Trial Outcomes & Findings for Exploratory Study of IFX-1 in Patients With Pyoderma Gangrenosum (NCT NCT03971643)

Last Updated: 2023-09-14

Results Overview

Number of patients with treatment-emergent adverse events (TEAEs), related TEAEs, serious TEAEs, and adverse events of special interest (AESIs) TEAEs are defined as adverse events that start at or after the first administration of study drug. Related TEAEs are defined as all TEAEs considered by the investigator to have at least a 'possible' relationship with the study drug. AESIs are defined as infusion-related reactions, including acute and delayed hypersensitivity and anaphylactic reactions during or after infusion; Meningitis; Meningococcal septicaemia; Invasive infection. As adverse events will be reported in details in the safety section, no separate reporting on System Organ Class (SOC) or Preferred Term (PT) level etc. is done here.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

From treatment start until end of study (including observational visits), an average of 249 days

Results posted on

2023-09-14

Participant Flow

Participant milestones

Participant milestones
Measure	Vilobelimab 800 mg Q2W Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Overall Study STARTED	6	6	7
Overall Study Completed Until Day 99 (Evaluable)	6	4	7
Overall Study COMPLETED	1	3	6
Overall Study NOT COMPLETED	5	3	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

For two patients the measurements could not be obtained because photos were not taken correctly.

Baseline characteristics by cohort

PRIMARY outcome

Timeframe: From treatment start until end of study (including observational visits), an average of 249 days

Population: Safety set

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs) Treatment-emergent adverse events (TEAEs)	6 Participants	4 Participants	5 Participants
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs) Related TEAEs	0 Participants	2 Participants	2 Participants
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs) Serious TEAEs	1 Participants	1 Participants	2 Participants
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs) Adverse events of special interest (AESIs) as identified by investigator	0 Participants	1 Participants	1 Participants
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs) Adverse events of special interest (AESIs) as identified by sponsor	2 Participants	1 Participants	2 Participants

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety Set

Efficacy endpoint: Proportion of patients with a clinical response, defined as Physician's Global Assessment (PGA) score ≤3 of target ulcer at Visit V4, V6, V10 and V16 (End of Treatment \[EOT\]) (SAF). PGA values: 0 = Completely clear, 1 = Almost clear, 2 =Marked improvement, 3 = Moderate improvement, 4 = Slight improvement, 5 = No change from baseline, 6 = Worse

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment) V4	2 Participants	0 Participants	0 Participants
Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment) V6	1 Participants	1 Participants	3 Participants
Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment) V10	3 Participants	1 Participants	5 Participants
Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment) V16	0 Participants	1 Participants	5 Participants

SECONDARY outcome

Timeframe: From treatment start until end of study (including observational visits), an average of 249 days

Population: Safety Set

Efficacy endpoint: Kaplan-Meier analysis of time to first clinical remission (complete closure of target ulcer) defined as PGA score of ≤ 1, PGA values: 0 = Completely clear, 1 = Almost clear, 2 =Marked improvement, 3 = Moderate improvement, 4 = Slight improvement, 5 = No change from baseline, 6 = Worse

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Time to Complete Closure of Pyoderma Gangrenosum Target Ulcer (Investigator Assessment) [Days]	NA days Interval 62.0 to Not available as values were not calculable due to low number of responders	NA days Not available as values were not calculable due to low number of responders	126 days Interval 77.0 to 197.0

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety Set Only patients with an available wound area measurement at both relevant visits are included in the respective analysis. Wound area for single visits was missing because images were not taken correctly and the measurements could not be obtained or patients discontinued the study prior to the relevant visit.

Efficacy endpoint: Percentage change in wound area \[percent change\] of target ulcer by photographic assessment between Visits V1 and V4, between Visits V1 and V6, between Visits V1 and V16, between Visits V6 and V10, between Visits V10 and V16.

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Percentage Change in Wound Healing (Wound Area) by Photographic Assessment V1-V4	-13.612 percent change Standard Deviation 41.7862	16.099 percent change Standard Deviation 15.0992	-12.620 percent change Standard Deviation 31.9371
Percentage Change in Wound Healing (Wound Area) by Photographic Assessment V1-V6	-4.130 percent change Standard Deviation 74.1725	-8.413 percent change Standard Deviation 47.5261	-34.153 percent change Standard Deviation 53.3455
Percentage Change in Wound Healing (Wound Area) by Photographic Assessment V1-V16	234.068 percent change	-57.538 percent change Standard Deviation 60.0506	-90.918 percent change Standard Deviation 16.2280
Percentage Change in Wound Healing (Wound Area) by Photographic Assessment V6-V10	-22.803 percent change Standard Deviation 52.8937	-16.198 percent change Standard Deviation 58.8229	-63.059 percent change Standard Deviation 50.1507
Percentage Change in Wound Healing (Wound Area) by Photographic Assessment V10-V16	61.451 percent change	8.644 percent change Standard Deviation 5.2038	-51.141 percent change Standard Deviation 69.4133

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety Set Only patients with an available wound volume measurement at both relevant visits are included in the respective analysis. Wound volume for single visits was missing because images were not taken correctly and the measurements could not be obtained or patients discontinued the study prior to the relevant visit. Additionally, sometimes the pictures allowed to measure the wound area, but not the volume as several pictures had to be taken to estimate the volume adequately.

