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 Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation (NCT01746784), a randomized phase 1 study. The primary outcome is Safety and Tolerability.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01746784 |
| Phase | Phase 1 |
| Condition | Cystic Fibrosis |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 66 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Nivalis Therapeutics, Inc. |
| Results posted | Yes |
2. Clinical Question
Population
Adults with cystic fibrosis who were homozygous for the F508del-CFTR mutation. This population is specified in the study title and purpose. The registry record does not report further eligibility details, such as lung-function thresholds or other inclusion and exclusion criteria.
Intervention
N6022 administered by intravenous infusion once per day for 7 days. The study included dose groups described as 5 mg, 10 mg, 20mg, and 40 mg of N6022.
Comparator
Normal saline administered intravenously using the same volume as the active-drug group. The registry record describes the study as placebo-controlled.
Primary question
What safety, tolerability, and pharmacokinetic observations occur with N6022 in this population? The primary outcome is described as safety and tolerability, assessed through abnormal clinical evaluations, abnormal laboratory assessments, and adverse events. The study purpose also includes descriptive information on biomarkers of CFTR function and inflammation.
This is a Phase 1 study recorded as randomized, quadruple-masked, and parallel-group. Its stated focus is early evaluation of safety, tolerability, and pharmacokinetics, rather than a definitive comparison of clinical effectiveness. The listed secondary outcomes are change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) and change in biomarkers of CFTR function.
The study record describes N6022 as being given over 7 treatment days, with safety follow-up over 7 days. It does not report a primary hypothesis comparing a particular dose with saline, a prespecified clinically important difference, or a statistical decision threshold for those comparisons. The registry record therefore supports describing the question and outcomes, but not inferring a treatment-effect conclusion from the study’s design alone.
3. Trial Design
The registry record describes a randomized, double-blind, placebo-controlled, dose-escalation study with a parallel-group model. It is recorded as Phase 1, while the official title calls it Phase 1b. Quadruple masking is recorded, but the registry record does not specify which roles or groups were masked.
The study enrolled 66 participants and is marked completed. Its recorded start was 2014-02, and primary completion was 2014-04. Nivalis Therapeutics, Inc. is listed as the sponsor. The condition is cystic fibrosis, and the study population is described as adults homozygous for the F508del-CFTR mutation.
Participants assigned to N6022 received intravenous infusions once per day for 7 days. The dose groups are labeled 5 mg/N6022, 10 mg/N6022, 20mg/N6022, and 40 mg/N6022. The placebo group received normal saline intravenously in the same volume as the active-drug group. Although the study is described as dose escalation, the registry record does not report the rules for moving between doses or whether dose escalation depended on particular safety findings.
The registry record does not report the allocation ratio, the number assigned to each group as a planned design feature, or the sample-size justification. The group counts appearing with the adverse-event results should be read as the reported counts at risk for those safety summaries, not as a substitute for a full account of randomization or participant flow. Details such as withdrawals, protocol deviations, and the disposition of all enrolled participants are not reported in the registry record.
The study purpose includes pharmacokinetics and descriptive information on biomarkers of CFTR function and inflammation. The listed secondary outcomes specifically name change in Percent Predicted FEV1 and change in biomarkers of CFTR function. The registry record does not give enough detail to establish the full measurement schedule or the planned statistical approach for these outcomes.
