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 Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors (NCT03196297), a na phase 2 study. The primary outcome is The Number of Bleeding Episodes During at Least 24 Weeks From Treatment Onset.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT03196297 |
| Phase | Phase 2 |
| Condition | Haemostasis, Haemophilia A |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 36 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Novo Nordisk A/S |
| Results posted | Yes |
2. Clinical Question
Population
People with severe haemophilia A without inhibitors who took at least one dose of study drug. The registry describes the full analysis set as including all participants who took at least one dose. The trial was conducted in Asia, Europe and the United States of America.
Intervention
Daily subcutaneous concizumab. The record describes administration to both on-demand and prophylaxis patients, and reports concizumab dose groups in the main and extension parts.
Comparator
No randomized comparator is identified. Allocation is recorded as not applicable, and the registry cautions that the arms are not a like-for-like control comparison. Turoctocog alfa is listed as an intervention, but its role in the arm descriptions is not reported in the registry record.
Primary question
How many treated bleeding episodes occurred during at least 24 weeks from treatment onset, with the data presented for the last dose level being taken when the bleed occurred? The registry record gives the endpoint definition but does not report its numerical result.
The study aim was to assess the efficacy and safety of daily concizumab in preventing bleeding episodes. Statistically, the primary endpoint is a count of treated events over a stated observation period, rather than a direct comparison of randomized treatment groups. The registry record does not provide a numerical estimate of the primary outcome, its uncertainty, or a statistical test for it. As a result, the stated research question can be described, but the size and precision of the primary outcome cannot be assessed from the registry record.
3. Trial Design
This was a Phase 2, multicentre trial with a single-group model. The registry records allocation as not applicable and masking as none. It lists enrollment as 36 and gives the study status as completed. The study start date is 2017-08-16 and the primary completion date is 2018-06-22. These dates and the enrollment figure describe the registry entry; they do not by themselves establish how many participants contributed data to each outcome or dose group.
The stated aim was to assess daily subcutaneous concizumab in people with severe haemophilia A without inhibitors. The brief summary says the trial took place in Asia, Europe and the United States of America. The arm description refers to daily concizumab administration to both on-demand and prophylaxis patients. Dose groups are identified for a main part and an extension part. The record also lists turoctocog alfa as an intervention, but its use or role in the described arms is not reported in the registry record.
The registry explicitly warns that the arms are not a like-for-like control comparison because this was not a randomized comparison. Therefore, differences between dose groups or between the main and extension parts should not be interpreted as randomized treatment effects. The record does not report the allocation process between dose levels, the reasons participants entered particular groups, or how the extension participants were selected. Those design details are not reported in the registry record.
The primary endpoint was the number of treated bleeding episodes during at least 24 weeks from treatment onset, with data presented according to the last dose level being taken when the bleed occurred. Several secondary outcomes concern bleeding, spontaneous bleeding, treatment-emergent adverse events, anti-concizumab antibodies and fibrinogen over stated follow-up periods. The registry lists 30 outcome measures, but the primary endpoint's numerical result 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.
Before assignment: The trial consisted of two treatment periods: main part which lasted at least 24 weeks for all participants in the trial and an extension part which was up to 102 weeks.
Main Period
| Stage | Concizumab |
|---|---|
| STARTED | 36 |
| Full Analysis Set (FAS) | 36 |
| Subject Analysis Set (SAS) | 36 |
| COMPLETED | 32 |
| NOT COMPLETED | 4 |
Reasons for not completing this period:
| Reason | Concizumab |
|---|---|
| Withdrawal by Subject | 4 |
Extension Period
| Stage | Concizumab |
|---|---|
| STARTED | 32 |
| FAS | 32 |
| SAS | 32 |
| COMPLETED | 29 |
| NOT COMPLETED | 3 |
Reasons for not completing this period:
| Reason | Concizumab |
|---|---|
| Withdrawal by Subject | 1 |
| Lack of Efficacy | 2 |
Analysis population for the primary outcome, as defined in the registry: The FAS included all participants who took al least one dose of the study drug.