Efficacy endpoint: Percentage change in wound volume \[percent change\] of target ulcer by photographic assessment between Visits V1 and V4, between Visits V1 and V6, between Visits V1 and V16, between Visits V6 and V10, between Visits V10 and V16.

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment V1-V4	-19.733 percent change Standard Deviation 53.4282	74.918 percent change	-36.328 percent change Standard Deviation 32.3445
Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment V1-V6	-17.300 percent change Standard Deviation 90.9283	—	-58.959 percent change Standard Deviation 46.7969
Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment V1-V16	786.937 percent change	—	-92.639 percent change Standard Deviation 15.6580
Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment V6-V10	-38.033 percent change Standard Deviation 82.8031	-100.000 percent change	-82.525 percent change Standard Deviation 30.2680
Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment V10-V16	150.857 percent change	—	4.167 percent change Standard Deviation 177.7658

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety Set Only patients with an available wound area measurement at both relevant visits (V1 and V16) are included in this analysis. Wound area for single visits was missing because images were not taken correctly and the measurements could not be obtained or patients discontinued the study prior to the relevant visit.

Efficacy endpoint: Rate of change per day in area of target ulcer \[mm²/day\] between Visits V1 and V16 (photographic assessment)

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=1 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=2 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=6 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Rate of Change Per Day in Area of Target Ulcer by Photographic Assessment From V1 to V16	15.14 mm²/day	-12.04 mm²/day Standard Deviation 9.638	-9.58 mm²/day Standard Deviation 10.751

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety Set Only patients with an available wound area measurement at baseline and V16 are included in this analysis. Wound area for single visits was missing because images were not taken correctly and the measurements could not be obtained or patients discontinued the study prior to the relevant visit.

Efficacy endpoint: Number of patients with a decrease in area of target ulcer of ≥ 50% by photographic assessment at Visit V16 (EOT) compared to Baseline

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=1 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=2 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=6 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Number of Patients With ≥ 50% Decrease in Area of Target Ulcer by Photographic Assessment at V16	0 Participants	1 Participants	6 Participants

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Efficacy endpoint: Number of patients with a decrease in area of target ulcer of 100% at Visit V16 (EOT) compared to Baseline (remission) (photographic assessment)

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=1 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=2 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=6 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Number of Patients With 100% Decrease in Area of Target Ulcer at V16	0 Participants	1 Participants	2 Participants

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety set

Efficacy endpoint: Degree of erythema of the target ulcer at Visit V4, V6, V10 and V16 (EOT) (investigator assessment)

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V6 · Moderate (2)	2 Participants	2 Participants	2 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V6 · Severe (3)	2 Participants	2 Participants	3 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V6 · Very severe (4)	1 Participants	0 Participants	1 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V10 · None (0)	1 Participants	0 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V10 · Slight (1)	0 Participants	1 Participants	3 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V10 · Moderate (2)	3 Participants	1 Participants	1 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V10 · Severe (3)	2 Participants	2 Participants	2 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V10 · Very severe (4)	0 Participants	0 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V16 · None (0)	0 Participants	1 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V16 · Slight (1)	0 Participants	1 Participants	5 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V16 · Moderate (2)	0 Participants	0 Participants	1 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V16 · Severe (3)	1 Participants	1 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V16 · Very severe (4)	0 Participants	0 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V4 · None (0)	0 Participants	0 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V4 · Slight (1)	0 Participants	0 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V4 · Moderate (2)	2 Participants	2 Participants	1 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V4 · Severe (3)	3 Participants	3 Participants	4 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V4 · Very severe (4)	1 Participants	1 Participants	2 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V6 · None (0)	1 Participants	0 Participants	0 Participants
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16 V6 · Slight (1)	0 Participants	0 Participants	1 Participants

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety Set

Efficacy endpoint: Border elevation of the target ulcer at visits V4, V6, V10 and V16 (EOT) (investigator assessment)