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: There were 4 ascending dose cohorts starting at 5 mg and continuing with 10 mg, 20 mg and 40 mg. There was a fifth confirmatory cohort at the maximum tested dose, 40 mg. In Cohorts 1-4 approximately 9 subjects received N6022 and 3 received placebo. In the fifth cohort 11 subjects received N6022 and 7 subjects receive placebo
Overall Study
| Stage | Placebo/Saline | 5 mg/N6022 | 10 mg/N6022 | 20mg/N6022 | 40 mg/N6022 |
|---|---|---|---|---|---|
| STARTED | 19 | 10 | 9 | 9 | 19 |
| Enrolled | 19 | 10 | 9 | 9 | 19 |
| COMPLETED | 19 | 10 | 9 | 9 | 19 |
| NOT COMPLETED | 0 | 0 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: Treatment emergent adverse events by Grade 1 (mild) to 5 (fatal)
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | Normal Saline | 5mg/N6022 | 10mg/N6022 | 20mg/N6022 | 40mg/N6022 | Total |
|---|---|---|---|---|---|---|---|
| Participants analysed | 19 | 10 | 9 | 9 | 19 | 66 | |
| Age, Continuous | years | 29.0 (SD 7.27) | 26.9 (SD 6.33) | 32.3 (SD 7.84) | 28.6 (SD 10.3) | 28.4 (SD 8.46) | 28.9 (SD 7.93) |
| Sex: Female, Male: Female | Participants | 10 | 6 | 6 | 7 | 11 | 40 |
| Sex: Female, Male: Male | Participants | 9 | 4 | 3 | 2 | 8 | 26 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 19 | 10 | 9 | 9 | 19 | 66 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 19 | 10 | 9 | 9 | 19 | 66 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 | 0 |
| Region of Enrollment: United States | participants | 19 | 10 | 9 | 9 | 19 | 66 |
| Body Mass Index (BMI) | kg/^m2 | 21.4 (SD 3.25) | 21.7 (SD 4.59) | 21.7 (SD 2.27) | 21.0 (SD 3.80) | 22.5 (SD 2.62) | 21.9 (SD 3.25) |
| Weight | kg | 62.9 (SD 11.50) | 61.8 (SD 11.73) | 59.1 (SD 9.06) | 56.3 (SD 13.59) | 63.8 (SD 12.53) | 61.0 (SD 11.96) |
| FEV1 (L) | L | 2.7 (SD 0.85) | 2.6 (SD 0.88) | 2.2 (SD 0.91) | 2.1 (SD 0.66) | 2.8 (SD 1.12) | 2.5 (SD 0.97) |
| FEV1 Percent Predicted | percent | 72.5 (SD 23.7) | 70.7 (SD 19.5) | 63.3 (SD 17.5) | 63.2 (SD 21.1) | 73.9 (SD 21.9) | 70.1 (SD 21.3) |
| Sweat Chloride | mEq/L | 100.4 (SD 12.9) | 103.3 (SD 6.9) | 107.3 (SD 6.7) | 93.1 (SD 9.2) | 100.3 (SD 8.3) | 100.8 (SD 10.7) |
6. Primary Endpoint
The registry lists the primary outcome as Safety and Tolerability, measured over Over 7 treatment days and 7 days of follow-up.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | participants |
| Endpoint classification | Binary Endpoint |
The primary outcome is titled Safety and Tolerability. Its description says assessments are based on the number of participants with abnormal clinical evaluations, abnormal laboratory assessments, and adverse events. The population description focuses on treatment-emergent adverse events graded from Grade 1 (mild) to 5 (fatal), and the timeframe is over 7 treatment days and 7 days of follow-up.
In general, safety outcomes are used to describe the kinds and frequency of unwanted events observed during a study. Counts can show how many participants in each group were recorded as affected, while the event descriptions and severity grades provide context that a count alone cannot convey. These measures are particularly relevant to an early-phase study whose stated purpose includes examining safety and tolerability.
The posted safety results include counts of participants with serious adverse events and counts with other adverse events across the saline and N6022 dose groups. They do not supply a complete breakdown by event grade, clinical evaluation, or laboratory assessment in the registry record used for this description. The registry record also does not report a single prespecified statistical contrast for the primary outcome. Consequently, the safety counts are best read as descriptive observations rather than as a complete inferential comparison of the groups.
Safety and tolerability do not answer the same question as the secondary FEV1 and CFTR-biomarker outcomes. The registry record names those outcomes but does not provide their results in the information available for this account. Pharmacokinetic measurements are part of the study purpose, but their measures and results are not reported in the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted safety counts are distributed across saline and four N6022 dose groups. For serious adverse events, the reported affected and at-risk counts are 1 of 19 in Placebo/Saline, 2 of 10 in 5 mg/N6022, 0 of 9 in 10 mg/N6022, 0 of 9 in 20mg/N6022, and 1 of 19 in 40 mg/N6022. These are descriptive group-level observations. They do not by themselves establish whether the groups differ beyond what could occur through chance variation, nor do they establish a causal effect of dose.