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 (FAS) included all dosed participants.
| Characteristic | Unit | Concizumab |
|---|---|---|
| Participants analysed | 36 | |
| Age, Continuous | Years | 36.9 (SD 12.9) |
| Sex: Female, Male: Female | Participants | 0 |
| Sex: Female, Male: Male | Participants | 36 |
| Ethnicity (NIH/OMB): Hispanic or Latino | Participants | 3 |
| Ethnicity (NIH/OMB): Not Hispanic or Latino | Participants | 30 |
| Ethnicity (NIH/OMB): Unknown or Not Reported | Participants | 3 |
| Race/Ethnicity, Customized: Asian | Participants | 8 |
| Race/Ethnicity, Customized: White | Participants | 24 |
| Race/Ethnicity, Customized: Other | Participants | 1 |
| Race/Ethnicity, Customized: Not applicable | Participants | 3 |
6. Primary Endpoint
The registry lists the primary outcome as The Number of Bleeding Episodes During at Least 24 Weeks From Treatment Onset, measured over During at least 24 weeks from treatment onset.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | Episodes |
| Endpoint classification | not reduced to a single comparison |
The primary endpoint counts bleeding episodes that were treated during at least 24 weeks from treatment onset. The registry states that the data are presented while on the last dose level when the bleed occurred. The full analysis set is described as including all participants who took at least one dose of study drug. The endpoint is therefore an event count over a follow-up period, not a count of participants who experienced at least one bleed.
General statistical context: event counts can describe the frequency of recurrent events, so they retain information about more than whether a participant had any event. They are also influenced by the time each participant is observed. A count can be presented as a total, or analyzed with a model that accounts for exposure time and variation in event rates. Which approach was used for this endpoint is not reported in the registry record.
General rationale: a treated-bleed count is relevant to a study whose stated aim concerns bleeding episodes, because it measures the event type of interest during a specified period. However, interpretation depends on clear definitions of a treated bleed, the time under observation, and how treatment changes over time are handled. The endpoint description provides the treatment and follow-up context but does not report a numerical count, a rate, an uncertainty interval or a statistical test. Accordingly, the magnitude and precision of the endpoint result cannot be assessed from the registry record.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry describes a single-group study and explicitly cautions that the arms are not a like-for-like control comparison. This is central to interpretation. The reported groups differ by concizumab dose and by main or extension part, but the record does not identify randomized allocation. Any differences between these groups could reflect differences in who entered them, when they were observed, their dose histories or other factors, rather than a causal effect of dose. The registry record does not report enough design detail to separate these possibilities.
The primary outcome is a count of treated bleeding episodes during at least 24 weeks from treatment onset. Its numerical result is not reported in the registry record. There is consequently no primary estimate in this record from which to describe the observed event burden, its variation, or the precision of an estimate. A claim that bleeding was reduced or that a particular dose was better supported would go beyond the reported information. The aim is described, but an aim is not an outcome result.
The safety record lists serious adverse events by dose group and study part. In the main part, serious events are recorded as affecting 0 of 36 participants in the Concizumab 0.15 mg/kg group, 0 of 15 in the Concizumab 0.20 mg/kg group, and 0 of 8 in the Concizumab 0.25 mg/kg group. In the extension part, the corresponding entries are 2 of 19, 1 of 14 and 2 of 10. Deaths are recorded as 0 in each of these groups. These are descriptive group entries, not a randomized comparison, and they do not establish whether an event was caused by treatment.
The registry also lists serious-event terms with percentages: atypical pneumonia and gastrointestinal haemorrhage at 10.0% each, ligament sprain at 7.1%, and gastrointestinal infection and pharyngeal haemorrhage at 5.3% each. The record does not give the denominators or further context for these term percentages in the information available for this interpretation. They should not be treated as directly comparable across groups or as measures of treatment effect.
Overall, the record supports a description of the study design, endpoint definition and certain safety entries. It does not provide a numerical primary outcome result or a controlled estimate of concizumab's effect. The distinction between descriptive observations and comparative evidence is especially important for this single-group, unmasked trial.
9. Statistical Methods Explained
The statistical methods that can be discussed from the registry record are limited. It identifies the study as Phase 2 and single-group, describes a count primary endpoint, and provides several safety entries. It does not report the statistical model, an analysis plan, a primary effect estimate, an uncertainty interval or a hypothesis test for the primary endpoint. The following general explanations clarify the kinds of issues that matter for these data; they do not establish which methods the investigators used.
