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 A Phase II Study to Evaluate the Efficacy of IdeS to Desensitize Transplant Patients With a Positive Crossmatch Test (NCT02790437), a na phase 2 study. The primary outcome is Number of Patients With Crossmatch Conversion (Positive to Negative).
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02790437 |
| Phase | Phase 2 |
| Condition | Kidney Failure, Chronic |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 19 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Hansa Biopharma AB |
| Results posted | Yes |
2. Clinical Question
Population
People with chronic kidney failure who were on the kidney transplant waiting list and had an available live or deceased donor with a positive crossmatch test. The study was intended for patients who had previously undergone unsuccessful desensitization or for whom effective desensitization was considered highly unlikely. The primary endpoint description refers to patients with donor-specific antibodies and a positive crossmatch before treatment.
Intervention
IdeS, administered by intravenous infusion. The registry record also lists kidney transplantation as a procedure, but it does not establish that every participant underwent transplantation.
Comparator
There is no randomized control group described. The study is classified as single-group, and allocation is listed as NA. The one-dose and two-dose safety groups are not a like-for-like control comparison.
Primary question
Could IdeS convert a positive donor crossmatch test to a negative result within 24 hours of dosing? The endpoint counted patients meeting the conversion definition, which required at least one positive crossmatch before dosing and a negative result on the last test within 24 hours. The registry record does not report the observed number of patients who met this endpoint.
3. Trial Design
This completed phase II study evaluated IdeS in transplant candidates with a positive crossmatch to an available kidney donor. The registry record lists chronic kidney failure as the condition and Hansa Biopharma AB as the sponsor. The study start is recorded as 2016-06, and primary completion as 2017-12-12. Enrollment is listed as 19.
The design is recorded as single-group, with allocation listed as NA and masking as none. Accordingly, the record does not describe a randomized comparison or a masked treatment assignment. The one-dose and two-dose groups listed in the safety results reflect dosing categories; they should not be treated as equivalent to randomized treatment arms or as a control comparison.
The treatment arm is described as an intravenous IdeS infusion. Kidney transplantation is also listed as an intervention, but the record does not establish that transplantation occurred for every participant. The study objective was to assess whether IdeS could reduce donor-specific antibodies and convert a positive crossmatch test to a negative one, as well as to assess safety.
The primary outcome was assessed within 24 hours of dosing. The endpoint description says the crossmatch tests included FACS and CDC methods and defines conversion using a positive pre-dose test and a negative last test within the specified period. The record lists secondary outcomes covering donor-specific antibody levels, time to negative crossmatch tests, kidney function, serum IgG, and pharmacokinetic measures.
The registry record does not report a randomized comparator, a detailed statistical analysis plan, or the primary endpoint result. The planned-dose analysis-set description is incomplete: it ends after stating that the planned dose was defined as 1 administration in. The full rule is not reported in the registry record.
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 | One Dose of IdeS (0.25 mg/kg BW) | Two Doses of IdeS (2 x 0.25 mg/kg BW) |
|---|---|---|
| STARTED | 16 | 3 |
| COMPLETED | 13 | 3 |
| NOT COMPLETED | 3 | 0 |
Reasons for not completing this period:
| Reason | One Dose of IdeS (0.25 mg/kg BW) | Two Doses of IdeS (2 x 0.25 mg/kg BW) |
|---|---|---|
| Patient graft failure - nephrectomy | 1 | 0 |
| Adverse Event | 1 | 0 |
| Withdrawal by Subject | 1 | 0 |
Analysis population for the primary outcome, as defined in the registry: The full analysis set (FAS) comprises data from all patients in the safety analysis set (SAS) with available post-dose efficacy data. Before analysis it was specified that the analysis set should include all patients combined who had received the planned dose which was defined as 1 administration initially which could be repeated if needed. The single as well as the repeated administration are defined as the only planned dose. Consequently, the results are presented for all patients together.
5. Baseline Characteristics
The table reproduces the baseline characteristics posted in the registry, which describe the participants analysed at baseline and help show whether the randomized groups were comparable.