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V4 · None (0)	0 Participants	0 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V4 · Slight (1)	2 Participants	0 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V4 · Moderate (2)	1 Participants	5 Participants	4 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V4 · Severe (3)	3 Participants	0 Participants	3 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V4 · Very severe (4)	0 Participants	1 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V6 · None (0)	2 Participants	0 Participants	1 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V6 · Slight (1)	1 Participants	0 Participants	1 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V6 · Moderate (2)	2 Participants	3 Participants	4 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V6 · Severe (3)	1 Participants	1 Participants	1 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V6 · Very severe (4)	0 Participants	0 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V10 · None (0)	1 Participants	0 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V10 · Slight (1)	2 Participants	1 Participants	6 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V10 · Moderate (2)	3 Participants	3 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V10 · Severe (3)	0 Participants	0 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V10 · Very severe (4)	0 Participants	0 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V16 · None (0)	0 Participants	1 Participants	1 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V16 · Slight (1)	0 Participants	1 Participants	4 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V16 · Moderate (2)	1 Participants	1 Participants	1 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V16 · Severe (3)	0 Participants	0 Participants	0 Participants
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16 V16 · Very severe (4)	0 Participants	0 Participants	0 Participants

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety set Only patients with an available NRS at both relevant visits are included in the respective analysis.

Efficacy endpoint: Percentage change from Baseline in pain assessed by Numeric Rating Scale (NRS) \[percent change\] at visits V4, V6, V10 and V16 (EOT) Mean (SD) The NRS is an 11-point scale (from 0 represent no pain to 10 representing the worst pain the subject can imagine).

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16 V1-V4	-41.6 percent change Standard Deviation 31.29	10.6 percent change Standard Deviation 19.88	-41.4 percent change Standard Deviation 33.77
Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16 V1-V6	-43.3 percent change Standard Deviation 16.43	1.7 percent change Standard Deviation 22.19	-45.0 percent change Standard Deviation 45.23
Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16 V1-V10	-64.3 percent change Standard Deviation 32.55	32.5 percent change Standard Deviation 89.87	-72.2 percent change Standard Deviation 32.07
Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16 V1-V16	-22.2 percent change	5.6 percent change Standard Deviation 141.75	-51.6 percent change Standard Deviation 33.89

SECONDARY outcome

Timeframe: From treatment start until V16 (Day 189)

Population: Safety set Only patients with an available DLQI at both relevant visits are included in the respective analysis.

Efficacy endpoint: Percentage change from Baseline in Dermatology Life Quality Index (DLQI) \[percent change\] at visits V4, V6, V10, and V16 (EOT) The DLQI comprises 10 questions with single scores 0 (No effect on patient's life) to 3 (large effect on patient's life). The DLQI total score is calculated by summing up the score of each question resulting in a minimum of 0 (best) and a maximum of 30 (worst).

Outcome measures

Outcome measures
Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W),with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16 V1-V4	-29.9 percent change Standard Deviation 22.75	-3.8 percent change Standard Deviation 51.05	-33.1 percent change Standard Deviation 28.61
Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16 V1-V6	-31.4 percent change Standard Deviation 20.29	-33.2 percent change Standard Deviation 29.68	-24.7 percent change Standard Deviation 34.30
Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16 V1-V10	-50.0 percent change Standard Deviation 28.01	-33.9 percent change Standard Deviation 31.26	-43.1 percent change Standard Deviation 28.17
Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16 V1-V16	4.5 percent change	-2 percent change Standard Deviation 56.07	-50.8 percent change Standard Deviation 45.10

Adverse Events

Vilobelimab 800 mg Q2W

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Vilobelimab 1600 mg Q2W

Serious events: 1 serious events

Other events: 3 other events

Deaths: 0 deaths

Vilobelimab 2400 mg Q2W

Serious events: 2 serious events

Other events: 5 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Vilobelimab 800 mg Q2W n=6 participants at risk Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 participants at risk Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 participants at risk Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Infections and infestations Endocarditis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Erysipelas	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Intervertebral discitis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Pneumonia	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Sepsis	16.7% 1/6 • Number of events 2 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Superinfection bacterial	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Pyoderma gangrenosum	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Rash	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days

Other adverse events

Additional Information

Prof. Dr. Niels C. Riedemann

InflaRx

Phone: +49-(0) 3641-508 180

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Publication of the results of the Study conducted at the Study Site shall not be made before the first multi-site publication by Sponsor occured within 18 months
Publication restrictions are in place