The counts for participants affected by other adverse events are 18 of 19 in Placebo/Saline, 9 of 10 in 5 mg/N6022, 9 of 9 in 10 mg/N6022, 9 of 9 in 20mg/N6022, and 15 of 19 in 40 mg/N6022. The registry record does not provide the event-level detail needed to interpret these counts as a comparison of specific symptoms, severity, duration, or clinical consequences. The word “other” should not be taken to mean that all these events had the same nature or importance.
Appendicitis and Pulmonary Exacerbation of CF are each listed at 10.0% among the top serious terms. The registry record does not identify the group assignment associated with these terms in the registry record for this account, so they should not be attributed to a particular dose group. Nor does their listing alone establish whether an event was caused by study treatment.
In general, a difference in observed counts is not automatically evidence of a treatment effect. Small groups can yield unstable estimates: a single participant’s event status can materially change a group summary. Confidence intervals, when available, describe the precision of an estimate under the assumptions of the analysis. The registry record does not report confidence intervals or a formal statistical comparison for these safety counts.
The study has several dose groups and multiple stated outcomes, including FEV1 and CFTR biomarkers. A dose-by-dose pattern in safety counts should not be treated as a confirmed trend without the relevant analysis plan and uncertainty estimates. No formal analyses are reported in the registry record. This absence in the record does not establish that no analyses were conducted; it means their methods and results cannot be evaluated from the registered information.
The counts therefore describe what was recorded in the posted safety results, not a complete account of the balance of benefits and harms or a definitive test of efficacy. The registry record does not report the FEV1 and biomarker results, detailed pharmacokinetic results, or a prespecified statistical comparison. Any interpretation beyond the descriptive safety observations would require information not reported in the registry record.
9. Statistical Methods Explained
Randomization is intended, in general, to make treatment assignment independent of participant characteristics, reducing systematic differences between groups at the start of a trial. The registry record identifies this study as randomized, but does not report the allocation procedure, allocation ratio, or sequence-generation details. The group counts in the adverse-event results are not enough to reconstruct those design details.
Masking is used in general to reduce the influence of knowledge about assigned treatment on participant experience, care, or outcome assessment. The record labels the study quadruple-masked and double-blind in the official title. It does not identify the roles covered by quadruple masking or explain how masking was maintained. The saline comparator was administered by the same route and at the same volume as the active-drug group, according to the record; no further masking procedures are described.
The parallel-group model means that participants are assigned to a group and assessed under that assigned regimen, rather than each participant necessarily receiving every regimen in sequence. This is a general explanation of the design label. The record does not report crossover periods or within-participant comparisons. The study is also described as dose escalation, but operational details of escalation are not reported.
The primary outcome is a safety and tolerability assessment, with counts of participants described as having abnormal clinical evaluations, abnormal laboratory assessments, and adverse events. The posted adverse-event summary separates serious events from other events. These are count data: they record how many participants were affected, rather than measuring an average continuous response. A count can be useful for describing observed events, but its interpretation depends on the event definition, observation period, completeness of follow-up, and how repeated events in one participant were handled. Those details are not fully reported in the registry record.
In general, a rate or proportion compares an event count with the number of participants at risk. The posted serious-event information includes both affected and at-risk counts by group. Such summaries make the denominator visible, but they do not by themselves account for differences in follow-up time, participant characteristics, or event recurrence. The record does not report a time-to-event analysis or a model adjusting for other factors.
Confidence intervals are, in general, a way to express uncertainty around an estimated quantity. Wider intervals indicate less precision, while narrower intervals indicate more precision under the method’s assumptions. For rare events or small groups, estimates can be particularly imprecise. No confidence intervals are reported for the adverse-event counts in the registry record, so their statistical precision cannot be assessed from those results alone.
