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 New Onset Type 1 Diabetes: Role of Exenatide (NCT01269034), a randomized phase 4 study. The primary outcome is The Role of Exenatide as Compared to Insulin Monotherapy in Reducing Postprandial Hyperglycemia..
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT01269034 |
| Phase | Phase 4 |
| Condition | Type 1 Diabetes |
| Allocation | Randomized |
| Design model | Crossover |
| Masking | None (open label) |
| Enrollment | 13 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Albert Einstein College of Medicine |
| Results posted | Yes |
2. Clinical Question
Population
The study concerns people with new-onset type 1 diabetes. The brief summary says participants were to be diagnosed within 3 months and that some would be children aged 12-18 years. The registry records enrollment of 13. Healthy controls are also described, but they are not the target population for the primary treatment comparison.
Intervention
The registry describes exenatide given with insulin before a meal challenge called the “boost.” The brief summary specifies exenatide at 1.25 mcg and long-acting insulin. Another intervention description includes rapid- and long-acting insulin with 1.25 mcg exenatide. The precise relationship between the intervention descriptions is not clarified in the registry record.
Comparator
The primary question compares exenatide with insulin monotherapy in reducing postprandial hyperglycemia. The listed arms include insulin treatment without exenatide, as well as exenatide-plus-insulin conditions. Healthy controls without medication are also described, but they are not the comparator named in the primary outcome.
Primary question
The study asks whether adding exenatide to insulin changes post-meal hyperglycemia in people with new-onset type 1 diabetes. The registry gives the primary outcome’s title and a timeframe of February 2013, but no outcome measurements or treatment-effect estimate are available. Consequently, the record does not establish the direction or size of any difference.
3. Trial Design
The registry identifies NCT01269034 as a randomized, Phase 4, crossover study of exenatide in new-onset type 1 diabetes. It records enrollment of 13 and a completed status. The study start is listed as 2010-12, and primary completion as 2017-01-31. These administrative dates describe the registered study; they do not provide information about when individual participants entered each treatment condition.
A crossover design generally means that participants receive more than one treatment condition at different times, allowing comparisons within participants. This can reduce the influence of stable differences between people, but the interpretation depends on details such as treatment order, period length, washout, and how carryover is handled. Those details are not reported in the registry record. The record also does not provide the randomization sequence or explain how assignment was implemented.
The listed conditions include exenatide with insulin, insulin without exenatide, and healthy controls without medication before the “boost.” The brief summary describes a meal-related assessment with blood tests for glucose, insulin, and other hormones, but does not provide a full schedule or specify all measurements. The primary outcome compares exenatide with insulin monotherapy, rather than with the healthy-control condition.
The registry classifies masking as none. In contrast, the brief summary says that participants and researchers would not know which dose was being taken at any visit. These descriptions do not align, and the record does not explain the difference. The registry’s enrollment is 13, while the brief summary describes a planned total of 20 participants. The record does not explain why the final enrollment differs from that planned figure.
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: "Each participant only received one part (A,B, or C) and did not cross over to the subsequent parts
Overall Study
| Stage | Part A | Part B | Part C | Healthy Controls |
|---|---|---|---|---|
| STARTED | 1 | 2 | 1 | 9 |
| COMPLETED | 0 | 2 | 1 | 9 |
| NOT COMPLETED | 1 | 0 | 0 | 0 |
Analysis population for the primary outcome, as defined in the registry: The original PI and their entire team left the institution. Despite repeated efforts to contact the PI and study team members, the PI and study team members refused to disclose any collected data. Additionally, no secondary information sources such as study publications exist from which to summarize collected data
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: Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted.
| Characteristic | Unit | Part A | Part B | Part C | Healthy Controls | Total |
|---|---|---|---|---|---|---|
| Participants analysed | 1 | 2 | 1 | 9 | 13 | |
| Age, Categorical: <=18 years | Participants | 0 | 1 | 1 | 6 | 8 |
| Age, Categorical: Between 18 and 65 years | Participants | 1 | 1 | 0 | 3 | 5 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 | 0 | 0 |
| Sex: Female, Male: Female | Participants | 1 | 1 | 0 | 5 | 7 |
| Sex: Female, Male: Male | Participants | 0 | 1 | 1 | 4 | 6 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 1 | 1 | 0 | 5 | 7 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 0 | 1 | 1 | 4 | 6 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Asian | Participants | 0 | 0 | 0 | 1 | 1 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): White | Participants | 1 | 2 | 1 | 8 | 12 |
| Race (NIH/OMB): More than one race | Participants | 0 | 0 | 0 | 0 | 0 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as The Role of Exenatide as Compared to Insulin Monotherapy in Reducing Postprandial Hyperglycemia., measured over February 2013.