What does a count endpoint represent?
General explanation: a count endpoint records how many events occur during a defined period. It differs from a binary outcome, which records only whether an event occurred, and from a time-to-event outcome, which focuses on how long it takes until an event. A count can reflect recurrent episodes in the same participant. For that reason, the total number of episodes and the number of participants with episodes answer different questions.
The registry's primary outcome is the number of treated bleeding episodes during at least 24 weeks from treatment onset. It specifies that data are presented while on the last dose level when the bleed occurred. That wording makes dose at the time of an event relevant to describing the outcome. The record does not explain how observation time was handled when participants had different follow-up durations or changed dose levels. Nor does it report whether the endpoint was analyzed as a raw count, a rate, or with another approach.
Why can count data need special care?
General explanation: simple count models often assume that the variability in counts follows a particular relationship to their average. In clinical data, counts may vary more than that assumption allows, a feature called over-dispersion. When over-dispersion is present, a model that does not account for it may understate uncertainty. Repeated events within a participant can also be related to one another, rather than behaving like unrelated observations. Analysts may use methods that account for exposure time, participant-level clustering or extra variability, depending on the design and data.
These are general considerations, not claims about this trial's data. The registry record does not report whether over-dispersion was assessed, whether repeated events were modeled as correlated, or what distribution or link function was used. It also does not report an event-rate estimate or an uncertainty interval that would allow readers to evaluate the precision of the primary endpoint.
What does a confidence interval tell a reader?
General explanation: a confidence interval expresses the uncertainty around an estimate under the assumptions of the statistical method. A narrow interval indicates greater precision than a wide interval, but neither an interval nor a point estimate alone establishes a causal effect. In a non-randomized, single-group setting, uncertainty intervals do not remove confounding or create a control group. The registry record does not report a confidence interval for the primary bleeding endpoint.
How should the group entries be read?
The serious adverse event information is organized by dose group and study part, with participants at risk, participants affected and deaths recorded. These entries are descriptive. The fact that the counts are displayed by dose does not make them randomized comparisons. Group sizes differ, and the registry cautions that the arms are not a like-for-like control comparison. Directly comparing the entries as if dose assignment were randomized would therefore be unjustified.
General explanation: a participant count with an event is not the same measure as an event count. A participant may experience more than one event, while a participant-level summary generally records whether the person experienced at least one event in the category. The adverse-event entries use affected-participant counts; the primary endpoint is described as a number of bleeding episodes. These outcome types should not be interchanged.
What can the planning assumptions establish?
The registry describes a planning illustration based on the observed effect, with two-sided significance level 0.05, equal allocation between two arms, and a normal approximation. It says the effect to detect is a stated fraction of the observed effect. For time-to-event outcomes the planning output is defined as a total number of events; other outputs are defined as participants per arm. These are planning conventions, not observed trial outcomes or observed power. The record does not report the numerical planning outputs in the information summarized for this interpretation, and the two-arm equal-allocation assumptions should not be mistaken for the design of this single-group trial.
More broadly, a planning calculation depends on assumptions about the effect to detect, variability, allocation and analysis method. A calculation based on an observed effect is not independent confirmation of that effect. It is an illustration of what a future design might require under the stated assumptions. It does not replace a controlled trial or establish that the effect is reliable.
The registry record lists 30 outcome measures but no analysis entries. It does not report which statistical model was used for the primary endpoint, how missing observations were handled, whether any covariates were included, or how multiplicity was addressed. Readers should therefore treat the reported design details and safety counts as descriptive, while recognizing that the inferential methods and numerical primary result are not reported in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 30 outcome measures, including the primary treated-bleeding count and secondary measures concerning bleeding, spontaneous bleeding, treatment-emergent adverse events, anti-concizumab antibodies and fibrinogen. Testing or estimating many outcomes can raise multiplicity concerns. General explanation: when many statistical tests are conducted, the chance of at least one apparently notable result can increase unless the analysis plan addresses how results will be interpreted. The registry record does not report a multiplicity adjustment, a hierarchy of outcomes, or which outcomes were intended to support formal claims.
