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 Study of PRN1008 in Adult Patients With Pemphigus Vulgaris (NCT02704429), a na phase 2 study. The primary outcome is Percentage of Participants With Treatment-emergent Adverse Events.
| Feature | Registry value |
|---|---|
| ClinicalTrials.gov identifier | NCT02704429 |
| Phase | Phase 2 |
| Condition | Pemphigus Vulgaris |
| Allocation | Not applicable (single group) |
| Design model | Single Group |
| Masking | None (open label) |
| Enrollment | 42 |
| Arms in the primary outcome | n/a |
| Lead sponsor | Principia Biopharma, a Sanofi Company |
| Results posted | Yes |
2. Clinical Question
Population
Adults with newly diagnosed or relapsing pemphigus vulgaris. The registry lists an enrollment of 42 and describes a safety analysis population consisting of patients who received at least 1 dose of rilzabrutinib.
Intervention
Open-label oral PRN1008, with dose adjustment within the participant based on clinical response and BTK occupancy. Conventional immunosuppressive rescue treatment could be used if indicated.
Comparator
There was no concurrent control group. Part A and Part B describe study phases, not randomized treatment arms, so they are not like-for-like comparator groups.
Primary question
What percentage of participants experienced treatment-emergent adverse events during the specified study periods? The registry defines these events as adverse events that developed, worsened, or became serious during the on-treatment phase.
The clinical question is primarily about safety: the listed primary endpoint counts participants with treatment-emergent adverse events, including clinically significant changes in physical examination, laboratory tests, and vital signs. An adverse event did not have to be caused by the study treatment to meet the registry definition.
The study also listed clinical activity outcomes, including control of disease activity, complete response, complete remission, time-to-event outcomes, relapse after treatment discontinuation, and cumulative corticosteroid use. These outcomes describe different aspects of disease control and treatment exposure. Their presence in the registry does not establish that results for them were reported. The registry record does not report results for those secondary outcomes.
Because the study was open-label and single-group, the main question is descriptive: what events were recorded among treated participants? It cannot, by itself, separate events associated with treatment from events that might have occurred without it. Nor does a safety endpoint answer whether clinical activity changed relative to an untreated or alternative-treatment group.
3. Trial Design
This was an open-label, phase 2 pilot study of oral PRN1008 in adults with newly diagnosed or relapsing pemphigus vulgaris. The registry lists the study as completed and records enrollment as 42. The study had a single-group model and no masking; allocation is listed as not applicable. Accordingly, the record does not describe a randomized control group or blinded assessment.
The treatment plan had two parts. Part A consisted of open-label PRN1008 for 12 weeks followed by 12 weeks of follow-up. Part B consisted of open-label PRN1008 for 24 weeks followed by 4 weeks of follow-up. The primary safety endpoint has a timeframe of Part A until 24 weeks and Part B until 28 weeks. These study periods should not be read as two independent randomized arms: Part A and Part B are phase labels, and the record does not describe a like-for-like control comparison between them.
Dose adjustment within a participant was based on clinical response and BTK occupancy. Conventional immunosuppressive rescue treatment could be used if indicated. This makes the treatment experience broader than exposure to a fixed dose alone: response-based adjustment and rescue treatment were part of the study description. The registry record does not report further details about how often dose adjustments or rescue treatment occurred.
The stated safety analysis population included all patients who received at least 1 dose of rilzabrutinib and was used for safety analyses. The primary endpoint was the percentage of participants with treatment-emergent adverse events. The registry record gives adverse-event counts for Part A and Part B, but does not report the primary endpoint percentage itself. It also does not report a statistical analysis plan or the rationale for the study's enrollment target.
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: A total of 69 patients were screened for the study (52 patients in Part A and 18\* patients in Part B). Of these, 41 unique patients were enrolled in the study (27 patients in Part A and 15\* patients in Part B). \*One patient who completed Part A of the study and later relapsed was enrolled in Part B.
