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 Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease (NCT02187003), a randomized phase 3 study. The primary outcome is Time to Readiness for Discharge From Hospital.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02187003 |
| Phase | Phase 3 |
| Condition | Anemia, Sickle Cell |
| Allocation | Randomized |
| Design model | Parallel |
| Masking | Quadruple |
| Enrollment | 345 |
| Arms in the primary outcome | 2 |
| Lead sponsor | Biossil Inc. |
| Results posted | Yes |
2. Clinical Question
Population
The trial concerned people with sickle cell disease who were hospitalized for a vaso-occlusive crisis. The registry summary describes eligibility from age 6 years. Participants aged 12 years and above were identified as Cohort 1, while those aged 6-11 years were identified as Cohort 2. The primary analysis and study conclusions were based on the full study population.
Intervention
The intervention was rivipansel, also identified as GMI-1070. The registry describes the study drug as administered by infusion, including a loading dose used as the time origin for the primary endpoint. The treatment-arm description does not report further dosing details in the registry record.
Comparator
The comparator was placebo. The study used randomized assignment and a parallel-group design. The registry identifies quadruple masking, but does not report the specific roles included in that masking designation in the registry record.
Primary question
The primary question was whether the time from the first infusion of study drug to meeting all applicable readiness-for-discharge criteria differed between the rivipansel and placebo groups. The posted primary analysis reports a hazard ratio, confidence interval, and p-value from a log-rank analysis. Those statistics describe the trial’s comparison; they do not by themselves establish a treatment effect or its clinical importance.
3. Trial Design
This was a phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group study of rivipansel for hospitalized people with sickle cell disease experiencing a vaso-occlusive crisis. The registry lists an enrollment of 345 and records the study as completed. The listed start date is 2015-06-17, and the primary completion date is 2019-05-03. Biossil Inc. is named as sponsor.
The registry identifies two treatment arms: rivipansel and placebo. It describes the masking as quadruple, but the specific people or roles covered by that term are not reported in the registry record. The arm descriptions do not provide additional treatment or placebo details. The registry summary says participants were 6 years of age or older and had a pain crisis requiring hospitalization. The primary population included randomized participants; age cohorts were identified as 12 years and above and 6-11 years, while the primary analysis and study conclusions were based on the full study population.
The primary endpoint was time to readiness for discharge from hospital, measured in hours from the start of the first infusion to the time all applicable readiness criteria were met. The registry also names outcomes concerning hospital discharge, intravenous opioid use, and rehospitalization for vaso-occlusive crisis. Although those outcomes describe different aspects of the hospital course, their definitions and analytic details are not all reported in the registry record.
The registry record does not report the rationale for the planned enrollment, a detailed statistical analysis plan, or details about interim analyses. The posted results should therefore be interpreted using the analyses and population descriptions actually recorded, rather than assumptions about unreported design choices.
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: Due to the short duration of time allowed to dose upon a VOC event, an optional screening may have occurred while the participant was well. Patients were neither enrolled nor randomized to study treatment until a VOC occurred, at which time the formal assessment of eligibility for enrollment into the study was performed. Of the 475 screened participants, 345 participants experiencing an acute VOC event were randomized.
Overall Study
| Stage | Rivipansel Treatment Arm | Placebo Treatment Arm |
|---|---|---|
| STARTED | 173 | 172 |
| Treated | 162 | 158 |
| COMPLETED | 146 | 141 |
| NOT COMPLETED | 27 | 31 |
Reasons for not completing this period:
| Reason | Rivipansel Treatment Arm | Placebo Treatment Arm |
|---|---|---|
| Adverse Event | 2 | 0 |
| Lost to Follow-up | 14 | 10 |
| Withdrawal by Subject | 3 | 7 |
| Protocol Violation | 2 | 4 |
| Other | 5 | 5 |
| Withdrawal by Parent/Guardian | 1 | 5 |
Analysis population for the primary outcome, as defined in the registry: Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective.
