Adults 18 to 80, any sex, with Pemphigus Vulgaris. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Percentage of Participants With Treatment-emergent Adverse EventsPrimary· Part A: until 24 weeks and Part B: until 28 weeks
Treatment-emergent adverse events (TEAEs) including clinically significant changes in physical examination, laboratory tests, and vital signs. An AE was defined as any untoward medical occurrence in a participant who received study drug and did not necessarily had to have a causal relationship with the treatment. TEAEs were defined as AEs that developed or worsened or became serious during on-treatment phase that was defined as the time from the start of study drug up to study completion.
Any TEAE
Group
Value
95% CI
Part A
74.1
Part B
86.7
Any serious TEAE
Group
Value
95% CI
Part A
11.1
Part B
0
Any TEAE leading to treatment discontinuation
Group
Value
95% CI
Part A
11.1
Part B
0
Percentage of Participants Who Are Able to Achieve Control of Disease Activity (CDA) Within 4 Weeks of Starting PRN1008 Treatment Without the Need for Doses of Prednisone or Prednisolone >0.5 mg/kgPrimary· 4 weeks
CDA was defined as the time at which new lesions cease to form and established lesions begin to heal.
Group
Value
95% CI
Part A
51.9
31.9 – 71.3
Part B
60.0
32.29 – 83.66
Percentage of Participants Able to Achieve Control of Disease Activity (CDA) Without Corticosteroids Within 4 WeeksSecondary· 4 weeks
CDA was defined as the time at which new lesions cease to form and established lesions begin to heal.
Group
Value
95% CI
Part A
11.1
2.4 – 29.2
Part B
6.7
0.17 – 31.95
Percentage of Participants Able to Achieve a Complete Response (CR) Without CorticosteroidsSecondary· Part A: 12 weeks treatment and Part B: 24 weeks treatment
CR was defined as complete healing of all lesions and the absence of new lesions.
Group
Value
95% CI
Part A
0
Part B
0
Percentage of Participants Able to Achieve Complete Remission (CR) Without the Need for Doses of Prednisone or Prednisolone of Greater Than 0.5mg/kgSecondary· Part A: 12 weeks treatment and Part B: 24 weeks treatment
CR was defined as complete healing of all lesions and the absence of new lesions.
Group
Value
95% CI
Part A
14.8
4.2 – 33.7
Part B
33.3
11.82 – 61.62
Time to Control of Disease Activity (CDA)Secondary· Part A: until 24 weeks and Part B: until 28 weeks
CDA was defined as the time at which new lesions cease to form and established lesions begin to heal. Kaplan-Meier estimate median time is reported.
Group
Value
95% CI
Part A
33.0
29.0 – 58.0
Part B
29.0
15.0 – 34.0
Time to End of Consolidation Phase (ECP)Secondary· Part A: until 24 weeks and Part B: until 28 weeks
ECP was defined as the time at which no new lesions have developed for minimum of 2 weeks, and approximately 80% of existing lesions have healed. The median time to ECP was based on Kaplan-Meier estimate which considered censoring at end of follow-up for patients who didn't have an event. A +/-3 days window for visits at Week 3 and 5, and +/-7 days window for visits at Week 9, 13, 17, 21, 25.
Group
Value
95% CI
Part A
170.0
95.0 – NA
Part B
58.0
48.0 – NA
Time to Complete Remission (CR)Secondary· Part A: until 24 weeks and Part B: until 28 weeks
CR was defined as complete healing of all lesions and the absence of new lesions.
Group
Value
95% CI
Part A
NA
NA – NA
Part B
NA
NA – NA
Time to Relapse After PRN1008 Treatment DiscontinuationSecondary· Part A: until 24 weeks and Part B: until 28 weeks
Relapse was defined as appearance of ≥3 new lesions/month that do not heal spontaneously within 1 week, or by extension of established lesions, in a patient who has achieved disease control. The median time to relapse was based on Kaplan-Meier estimate which considered censoring at end of follow-up for patients who didn't have an event. A +/-3 days window for visits at Week 3 and 5, and +/-7 days window for visits at Week 9, 13, 17, 21, 25.
Group
Value
95% CI
Part A
96.0
27.0 – 99.0
Part B
198.0
41.0 – 209.0
Cumulative Corticosteroid UsageSecondary· Part A: until 24 weeks and Part B: until 28 weeks
Cumulative corticosteroid usage
Group
Value
95% CI
Part A
983.43
± 827.676
Part B
2089.600
± 1274.011
Percentage Change From Baseline in Pemphigus Disease Area Index (PDAI) Total Activity ScoresSecondary· Part A: until 24 weeks and Part B: until 28 weeks
The PDAI questionnaire has 2 components including activity and damage. The activity component consists of skin, scalp and mucosa parts, and the damage component consists of skin and scalp parts. The total activity score was used for the summary of PDAI scores. PDAI total activity score = Total skin activity + Total scalp activity + Total mucosa activity. PDAI Total Activity Score ranged from 0 to 250 points representing disease activity (higher scores mean a worse outcome). Negative change in total activity score from baseline indicates improvement in pemphigus activity.
end of treatment
Group
Value
95% CI
Part A
-55.7
± 40.91
Part B
-78.60
± 35.839
end of follow-up
Group
Value
95% CI
Part A
-57.7
± 38.45
Part B
-59.68
± 48.403
Change From Baseline in Autoimmune Bullous Skin Disorder Intensity Score (ABSIS) Total Activity ScoreSecondary· Part A: until 24 weeks and Part B: until 28 weeks
ABSIS Total Activity Score ranged from 0 to 206 points representing disease activity (higher scores mean a worse outcome). Negative change in total activity score from baseline indicates improvement in pemphigus activity.
end of treatment
Group
Value
95% CI
Part A
-8.18
± 14.475
Part B
-6.591
± 4.7664
end of follow-up
Group
Value
95% CI
Part A
-9.98
± 18.369
Part B
-5.705
± 4.6248
Adverse events — posted to ClinicalTrials.gov
Time frame: Part A: until 24 weeks and Part B: until 28 weeks.
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Open-label cohort study in adult patients with newly diagnosed or relapsing pemphigus vulgaris, with intra-patient dose-adjustment based on clinical response and BTK occupancy, and with conventional immunosuppressive "rescue treatment", if indicated. The duration of therapy in Part A will be 12 weeks, followed by 12 weeks of follow up. The extension phase, Part B includes 24 weeks of therapy, followed by 4 weeks of follow-up.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
Pemphigus.
Kasperkiewicz M, Ellebrecht CT, Takahashi H, Yamagami J, et al ·
· 2017
· cited 365×
· PMID 28492232
· DOI 10.1038/nrdp.2017.26
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Principia Biopharma, a Sanofi Company
Last refreshed: 13 February 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT02704429.