Part A: 24 Weeks
| Stage | Part A | Part B |
|---|---|---|
| STARTED | 27 | 0 |
| COMPLETED | 24 | 0 |
| NOT COMPLETED | 3 | 0 |
Reasons for not completing this period:
| Reason | Part A | Part B |
|---|---|---|
| Adverse Event | 3 | 0 |
Part B: 28 Weeks
| Stage | Part A | Part B |
|---|---|---|
| STARTED | 0 | 15 |
| COMPLETED | 0 | 14 |
| NOT COMPLETED | 0 | 1 |
Reasons for not completing this period:
| Reason | Part A | Part B |
|---|---|---|
| Worsening of pemphigus | 0 | 1 |
Analysis population for the primary outcome, as defined in the registry: Safety Analysis population: All patients who received at least 1 dose of rilzabrutinib; used for all safety analyses
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: One patient who completed Part A of the study and later relapsed was enrolled in Part B. Therefore baseline results of one patient are repeated in Part B and total patient number is 15.
| Characteristic | Unit | Part A | Part B | Total |
|---|---|---|---|---|
| Participants analysed | 27 | 15 | 42 | |
| Age, Categorical: Part A – <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Part A – Between 18 and 65 years | Participants | 24 | 0 | 24 |
| Age, Categorical: Part A – >=65 years | Participants | 3 | 0 | 3 |
| Age, Categorical: Part B – <=18 years | Participants | 0 | 0 | 0 |
| Age, Categorical: Part B – Between 18 and 65 years | Participants | 0 | 15 | 15 |
| Age, Categorical: Part B – >=65 years | Participants | 0 | 0 | 0 |
| Sex: Female, Male: Part A – Female | Participants | 15 | 0 | 15 |
| Sex: Female, Male: Part A – Male | Participants | 12 | 0 | 12 |
| Sex: Female, Male: Part B – Female | Participants | 0 | 7 | 7 |
| Sex: Female, Male: Part B – Male | Participants | 0 | 8 | 8 |
| Race (NIH/OMB): Part A – American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part A – Asian | Participants | 1 | 0 | 1 |
| Race (NIH/OMB): Part A – Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part A – Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part A – White | Participants | 22 | 0 | 22 |
| Race (NIH/OMB): Part A – More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part A – Unknown or Not Reported | Participants | 4 | 0 | 4 |
| Race (NIH/OMB): Part B – American Indian or Alaska Native | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part B – Asian | Participants | 0 | 2 | 2 |
| Race (NIH/OMB): Part B – Native Hawaiian or Other Pacific Islander | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part B – Black or African American | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part B – White | Participants | 0 | 8 | 8 |
| Race (NIH/OMB): Part B – More than one race | Participants | 0 | 0 | 0 |
| Race (NIH/OMB): Part B – Unknown or Not Reported | Participants | 0 | 5 | 5 |
6. Primary Endpoint
The registry lists the primary outcome as Percentage of Participants With Treatment-emergent Adverse Events, measured over Part A: until 24 weeks and Part B: until 28 weeks.
| Element | Registry entry |
|---|---|
| Result type | Number |
| Measure of spread | not applicable |
| Unit | percentage of participants |
| Endpoint classification | Binary Endpoint |
The primary endpoint is the percentage of participants with treatment-emergent adverse events. The registry describes these as adverse events that developed, worsened, or became serious during the on-treatment phase, defined from the start of study drug up to study completion. Clinically significant changes in physical examination, laboratory tests, and vital signs were included. An adverse event did not need to have a causal relationship with treatment to qualify.
This endpoint is used to describe the safety experience during treatment. Counting the percentage of participants with at least one event gives a participant-level summary: it distinguishes whether an event was reported for a participant, rather than simply counting every event occurrence. That distinction matters because one participant may experience more than one event. The registry record does not specify whether repeated events were handled in any additional way for the posted primary result.
The endpoint is descriptive in this single-group, open-label study. Without a concurrent control group, the count cannot establish how many events would have occurred in the absence of PRN1008 or under another treatment. Also, the definition includes events regardless of whether a causal link to treatment was established. The registry record does not report the overall primary endpoint percentage, confidence interval, or a comparative estimate. The available adverse-event counts therefore provide safety context but do not substitute for a complete estimate of the stated primary endpoint.