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: Full analysis set population included all participants who were randomized in the study. Participants aged 12 years and above were identified as Cohort 1 and participants aged 6-11 years were identified as Cohort 2; however, the primary analysis and study conclusions were based on the full study population in alignment with the primary study objective. Full analysis set population included all participants who were randomized in the study.
| Characteristic | Unit | Rivipansel Treatment Arm | Placebo Treatment Arm | Total |
|---|---|---|---|---|
| Participants analysed | 173 | 172 | 345 | |
| Age, Continuous | years | 22.00 (SD 10.61) | 21.34 (SD 10.20) | 21.67 (SD 10.40) |
| Sex: Female, Male: Female | Participants | 84 | 99 | 183 |
| Sex: Female, Male: Male | Participants | 89 | 73 | 162 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 7 | 16 | 23 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 165 | 155 | 320 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 1 | 1 | 2 |
| Race/Ethnicity, Customized: Race: Black | Participants | 167 | 159 | 326 |
| Race/Ethnicity, Customized: Race: White | Participants | 0 | 6 | 6 |
| Race/Ethnicity, Customized: Race: Other | Participants | 6 | 7 | 13 |
6. Primary Endpoint
The registry lists the primary outcome as Time to Readiness for Discharge From Hospital, measured over Day 1 up to the latest day when all 6 criteria of readiness-for-discharge were met (up to an average of Day 8).
| Element | Registry entry |
|---|---|
| Result type | Median |
| Measure of spread | 95% Confidence Interval |
| Unit | Hours |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint was time to readiness for discharge from hospital, measured in hours. The clock began at the date and time of the first study-drug infusion, described as the loading dose. The endpoint time was reached when all applicable readiness-for-discharge criteria had been documented. The criteria addressed oral pain medication, resolution of acute complications related to the crisis, discontinuation of intravenous opioids, intravenous hydration and intravenous antibiotics, and no longer requiring red blood cell transfusion for treatment of the crisis.
This endpoint combines several components of the hospital course into a time-to-event measure. In general, time-to-event analysis is useful when the outcome is the time until a defined event occurs and participants may reach that event at different times. A readiness criterion can also capture more than the physical act of leaving hospital: the registry definition requires that all applicable clinical and treatment conditions be met. That distinction matters when interpreting the endpoint, because readiness for discharge and actual discharge are not identical outcomes.
The registry gives the timeframe as Day 1 up to the latest day when all 6 criteria were met, up to an average of Day 8. The endpoint is reported as a median with a 95% confidence interval for each arm. The registry record does not report further details about handling participants who did not meet the endpoint or about missing endpoint data.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The posted median time to readiness for discharge was 87.78 hours in the rivipansel arm, with a 95% confidence interval from 65.68 to 100.2 hours. In the placebo arm, the median was 93.47 hours, with a 95% confidence interval from 74.67 to 109.7 hours. These arm-specific summaries describe the observed time distributions; they are not, on their own, a test of the between-arm difference.
The registry’s primary comparison used a log-rank method and reported a hazard ratio of 0.97, with a 95% confidence interval from 0.77 to 1.22 and p = 0.794. In general, a hazard ratio compares the rates of reaching the defined event over time, subject to the model and analysis assumptions. Its interpretation depends on how the event and comparison are defined. The confidence interval includes values on either side of 1, so the estimate is compatible with a range of possible relative event rates. The p-value is not evidence that the groups were identical; it describes how unusual the observed data would be under the test’s null hypothesis and assumptions.
The interval is also important for judging precision. It spans estimates that could correspond to different directions and magnitudes of association. The reported p-value does not resolve that uncertainty, nor does it establish whether any difference would be clinically important. The registry does not report a clinically meaningful threshold for this endpoint, so the clinical importance of particular values cannot be determined from the posted record alone.
The reported medians and the log-rank hazard ratio answer related but not identical questions. Medians locate the middle of each observed time distribution, while the log-rank analysis compares event timing across follow-up. The reported summaries should therefore be read together without treating the difference between the medians as a substitute for the sponsor’s time-to-event analysis. No new contrast between the medians is calculated.
