Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study: Complete Statistical Analysis

1. Trial at a Glance

This page covers the registry results for Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study (NCT02750501), a na na study. The primary outcome is Change From Baseline of Erythrocyte Omega-3 Index % (DHA+EPA).

2. Clinical Question

The study was intended to evaluate fat absorption alongside safety and tolerability during sustained RELiZORB use in patients with cystic fibrosis and exocrine pancreatic insufficiency. The posted primary outcome focuses on a blood-cell fatty-acid composition measure as an endpoint related to omega-3 status. It is not a direct numerical measure of all aspects of fat absorption, and the record does not give a broader clinical outcome as the primary endpoint.

Because the study has a single group, the primary question is descriptive: how did the measured endpoint change within the analyzed participants over the stated period? Without a concurrent comparator, a change over time cannot by itself distinguish the effect of the device and feeding regimen from other influences or the course of participants’ condition. The record does not report a numerical endpoint result, so the direction and magnitude of any observed change cannot be assessed.

3. Trial Design

ASSURE is described as a multicenter, open-label study of sustained RELiZORB use during enteral feeding in patients with cystic fibrosis and exocrine pancreatic insufficiency. The registry lists the study as completed and records enrollment of 49. It identifies one arm, with RELiZORB used alongside Impact Peptide 1.5. The arm description specifies enteral feedings ranging from 500 mL to 1,000 mL per feeding for 90 days.

The design is single group, with masking listed as none and allocation listed as NA. There is no concurrent control arm in the record. Thus, the study can describe measurements and events observed in the participants who received the regimen, but its design does not provide a randomized between-group contrast. The study title and summary emphasize absorption, safety, and tolerability with sustained use; the primary outcome listed is change in erythrocyte omega-3 index from Day 0 to Day 90.

The primary-outcome description says the intent-to-treat population comprised 39 subjects who received at least one exposure to RELiZORB. It also identifies an analysis group of 38 because one subject discontinued before Visit 3. These are the populations described for the primary analysis, not the same number as overall registry enrollment. The record does not explain the disposition of every enrolled participant or provide a participant flow account.

The adverse-event record separates an observation and run-in period, Day -14 to Day -1, from the RELiZORB treatment period, Day 0 to Day 90. The numbers at risk differ between these periods. These safety summaries should therefore be read within their stated periods and denominators, not as a controlled comparison. The registry lists eight outcome measures and zero analyses in the information. It does not report the protocol’s sample-size justification, a detailed statistical analysis plan, interim analyses, or a more complete account of endpoint analysis procedures.

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: 7-day Observation Period: Maintain usual enteral feeding (EF) regimen. 7-day Run-in period: Maintain usual EF volume up to a max of 1000 mL using standard formula for at least 5 days. 49 subjects signed consent; 5 screen failed; 44 started the observation period; 39 completed observation and run-in periods entering the treatment period.

Overall Study

Reasons for not completing this period:

Analysis population for the primary outcome, as defined in the registry: Intent to treat population = 39 subjects who received at least one exposure to RELiZORB. Analysis group is 38 due to one subject discontinuing prior to Visit 3.

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.

6. Primary Endpoint

The registry lists the primary outcome as Change From Baseline of Erythrocyte Omega-3 Index % (DHA+EPA), measured over Day 0 to Day 90.

The primary endpoint is change from baseline in erythrocyte omega-3 index, defined in the record as the percentage of omega-3 composition represented by DHA plus EPA in erythrocyte tissue. The measurement period is Day 0 to Day 90. The record specifies a least-squares mean as the parameter and standard error as the measure of dispersion, but it does not provide the estimated change or its standard error.

As a general statistical concept, a change-from-baseline endpoint compares a participant’s later measurement with their starting measurement. This can describe how a measure varies over the study period while taking the starting value into account. The erythrocyte measure offers a way to summarize DHA and EPA composition in red blood cells; it is the endpoint chosen in this record to represent change in omega-3 index during the feeding regimen. Its interpretation is limited to that measured composition and timeframe. It does not, on its own, establish a change in every aspect of fat absorption, symptoms, or health.