7. Why No Re-analysis Is Shown
8. Statistical Interpretation
The registry record describes a single-group safety study, not a randomized treatment comparison. Its primary endpoint asks for the percentage of participants with treatment-emergent adverse events, while the posted adverse-event summary gives counts for Part A and Part B. In Part A, 3 of 27 participants at risk are reported with serious adverse events, and 20 of 27 with other adverse events. In Part B, 0 of 15 are reported with serious adverse events, and 13 of 15 with other adverse events. These counts describe the recorded experience in each part; they do not establish a causal effect of PRN1008.
The Part A and Part B figures should not be interpreted as a randomized comparison. The study was open-label, allocation is listed as not applicable, and the design is single-group. The parts also have different treatment and follow-up schedules. The registry record does not establish that their participants, observation time, or clinical circumstances are directly comparable. A difference between the counts would therefore not be an estimate of treatment benefit or harm attributable to one part versus the other.
The primary endpoint result itself is not reported in the registry record. The posted counts include serious and other adverse-event categories, but they do not supply the overall percentage of participants with any treatment-emergent adverse event as defined for the primary endpoint. No confidence interval or comparative statistical analysis for that endpoint is reported. The record lists 15 outcome measures and 0 analyses, so it does not provide a set of inferential results for interpreting the safety endpoint alongside the secondary outcomes.
The reported serious-event terms are Pulmonary sequestration, Pancreatic pseudocyst, Cellulitis, and Pneumonitis, each listed at 3.7%. Those values describe entries in the registry's serious-event summary. The record does not supply enough detail in the registry record to establish the event timing, participant overlap, clinical course, or causal attribution. The term percentages should not be treated as proof that the events were caused by study treatment.
In general, an adverse-event count is most informative when read with the event definition, the population at risk, the observation period, and the rules used to collect and classify events. The registry gives a broad treatment-emergent definition and different timeframes for the study parts, but the record does not report a fuller analysis explaining how exposure duration, repeated events, or missing safety observations were handled.
The results are therefore best read as descriptive safety information from an open-label pilot study. They can convey which event categories were recorded and how many participants were affected in the stated parts. They do not, on their own, quantify a causal effect, establish comparative safety, or answer the clinical activity questions listed as secondary outcomes.
9. Statistical Methods Explained
Statistical methods should be interpreted in light of the study design and the outcome definition. This trial is listed as open-label and single-group, with allocation not applicable. It does not have a concurrent randomized control group. That design supports a description of events observed during the study, but it does not support a direct randomized estimate of how PRN1008 compares with another treatment or with no treatment.
What does a participant percentage represent?
For a binary safety outcome, each participant is classified according to whether the event definition was met during the specified observation period. A percentage of participants with an event is therefore a participant-level summary, not a count of every event episode. In general, a participant who has repeated events can still contribute once to a measure of whether that participant experienced at least one event. The registry describes the primary endpoint as the percentage with treatment-emergent adverse events, but does not report that overall percentage.
The listed adverse-event figures distinguish serious adverse events from other adverse events. Those categories should be read according to the posted labels, not presumed to be mutually exclusive across all possible event summaries unless the registry specifies that. A person-level count and an event-term count answer different questions: the former concerns how many participants were affected, while the latter can describe which kinds of events were recorded. The registry record does not report additional rules for repeated terms or overlap between event categories.
Why does the design affect interpretation?
Randomization, when used, helps create groups that can be compared while limiting systematic differences in treatment assignment. This study's registry description does not include random allocation or a concurrent control. Consequently, observed events may reflect the underlying condition, other care, or other circumstances as well as treatment exposure. The open-label design also means that treatment and outcome awareness were not masked, according to the registry's masking field. These design features do not make the descriptive counts unusable, but they limit causal interpretation.
Part A and Part B have distinct schedules: Part A includes 12 weeks of therapy and 12 weeks of follow-up, while Part B includes 24 weeks of therapy and 4 weeks of follow-up. The primary endpoint timeframe is specified as until 24 weeks for Part A and until 28 weeks for Part B. In general, event counts depend on the time participants are observed and on how events are collected. Comparisons between parts therefore require caution, particularly when the observation schedules differ. The registry record does not report an adjustment that would make the part-specific counts comparable by exposure time.