The primary analysis population is described as the full analysis set, including all participants randomized in the study. The record separately identifies the age cohorts but says the primary analysis and study conclusions were based on the full study population. Further details about missing data, censoring, or adjustments are not reported in the registry record. Those omissions limit assessment of how the analysis handled participants whose readiness time may not have been observed in the same way as others.
9. Statistical Methods Explained
The registry reports the primary comparison using a log-rank method and gives a hazard ratio with a 95% confidence interval and p-value. The outcome is time from the first infusion to meeting the readiness-for-discharge definition. In general, time-to-event methods use both whether an event occurred and when it occurred. They can account for observations that end before the event is recorded, but the specific handling of such observations must be reported to evaluate the analysis fully. That handling is not reported in the registry record.
What does the hazard ratio describe?
In general, a hazard is the rate at which an event occurs among participants who have not yet experienced it, at a particular point in follow-up. A hazard ratio compares those rates between groups according to the analysis definition. A value of 1 represents equal rates under that parameterization; values on either side represent different relative rates. The registry reports a hazard ratio of 0.97. This estimate is close to 1, but its uncertainty must be considered rather than reading the point estimate alone.
The 95% confidence interval is 0.77 to 1.22. In general, a confidence interval expresses uncertainty in an estimate under the method’s assumptions. It is not a guarantee that the true value lies in the interval, and it does not assign a probability to the true value after the data have been observed. Here, the interval spans 1 and includes values on both sides. The result therefore does not precisely distinguish the estimated relative event rate from equality, and it remains compatible with a range of associations.
Why also report medians?
A median is the point that divides an observed distribution so that half of the observations are on either side, according to the applicable definition. The registry reports medians of 87.78 hours for rivipansel and 93.47 hours for placebo, each with a 95% confidence interval. These are summaries of each arm separately. The log-rank test instead uses event timing across the follow-up period. In general, a comparison based on the full event-time information can differ from a comparison based only on the two medians. The registry record does not report enough detail to reconstruct the full event-time analysis.
How should the p-value be read?
The primary p-value is 0.794. In general, a p-value measures how compatible the observed data are with a specified null hypothesis, given the statistical method and its assumptions. It is not the probability that the null hypothesis is true, and it does not measure the size or importance of an effect. A relatively large p-value does not prove the groups are equivalent. Equivalence requires a suitable design, a prespecified margin, and an analysis directed at that question; those details are not reported in the registry record.
The registry also reports a secondary comparison of rehospitalization within 3 days of discharge. A simple unadjusted re-calculation uses the posted counts and arm sizes and reports a risk difference of -0.012, with a confidence interval from -0.043 to 0.02 and p = 0.469. The registry analysis for this endpoint uses the Chan and Zhang method and reports a difference in percentage of participants of -1.17, with a 95% confidence interval from -5.19 to 2.46 and p = 0.535. These analyses use different methods and display the estimate on different scales. The registry’s own analysis should be distinguished from the unadjusted re-calculation.
Why does over-dispersion matter in general?
Over-dispersion occurs when observed variation is greater than a statistical model expects. If a model assumes less variability than the data contain, standard errors can be too small and confidence intervals too narrow, making results appear more precise than warranted. This is a general issue for analyses of counts or rates when the model’s variance assumptions may not fit. The registry record does not identify an over-dispersion issue or describe a related model for the primary endpoint; it should not be presumed to have occurred in this trial.
What do planning illustrations mean?
In general, sample-size planning estimates how many participants per arm might be needed to detect a specified effect under stated assumptions. The registry’s planning illustration is based on the observed rehospitalization effect, uses a two-sided significance level of 0.05, assumes equal allocation, and uses a normal approximation. It gives scenarios for planning for 100%, 75%, and 50% of the observed effect. The listed planning values are participants per arm for 80% power and participants per arm for 90% power. They are illustrations, not observed power, and they are not evidence that the original study was designed using those assumptions. The trial’s own sample-size justification is not reported in the registry record.