| Characteristic | Unit | One Dose of IdeS (0.25 mg/kg BW) | Two Doses of IdeS (2 x 0.25 mg/kg BW) | Total |
|---|---|---|---|---|
| Participants analysed | 16 | 3 | 19 | |
| Age, Categorical: <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Between 18 and 65 years | Participants | 16 | 3 | 19 |
| Age, Categorical: >=65 years | Participants | 0 | 0 | 0 |
| Age, Continuous | years | 40 (20 to 64) | 45 (39 to 45) | 40 (20 to 64) |
| Sex: Female, Male: Female | Participants | 5 | 1 | 6 |
| Sex: Female, Male: Male | Participants | 11 | 2 | 13 |
| Race/Ethnicity, Customized: Asian | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Black or african american | participants | 2 | 2 | 4 |
| Race/Ethnicity, Customized: Hispanic | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: Indian | participants | 1 | 0 | 1 |
| Race/Ethnicity, Customized: White | participants | 11 | 1 | 12 |
| Region of Enrollment: Sweden | participants | 1 | 1 | 2 |
| Region of Enrollment: United States | participants | 12 | 2 | 14 |
| Region of Enrollment: France | participants | 3 | 0 | 3 |
| Weight | kg | 74.05 (45.1 to 107.4) | 68.0 (62.6 to 70.3) | 71.6 (45.1 to 107.4) |
6. Primary Endpoint
The registry lists the primary outcome as Number of Patients With Crossmatch Conversion (Positive to Negative), measured over Within 24 hours of IdeS dosing.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Patients |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the number of patients with crossmatch conversion from positive to negative within 24 hours of IdeS dosing. The endpoint description requires at least one positive crossmatch before dosing and a negative result on the last test within 24 hours. Crossmatch testing was performed using FACS and CDC methods.
These tests assess different features of the donor-recipient antibody interaction. The FACS procedure uses recipient serum to stain donor cells and identify donor-specific antibodies. The CDC test assesses the cytotoxic capacity of those antibodies by mixing recipient serum with donor cells before adding complement and scoring live and dead cells. The record cautions that CDC crossmatch amplified with anti-human globulin is not compatible with imlifidase and should not be used.
As a binary endpoint, conversion is counted as an event: each patient either meets the stated definition or does not. This makes the endpoint a direct measure of whether the specified laboratory change occurred within the assessment window. It does not, by itself, measure the duration of a negative result, transplant success, or longer-term kidney function. The registry record does not report the observed conversion count or an estimate of its precision.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a clinically focused laboratory endpoint: conversion from a positive to a negative donor crossmatch within 24 hours of dosing. However, it does not report the number of patients who met that endpoint, the endpoint estimate, or a confidence interval. Therefore, the posted registry information does not permit a numerical interpretation of the primary efficacy result or its precision.
The listed enrollment is 19, but that figure should not automatically be treated as the primary endpoint denominator. The endpoint population is described as the full analysis set, comprising patients in the safety analysis set with available post-dose efficacy data. The record also says that the analysis set was to include patients combined who received the planned dose, but the definition of that dose is cut off. The number included in the primary analysis and the handling of participants without post-dose efficacy data are not reported in the registry record.
The safety record distinguishes one-dose, two-dose, and all-patient groups. In the all-patient group, serious adverse events are recorded for 15 of 19, and other adverse events for 18 of 19; deaths are recorded as 0. In the one-dose group, the corresponding counts are 12 of 16 and 15 of 16, with deaths recorded as 0. In the two-dose group, the counts are 3 of 3 and 3 of 3, with deaths recorded as 0. These are descriptive counts, not evidence from a randomized comparison. The registry record does not give a basis for attributing events to IdeS or separating treatment effects from the clinical circumstances of transplantation.
The listed serious-event terms include transplant rejection at 66.7%, and blood creatine increased, thrombotic microangiopathy, upper abdominal pain, toxic nephropathy, and tubulointerstitial nephritis at 33.3% each. The denominator underlying these percentages is not reported in the registry record, so they should be read as the percentages recorded for those terms, not as independently reconstructed risks.
In a single-group study, observed changes can describe what happened after treatment, but they cannot by themselves establish what would have happened without treatment. The registry record does not report a concurrent control group, adjusted comparison, or primary efficacy estimate. Consequently, it supports description of the study question and the posted safety counts, but not a comparative conclusion about efficacy.
9. Statistical Methods Explained
What does a binary endpoint represent?
General statistical explanation: A binary endpoint classifies each participant into one of two outcome categories, such as meeting or not meeting a prespecified definition. The trial’s primary endpoint uses this structure: a participant meets the endpoint if the pre-dose and post-dose crossmatch conditions are satisfied within the stated time window. A count of participants meeting the definition is easiest to interpret when its analysis denominator and missing-outcome handling are also clear.
The registry record describes the endpoint definition but does not report the observed count, analysis denominator, or confidence interval. Without those, it is not possible to assess the magnitude of the observed conversion result or how precisely it was estimated. The enrollment figure alone does not establish how many participants had evaluable post-dose efficacy data.