The record lists no analysis entries. This does not identify what analyses may have been conducted outside the registry; it means that statistical tests and analysis methods are not reported in the registry record. The primary endpoint's numerical result and uncertainty are also not reported in the information. As a result, the evidence cannot be assessed through an estimate, interval or p-value for that outcome.
The full analysis set is described as including all participants who took at least one dose of study drug. That definition identifies who is intended to be included in that population, but it does not explain how missing outcome observations, incomplete follow-up, withdrawal or changes in dose were handled. The registry record does not report the amount or pattern of missing data, imputation procedures, sensitivity analyses, or whether the primary outcome analysis used a different population.
General explanation: missing data can affect both the estimate and its uncertainty, especially if the chance of missingness is related to health status or outcome experience. The appropriate analysis depends on why observations are missing and what assumptions can be defended. Without information on missingness and its handling, the completeness of outcome estimates cannot be evaluated. No assumptions about the protocol, interim analyses or statistical plan can be made from the registry record.
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 |
|---|---|---|---|
| Concizumab 0.15 mg/kg - Main Part | 0 | 36 | 0.0% |
| Concizumab 0.20 mg/kg - Main Part | 0 | 15 | 0.0% |
| Concizumab 0.25 mg/kg - Main Part | 0 | 8 | 0.0% |
| Concizumab 0.15 mg/kg - Extension Part | 2 | 19 | 10.5% |
| Concizumab 0.20 mg/kg - Extension Part | 1 | 14 | 7.1% |
| Concizumab 0.25 mg/kg - Extension Part | 2 | 10 | 20.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Concizumab 0.15 mg/kg - Main Part | 0 of 36 |
| Concizumab 0.20 mg/kg - Main Part | 0 of 15 |
| Concizumab 0.25 mg/kg - Main Part | 0 of 8 |
| Concizumab 0.15 mg/kg - Extension Part | 0 of 19 |
| Concizumab 0.20 mg/kg - Extension Part | 0 of 14 |
| Concizumab 0.25 mg/kg - Extension Part | 0 of 10 |
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 |
|---|---|
| Concizumab 0.15 mg/kg - Main Part | 26 of 36 |
| Concizumab 0.20 mg/kg - Main Part | 7 of 15 |
| Concizumab 0.25 mg/kg - Main Part | 3 of 8 |
| Concizumab 0.15 mg/kg - Extension Part | 16 of 19 |
| Concizumab 0.20 mg/kg - Extension Part | 9 of 14 |
| Concizumab 0.25 mg/kg - Extension Part | 7 of 10 |
Most frequent serious adverse event terms
| Event term | Organ system | Concizumab 0.15 mg/kg - Main Part | Concizumab 0.20 mg/kg - Main Part | Concizumab 0.25 mg/kg - Main Part | Concizumab 0.15 mg/kg - Extension Part | Concizumab 0.20 mg/kg - Extension Part | Concizumab 0.25 mg/kg - Extension Part |
|---|---|---|---|---|---|---|---|
| Atypical pneumonia | Infections and infestations | 0 of 36 | 0 of 15 | 0 of 8 | 0 of 19 | 0 of 14 | 1 of 10 |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | 0 of 36 | 0 of 15 | 0 of 8 | 0 of 19 | 0 of 14 | 1 of 10 |
| Gastrointestinal infection | Infections and infestations | 0 of 36 | 0 of 15 | 0 of 8 | 1 of 19 | 0 of 14 | 0 of 10 |
| Ligament sprain | Injury, poisoning and procedural complications | 0 of 36 | 0 of 15 | 0 of 8 | 0 of 19 | 1 of 14 | 0 of 10 |
| Pharyngeal haemorrhage | Respiratory, thoracic and mediastinal disorders | 0 of 36 | 0 of 15 | 0 of 8 | 1 of 19 | 0 of 14 | 0 of 10 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Concizumab 0.15 mg/kg - Main Part | Concizumab 0.20 mg/kg - Main Part | Concizumab 0.25 mg/kg - Main Part | Concizumab 0.15 mg/kg - Extension Part | Concizumab 0.20 mg/kg - Extension Part | Concizumab 0.25 mg/kg - Extension Part |
|---|---|---|---|---|---|---|---|
| Nasopharyngitis | Infections and infestations | 9 of 36 | 1 of 15 | 0 of 8 | 6 of 19 | 1 of 14 | 0 of 10 |
| Fibrin D dimer increased | Investigations | 3 of 36 | 2 of 15 | 2 of 8 | 1 of 19 | 1 of 14 | 2 of 10 |
| Headache | Nervous system disorders | 7 of 36 | 1 of 15 | 0 of 8 | 2 of 19 | 1 of 14 | 0 of 10 |
| Prothrombin level increased | Investigations | 3 of 36 | 2 of 15 | 2 of 8 | 0 of 19 | 0 of 14 | 2 of 10 |