10. Analyses Reported by the Sponsor
| Method | Estimate | 95% CI | P-value |
|---|---|---|---|
| Log Rank | Hazard Ratio (HR) 0.97 | 0.77 to 1.22 | 0.794 |
The sponsor’s primary analysis is the registry-reported log-rank comparison for time to readiness for discharge: hazard ratio 0.97, 95% confidence interval 0.77 to 1.22, and p = 0.794. The arm-specific medians and confidence intervals provide descriptive context, while the hazard ratio and its p-value report the stated time-to-event comparison. The registry record does not report further details of the analysis plan that would allow a fuller audit of its implementation.
For rehospitalization within 3 days of discharge, the registry reports a Chan and Zhang analysis, with a difference in percentage of participants of -1.17, a 95% confidence interval from -5.19 to 2.46, and p = 0.535. The unadjusted re-calculation is a separate analysis of the posted summary counts. It reports a risk difference of -0.012, a confidence interval from -0.043 to 0.02, and p = 0.469. The estimates are displayed on different scales and arise from different methods, so they should not be treated as identical calculations. The registry analysis is the sponsor-reported result; the re-calculation is a supplementary, unadjusted reading of the posted summaries.
The record does not flag a discrepancy between the posted and recalculated result. Neither analysis should be interpreted as proving equivalence or as establishing a clinically important difference. The uncertainty intervals include no difference, and the registry’s endpoint-specific analysis remains the relevant reported analysis for the trial record.
11. Multiplicity, Missing Data and Analysis Populations
The registry record lists 15 outcome measures and 8 analyses. It identifies a primary endpoint and several secondary outcomes, but does not report a complete statistical analysis plan, a testing hierarchy, or an adjustment for multiplicity. In general, multiplicity matters when several hypotheses are tested: without a planned approach, isolated p-values can be difficult to interpret because repeated testing increases the chance of at least one apparently notable result under a collection of null hypotheses. The record does not establish whether or how this issue was addressed.
Details about missing data are also not reported in the registry record. For the primary endpoint, the record defines the time origin and readiness event and identifies the full analysis set as including randomized participants. It does not describe how missing readiness assessments, incomplete follow-up, or participants who did not meet the endpoint were handled. The specific censoring rules and any sensitivity analyses are not reported in the registry record. These details matter in general because time-to-event estimates depend on how observation periods end and on the assumptions used for participants without a recorded event.
The registry describes the primary analysis population as the full analysis set, including all randomized participants. It also identifies Cohort 1 as participants aged 12 years and above and Cohort 2 as participants aged 6-11 years, while stating that the primary analysis and study conclusions were based on the full study population. The record does not provide additional subgroup estimates in the registry record reported for this analysis, and no age-specific conclusions can be drawn from the overall result alone.
The registry does not report interim analyses, stopping rules, or a sample-size justification. The planning illustration based on the observed rehospitalization effect is not a substitute for a trial-design justification and should not be interpreted as observed power. Without these details, the posted record permits interpretation of the reported estimates but not a complete assessment of all design and analysis decisions that may affect their evidential strength.
12. 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 |
|---|---|---|---|
| Rivipansel Treatment Arm | 52 | 162 | 32.1% |
| Placebo Treatment Arm | 49 | 158 | 31.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Rivipansel Treatment Arm | 0 of 162 |
| Placebo Treatment Arm | 0 of 158 |
Other (non-serious) adverse events
Participants with at least one non-serious event at or above the registry's 2% reporting threshold.
