time in calendar days from consent to reporting genomic results to a physician. Its title states a target of no more than 21 days in at least 90% of patients, while the outcome description specifies an average and full range. The registry record does not report the observed primary result or any analysis. It lists secondary outcomes related to actionable genomic results, biopsy-related adverse events, and patient and physician experience, but does not report their results.

The adverse-event entries report 2 affected among 49 at risk for serious events and 6 affected among 49 at risk for other events; Grade 2 pneumothorax and Grade 3 cellulitis are listed at 2.0% each. The record does not establish event attribution. Overall, the registry describes the feasibility question and limited safety information, but does not provide results needed to evaluate the primary target or the secondary outcomes.