| Element | Registry entry |
|---|---|
| Result type | N/A |
| Measure of spread | not applicable |
| Unit | not stated |
| Endpoint classification | not reduced to a single comparison |
The primary outcome is titled “The Role of Exenatide as Compared to Insulin Monotherapy in Reducing Postprandial Hyperglycemia.” Postprandial hyperglycemia refers generally to elevated blood glucose after eating. Measuring glucose after a meal or meal challenge can address a question that a fasting measurement alone may not answer: how glucose changes in the period following food intake. The brief summary describes a “boost” and serial blood tests, but the exact endpoint definition, measurement scale, analysis time point, and summary measure are not reported in the registry record.
The endpoint is clinically and physiologically relevant to the study’s stated question because the summary focuses on after-meal glucose and describes exenatide as a possible addition to insulin. However, relevance does not establish that the endpoint was measured as planned or that a particular treatment effect occurred. The registry gives a timeframe of February 2013, but it reports no primary outcome value, variability measure, effect estimate, or confidence interval. It states that the data are no longer accessible after the principal investigator and study team left the institution, and that efforts to locate them were exhausted. The endpoint therefore cannot be interpreted quantitatively from this record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The central limitation for interpretation is the absence of primary outcome measurements. The registry names postprandial hyperglycemia as the primary question and gives February 2013 as its timeframe, but explicitly says the data are no longer accessible. No glucose values, contrast between treatment conditions, uncertainty interval, or test result are available. The direction and magnitude of the primary effect therefore cannot be established.
This absence should not be read as evidence of no effect. A null or inconclusive result would require outcome data and an analysis; neither is reported. Likewise, the lack of an estimate does not support a claim that exenatide improved or worsened post-meal glucose. The record says the principal investigator and team left the institution, that repeated efforts to contact them did not yield the collected data, and that no secondary information sources such as study publications exist from which to summarize the results.
The registry records enrollment of 13, while its brief summary had described a total of 20 participants. The record does not explain this difference or provide participant flow, treatment exposure, withdrawals, or the number included in the primary analysis. Those omissions prevent assessment of how many participants contributed information to each treatment comparison. They also limit evaluation of whether the crossover design was completed as intended.
Although adverse-event counts are listed, the reported at-risk groups are small and differ across the named conditions. These safety entries cannot substitute for the unavailable efficacy data, and they do not support broad conclusions about safety. The record gives no event descriptions among the serious adverse-event terms listed, and no deaths are recorded in the displayed groups.
For statistical interpretation, the distinction between a missing estimate and a precisely estimated null effect is essential. A confidence interval would communicate the range of effects reasonably compatible with the data under an analysis model; no such interval is available. The registry record therefore documents the research question and some design and safety details, but it does not permit a treatment-effect conclusion or an assessment of its precision.
9. Statistical Methods Explained
Randomization is intended to reduce systematic differences between treatment assignments. In a crossover study, comparisons may also use information from the same participant under different conditions. This can be statistically efficient when the measurements within a participant are meaningfully related. It also requires careful attention to treatment order, period effects, and carryover: a prior treatment may influence measurements made during a later period. These are general features of crossover analysis. The registry does not report the treatment sequence, washout procedures, period-specific measurements, or the method used to account for these issues.
The registry identifies the study as randomized and crossover, but does not report a statistical analysis plan. In particular, the analysis model, covariates, handling of repeated measurements, and method for estimating the primary treatment contrast are not reported in the registry record. A reader therefore cannot determine whether the primary comparison was based on within-participant contrasts, a model incorporating period and sequence, or another approach. The primary outcome data are also inaccessible, so no independent recalculation from summary statistics is possible.
For a continuous glucose outcome, an analysis would ordinarily need to define which measurements contribute to the post-meal summary and how those measurements are combined. A mean, a peak value, or an area-under-the-curve summary can represent different features of a glucose response. This is general statistical context, not a description of the analysis used in this trial: the registry does not state the outcome’s unit, parameter, dispersion measure, or precise endpoint calculation. Without that definition, even a reported number could be difficult to interpret or reproduce.