A least-squares mean is generally an estimated mean from a statistical model, potentially adjusted for terms specified in that model. The registry record does not report the model terms or other details needed to explain what adjustment, if any, was used. A standard error describes the precision of an estimate under the analysis; it is not the same as the spread of individual participants’ measurements. Since neither the estimate nor its standard error is reported, the endpoint’s observed magnitude and precision cannot be evaluated from the registry summary.

7. Why No Re-analysis Is Shown

8. Statistical Interpretation

9. Statistical Methods Explained

What does a single-group design estimate?

In a single-group study, measurements are collected in participants receiving the study regimen, without a concurrent control group. The design can describe what was observed in that group over time. It cannot directly estimate how the same participants would have changed under a different regimen or without the intervention. This distinction matters when interpreting a change-from-baseline endpoint: a within-group change is not automatically a causal treatment effect.

ASSURE is listed as single group, open label, with allocation recorded as NA and masking recorded as none. The study description specifies RELiZORB and Impact Peptide 1.5 during enteral feeding. The primary endpoint covers Day 0 to Day 90. Because the record does not describe a control arm, the primary endpoint is best understood as a planned assessment of change during use rather than a between-group comparison.

What is a change-from-baseline outcome?

A change-from-baseline outcome is formed by comparing a later measurement with the participant’s starting measurement. It can summarize the direction and average size of change across the analyzed population. The outcome is erythrocyte omega-3 index, expressed as percentage of omega-3 composition and described as DHA plus EPA. It represents a specific biological measurement, not a complete measure of clinical status or all dimensions of fat absorption.

General statistical interpretation depends on how the endpoint is summarized and modeled. The registry identifies the parameter as a least-squares mean. Generally, a least-squares mean is a model-based estimate of a mean, but its exact interpretation depends on the model and terms included. Those details are not reported in the registry record, so readers cannot determine from this entry whether the estimate was adjusted or what model assumptions were used.

What does the standard error tell us?

The record names standard error as the dispersion measure for the primary endpoint. In general, a standard error describes the estimated precision of a statistic, such as a mean, under the analysis. It differs from a standard deviation, which describes variability among individual observations. A smaller standard error generally indicates a more precise estimate under the assumed model, while a larger one indicates more uncertainty. The registry does not provide the endpoint estimate or its standard error, so precision cannot be assessed.

Confidence intervals are another general way to express uncertainty around an estimate. They combine a point estimate with its uncertainty to show a range compatible with the data and method. No confidence interval is reported for the primary outcome in this record. Nor is a p-value or test result posted in the posted summary, so statistical significance cannot be evaluated from this entry.

Why does the analysis population matter?

The primary-outcome description defines an intent-to-treat population as 39 subjects who received at least one exposure to RELiZORB. It separately states that the analysis group is 38 because one subject discontinued before Visit 3. These descriptions indicate that the analyzed population was not identical to the exposure-defined group. The record does not state how the distinction was handled in the statistical model, whether any measurements were missing, or what method was used to address missing data.

In general, an analysis population determines which participants contribute to a particular estimate. Excluding participants or having incomplete outcome measurements can affect both the estimate and its precision, especially if the reasons for missing data relate to outcomes. The registry does not provide enough information to assess those possibilities. The stated group sizes should therefore be reported as written, without assuming that all participants had complete measurements or that a particular missing-data method was used.

How should the safety periods be read?

The safety information is divided into an observation and run-in period, Day -14 to Day -1, and a RELiZORB treatment period, Day 0 to Day 90. Each period has its own number at risk and counts of participants with serious and other adverse events. These are period-specific descriptive counts. They are not a randomized comparison, and the change in denominators means the periods should not be treated as if they involved identical participant sets. General comparisons of safety rates would require careful attention to exposure time, definitions, and participant follow-up; those details are not reported.

The posted sample-size section is a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation. It describes planning for a stated fraction of the observed effect. The values are planning illustrations, not observed power, and they do not change the fact that this trial itself is single group. The record does not report the trial’s own sample-size justification or enough primary outcome detail to independently assess the assumptions behind the illustration.