Hypothesis tests, in general, evaluate data against a stated null hypothesis using a prespecified analysis. A p-value does not measure the size or clinical importance of an effect, and it does not replace an estimate with an uncertainty interval. The registry record reports no analyses, and does not specify a primary statistical contrast, test, model, or inferential threshold for the outcomes described. No p-value or conclusion of statistical significance should be inferred from the event counts.
The study names FEV1 and CFTR-function biomarkers as secondary outcomes. In general, continuous outcomes such as a lung-function measure may be summarized with a change from baseline and an estimate of the difference between groups. The appropriate analysis depends on the measurement schedule, baseline values, missing observations, and prespecified model. These details and the outcome results are not reported in the registry record. Biomarker outcomes likewise require attention to how measurements were defined and analyzed, details that are not available in the record.
Multiplicity arises in general when several outcomes, doses, or comparisons are examined. Testing many hypotheses can increase the chance of at least one apparently unusual result if no adjustment or prioritization is planned. This study includes multiple dose groups and lists secondary outcomes, but the registry record does not report a multiplicity strategy or an analysis hierarchy. Therefore, no adjustment method or control of family-wise error can be attributed to the study based on the registered information.
The record includes planning assumptions for sample-size illustrations: a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with effects framed as fractions of an observed effect. Such calculations are planning illustrations, not observed power and not evidence that the trial was designed or powered according to those assumptions. The trial’s own sample-size justification and its underlying effect estimate are not reported in the registry record. The listed illustration assumptions also do not establish that a two-arm comparison captures the study’s multi-dose design.
Over-dispersion is a general concern when count data vary more than a chosen statistical model expects. If a model assumes more uniformity than the data have, uncertainty can be understated. The registry record does not identify a count-data model, and the posted safety information is descriptive; therefore, no conclusion about over-dispersion or model adequacy can be made for this trial. Similarly, handling of missing data, analysis populations, and sensitivity analyses is not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists change in Percent Predicted FEV1 and change in biomarkers of CFTR function as secondary outcomes, alongside the primary safety and tolerability outcome. It also describes pharmacokinetics and biomarkers of inflammation as part of the study purpose. The number of outcome measures is recorded as 3, while no analyses are reported in the registry record. The record does not provide an analysis hierarchy, identify which outcomes were confirmatory, or describe a method for handling multiple outcomes or dose comparisons.
In general, testing multiple outcomes or comparing several groups can create multiplicity: the more questions examined, the greater the chance of an apparently notable result arising by chance. A prespecified hierarchy or adjustment can help define how such results should be interpreted. No such approach is reported for this study, so the presence or absence of multiplicity control cannot be determined from the registry record.
Missing-data handling matters because incomplete measurements can affect the groups being compared and the precision of estimates. The registry record does not describe missing-data rules, participant-level outcome availability, imputation, or sensitivity analyses. It also does not report analysis populations, such as whether results were based on all randomized participants or a subset defined by treatment received or outcome availability.
These omissions do not prove that the study team did not use analysis procedures that are not included in the registry record. They mean that readers cannot assess from this record which populations, missing-data assumptions, or multiplicity procedures informed the analyses.