Why do the crossmatch methods matter?
General statistical explanation: An outcome’s measurement procedure is part of its definition. When tests use different procedures, a result should be interpreted in light of which procedure produced it and how the endpoint combines results. The registry record says that both FACS and CDC crossmatch tests were used and defines conversion as requiring at least one positive pre-dose test and a negative last test within 24 hours. It also says CDC crossmatch amplified with anti-human globulin is not compatible with imlifidase and should not be used. The record does not report the endpoint results separately by test method.
How should counts and percentages be read?
General statistical explanation: A count reports how many participants experienced an outcome; its denominator identifies the group in which those participants were assessed. Counts should be read with their denominators, particularly when group sizes differ. Percentages can make groups easier to compare, but they do not remove uncertainty, and a percentage without a clear denominator is difficult to interpret.
The safety results give counts for participants with serious and other adverse events in the one-dose, two-dose, and all-patient groups. The serious-event terms are also accompanied by percentages, but the denominator for those term-specific percentages is not reported in the registry record. The counts and listed percentages are descriptive. They do not establish causation or provide a randomized comparison between dosing groups.
What does a confidence interval add?
General statistical explanation: A confidence interval expresses the uncertainty around an estimated effect under a statistical model. A narrower interval indicates greater precision under that model; a wider interval indicates less precision. A confidence interval does not correct for bias, incomplete outcome data, or the absence of a control group. The registry record does not report a confidence interval for the primary endpoint, so its precision cannot be assessed from the posted registry information.
What is the role of a planning illustration?
General statistical explanation: Sample-size planning uses assumptions about the effect to detect, the significance level, allocation, and statistical method to estimate how many participants a future study might need. Such calculations are planning illustrations, not observed evidence and not a measure of the power achieved by a completed study. The stated planning assumptions use a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation; the effect to detect is defined as a fraction of the observed effect. For time-to-event calculations, the stated planning quantity is the total number of events; other calculations are expressed as participants per arm. These assumptions do not make this single-group study a randomized comparison. The registry record does not report the study’s own sample-size justification.
What can the design establish?
General statistical explanation: A single-group study can describe outcomes observed in the enrolled group, but without a concurrent comparison group it cannot isolate the effect of treatment from other explanations for those outcomes. This matters especially when the endpoint is measured after treatment: a before-and-after change may be consistent with a treatment effect but does not alone prove that treatment caused the change. Here, the registry record classifies the design as single-group and provides no randomized comparator or adjusted comparative analysis. It also reports no primary endpoint estimate, leaving the central efficacy result unavailable for statistical interpretation.
10. Multiplicity, Missing Data and Analysis Populations
The registry record lists 15 outcome measures and 0 analyses. It names secondary outcomes involving donor-specific antibodies, timing of negative crossmatch results, kidney function assessed by eGFR, serum IgG, and pharmacokinetic measures. It does not report how many outcomes were analyzed, which analyses were prespecified, or how the risk of false-positive findings across multiple outcomes was addressed. A multiplicity adjustment or testing hierarchy is not reported in the registry record.
The primary analysis population is described as patients in the safety analysis set with available post-dose efficacy data. The record further says that the analysis set should include all patients combined who received the planned dose, but the dose definition is incomplete, ending after 1 administration in. The complete planned-dose criterion is not reported in the registry record.
The number of patients with available post-dose efficacy data is not reported, nor is the number missing primary outcome data. The record does not describe imputation, exclusion rules for missing measurements, or sensitivity analyses to assess the effect of missing outcomes. It therefore cannot be established from the registry information whether the primary endpoint analysis included every enrolled participant or how missing data were handled.
These omissions limit interpretation of both the analysis population and the certainty of any reported outcome. A clear account of prespecified outcomes, analysis populations, missing-data procedures, and multiplicity handling would be needed to judge whether the analyses followed a defined plan and how robust their conclusions are.