| Back pain | Musculoskeletal and connective tissue disorders | 4 of 36 | 0 of 15 | 0 of 8 | 1 of 19 | 2 of 14 | 0 of 10 |
| Injection site bruising | General disorders | 5 of 36 | 0 of 15 | 0 of 8 | 1 of 19 | 0 of 14 | 0 of 10 |
| Upper respiratory tract infection | Infections and infestations | 2 of 36 | 0 of 15 | 0 of 8 | 1 of 19 | 2 of 14 | 1 of 10 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 1 of 36 | 0 of 15 | 1 of 8 | 0 of 19 | 2 of 14 | 1 of 10 |
| Injection site haematoma | General disorders | 4 of 36 | 0 of 15 | 0 of 8 | 1 of 19 | 0 of 14 | 0 of 10 |
| Injection site haemorrhage | General disorders | 3 of 36 | 0 of 15 | 0 of 8 | 0 of 19 | 1 of 14 | 1 of 10 |
Serious events with the highest arm-level rate: atypical pneumonia (10.0%), gastrointestinal haemorrhage (10.0%), ligament sprain (7.1%), gastrointestinal infection (5.3%), pharyngeal haemorrhage (5.3%).
The serious adverse event entries should be read as participant counts within the named dose group and study part. In the main part, the registry records 0 affected participants among 36 at Concizumab 0.15 mg/kg, 0 among 15 at Concizumab 0.20 mg/kg, and 0 among 8 at Concizumab 0.25 mg/kg. In the extension part, it records 2 among 19, 1 among 14, and 2 among 10, respectively. Deaths are recorded as 0 for each group. These figures describe the registry entries; they do not by themselves show whether events were caused by concizumab.
The record lists atypical pneumonia and gastrointestinal haemorrhage at 10.0% each among the top serious terms, ligament sprain at 7.1%, and gastrointestinal infection and pharyngeal haemorrhage at 5.3% each. The denominators and details of how these term percentages were derived are not reported in the registry record. These percentages should therefore be read as listed event-term summaries, not as comparative risks between dose groups.
General explanation: serious-event counts are distinct from counts of all adverse events, and participant counts are distinct from the total number of event occurrences. A zero serious-event count in a group does not prove that no risk exists, particularly when the number observed and follow-up may be limited. The registry's non-randomized design also prevents a reliable like-for-like safety comparison across groups.
12. What the Result Does, and Does Not, Mean
The registry record establishes that a Phase 2, single-group study assessed daily subcutaneous concizumab in people with severe haemophilia A without inhibitors. It defines the primary endpoint as treated bleeding episodes during at least 24 weeks from treatment onset and describes the full analysis set as participants who took at least one dose. It also provides serious adverse event entries by dose group and study part. These registry entries allow readers to understand the research question and the structure of some reported safety information.
The primary endpoint's numerical result is not reported in the registry record. Without that result, there is no basis for describing the observed number or rate of treated bleeds, the precision of an estimate, or a difference from another treatment. The trial's stated aim is not evidence that the aim was achieved. Likewise, the presence of multiple dose groups does not create a controlled dose comparison, because the registry identifies the study as single-group and cautions that the arms are not like-for-like controls.
The serious adverse event counts are descriptive entries and cannot establish causation. The record lists zero deaths in each dose group, but that observation alone cannot determine the broader safety profile or exclude uncommon events. The listed serious-event percentages also lack denominator context in the registry information used.
General explanation: a controlled randomized comparison can help distinguish treatment effects from differences in participant characteristics and other factors. In its absence, outcomes observed during treatment can be described, but attribution is more limited. A statistical planning illustration is also not an outcome: it concerns assumptions for a possible design and does not show how precisely the trial estimated an effect.
