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 Study to Evaluate the Safety and Efficacy of Relamorelin in Participants With Diabetic Gastroparesis Study 02 (NCT03426345), a randomized phase 3 study. The primary outcome is Change From Baseline to Week 12 in the Weekly Diabetic Gastroparesis Symptom Severity Score (DGSSS).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03426345 |
| Phase | Phase 3 |
| Condition | Gastroparesis, Diabetes Mellitus |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Double |
| Enrollment | 311 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Allergan |
| Results posted | Yes |
2. Clinical Question
Population
Participants with diabetic gastroparesis were enrolled. The registry record reports enrollment of 311 participants and defines the primary-outcome analysis population as the modified intent-to-treat (mITT) population: randomized participants with at least one postbaseline assessment of the Diabetic Gastroparesis Symptom Severity Diary (DGSSD).
Intervention
Participants assigned to relamorelin received 10 μg by subcutaneous injection twice daily for up to 12 weeks, after a 2-week placebo run-in.
Comparator
The comparator was placebo-matching relamorelin, injected subcutaneously twice daily for up to 12 weeks after the same 2-week placebo run-in.
Primary question
The primary question was whether the change from baseline to Week 12 in the weekly Diabetic Gastroparesis Symptom Severity Score (DGSSS) differed between the relamorelin and placebo groups. A negative change from baseline indicates improvement on the score. The registry record does not report the arm-specific changes, the between-group comparison, or a statistical test result, so the clinical question cannot be answered from the posted results.
3. Trial Design
This was a Phase 3, randomized, double-blind, parallel-group, placebo-controlled study of relamorelin in participants with diabetic gastroparesis. The registry record identifies Allergan as the sponsor and reports enrollment of 311 participants. The study status is listed as terminated. The recorded study start was 2018-02-16, and the primary completion date was 2020-07-16. The registry record does not report why the study was terminated.
Before treatment, participants completed a 2-week placebo run-in. The relamorelin arm received 10 μg by subcutaneous injection twice daily for up to 12 weeks. The comparison arm received placebo-matching relamorelin by subcutaneous injection twice daily for up to 12 weeks. Randomization and double masking are reported, but the registry record does not describe the randomization method, allocation concealment, who was masked, or how masking was maintained.
The primary outcome was change from baseline to Week 12 in the weekly DGSSS. Participants recorded symptom severity daily in an electronic diary. The registry record also lists secondary outcomes concerning nausea, abdominal pain, bloating, and postprandial fullness responder criteria during each of the last 6 weeks of treatment, as well as several safety outcomes. It reports 12 outcome measures overall.
The registry record does not report the planned sample-size justification, a detailed statistical analysis plan, or outcome analysis results. No statistical analysis entries are reported in the record. The study design therefore identifies the intended comparison, but the posted information does not establish how the primary comparison was estimated or tested.
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
| Stage | Placebo | Relamorelin 10 μg |
|---|---|---|
| STARTED | 155 | 156 |
| Safety Population | 152 | 155 |
| COMPLETED | 137 | 139 |
| NOT COMPLETED | 18 | 17 |
Reasons for not completing this period:
| Reason | Placebo | Relamorelin 10 μg |
|---|---|---|
| Adverse Event | 4 | 4 |
| Lack of Efficacy | 1 | 0 |
| Withdrawal by Subject | 5 | 6 |
| Lost to Follow-up | 3 | 1 |
| Protocol Deviation | 3 | 4 |
| Reason Not Specified | 1 | 2 |
| Missing Completion Status | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: Modified Intent-to-treat (mITT) Population included all randomized participants with ≥1 postbaseline assessment of DGSSD.
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: Intent-to-treat (ITT) Population included all randomized participants.
| Characteristic | Unit | Placebo | Relamorelin 10 μg | Total |
|---|---|---|---|---|
| Participants analysed | 155 | 156 | 311 | |
| Age, Continuous | years | 54.1 (SD 12.13) | 55.8 (SD 12.07) | 55.0 (SD 12.11) |
| Sex: Female, Male: Female | Participants | 112 | 114 | 226 |
| Sex: Female, Male: Male | Participants | 43 | 42 | 85 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 54 | 51 | 105 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 101 | 105 | 206 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): American Indian or Alaska Native | Participants | 4 | 7 | 11 |
| Race (NIH/OMB): Asian | Participants | 1 | 2 | 3 |
| Race (NIH/OMB): Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Black or African American | Participants | 25 | 17 | 42 |
| Race (NIH/OMB): White | Participants | 123 | 127 | 250 |
| Race (NIH/OMB): More than one race | Participants | 2 | 3 | 5 |
| Race (NIH/OMB): Unknown or Not Reported | Participants | 0 | 0 | 0 |
6. Primary Endpoint
The registry lists the primary outcome as Change From Baseline to Week 12 in the Weekly Diabetic Gastroparesis Symptom Severity Score (DGSSS), measured over Baseline (Day-14 to Day-1) to Week 12.