A confidence interval is a way to express uncertainty around an estimated treatment contrast. Its width depends in part on the amount of information and the variability of the measurements. A point estimate alone would not show how precisely the effect was estimated; a p-value alone would not describe the range of plausible effects. Neither an estimate nor its uncertainty is available for the primary outcome in this record. The statistical significance level named in the planning assumptions is 0.05, but that planning value is not evidence that a particular primary analysis was conducted using that threshold.
Over-dispersion is a general concern when observed variability exceeds what a statistical model assumes. If a model understates variability, standard errors can be too small and uncertainty can appear narrower than warranted. Conversely, a model suited to the data can better represent their variability. The registry does not report the type of primary outcome data, a fitted model, or diagnostics, so there is no basis for evaluating over-dispersion or other model assumptions in this study.
Sample-size calculations are planning tools, not evidence about the results. The registry’s planning assumptions describe a two-sided significance level of 0.05, equal allocation between the two arms, and a normal approximation. The calculation is described as using a stated fraction of the observed effect. Such calculations can illustrate how a target effect and design assumptions relate to a required sample size, but they cannot replace an analysis of observed outcomes. In particular, a sample size associated with a target power is not “observed power” and does not establish whether the trial detected an effect. The registry does not report a trial-specific sample-size justification, and the primary outcome effect needed to interpret the stated illustration is unavailable.
The record lists zero analyses. This indicates that no analysis entries are recorded in the registry, not that the study necessarily had no analyses outside the registry. The statistical plan, analysis population, treatment of missing measurements, and any interim analyses are not reported in the registry record. These gaps, together with unavailable primary data, prevent reconstruction or verification of the planned and performed analyses.
10. Multiplicity, Missing Data and Analysis Populations
The record names a primary outcome concerning postprandial hyperglycemia and secondary outcomes concerning postprandial glucagon, gastric emptying, glucose excursions, and measures in healthy controls. When a study evaluates multiple endpoints, time points, or comparisons, the chance of a chance-positive finding can increase if results are interpreted without accounting for the multiplicity. A prespecified hierarchy or adjustment can help address this issue. The registry does not report a multiplicity strategy, and no outcome analysis is available to determine how multiple measurements or comparisons were handled.
The record gives no analysis population definition, such as whether the primary analysis included all randomized participants or only those completing treatment conditions. It also does not report participant flow, missing outcome counts, reasons for missingness, or an imputation method. The primary outcome description says the data are no longer accessible; that is a barrier to evaluating the results, not a reported statistical method for handling participant-level missing observations.
The registry records zero analyses and does not give an analysis plan, interim-analysis information, or details of how crossover periods were handled. These items are not reported in the registry record. Without them, it is not possible to assess whether the reported outcome set reflects prespecified analyses, whether all randomized participants contributed to the intended contrast, or whether missing measurements could have affected an estimate. The named secondary outcomes do not resolve the absence of the primary data.
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 |
|---|---|---|---|
| Part A | 0 | 1 | 0.0% |
| Part B | 0 | 2 | 0.0% |
| Part C | 0 | 1 | 0.0% |
| Healthy Controls | 0 | 9 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Part A | 0 of 1 |
| Part B | 0 of 2 |
| Part C | 0 of 1 |
| Healthy Controls | 0 of 9 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 0% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Part A | 0 of 1 |
| Part B | 0 of 2 |
| Part C | 0 of 1 |
| Healthy Controls | 0 of 9 |
The registry lists serious adverse events, other adverse events, and deaths separately for Part A, Part B, Part C, and healthy controls. For Part A, 1 participant is listed as at risk for each event category, with 0 serious events, 0 other events, and 0 deaths. For Part B, 2 are listed as at risk for each event category, with 0 serious events, 0 other events, and 0 deaths. For Part C, 1 is listed as at risk for each event category, with 0 serious events, 0 other events, and 0 deaths. For healthy controls, 9 are listed as at risk for each event category, with 0 serious events, 0 other events, and 0 deaths.
These are the registry’s reported counts, not event rates or evidence that an event was impossible. The at-risk numbers are limited and differ by group, and the record does not explain the relationship between these group counts and total enrollment. No serious adverse-event terms are listed. The entries should therefore be read as a narrow account of what was recorded in these registry fields, not as a comprehensive safety assessment or a comparison establishing relative risk.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a randomized Phase 4 crossover study was registered to examine exenatide with insulin in new-onset type 1 diabetes, with a primary question about postprandial hyperglycemia compared with insulin monotherapy. It also documents the study’s completed status, enrollment of 13, named intervention conditions, and limited adverse-event counts. These are details about the record and its reporting, not a quantitative account of the primary treatment effect.