What analysis details remain unavailable?

The registry lists eight outcome measures and zero analyses in the information. It does not report the statistical test for the primary endpoint, model specification, handling of repeated measurements, covariate adjustment, confidence intervals, or multiplicity strategy. Those methods should not be inferred from the least-squares mean label alone. General principles can explain what these methods might do, but the method actually used in this study is not reported in the registry record.

10. Confidence Intervals for Each Arm

No two-arm comparison is shown for this trial, but each arm's mean can be given with a t-based 95% confidence interval computed from the posted mean, spread and number of participants.

R code and output

arm <- c("RELiZORB Cartridge With Standard Enteral Formula")
n   <- c(38)
m   <- c(5.01)
sp  <- c(0.4)
sd  <- sp * sqrt(n)      # standard errors converted to SDs
for (i in seq_along(n)) {
  se <- sd[i] / sqrt(n[i]); h <- qt(0.975, n[i] - 1) * se
  cat(sprintf("CARM|%s|%d|%.3f|%.3f|%.3f|%.3f\n", arm[i], n[i], m[i], sd[i], m[i] - h, m[i] + h))
}

CARM  RELiZORB Cartridge With Standard Enteral Formula  38  5.010  2.466  4.200  5.820

11. Multiplicity, Missing Data and Analysis Populations

The registry lists eight outcome measures, including the primary erythrocyte omega-3 index endpoint and secondary measures related to adverse device effects, plasma DHA plus EPA, erythrocyte DHA and EPA composition, and fatty-acid ratios. It does not report how statistical multiplicity across outcomes was handled. In general, testing or estimating many outcomes can increase the chance of apparently notable findings arising by chance. A prespecified hierarchy, adjustment, or descriptive-only approach may address this issue, but the approach used is not reported in the registry record.

The record identifies an intent-to-treat population of 39 subjects exposed to RELiZORB and an analysis group of 38, with one subject discontinuing before Visit 3. It does not describe the endpoint data available for each participant, the reason for discontinuation, or how missing outcome measurements were handled. No imputation method or sensitivity analysis is reported. Therefore, readers cannot evaluate whether missing data affected the primary estimate or whether conclusions would be robust to alternative assumptions.

The posted information also does not report the primary statistical model, a detailed analysis plan, interim analyses, or the trial’s own sample-size justification. These omissions limit assessment of prespecification and of how the analysis was conducted. They do not establish that a particular method was or was not used; those details are simply not reported in the registry record.

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: infectious colitis (2.3%), infective pulmonary exacerbation of cystic fibrosis (2.3%).

The safety table separates the observation and run-in period, Day -14 to Day -1, from the RELiZORB treatment period, Day 0 to Day 90. In the run-in period, 2 of 44 participants at risk had serious adverse events and 4 of 44 had other adverse events; no deaths are recorded. In the treatment period, 0 of 39 participants at risk had serious adverse events and 23 of 39 had other adverse events; no deaths are recorded. These are counts of participants with events, not estimates of comparative risk.

The record lists Infectious colitis and Infective pulmonary exacerbation of cystic fibrosis among the top serious-event terms, each at 2.3%, for the run-in period. The table does not give a corresponding treatment-period serious-event term because no participants in that period are recorded with a serious adverse event. The event summaries do not show whether an event was caused by the device or feeding regimen. In general, an adverse event occurring during a study is not by itself evidence of causation.

The denominators differ between periods, and the record does not provide a controlled comparison or detailed exposure-adjusted analysis. The figures should therefore be read as descriptive safety observations in the stated periods, not as proof of comparative safety or risk.

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

This record documents a completed, open-label, single-group study of RELiZORB used with Impact Peptide 1.5 during enteral feedings in patients with cystic fibrosis. The arm description specifies feedings ranging from 500 mL to 1,000 mL per feeding over 90 days. The primary endpoint is change in erythrocyte omega-3 index from Day 0 to Day 90, defined as the percentage of DHA plus EPA in erythrocyte tissue composition.