| Element | Registry entry |
|---|---|
| Result type | Mean |
| Measure of spread | Standard Deviation |
| Unit | score on a scale |
| Endpoint classification | Continuous Endpoint |
The primary endpoint was change from baseline to Week 12 in the weekly Diabetic Gastroparesis Symptom Severity Score. The DGSSS was derived from the DGSSD, a daily electronic diary in which participants assessed nausea, abdominal pain, postprandial fullness, and bloating. The score was calculated from the weekly averages of those four symptom items.
Each symptom item used an 11-point ordinal scale, from 0, meaning no or no discomfort, to 10, meaning worst possible or most uncomfortable. The total DGSSS could range from 0, best, to 40, worst. A negative change from baseline indicates improvement. Baseline was defined as the average of the two weekly DGSSS values from the run-in period; the endpoint timeframe was Baseline (Day-14 to Day-1) to Week 12.
A composite symptom score can describe overall symptom burden across the selected symptoms, while daily diary entries provide repeated reports over the treatment period. In general, using a prespecified composite can make a broad symptom question measurable, but the interpretation depends on how the component items are combined and on the quality and completeness of diary assessments. The registry record gives the score construction but does not report the primary results, so the size and precision of any treatment-group difference cannot be assessed.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record defines a clear primary endpoint: change from baseline to Week 12 in the weekly DGSSS. It also describes the scoring direction, component symptoms, and mITT population. However, it does not report the observed endpoint values for either group, the estimated difference between groups, a confidence interval, or a p-value. Without those results, there is no posted estimate of the direction, magnitude, or statistical precision of the primary comparison to interpret.
The score ranges from 0, best, to 40, worst, and a negative change from baseline indicates improvement. That direction applies to a within-participant change. To assess the randomized comparison, the relevant evidence would include how change differed between the relamorelin and placebo groups, together with uncertainty around that estimate. The registry record does not report those quantities. A change within one group alone, even if it were reported, would not establish that the groups differed; the placebo group provides the randomized comparator for interpreting the treatment contrast.
The record describes the analysis population as randomized participants with at least one postbaseline DGSSD assessment. It does not report how many participants in each group contributed to the primary analysis, how many lacked postbaseline assessments, or how missing diary data were handled. The enrollment total of 311 is not itself a count of participants analyzed for the primary endpoint. The distinction matters because the analysis population and the handling of missing outcome data affect which participants contribute to the estimate and how robust that estimate may be.
The study is listed as terminated, but the registry record does not state the reason. Termination alone does not establish whether the primary endpoint was analyzed, whether the study stopped early for a particular reason, or whether the planned analysis changed. The record also does not report an interim analysis, stopping rule, or final statistical analysis plan. No conclusions about the cause of termination or its implications for the results can therefore be drawn from the posted information.
The record lists secondary symptom responder outcomes for nausea, abdominal pain, bloating, and postprandial fullness during each of the last 6 weeks of treatment. It does not report the responder definitions, group results, or statistical tests for those outcomes. It also lists safety outcomes, but the posted numerical safety information is limited to counts of participants affected by serious and other adverse events and deaths. The record does not provide primary efficacy estimates alongside those safety counts.
Accordingly, the posted information supports a description of the trial question, endpoint, and design, but not a quantitative assessment of efficacy. It does not establish whether the groups differed, how large any difference was, or how precisely it was estimated. Those conclusions require the outcome estimates and their uncertainty, which are not reported in the registry record.
9. Statistical Methods Explained
The registry record describes a randomized, double-blind, parallel-group comparison of relamorelin with placebo. Randomization is intended, in general, to make treatment assignment independent of participants’ characteristics, reducing systematic differences between groups at the start of a trial. It does not guarantee that groups will be identical, and its interpretation depends on the allocation process and analysis. The record does not describe the randomization sequence, allocation concealment, or methods used to preserve masking.