Restriction type: OTHER

Measure	Vilobelimab 800 mg Q2W n=6 Participants Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 Participants Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 Participants Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).	Total n=19 Participants Total of all reporting groups
Race (NIH/OMB) Black or African American	0 Participants n=6 Participants	2 Participants n=6 Participants	0 Participants n=7 Participants	2 Participants n=19 Participants
Race (NIH/OMB) White	6 Participants n=6 Participants	4 Participants n=6 Participants	7 Participants n=7 Participants	17 Participants n=19 Participants
Race (NIH/OMB) More than one race	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=7 Participants	0 Participants n=19 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=7 Participants	0 Participants n=19 Participants
Baseline Disease Characteristic: Duration of pyoderma gangrenosum	1.7 years STANDARD_DEVIATION 1.86 • n=6 Participants	5.7 years STANDARD_DEVIATION 9.54 • n=6 Participants	3.4 years STANDARD_DEVIATION 6.05 • n=7 Participants	3.6 years STANDARD_DEVIATION 6.41 • n=19 Participants
Age, Continuous	59.5 years STANDARD_DEVIATION 11.41 • n=6 Participants	60.5 years STANDARD_DEVIATION 12.36 • n=6 Participants	43.0 years STANDARD_DEVIATION 15.00 • n=7 Participants	53.7 years STANDARD_DEVIATION 14.99 • n=19 Participants
Sex: Female, Male Female	4 Participants n=6 Participants	3 Participants n=6 Participants	3 Participants n=7 Participants	10 Participants n=19 Participants
Sex: Female, Male Male	2 Participants n=6 Participants	3 Participants n=6 Participants	4 Participants n=7 Participants	9 Participants n=19 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=6 Participants	0 Participants n=6 Participants	1 Participants n=7 Participants	1 Participants n=19 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	6 Participants n=6 Participants	6 Participants n=6 Participants	6 Participants n=7 Participants	18 Participants n=19 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=7 Participants	0 Participants n=19 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=7 Participants	0 Participants n=19 Participants
Race (NIH/OMB) Asian	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=7 Participants	0 Participants n=19 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=6 Participants	0 Participants n=6 Participants	0 Participants n=7 Participants	0 Participants n=19 Participants
Baseline Disease Characteristic: Wound area at baseline	1692.374 area [mm²] STANDARD_DEVIATION 1391.4307 • n=5 Participants • For two patients the measurements could not be obtained because photos were not taken correctly.	7898.952 area [mm²] STANDARD_DEVIATION 5844.5941 • n=5 Participants • For two patients the measurements could not be obtained because photos were not taken correctly.	1827.553 area [mm²] STANDARD_DEVIATION 1901.2830 • n=7 Participants • For two patients the measurements could not be obtained because photos were not taken correctly.	3573.500 area [mm²] STANDARD_DEVIATION 4320.3723 • n=17 Participants • For two patients the measurements could not be obtained because photos were not taken correctly.

Measure	Vilobelimab 800 mg Q2W n=6 participants at risk Group 1 (N=6) continued to receive vilobelimab 800 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 1600 mg every Q2W.	Vilobelimab 1600 mg Q2W n=6 participants at risk Group 2 (N=6) received vilobelimab 1600 mg every 2 weeks (Q2W), with option to increase dose from Day 57 to 2400 mg every Q2W.	Vilobelimab 2400 mg Q2W n=7 participants at risk Group 3 (N=7) received vilobelimab 2400 mg every 2 weeks (Q2W).
Infections and infestations Wound infection	33.3% 2/6 • Number of events 3 • From treatment start until end of study (including observational visits), an average of 249 days	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Wound infection pseudomonas	33.3% 2/6 • Number of events 2 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Abscess limb	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Cellulitis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Gingivitis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Impetigo	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Nasopharyngitis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Oral herpes	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Pneumonia	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Infections and infestations Urinary tract infection	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Dermatitis contact	33.3% 2/6 • Number of events 2 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Pyoderma gangrenosum	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	28.6% 2/7 • Number of events 2 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Actinic keratosis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Dermatitis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Dry skin	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Lichen planus	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Seborrhoeic dermatitis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Skin and subcutaneous tissue disorders Urticaria	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Injury, poisoning and procedural complications Traumatic haematoma	33.3% 2/6 • Number of events 2 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Injury, poisoning and procedural complications Burns second degree	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Injury, poisoning and procedural complications Contusion	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Injury, poisoning and procedural complications Head injury	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Injury, poisoning and procedural complications Skin laceration	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Gastrointestinal disorders Diarrhoea	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Gastrointestinal disorders Glossitis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Gastrointestinal disorders Peptic ulcer	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Gastrointestinal disorders Vomiting	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
General disorders Influenza like illness	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
General disorders Oedema peripheral	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
General disorders Pyrexia	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	16.7% 1/6 • Number of events 2 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Investigations Blood creatinine increased	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Investigations Haemoglobin decreased	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Vascular disorders Hypertension	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Vascular disorders Superficial vein thrombosis	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Endocrine disorders Hypothyroidism	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days
Nervous system disorders Syncope	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Psychiatric disorders Alcohol abuse	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	14.3% 1/7 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days
Renal and urinary disorders Haematuria	16.7% 1/6 • Number of events 1 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/6 • From treatment start until end of study (including observational visits), an average of 249 days	0.00% 0/7 • From treatment start until end of study (including observational visits), an average of 249 days