The registry record does not report the numerical primary endpoint result, its standard error, a confidence interval, or a p-value. It therefore does not show how large or precise the endpoint change was. This absence should not be interpreted as evidence that there was no change. The posted information simply does not provide the result needed to describe it.

Even if a numerical before-and-after change were available, this design would not establish that the change was caused by the study regimen. There is no concurrent control group, and the study is not described as randomized. A single-group result can describe what happened among participants during the study period, but cannot separate the intervention from other influences on change over time.

The safety table reports period-specific counts, including zero serious adverse events among 39 participants at risk during the treatment period, and 23 participants with other adverse events among 39 at risk. The run-in period has different denominators and separate event counts. These observations do not provide a controlled safety comparison, nor do they establish whether events were related to the regimen.

In short, the registry entry provides the design, population definitions, endpoint description, and selected safety counts. It does not provide the numerical efficacy result or enough analysis detail to evaluate its precision, and its single-group design limits causal interpretation.

14. Why This Trial Matters Statistically

This study illustrates why outcome definitions and reported estimates need to be read together. The registry names a specific primary endpoint, describes it as a least-squares mean change, and identifies standard error as the dispersion measure. Those details tell readers what the investigators intended to summarize and how precision might be expressed. Yet without the estimated change and uncertainty, the endpoint’s observed result cannot be interpreted quantitatively.

The record also makes the distinction between enrollment and analysis populations important. Overall enrollment is 49, while the primary-outcome description identifies 39 exposed subjects and an analysis group of 38. The stated reason for the difference between the latter groups is one discontinuation before Visit 3. Transparent accounting of who contributes to an endpoint helps readers judge the scope of an estimate; further participant flow and missing-data details are not reported.

Finally, this single-group, open-label design shows why a within-group change should not be confused with a controlled treatment effect. The study can describe outcomes and adverse events observed during sustained use, but there is no concurrent control arm for a like-for-like comparison. Statistical interpretation must reflect that design constraint, as well as the absence of a posted primary result and analysis details. The planning illustration elsewhere on the page is not observed power and does not substitute for an outcome estimate or a control group.

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 for interpreting the primary endpoint is that its numerical result is not reported in the registry record. Although the endpoint, timeframe, parameter, and dispersion measure are named, there is no posted estimate, standard error, confidence interval, or p-value. The direction, magnitude, and precision of change therefore cannot be assessed from this record.

The design is single group and open label, with no concurrent control arm. This limits causal interpretation of change over time and makes a like-for-like comparative treatment estimate unavailable. The registry does not report randomization, and allocation is listed as NA. It also does not report details of the statistical model or the analysis plan.

The primary-outcome description gives 39 exposed subjects and an analysis group of 38, noting one discontinuation before Visit 3. The record does not explain the disposition of all enrolled participants, provide a full participant flow, or report how missing outcome data were handled. It also does not describe the prespecified approach to multiple outcomes or provide the trial’s sample-size justification.

Safety results are descriptive and split between run-in and treatment periods with different denominators. The registry does not provide a controlled or exposure-adjusted safety comparison, and the event counts alone do not establish causation. These limitations are specific to the information and design described in this record; additional details are not reported in the registry record.

16. Related Tutorials

17. Related Statistical Calculators

18. Sources

• ClinicalTrials.gov: Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study — NCT02750501.
• Registry record last updated 2018-08-15; status last verified 2018-06.
• Results first posted 2018-07-18.
• 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

ASSURE is a completed, multicenter, open-label, single-group study with enrollment of 49. It evaluated sustained use of a RELiZORB immobilized lipase cartridge with Impact Peptide 1.5 during enteral feeding in patients with cystic fibrosis and exocrine pancreatic insufficiency. The arm description specifies feedings from 500 mL to 1,000 mL per feeding for 90 days.

The primary endpoint was change from baseline in erythrocyte omega-3 index, DHA plus EPA, from Day 0 to Day 90. The record identifies 39 exposed subjects and an analysis group of 38, but does not report the numerical primary result. Safety counts are reported separately for run-in and treatment periods. The registry does not describe a control group, detailed analysis methods, or missing-data handling.