What does the primary score represent?
The primary measure is a continuous score formed from weekly averages of four symptom diary items. The listed summary measure is the mean, with standard deviation as the dispersion measure. In general, a mean describes the average score in a group, while a standard deviation describes the spread of individual scores around that mean. These summaries do not by themselves show the precision of a treatment comparison. Precision is commonly conveyed by a confidence interval around the estimated difference; no such estimate or interval is reported in the registry record.
The DGSSS combines nausea, abdominal pain, postprandial fullness, and bloating. Each symptom is rated on an 11-point ordinal scale, and the total score has a stated range from 0 to 40, with lower scores representing less symptom severity. A negative change from baseline indicates improvement. This direction is useful for reading an estimate, but no group-specific change or between-group estimate is reported, so the direction of the observed treatment contrast is not available.
Why compare change between randomized groups?
In general, a randomized treatment comparison focuses on the difference between outcomes in the assigned groups, rather than treating a change within one group as evidence of a treatment effect. Participants may change over time for reasons unrelated to the study treatment, including the course of the condition, participation in a trial, or other influences. A concurrent placebo group helps distinguish the treatment-group pattern from changes shared by both groups. The registry record identifies placebo as the comparator but does not report the estimated contrast or its uncertainty.
Baseline was defined using the average of the two weekly DGSSS values during the run-in period. Comparing change from that baseline to Week 12 makes the endpoint a measure of change over the specified treatment timeframe. The record does not state whether the primary analysis adjusted for baseline score, what model was used, or how repeated diary measurements were incorporated into the analysis. Those choices are not reported in the registry record and should not be inferred from the endpoint description.
What does a confidence interval add?
In general, a confidence interval gives a range of values compatible with the observed data under the statistical model used. It helps readers assess the precision of an estimate and the range of effects that remain plausible. It is not a guarantee that the true effect lies within that particular interval. A p-value, when reported, addresses compatibility of the data with a specified null hypothesis under the analysis assumptions; it does not measure the size or importance of an effect. Neither a confidence interval nor a p-value for the primary comparison is reported in the registry record.
What is the role of dispersion?
The listed endpoint uses a mean and standard deviation. In general, variation in individual scores affects how precisely a group mean or group difference can be estimated. If the distribution is markedly asymmetric or has features poorly represented by a mean and standard deviation, alternative summaries or models may be useful. The registry record does not report score distributions, diagnostic checks, or sensitivity analyses, so it is not possible to assess those features from the posted information.
For count outcomes, over-dispersion is a general concern when observed variability exceeds what a model assumes. It can make standard errors too small if ignored. This concept is not a reported finding for this study: the registry record does not provide models or dispersion diagnostics for count outcomes. The primary endpoint is described as a continuous score, and the record does not specify the statistical model used for it.
What do the planning assumptions mean?
The stated sample-size assumptions describe a planning illustration based on the observed effect, with a two-sided significance level of 0.05, equal allocation, and a normal approximation; the effect to detect is a stated fraction of the observed effect. Such calculations are planning illustrations, not observed power and not evidence that a trial had a particular chance of finding a result after its data were collected. They should not be mistaken for the original trial’s design justification. The registry record does not report the trial’s own sample-size justification, and it does not report an observed primary effect from which to interpret a planning illustration.
The record reports no statistical analysis entries. It therefore does not establish which model, variance assumptions, confidence level, hypothesis test, or covariate adjustment was used for the primary endpoint. Those methodological details are not reported in the registry record. The endpoint description alone is not enough to reconstruct the analysis or recalculate a valid treatment comparison.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists multiple outcomes, including the primary DGSSS change and secondary responder outcomes for nausea, abdominal pain, bloating, and postprandial fullness. The responder outcomes are described across each of the last 6 weeks of the 12-week treatment period. It also lists safety outcomes. Testing several outcomes or time-specific comparisons can increase the chance of at least one apparently unusual result when all tests are considered together. In general, statistical plans may address this through a prespecified testing sequence or adjustment, but the registry record does not report a multiplicity strategy.
No statistical analysis entries are reported, and the registry record does not specify which secondary outcomes were formally tested, how tests were ordered, or whether any adjustment was planned. It also does not report the responder definitions or results. Consequently, the multiplicity implications of the planned or completed analyses cannot be evaluated from the posted information.