How should uncertainty be understood?
In general, a confidence interval describes the range of estimates compatible with the observed data under a specified statistical method. A narrow interval indicates greater precision under that method; a wide interval indicates more uncertainty. It is not a guarantee that the true value lies in the interval. The registry record does not report a confidence interval for the primary endpoint, so its precision cannot be assessed from a reported interval. No p-value or primary endpoint analysis is reported in the registry record either.
In general, over-dispersion is an issue when observed variation is greater than a statistical model expects. It is especially relevant for count outcomes analyzed with models that assume a particular relationship between the mean and variance. The primary endpoint is described as a percentage of participants, and the registry entries do not identify an over-dispersion model or any fitted model. The registry record does not report methods for modeling repeated events or event counts. General discussion of such methods should not be mistaken for an analysis that was used in this study.
What do sample-size illustrations mean?
Any sample-size illustrations associated with these results are planning exercises, not observed trial outcomes and not observed power. The stated assumptions are a two-sided significance level of 0.05, equal allocation between two arms, and a normal approximation, with the effect to detect expressed as a fraction of the observed effect. The stated convention is participants per arm for non-time-to-event outcomes, while time-to-event illustrations use the total number of events. These are generic planning assumptions attached to the illustration, not evidence that the study itself was designed as a two-arm randomized trial.
The registry record does not report the trial's own sample-size justification, and it does not report that these illustrative assumptions governed the actual study design. Since the trial has a single-group model, an equal-allocation two-arm planning calculation should not be read as describing its observed allocation. Sample-size calculations also do not show that the study had a particular probability of detecting an effect after completion. That probability cannot be inferred from a planning illustration alone.
What analysis details remain unavailable?
The registry record does not report the statistical analysis plan, inferential tests for the primary endpoint, confidence intervals, or methods for handling missing data. It does not report whether analyses were adjusted for baseline characteristics, nor does it provide a detailed account of how rescue treatment or dose adjustment entered any analysis. Those details matter when interpreting safety and clinical activity, but should not be filled in by assumption. The posted counts are therefore most appropriately treated as descriptive entries in the registry record.
10. Multiplicity, Missing Data and Analysis Populations
The registry lists 15 outcome measures, including the primary safety endpoint and secondary outcomes concerning disease control, response, remission, time to events, relapse, and corticosteroid use. Considering multiple outcomes can raise a multiplicity issue: in general, the more statistical tests or outcome comparisons made, the more likely it becomes that at least one result appears noteworthy by chance. How to address that concern depends on the prespecified analysis plan and the purpose of each outcome.
The registry record does not report a statistical analysis plan, a multiplicity adjustment, a hierarchy of outcomes, or inferential analyses. It lists 0 analyses. It is therefore not possible to determine from the record whether any adjustment was planned or applied, or how results across the outcome measures were intended to be interpreted together. The secondary outcome titles identify planned measures, but do not establish that results were available or statistically tested.
Missing-data handling is also not reported in the registry record. In general, missing outcome information can affect an estimate if participants with unobserved outcomes differ from those with observed outcomes. The registry entries do not describe imputation, censoring rules, sensitivity analyses, or the amount and reasons for missing outcome data. No approach should be inferred.
The safety population is described as all patients who received at least 1 dose of rilzabrutinib, and as the population used for all safety analyses. The record gives at-risk counts by study part, but does not report a broader accounting of participants excluded from safety analyses or why any data might be unavailable. These omissions limit assessment of how complete the recorded safety information was and how the endpoint was analyzed.