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 |
|---|---|---|---|
| Placebo/Saline | 1 | 19 | 5.3% |
| 5 mg/N6022 | 2 | 10 | 20.0% |
| 10 mg/N6022 | 0 | 9 | 0.0% |
| 20mg/N6022 | 0 | 9 | 0.0% |
| 40 mg/N6022 | 1 | 19 | 5.3% |
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 |
|---|---|
| Placebo/Saline | 18 of 19 |
| 5 mg/N6022 | 9 of 10 |
| 10 mg/N6022 | 9 of 9 |
| 20mg/N6022 | 9 of 9 |
| 40 mg/N6022 | 15 of 19 |
Most frequent serious adverse event terms
| Event term | Organ system | Placebo/Saline | 5 mg/N6022 | 10 mg/N6022 | 20mg/N6022 | 40 mg/N6022 |
|---|---|---|---|---|---|---|
| Pulmonary Exacerbation of CF | Infections and infestations | 1 of 19 | 1 of 10 | 0 of 9 | 0 of 9 | 1 of 19 |
| Appendicitis | Infections and infestations | 0 of 19 | 1 of 10 | 0 of 9 | 0 of 9 | 0 of 19 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Placebo/Saline | 5 mg/N6022 | 10 mg/N6022 | 20mg/N6022 | 40 mg/N6022 |
|---|---|---|---|---|---|---|
| Cough | Respiratory, thoracic and mediastinal disorders | 7 of 19 | 3 of 10 | 1 of 9 | 3 of 9 | 2 of 19 |
| Increased bronchial secretion | Respiratory, thoracic and mediastinal disorders | 3 of 19 | 2 of 10 | 2 of 9 | 2 of 9 | 1 of 19 |
| Headache | Nervous system disorders | 1 of 19 | 1 of 10 | 1 of 9 | 2 of 9 | 1 of 19 |
| Chest tightness | Cardiac disorders | 1 of 19 | 2 of 10 | 1 of 9 | 0 of 9 | 2 of 19 |
| Nasal Congestion | Respiratory, thoracic and mediastinal disorders | 1 of 19 | 3 of 10 | 0 of 9 | 0 of 9 | 1 of 19 |
| Rales | Respiratory, thoracic and mediastinal disorders | 0 of 19 | 3 of 10 | 0 of 9 | 1 of 9 | 0 of 19 |
| Fatigue | General disorders | 1 of 19 | 1 of 10 | 0 of 9 | 0 of 9 | 2 of 19 |
| Diarrhea | Gastrointestinal disorders | 2 of 19 | 0 of 10 | 1 of 9 | 0 of 9 | 0 of 19 |
| Nausea | Gastrointestinal disorders | 1 of 19 | 1 of 10 | 0 of 9 | 0 of 9 | 1 of 19 |
| Pyrexia | General disorders | 0 of 19 | 1 of 10 | 0 of 9 | 0 of 9 | 2 of 19 |
Serious events with the highest arm-level rate: appendicitis (10.0%), pulmonary exacerbation of cf (10.0%).
The serious adverse-event summaries give the number affected and the number at risk within each group. The reported figures are 1 of 19 for Placebo/Saline, 2 of 10 for 5 mg/N6022, 0 of 9 for 10 mg/N6022, 0 of 9 for 20mg/N6022, and 1 of 19 for 40 mg/N6022. These are counts of affected participants, not a statistical test of differences between groups.
For other adverse events, the reported counts are 18 of 19, 9 of 10, 9 of 9, 9 of 9, and 15 of 19 in the corresponding groups. The registry record does not supply event-specific descriptions for those group counts in the information, and the counts do not indicate how many events each affected participant experienced.
Appendicitis and Pulmonary Exacerbation of CF are each listed at 10.0% among the top serious terms. The registry record does not link those terms to specific groups in the registry record for this account. Serious-event counts should therefore be read alongside the study’s short stated observation period and the limits of the available detail, not as a complete account of all safety outcomes.
12. What the Result Does, and Does Not, Mean
The posted results offer a descriptive view of serious and other adverse events recorded in the saline and N6022 groups during the study’s stated safety timeframe. They show that the serious-event counts were not the same across every group, and that participants with other adverse events were recorded in each group. These observations describe the registered safety summaries; they do not establish that N6022 caused or prevented any particular event.
The study’s stated purpose also included pharmacokinetics and descriptive information on CFTR function and inflammation, and the registry lists FEV1 and CFTR biomarkers as secondary outcomes. The results for those outcomes are not reported in the registry record used for this account. The safety summaries therefore cannot answer whether lung function or biomarker measures changed, or how any such changes compared between groups.
In general, early-phase safety studies can characterize observed tolerability and identify events that warrant further evaluation, but a small group or an event count alone cannot establish the likelihood of uncommon harms or a definitive treatment effect. The record does not report confidence intervals or inferential analyses for the adverse-event counts. A zero recorded serious-event count in a group should not be interpreted as proof that the treatment carries no risk.