| Arm | Participants affected of at risk |
|---|---|
| Rivipansel Treatment Arm | 137 of 162 |
| Placebo Treatment Arm | 124 of 158 |
Most frequent serious adverse event terms
| Event term | Organ system | Rivipansel Treatment Arm | Placebo Treatment Arm |
|---|---|---|---|
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | 37 of 162 | 34 of 158 |
| Acute chest syndrome | Respiratory, thoracic and mediastinal disorders | 8 of 162 | 6 of 158 |
| Headache | Nervous system disorders | 2 of 162 | 1 of 158 |
| Anaemia | Blood and lymphatic system disorders | 0 of 162 | 2 of 158 |
| Constipation | Gastrointestinal disorders | 2 of 162 | 0 of 158 |
| Influenza | Infections and infestations | 1 of 162 | 1 of 158 |
| Pneumonia | Infections and infestations | 0 of 162 | 2 of 158 |
| Transaminases increased | Investigations | 1 of 162 | 1 of 158 |
| Bone infarction | Musculoskeletal and connective tissue disorders | 2 of 162 | 0 of 158 |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | 1 of 162 | 1 of 158 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Rivipansel Treatment Arm | Placebo Treatment Arm |
|---|---|---|---|
| Pyrexia | General disorders | 29 of 162 | 33 of 158 |
| Nausea | Gastrointestinal disorders | 26 of 162 | 27 of 158 |
| Constipation | Gastrointestinal disorders | 29 of 162 | 21 of 158 |
| Headache | Nervous system disorders | 18 of 162 | 29 of 158 |
| Pruritus | Skin and subcutaneous tissue disorders | 24 of 162 | 17 of 158 |
| Vomiting | Gastrointestinal disorders | 17 of 162 | 16 of 158 |
| Anaemia | Blood and lymphatic system disorders | 18 of 162 | 11 of 158 |
| Sickle cell anaemia with crisis | Blood and lymphatic system disorders | 12 of 162 | 17 of 158 |
| Haemoglobin decreased | Investigations | 9 of 162 | 12 of 158 |
| Chest pain | General disorders | 9 of 162 | 8 of 158 |
Serious events with the highest arm-level rate: sickle cell anaemia with crisis (22.8%), acute chest syndrome (4.9%), anaemia (1.3%), pneumonia (1.3%), respiratory disorder (1.3%).
The registry reports serious adverse events among participants at risk in each arm. In the rivipansel arm, 52 of 162 participants at risk were affected; in the placebo arm, 49 of 158 were affected. The registry also reports other adverse events: 137 of 162 participants at risk in the rivipansel arm and 124 of 158 in the placebo arm. No deaths are reported in either arm.
These counts describe the posted safety record, but they do not by themselves establish that an event was caused by study treatment. The number of affected participants is not the same as the number of event episodes, and the registry record does not provide further detail about event duration, severity beyond the serious-event classification, timing, or attribution. The listed serious-event terms include sickle cell anaemia with crisis, acute chest syndrome, anaemia, pneumonia, respiratory disorder, and constipation, with percentages reported for those terms in the registry.
Safety comparisons should be read in light of the population at risk and the way events were collected and classified. The posted figures do not provide a causal estimate or a complete account of clinical context. They are descriptive registry results rather than a basis for individual treatment decisions.
13. What the Result Does, and Does Not, Mean
The primary result is an estimate of time to a defined readiness-for-discharge event in the two randomized groups. The posted arm medians are 87.78 hours for rivipansel and 93.47 hours for placebo. The log-rank analysis reports a hazard ratio of 0.97, with a 95% confidence interval from 0.77 to 1.22 and p = 0.794. Taken together, these results do not provide precise evidence of a difference in event timing, while the confidence interval also does not establish that the groups are equivalent.
The distinction between absence of clear evidence of a difference and evidence of no difference is important. A non-significant p-value is not proof that two treatments have the same effect. A confidence interval that includes 1 for a hazard ratio indicates that equal event rates are among the values compatible with the analysis, but other values are compatible as well. The registry does not report a prespecified equivalence margin or a non-inferiority framework for this endpoint.
The endpoint itself is a composite readiness definition, not simply the time until hospital discharge. It depends on all applicable criteria being documented, including medication and support requirements and the status of acute complications. The registry also lists actual time to discharge and opioid-related outcomes as secondary endpoints, but results for those measures are not reported in the registry record in the registry record for this analysis. The primary endpoint therefore cannot answer every question about length of stay, opioid use, or later rehospitalization.
The secondary rehospitalization comparison is also uncertain, and the registry reports no numeric results for the other listed secondary outcomes in this record. Safety counts are descriptive and do not establish causation. Overall, the posted findings describe the measured outcomes and their statistical uncertainty; they do not establish a general conclusion about treatment benefit, lack of benefit, or safety beyond the recorded analyses.