The primary analysis population is described as the mITT population, including all randomized participants with at least one postbaseline DGSSD assessment. The record does not report the number of participants meeting that definition in either group or the number excluded from the primary analysis. It does not describe how missing diary entries, incomplete weekly scores, or missing Week 12 assessments were handled. Possible approaches can make different assumptions about unobserved outcomes; without the analysis details and missing-data information, the robustness of the primary result cannot be assessed.
The registry record also does not report whether analyses followed assigned treatment regardless of adherence, whether participants were excluded for protocol deviations, or whether sensitivity analyses examined alternative assumptions. These details are not reported in the registry record and cannot be inferred from the mITT label alone.
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 | 6 | 152 | 3.9% |
| Relamorelin 10 μg | 8 | 155 | 5.2% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Placebo | 0 of 155 |
| Relamorelin 10 μg | 0 of 156 |
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 | 14 of 152 |
| Relamorelin 10 μg | 13 of 155 |
Most frequent serious adverse event terms
| Event term | Organ system | Placebo | Relamorelin 10 μg |
|---|---|---|---|
| Cardiac failure congestive | Cardiac disorders | 1 of 152 | 1 of 155 |
| Dehydration | Metabolism and nutrition disorders | 1 of 152 | 1 of 155 |
| Diabetic ketoacidosis | Metabolism and nutrition disorders | 1 of 152 | 1 of 155 |
| Abdominal pain | Gastrointestinal disorders | 0 of 152 | 1 of 155 |
| Diarrhoea | Gastrointestinal disorders | 0 of 152 | 1 of 155 |
| Nausea | Gastrointestinal disorders | 0 of 152 | 1 of 155 |
| Small intestinal obstruction | Gastrointestinal disorders | 0 of 152 | 1 of 155 |
| Vomiting | Gastrointestinal disorders | 0 of 152 | 1 of 155 |
| COVID-19 | Infections and infestations | 0 of 152 | 1 of 155 |
| Pneumonia | Infections and infestations | 0 of 152 | 1 of 155 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Placebo | Relamorelin 10 μg |
|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | 9 of 152 | 5 of 155 |
| Urinary tract infection | Infections and infestations | 5 of 152 | 9 of 155 |
Serious events with the highest arm-level rate: vaginal haemorrhage (0.9%), cardiac failure congestive (0.7%), influenza (0.7%), sepsis (0.7%), upper respiratory tract infection (0.7%).
The registry record reports that 6 participants in the placebo group and 8 participants in the relamorelin 10 μg group experienced serious adverse events. For other adverse events, the corresponding counts were 14 and 13 participants. Deaths were reported for neither group. The numbers at risk for these summaries were 152 in the placebo group and 155 in the relamorelin group.
These are participant counts, not estimates of a causal effect. The counts alone do not describe event timing, duration, severity beyond the serious-event classification, recurrence, or the nature of each participant’s full clinical course. The registry record does not report statistical comparisons, confidence intervals, exposure-adjusted rates, or a detailed accounting of non-serious events. The counts should therefore be read as descriptive safety information, not as proof that one group had more or fewer risks.
The record lists serious adverse-event terms with percentages, including vaginal haemorrhage, cardiac failure congestive, influenza, sepsis, upper respiratory tract infection, and dehydration. These terms are not linked to specific treatment groups in the posted information. Their listed percentages cannot be interpreted as group-specific rates from the information reported.
12. What the Result Does, and Does Not, Mean
The study record establishes that a randomized, double-blind, placebo-controlled comparison was conducted to evaluate relamorelin in participants with diabetic gastroparesis. It defines a symptom-based primary endpoint, describes the run-in and treatment regimens, and identifies the population intended for the primary analysis. These registry entries allow readers to understand the trial’s question and the type of outcome it was designed to assess.
The record does not report the primary endpoint results. It therefore does not show whether the relamorelin group’s change differed from the placebo group’s change, what the estimated difference was, or how precisely that difference was estimated. The available information cannot establish a statistically significant difference, a clinically important difference, or the absence of a difference. A missing result in a registry record is not evidence that a treatment did or did not have an effect.
The registry record lists the study as terminated, but does not report the reason. That status does not by itself identify whether the planned analysis was completed, whether the trial ended early, or whether termination affected outcome reporting. The record also does not provide the statistical analysis plan or primary analysis results needed to evaluate those possibilities.