11. Safety Results
The registry reports serious adverse events as affected participants divided by participants at risk.
| Arm | Participants with serious adverse events | Participants at risk | Percentage |
|---|---|---|---|
| Part A | 3 | 27 | 11.1% |
| Part B | 0 | 15 | 0.0% |
Deaths
| Arm | Deaths (participants affected of at risk) |
|---|---|
| Part A | 0 of 27 |
| Part B | 0 of 15 |
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 |
|---|---|
| Part A | 20 of 27 |
| Part B | 13 of 15 |
Most frequent serious adverse event terms
| Event term | Organ system | Part A | Part B |
|---|---|---|---|
| Pulmonary sequestration | Congenital, familial and genetic disorders | 1 of 27 | 0 of 15 |
| Pancreatic pseudocyst | Gastrointestinal disorders | 1 of 27 | 0 of 15 |
| Cellulitis | Infections and infestations | 1 of 27 | 0 of 15 |
| Pneumonitis | Respiratory, thoracic and mediastinal disorders | 1 of 27 | 0 of 15 |
Most frequent non-serious adverse event terms
| Event term | Organ system | Part A | Part B |
|---|---|---|---|
| Nausea | Gastrointestinal disorders | 6 of 27 | 4 of 15 |
| Headache | Nervous system disorders | 4 of 27 | 1 of 15 |
| Abdominal pain upper | Gastrointestinal disorders | 3 of 27 | 1 of 15 |
| Dizziness | Nervous system disorders | 2 of 27 | 2 of 15 |
| Diarrhoea | Gastrointestinal disorders | 3 of 27 | 0 of 15 |
| Upper respiratory tract infection | Infections and infestations | 2 of 27 | 1 of 15 |
| Fatigue | General disorders | 2 of 27 | 1 of 15 |
| Arthralgia | Musculoskeletal and connective tissue disorders | 2 of 27 | 1 of 15 |
| Muscle spasms | Musculoskeletal and connective tissue disorders | 2 of 27 | 1 of 15 |
| Pain in extremity | Musculoskeletal and connective tissue disorders | 2 of 27 | 1 of 15 |
Serious events with the highest arm-level rate: pulmonary sequestration (3.7%), pancreatic pseudocyst (3.7%), cellulitis (3.7%), pneumonitis (3.7%).
For Part A, the registry reports 3 participants with serious adverse events among 27 at risk, and 20 with other adverse events among 27 at risk. For Part B, it reports 0 with serious adverse events among 15 at risk, and 13 with other adverse events among 15 at risk. These are counts attached to the two study parts, not a randomized comparison. The different schedules and the single-group design mean that the figures should be read descriptively.
The serious adverse-event terms listed are Pulmonary sequestration, Pancreatic pseudocyst, Cellulitis, and Pneumonitis; each is reported as 3.7%. These term entries do not by themselves establish the number of unique affected participants, whether terms occurred together, or whether an event was judged related to treatment. The registry's adverse-event definition says events did not necessarily have to be causally related to treatment.
No deaths are reported for either part. The registry record does not provide further detail in the registry record about event duration, outcomes, timing, or attribution. The serious-event counts should therefore be understood as safety reporting, not as a causal estimate or a complete characterization of each event.
12. What the Result Does, and Does Not, Mean
The posted safety information documents adverse events reported during an open-label study of PRN1008 in adults with newly diagnosed or relapsing pemphigus vulgaris. It gives serious and other adverse-event counts for Part A and Part B, along with several serious-event terms. This can help readers understand what was recorded in the trial's safety reporting and which events appear in the registry summary.
It does not establish that PRN1008 caused the reported events. The registry definition explicitly allows an adverse event to qualify without a causal relationship to treatment. A single-group study also lacks a concurrent control for estimating how the event experience differs from what would have occurred under another treatment or without study treatment. Open-label observation adds further limits to causal interpretation.
The record does not report the overall primary endpoint percentage, a confidence interval, or inferential analyses. The serious and other event counts should not be substituted for that missing primary endpoint result: the endpoint is defined as the percentage of participants with treatment-emergent adverse events, while the posted adverse-event summary divides counts into categories. The registry also lists clinical activity outcomes, but their results are not reported in the record.
Nor does the presence of secondary outcome measures demonstrate a change in disease activity, remission, or corticosteroid use. Those questions require their own reported outcome data and appropriate interpretation. The listed design features, including dose adjustment and possible rescue treatment, are relevant context for understanding the study experience, but the registry does not report how these features affected results.