14. Why This Trial Matters Statistically
This trial is statistically informative because its primary outcome is a time-to-event measure tied to a detailed clinical readiness definition. The distinction between readiness for discharge and actual discharge illustrates why endpoint definitions matter: a statistical comparison can only address the event that was defined and recorded. The endpoint combines several treatment and complication criteria, rather than relying on a single measure of the hospital course.
The result also demonstrates why a point estimate, confidence interval, and p-value should be read together. The hazard ratio is 0.97, but the 95% confidence interval from 0.77 to 1.22 allows a range of values. The p-value of 0.794 does not prove equivalence or absence of any effect. The arm medians add descriptive information, but they are not a replacement for the reported time-to-event comparison.
The record provides a further example of how sponsor analyses and simple recalculations can differ in method and scale. The rehospitalization re-calculation uses posted summary counts and is unadjusted; it is not the same analysis as the Chan and Zhang result reported in the registry. Keeping those analyses distinct helps avoid treating an independently recalculated summary as if it were the trial’s prespecified analysis.
Finally, the registry contains multiple outcome measures but does not report a full multiplicity strategy, missing-data handling, or sample-size justification. These gaps show why statistical interpretation depends not only on the values but also on transparent reporting of the design and analysis choices. A registry result can be read accurately while still recognizing what its record does not establish.
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.
Several trial-specific limitations of interpretation arise from the registry record. Although the study is described as randomized and quadruple-masked, the record does not identify the roles covered by the masking designation or provide details of the allocation procedure. Further information on the treatment regimens is not included in the arm descriptions. These omissions limit assessment of how the design was implemented.
The primary endpoint uses a defined set of readiness criteria and reports time from the first infusion to meeting all applicable criteria. The record does not report how incomplete assessments, events not observed during follow-up, or censoring were handled. It also does not provide a clinically meaningful threshold for interpreting a difference in time. As a result, statistical uncertainty can be described, but the clinical importance of particular differences cannot be determined from the registry record alone.
The full analysis set is described as including randomized participants, and the primary conclusions are based on the full study population across the identified age cohorts. The registry entries do not include age-specific primary estimates, so the overall result cannot establish whether estimates were similar across the cohorts. The registry also does not provide details about missing-data methods or sensitivity analyses that might assess the robustness of the primary finding.
Several secondary outcomes are listed, but numeric results for most are not reported in the registry record. The record does not explain whether secondary comparisons were adjusted for multiplicity or how their results were prioritized. It also does not report interim analyses, the trial’s sample-size justification, or a detailed analysis plan. The planning illustration based on the observed rehospitalization effect should not be taken as evidence about the original design or as observed power. These limits constrain how completely the posted results can be evaluated.
16. Related Tutorials
17. Related Statistical Calculators
18. Sources
- ClinicalTrials.gov: Efficacy and Safety of Rivipansel (GMI-1070) in the Treatment of Vaso-Occlusive Crisis in Hospitalized Subjects With Sickle Cell Disease — NCT02187003.
- Registry record last updated 2025-03-11; status last verified 2025-02.
- Results first posted 2025-03-11.
- 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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19. Record Summary
The registry describes a completed phase 3, multicenter, randomized, double-blind, placebo-controlled, parallel-group study of rivipansel in hospitalized participants with sickle cell disease and vaso-occlusive crisis. The primary endpoint was time to readiness for discharge, measured from the first infusion until all applicable criteria were met.
The reported primary log-rank analysis gives a hazard ratio of 0.97, a 95% confidence interval from 0.77 to 1.22, and p = 0.794. The arm medians were 87.78 hours for rivipansel and 93.47 hours for placebo. The record also lists secondary outcomes and safety results, but does not report numeric results for most listed secondary endpoints in the registry record reported for this analysis.
The posted estimates should be interpreted with their uncertainty and alongside the reporting gaps: details about missing-data handling, multiplicity, interim analyses, and the trial’s sample-size justification are not reported in the registry record.