Nor do these data establish comparative effectiveness. The primary endpoint is safety and tolerability, and the registry record does not provide an efficacy conclusion, a prespecified treatment-effect threshold, or complete results for the secondary outcomes. The study is best understood from this information as a randomized, dose-escalation investigation focused on safety, tolerability, and pharmacokinetics in a specified cystic fibrosis population, with limits on what can be concluded from the posted results.
13. Why This Trial Matters Statistically
This trial illustrates a common statistical challenge in early-phase studies: safety outcomes may be summarized across several dose groups while the number of participants in each reported group is limited. For event counts, the denominator is essential context. The posted serious-event results give affected and at-risk counts by group, allowing readers to see the scale of the observations without mistaking them for a definitive comparison.
The dose-escalation design also raises an interpretive question. A pattern across dose labels may be of interest, but a pattern in descriptive counts is not equivalent to a prespecified dose-response analysis. The registry record does not report escalation rules, an inferential model, or a statistical plan for comparing doses. That makes it important to distinguish exploration and description from formal evidence of a dose-related effect.
The registry record names several kinds of outcomes, including safety, pharmacokinetics, FEV1, and biomarkers. Statistical interpretation depends not only on the measurements but also on how outcomes and comparisons were prioritized, how missing data were handled, and how multiplicity was addressed. Those analysis details are not reported in the registry record. This limits the ability to judge the precision and robustness of any outcome beyond the posted counts.
More broadly, the trial demonstrates why a registry record should be read as a record of design and posted results rather than as a substitute for a complete statistical report. It identifies the population, regimens, comparator, and safety observations, while leaving important analysis and outcome details unreported.
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 registry record does not report a statistical analysis plan, sample-size justification, allocation ratio, or randomization procedure. The sample-size assumptions associated with planning illustrations do not establish the study’s own design rationale. Although the record identifies quadruple masking, it does not describe the roles masked or procedures used to maintain masking.
The safety results provide counts by group, but the record does not report confidence intervals, formal comparisons, or detailed event-level results for the group summaries. It does not give a full breakdown of the primary safety outcome by grade, abnormal clinical evaluation, or abnormal laboratory assessment. The top serious-event terms are listed without a group assignment in the information available for this account.
The registry record does not report results for the listed FEV1 and CFTR-biomarker outcomes or detailed pharmacokinetic results. It also does not describe participant flow, withdrawals, missing-data handling, analysis populations, or sensitivity analyses. The planned dose-escalation rules and statistical approach to multiple outcomes or dose comparisons are not reported in the registry record.
These limitations restrict interpretation of the posted results to the observations and study features described in the registry record. They do not establish that unreported procedures or analyses were absent from the conduct of the study.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Safety and Pharmacokinetic Study of N6022 in Subjects With Cystic Fibrosis Homozygous for the F508del-CFTR Mutation — NCT01746784.
- Registry record last updated 2014-11-24; status last verified 2014-11.
- Results first posted 2014-11-24.
- 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.
Continue through the Clinical Biostats statistical pathway
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18. Record Summary
This completed randomized, quadruple-masked, parallel-group Phase 1 study evaluated N6022 in adults with cystic fibrosis homozygous for the F508del-CFTR mutation. The official title describes it as Phase 1b. N6022 was administered by intravenous infusion once per day for 7 days across dose groups labeled 5 mg, 10 mg, 20mg, and 40 mg; the comparator was normal saline administered intravenously at the same volume.
The primary outcome was safety and tolerability, assessed through abnormal clinical evaluations, abnormal laboratory assessments, and treatment-emergent adverse events. The posted safety results give serious and other adverse-event counts by group. Secondary outcomes include change in Percent Predicted FEV1 and change in CFTR-function biomarkers, but their results are not reported in the registry record used for this account. Statistical analyses, missing-data methods, and the sample-size justification are also not reported in the registry record.