In general, a pilot study can describe observed experiences and inform questions for further research, but its design and reported results determine what can be concluded. For this record, the defensible interpretation is limited to the descriptive safety counts and terms that are listed. Comparative effects, treatment causality, and clinical activity conclusions are not established by these registry entries.
13. Why This Trial Matters Statistically
This trial is statistically informative because it illustrates the distinction between describing safety events and estimating a treatment effect. The registry identifies a participant-level safety endpoint and reports counts by study part, but the design is open-label and single-group. That combination makes clear why a numerical result cannot be interpreted in isolation from the way participants were assigned and observed.
The study also includes within-participant dose adjustment based on clinical response and BTK occupancy, as well as conventional immunosuppressive rescue treatment if indicated. These features show that the treatment experience was not described as an unchanging dose in isolation. For statistical interpretation, it matters that the registry record does not report how adjustments or rescue treatment were analyzed or how they relate to the outcomes.
The difference in schedules between Part A and Part B is another useful consideration. Part A has a treatment and follow-up schedule of 12 weeks each, while Part B has 24 weeks of therapy and 4 weeks of follow-up. Safety counts from differently structured periods are not automatically comparable. In general, observation time and event definitions influence the meaning of counts, and careful interpretation requires understanding both.
Finally, the record lists multiple outcome measures but does not report analyses or a statistical analysis plan. This makes multiplicity, missing-data handling, and the precision of the primary endpoint difficult to assess. The study therefore offers a practical example of how registry descriptions can identify questions and outcomes while leaving key inferential details unavailable. Its statistical value lies in reading the design, endpoint, and reporting limits together rather than treating event counts as a comparative conclusion.
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.
Several trial-specific limitations shape interpretation. The study is open-label and single-group, with allocation listed as not applicable. There is no concurrent control group in the registry description, so the adverse-event counts cannot estimate a comparative treatment effect. The Part A and Part B labels should not be treated as randomized arms, and their schedules differ.
The primary endpoint is defined, but its overall percentage is not reported in the registry record. The record also does not report confidence intervals, p-values, or an analysis of the primary endpoint. Although the adverse-event summary gives counts for serious and other events, those categories do not provide the complete endpoint estimate as defined.
The study description permits dose adjustment based on clinical response and BTK occupancy and allows conventional immunosuppressive rescue treatment if indicated. The registry record does not report how frequently these occurred, or how they were considered in the safety or clinical activity analyses. This limits interpretation of the treatment exposure represented by the summarized counts.
Other relevant details are also not reported in the registry record: the statistical analysis plan, sample-size justification, multiplicity approach, missing-data methods, and fuller information on event timing, duration, outcomes, or attribution. The listed enrollment of 42 does not resolve these analysis and reporting gaps. The record's counts are useful as descriptive safety information, but the absent details restrict assessment of precision, completeness, and causal meaning.
15. Related Tutorials
16. Related Statistical Calculators
17. Sources
- ClinicalTrials.gov: A Study of PRN1008 in Adult Patients With Pemphigus Vulgaris — NCT02704429.
- Registry record last updated 2023-02-13; status last verified 2022-12.
- Results first posted 2023-02-13.
- 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
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18. Record Summary
The registry identifies NCT02704429 as a completed, open-label, phase 2 pilot study of PRN1008 in adults with newly diagnosed or relapsing pemphigus vulgaris. It lists enrollment as 42 and describes a single-group design without masking or randomized allocation. Treatment was organized into Part A and Part B, with different treatment and follow-up schedules, and could include dose adjustment and conventional immunosuppressive rescue treatment if indicated.
The primary endpoint concerns the percentage of participants with treatment-emergent adverse events. The registry reports serious and other adverse-event counts by part, plus serious-event terms, but does not report the overall primary endpoint percentage. It lists secondary clinical activity outcomes, but their results and statistical analyses are not reported in the registry record. The available information supports a descriptive reading of the posted safety counts, not a randomized comparison or a causal conclusion.