Accordingly, the registry record supports a careful account of the design, endpoint definition and listed safety observations. It does not, in the information, support a conclusion about the magnitude or causal effect of concizumab on bleeding episodes.
13. Why This Trial Matters Statistically
This trial is statistically notable because it studies a recurrent-event outcome in a single-group design. Counting treated bleeding episodes is different from counting participants who ever experienced a bleed: recurrent events can contribute multiple times for the same participant. The endpoint also has a defined follow-up period and identifies the dose level being taken when a bleed occurred. These features make the choice of outcome summary and handling of observation time important.
General explanation: event counts often require attention to exposure time, variation in event rates and correlation among repeated events within a participant. A simple count may not fully describe differences in follow-up, while a rate may require an exposure-time denominator. The registry record does not report which method was used, so readers cannot assess those choices for the primary analysis.
The design also illustrates why dose groups should not automatically be treated as comparative arms. Although the registry lists groups by dose and study part, it identifies allocation as not applicable and explicitly says the arms are not a like-for-like control comparison. Descriptive group outcomes can help characterize the recorded experience, but they cannot isolate the causal effect of dose in the way a randomized comparison is intended to do.
The record's combination of a defined clinical endpoint, multiple secondary outcomes and adverse event summaries underscores the need for transparent reporting of the analysis population, missing data, multiplicity and uncertainty. Those methods are not reported in the registry record. The absence of the primary numerical result further limits statistical interpretation: without an estimate and its precision, readers cannot judge the observed event burden from the primary endpoint.
The statistical lesson is not a conclusion about treatment effect. It is that study design, outcome definition and analysis reporting determine what can be inferred. This record offers useful design and endpoint details, but leaves key inferential information unreported.
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 central limitation for interpreting efficacy is that the primary endpoint's numerical result is not reported in the registry record. The endpoint definition is available, but there is no reported count, rate, estimate of uncertainty or test result from which to assess the observed bleeding experience.
The design is single-group, with allocation recorded as not applicable. The registry warns that the arms are not a like-for-like control comparison. As a result, differences between dose groups or study parts cannot be treated as randomized treatment effects. The record does not report how participants were assigned to dose groups, how the transition into the extension part occurred, or whether group characteristics were comparable.
The endpoint is described over at least 24 weeks from treatment onset and is organized by the last dose level being taken when a bleed occurred. The registry record does not explain how varying follow-up, changes in dose, or incomplete observation were handled in the primary analysis. The full analysis set definition is given, but the number contributing to each outcome and any exclusions or missing observations are not reported.
Important statistical details are also absent: the model for the count outcome, treatment of repeated events, handling of exposure time, confidence intervals, multiplicity procedures and sensitivity analyses are not reported in the registry record. The record lists 30 outcome measures but no analysis entries. Thus, it is not possible to assess the analysis choices or the robustness of a primary estimate.
Safety entries are descriptive, and the denominators or derivation details for the listed serious-event term percentages are not reported in the registry information used. These limitations constrain both comparative safety interpretation and causal attribution.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors — NCT03196297.
- Registry record last updated 2021-11-16; status last verified 2021-11.
- Results first posted 2021-05-10.
- 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.
Continue through the Clinical Biostats statistical pathway
Use this trial's endpoint as a starting point for deeper study of effect measures, confidence intervals, and sample-size planning.
18. Record Summary
The registry record describes a completed Phase 2, multicentre, single-group trial of daily subcutaneous concizumab in people with severe haemophilia A without inhibitors. Enrollment is listed as 36, with study start on 2017-08-16 and primary completion on 2018-06-22. The record identifies no masking and warns that the dose groups are not a like-for-like control comparison.
The primary endpoint is the number of treated bleeding episodes during at least 24 weeks from treatment onset, presented according to the last dose level taken when a bleed occurred. Its numerical result is not reported in the registry record. Serious adverse event counts and deaths are listed for the main and extension dose groups, but these are descriptive observations. The registry lists 30 outcome measures and no analysis entries. Statistical methods, missing-data handling and multiplicity procedures are not reported in the registry record.