The safety counts offer descriptive information about participants affected by serious and other adverse events, and the record reports no deaths in either group. They do not provide a complete assessment of comparative safety, and they do not establish that an event was caused by treatment. The listed serious-event terms are not assigned to treatment groups in the posted information.
In general, a treatment effect should be interpreted using the estimated group contrast, its uncertainty, the analysis population, missing-data handling, and the prespecified analysis plan. Those elements are not reported for the primary outcome in this registry record. The appropriate conclusion is therefore limited: the trial’s design and outcome definitions are described, but its posted results do not permit a quantitative conclusion about the primary treatment comparison.
13. Why This Trial Matters Statistically
This trial illustrates why a clearly defined clinical endpoint is only one part of statistical interpretation. The registry record specifies how daily reports of nausea, abdominal pain, postprandial fullness, and bloating were combined into the weekly DGSSS, how baseline was defined, and what direction of change indicates improvement. Those details make the intended outcome understandable and help prevent misreading the score’s direction.
The randomized, double-blind, placebo-controlled design also identifies the intended basis for comparing groups. In general, randomization and masking can support a fair comparison by limiting systematic differences in assignment and reducing some sources of bias. But design features do not replace outcome estimates. Without the group results and their uncertainty, readers cannot assess whether the planned contrast was observed or how precisely it was estimated.
The record also makes visible several questions that matter for interpreting diary-based outcomes: which participants contributed to the analysis, how incomplete assessments were handled, what statistical model was used, and how multiple outcomes were addressed. The mITT population is defined, but the record does not report analysis counts, missing-data methods, or a multiplicity approach. These omissions limit reproducibility and make it difficult to judge the sensitivity of any result to analytic choices.
Finally, a terminated status and descriptive safety counts require careful interpretation. Neither explains the reason the study ended nor substitutes for a complete analysis of efficacy and safety. This record is a useful example of the distinction between a trial’s design and its results: the former can be described from registry information even when the latter are not reported.
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 main limitation for interpretation is that the registry record does not report the primary endpoint values or the comparison between groups. There is no reported estimate, confidence interval, or p-value for change in DGSSS from baseline to Week 12. The direction and magnitude of the observed primary outcome are therefore unknown from the posted information.
The record does not report why the study was terminated, whether the final analysis followed the original plan, or whether any interim analyses or stopping rules were used. It also does not report the randomization method, allocation concealment, or operational details of masking. Those omissions prevent fuller assessment of how the design was implemented.
The mITT definition is stated, but arm-specific analysis counts and missing-data methods are not reported. The record does not describe handling of incomplete diary entries, missing weekly scores, or participants without Week 12 assessments. It also does not report adherence, protocol deviations, baseline characteristics, or sensitivity analyses. Without these details, the composition of the analyzed groups and the robustness of any treatment estimate cannot be assessed.
The registry lists several secondary outcomes and safety measures but does not report their results, responder definitions, or a strategy for addressing multiple comparisons. The serious-event terms are not linked to treatment groups, and the safety summaries do not include statistical comparisons or exposure-adjusted rates. These limitations constrain interpretation of both efficacy and safety and should not be filled by assumptions about the protocol or analysis.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: Study to Evaluate the Safety and Efficacy of Relamorelin in Participants With Diabetic Gastroparesis Study 02 — NCT03426345.
- Registry record last updated 2021-08-06; status last verified 2021-07.
- Results first posted 2021-08-06.
- 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
NCT03426345 is a randomized, double-blind, parallel-group, placebo-controlled Phase 3 study of relamorelin in participants with diabetic gastroparesis. The registry record reports enrollment of 311 and lists Allergan as the sponsor. The study status is terminated; the recorded start was 2018-02-16, and the primary completion date was 2020-07-16.
After a 2-week placebo run-in, participants received relamorelin 10 μg or placebo-matching injections subcutaneously twice daily for up to 12 weeks. The primary endpoint was change from baseline to Week 12 in the weekly DGSSS, based on four daily diary symptom items. The primary analysis population was defined as randomized participants with at least one postbaseline DGSSD assessment.
The registry record does not report primary outcome results, inferential analyses, the reason for termination, or the planned sample-size justification. It reports descriptive adverse-event counts and no deaths in either group.