The most important boundary is that the primary outcome data are no longer accessible. There is no result from which to estimate how glucose differed between treatment conditions, how uncertain such an estimate might be, or whether the observed data were compatible with a range of effects. The record does not support a conclusion that exenatide improved, worsened, or had no effect on post-meal glucose. Absence of an estimate is not equivalent to a finding of equivalence or lack of effect.
The crossover design could, in general, support within-participant comparisons, but its interpretation depends on the treatment schedule and analysis. The registry does not report enough detail to assess period effects or carryover, and it does not provide participant flow or the number contributing to each contrast. The difference between the stated planned total of 20 and registry enrollment of 13 is not explained. The masking classification also differs from the masking described in the brief summary.
The adverse-event entries record zero events in the listed groups, but their small and varying at-risk counts limit what can be inferred. They do not provide a basis for a broad safety conclusion. Overall, the record describes an unanswered statistical question: the study’s registered objective is clear, but the outcome information needed to answer it is unavailable.
13. Why This Trial Matters Statistically
This trial is statistically notable because it illustrates the difference between a registered question and an interpretable result. A protocol-level objective can identify the population, treatment contrast, and outcome of interest, but a treatment-effect conclusion requires outcome data, a defined analysis, and an account of uncertainty. The registry describes the intended postprandial glucose comparison yet states that the primary data are no longer accessible. The question cannot be answered from registration details alone.
The crossover design also highlights why design labels are not enough to evaluate an analysis. Within-participant comparisons can be useful, but treatment order, period effects, and carryover may matter. The registry does not provide the information needed to assess these features or determine how they were modeled. The record’s classification as unmasked also contrasts with the brief summary’s statement that participants and researchers would not know the treatment at each visit. Clear, internally consistent design reporting is important for judging risk of bias and interpreting estimates.
Finally, small and unevenly reported safety denominators demonstrate why zero recorded events should be presented with their context. Counts alone do not establish the absence of risk, especially when the exposed group sizes are limited. The case also emphasizes the value of preserving outcome data and making analysis results available: without them, neither statistical precision nor the relationship between the study question and observed evidence can be assessed.
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 principal limitation is that the primary outcome data are unavailable. The registry states that the principal investigator and the full study team left the institution, that repeated efforts to contact them did not produce disclosure of collected data, and that efforts to locate the data were exhausted. It also states that no secondary information sources, such as study publications, exist from which to summarize the outcome. As a result, there is no primary estimate, uncertainty interval, or analysis to interpret.
Important design and reporting details are also absent. The registry does not describe treatment order, crossover periods, washout, carryover handling, the endpoint calculation, or the statistical model. It does not give participant flow, treatment exposure, missing measurement details, or the analysis population. The statistical plan and any interim analyses are not reported in the registry record.
Enrollment is recorded as 13, whereas the brief summary describes a planned total of 20 participants; the record does not explain the difference. The registry lists masking as none, while the brief summary describes participants and researchers as unaware of the treatment at a visit. The intervention descriptions also vary in how insulin and exenatide are combined, and their relationship is not clarified. These reporting inconsistencies limit reconstruction of the intended comparison.
The adverse-event entries give small, different at-risk counts across groups and no serious-event terms. They do not supply a broad safety profile. Taken together, the missing outcomes, absent analysis details, and inconsistencies in the descriptions prevent a reliable assessment of efficacy, precision, or the full conduct of the study.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: New Onset Type 1 Diabetes: Role of Exenatide — NCT01269034.
- Registry record last updated 2021-08-04; status last verified 2021-07.
- Results first posted 2021-08-04.
- 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.
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18. Record Summary
NCT01269034 is registered as a completed randomized Phase 4 crossover study sponsored by Albert Einstein College of Medicine. The registry records enrollment of 13, a study start of 2010-12, and primary completion on 2017-01-31. It concerns new-onset type 1 diabetes and compares exenatide with insulin monotherapy for a primary question about postprandial hyperglycemia.
The primary outcome timeframe is February 2013. The registry states that the primary data are no longer accessible and that no publication or other secondary source is available to summarize them. No primary effect estimate or analysis is reported. The registry lists zero serious adverse events, other adverse events, and deaths in the displayed groups, with small and varying at-risk counts. The record therefore documents the study’s objective and selected design and safety details, but does not permit quantitative interpretation of the primary outcome.