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 |
|---|---|---|---|
| SAS - One Dose of IdeS (0.25 mg/kg BW) | 12 | 16 | 75.0% |
| SAS - Two Doses of IdeS (2 x 0.25 mg/kg BW) | 3 | 3 | 100.0% |
| SAS - All Patients (One or Two Doses of IdeS (0.25 mg/kg BW)) | 15 | 19 | 78.9% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| SAS - One Dose of IdeS (0.25 mg/kg BW) | 0 of 16 |
| SAS - Two Doses of IdeS (2 x 0.25 mg/kg BW) | 0 of 3 |
| SAS - All Patients (One or Two Doses of IdeS (0.25 mg/kg BW)) | 0 of 19 |
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 |
|---|---|
| SAS - One Dose of IdeS (0.25 mg/kg BW) | 15 of 16 |
| SAS - Two Doses of IdeS (2 x 0.25 mg/kg BW) | 3 of 3 |
| SAS - All Patients (One or Two Doses of IdeS (0.25 mg/kg BW)) | 18 of 19 |
Most frequent serious adverse event terms
| Event term | Organ system | SAS - One Dose of IdeS (0.25 mg/kg BW) | SAS - Two Doses of IdeS (2 x 0.25 mg/kg BW) | SAS - All Patients (One or Two Doses of IdeS (0.25 mg/kg BW)) |
|---|---|---|---|---|
| Transplant rejection | Immune system disorders | 7 of 16 | 2 of 3 | 9 of 19 |
| Blood creatine increased | Investigations | 1 of 16 | 1 of 3 | 2 of 19 |
| Nephropathy toxic | Renal and urinary disorders | 1 of 16 | 1 of 3 | 2 of 19 |
| Urinary tract infection | Infections and infestations | 2 of 16 | 0 of 3 | 2 of 19 |
| Axillary vein thrombosis | Vascular disorders | 1 of 16 | 0 of 3 | 1 of 19 |
| Superior vena cava syndrome | Vascular disorders | 1 of 16 | 0 of 3 | 1 of 19 |
| Infusion related reaction | Injury, poisoning and procedural complications | 1 of 16 | 0 of 3 | 1 of 19 |
| Transplant failure | Injury, poisoning and procedural complications | 1 of 16 | 0 of 3 | 1 of 19 |
| Weaning failure | Injury, poisoning and procedural complications | 1 of 16 | 0 of 3 | 1 of 19 |
| Donor specific antibody present | Investigations | 1 of 16 | 0 of 3 | 1 of 19 |
Most frequent non-serious adverse event terms
| Event term | Organ system | SAS - One Dose of IdeS (0.25 mg/kg BW) | SAS - Two Doses of IdeS (2 x 0.25 mg/kg BW) | SAS - All Patients (One or Two Doses of IdeS (0.25 mg/kg BW)) |
|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | 9 of 16 | 2 of 3 | 11 of 19 |
| Constipation | Gastrointestinal disorders | 7 of 16 | 2 of 3 | 9 of 19 |
| Hypomagnesaemia | Metabolism and nutrition disorders | 6 of 16 | 3 of 3 | 9 of 19 |
| Delayed graft function | Injury, poisoning and procedural complications | 7 of 16 | 1 of 3 | 8 of 19 |
| Nausea | Gastrointestinal disorders | 7 of 16 | 1 of 3 | 8 of 19 |
| Diarrhoea | Gastrointestinal disorders | 7 of 16 | 0 of 3 | 7 of 19 |
| Vomiting | Gastrointestinal disorders | 7 of 16 | 0 of 3 | 7 of 19 |
| Hyperkalaemia | Metabolism and nutrition disorders | 5 of 16 | 2 of 3 | 7 of 19 |
| Hypertriglyceridaemia | Metabolism and nutrition disorders | 4 of 16 | 3 of 3 | 7 of 19 |
| Metabolic acidosis | Metabolism and nutrition disorders | 5 of 16 | 2 of 3 | 7 of 19 |
Serious events with the highest arm-level rate: transplant rejection (66.7%), blood creatine increased (33.3%), thrombotic microangiopathy (33.3%), abdominal pain upper (33.3%), nephropathy toxic (33.3%).
The safety results are organized by one dose, two doses, and all patients. For serious adverse events, the recorded counts are 12 of 16 in the one-dose group, 3 of 3 in the two-dose group, and 15 of 19 in the all-patient group. For other adverse events, the counts are 15 of 16, 3 of 3, and 18 of 19, respectively. Deaths are recorded as 0 in each group.
These are descriptive counts. The one-dose and two-dose groups are not randomized comparison arms, and the record does not establish that their participants were otherwise comparable. The counts therefore should not be used to infer that one dosing category caused more or fewer events than another. Nor do the figures alone establish that an event was caused by IdeS.
The serious-event terms are listed with percentages: transplant rejection at 66.7%, and blood creatine increased, thrombotic microangiopathy, upper abdominal pain, toxic nephropathy, and tubulointerstitial nephritis at 33.3% each. The denominator for these term-specific percentages is not reported in the registry record. They should not be converted into participant counts or interpreted as risks for a defined group without that denominator.
12. What the Result Does, and Does Not, Mean
The study addresses whether IdeS can be followed by conversion of a positive donor crossmatch to a negative result within 24 hours. The registry record gives a specific endpoint definition and describes the crossmatch procedures used. It also lists safety counts for serious and other adverse events and records 0 deaths in each displayed safety group.
However, the primary endpoint result itself is not reported in the registry record. The record does not give the number of patients who achieved conversion, the primary analysis denominator, or a confidence interval. It is therefore not possible to state from the posted results how often conversion occurred or how precisely that result was estimated.
The single-group design also limits what the study can establish. Without a randomized comparator, an observed change after dosing cannot by itself show what would have happened without IdeS. The one-dose and two-dose safety groups do not provide a like-for-like control comparison. The registry record does not report a comparative efficacy analysis.
The safety counts describe events recorded in the listed groups; they do not establish that IdeS caused those events. The term-specific percentages lack a reported denominator, which limits their interpretation. The record also does not provide enough information to assess missing outcome data, the complete analysis population, or how multiple outcomes were handled.
Thus, the registry information defines the question and reports selected safety findings, but it does not provide the primary efficacy estimate needed to evaluate the frequency or precision of crossmatch conversion. It cannot support a claim that treatment works or does not work, or a comparative conclusion about different dosing categories.
13. Why This Trial Matters Statistically
This trial is statistically notable because it focuses on a defined laboratory endpoint tied to a specific clinical setting: a positive crossmatch between a transplant candidate and an available kidney donor. The endpoint is operationalized by requiring a positive pre-dose test and a negative last test within 24 hours. A clear operational definition helps readers understand what counts as an event, but interpretation still depends on the observed result, the analysis denominator, and the precision estimate.
The registry record also illustrates the distinction between a single-group study and a controlled comparison. A single group can describe outcomes after an intervention, while a control group is needed to make a direct comparison of outcomes under different treatment conditions. Here, allocation is listed as NA and the model as single-group. The dose categories in the safety results do not substitute for a randomized control group.
Safety reporting presents another useful statistical lesson: counts, denominators, and percentages must be read together. The registry lists adverse-event counts by dosing category, but the percentages attached to serious-event terms do not have a reported denominator. This leaves uncertainty about the population represented by those term-level figures.
Finally, the record shows why outcome definitions alone are not enough for a full statistical assessment. The primary outcome is described, but its observed result, confidence interval, missing-data handling, and full planned-dose analysis rule are not reported. Without those elements, readers can understand the intended measurement but cannot assess the magnitude or precision of the primary finding.
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 primary limitation for interpreting efficacy is that the registry record does not report the primary crossmatch-conversion result. It provides neither the number meeting the endpoint nor an estimate of precision. The enrollment figure does not resolve this gap because the primary analysis population is described as patients with available post-dose efficacy data, and the number with such data is not reported.
The analysis-population description is incomplete. It says the full analysis set includes patients in the safety analysis set with available post-dose efficacy data and refers to a planned-dose rule, but the rule ends mid-sentence. The record does not explain how missing post-dose results were handled or report sensitivity analyses.
The study is single-group and has no randomized control comparison described. Accordingly, changes in crossmatch results cannot be separated from other possible explanations using the registry information alone. The safety dosing groups should not be treated as comparable treatment arms.
The record lists multiple outcome measures but does not report a multiplicity strategy, testing hierarchy, or detailed statistical analysis plan. It does not report the sample-size justification used for the trial. The listed planning assumptions are illustrations and do not establish the trial’s design rationale or observed power.
For safety, the registry reports event counts by group, but the denominator behind the serious-event term percentages is not reported. The information also does not establish whether each event was considered treatment-related. These limits constrain interpretation of the listed safety findings.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Phase II Study to Evaluate the Efficacy of IdeS to Desensitize Transplant Patients With a Positive Crossmatch Test — NCT02790437.
- Registry record last updated 2021-05-20; status last verified 2021-04.
- Results first posted 2021-05-20.
- 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
This completed phase II study evaluated intravenous IdeS in transplant candidates with chronic kidney failure and a positive crossmatch to an available kidney donor. The registry lists enrollment of 19, a single-group model, no masking, and no randomized allocation. Kidney transplantation is also listed as an intervention, but the record does not establish that every participant received a transplant.
The primary endpoint was crossmatch conversion within 24 hours, defined by a positive pre-dose result and a negative last test in that period. The primary efficacy result, its analysis denominator, and its precision are not reported in the registry record. The safety record lists serious and other adverse-event counts by one-dose, two-dose, and all-patient groups, with deaths recorded as 0 in each group. The registry record does not provide a control comparison or a complete account of missing-data and multiplicity methods.