Back to resultsExploratory Study of IFX-1 in Patients With Pyoderma Gangrenosum
Primary Purpose
Pyoderma Gangrenosum
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
vilobelimab
Sponsored by
About this trial
This is an interventional treatment trial for Pyoderma Gangrenosum
Eligibility Criteria
Inclusion Criteria:
- Diagnosis of an ulcerative form of pyoderma gangrenosum confirmed by the investigator
In addition, the subject must fulfill at least 3 of the following 6 criteria at screening:
History of
- Pathergy (ulcer occurring at the sites of trauma)
- Personal history of inflammatory bowel disease or inflammatory arthritis
- History of papule, pustule or vesicle that rapidly ulcerated
Clinical examination (or photographic evidence) of
- Peripheral erythema, undermining border, and tenderness at site of ulceration
- Multiple ulcerations (at least 1 occurring on the lower leg)
- Cribriform or "wrinkled paper" scar(s) at sites of healed ulcers
Subject has a minimum of 1 evaluable ulcer (≥2 cm2) on the lower extremity at screening
Exclusion Criteria:
- Pyoderma gangrenosum target ulcer for more than 3 years before screening
- Surgical wound debridement within the previous 2 weeks before screening
- Use of intravenous antibacterials, antivirals, anti-fungals, or anti-parasitic agents within 30 days before screening
- Any drug treatment for pyoderma gangrenosum including corticosteroids (>10 mg), intralesional steroids, cyclosporine A, biologicals and immunosuppressives (with the exception of antibiotics for wound superinfection) used within a time of 5 half-lives of the drug before screening
Sites / Locations
- InflaRx Site #07
- InflaRx Site #08
- InflaRx Site #03
- InflaRx Site #10
- InflaRx Site #05
- InflaRx Site #12
- InflaRx Site #09
- InflaRx Site #01
- InflaRx Site #21
- InflaRx Site #20
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Experimental
Arm Label
vilobelimab 800 mg Q2W
vilobelimab 1600 mg Q2W
vilobelimab 2400 mg Q2W
Arm Description
Dose finding with a total of 15 doses of vilobelimab. Vilobelimab: IV infusions of vilobelimab diluted in sodium chloride. All patients received vilobelimab 800 mg three times during the first week (Days 1, 4, and 8). Starting at Day 15: 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.
Dose finding with a total of 15 doses of vilobelimab. Vilobelimab: IV infusions of vilobelimab diluted in sodium chloride. All patients received vilobelimab 800 mg three times during the first week (Days 1, 4, and 8). Starting at Day 15: 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.
Dose finding with a total of 15 doses of vilobelimab. Vilobelimab: IV infusions of vilobelimab diluted in sodium chloride. All patients received vilobelimab 800 mg three times during the first week (Days 1, 4, and 8). Starting at Day 15: Group 3 (N=7) received vilobelimab 2400 mg every Q2W.
Outcomes
Primary Outcome Measures
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs)
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.
Secondary Outcome Measures
Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment)
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
Time to Complete Closure of Pyoderma Gangrenosum Target Ulcer (Investigator Assessment) [Days]
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
Percentage Change in Wound Healing (Wound Area) by Photographic Assessment
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.
Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment
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.
Rate of Change Per Day in Area of Target Ulcer by Photographic Assessment From V1 to V16
Efficacy endpoint: Rate of change per day in area of target ulcer [mm²/day] between Visits V1 and V16 (photographic assessment)
Number of Patients With ≥ 50% Decrease in Area of Target Ulcer by Photographic Assessment at V16
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
Number of Patients With 100% Decrease in Area of Target Ulcer at V16
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)
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16
Efficacy endpoint: Degree of erythema of the target ulcer at Visit V4, V6, V10 and V16 (EOT) (investigator assessment)
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16
Efficacy endpoint: Border elevation of the target ulcer at visits V4, V6, V10 and V16 (EOT) (investigator assessment)
Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16
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).
Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16
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).
Full Information
NCT ID
NCT03971643
First Posted
May 28, 2019
Last Updated
September 4, 2023
Sponsor
InflaRx GmbH
Collaborators
Innovaderm Research Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03971643
Brief Title
Exploratory Study of IFX-1 in Patients With Pyoderma Gangrenosum
Official Title
Open Label Exploratory Phase IIa Trial to Investigate the Safety and Efficacy of IFX-1 in Treating Patients With Pyoderma Gangrenosum (OPTIMA)
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
May 16, 2019 (Actual)
Primary Completion Date
January 3, 2022 (Actual)
Study Completion Date
January 3, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
InflaRx GmbH
Collaborators
Innovaderm Research Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. Study Description
Brief Summary
The purpose of this study is to determine whether vilobelimab (development name: IFX-1) is safe and effective in the treatment of pyoderma gangrenosum.
Detailed Description
Neutrophilic dermatoses are a spectrum of inflammatory disorders characterized by skin lesions resulting from a neutrophil-rich inflammatory infiltrate in the absence of infection. Pyoderma gangrenosum is associated with a neutrophilic leukocytosis, which is likely to be triggered by C5a. This study is set up based on the hypothesis that vilobelimab might be able to block C5a induced pro-inflammatory effects such as neutrophil activation and cytokine generation, potentially contributing to the local skin inflammation and tissue damage.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Pyoderma Gangrenosum
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
IV infusions of vilobelimab diluted in sodium chloride
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
19 (Actual)
8. Arms, Groups, and Interventions
Arm Title
vilobelimab 800 mg Q2W
Arm Type
Experimental
Arm Description
Dose finding with a total of 15 doses of vilobelimab.
Vilobelimab: IV infusions of vilobelimab diluted in sodium chloride.
All patients received vilobelimab 800 mg three times during the first week (Days 1, 4, and 8).
Starting at Day 15:
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.
Arm Title
vilobelimab 1600 mg Q2W
Arm Type
Experimental
Arm Description
Dose finding with a total of 15 doses of vilobelimab.
Vilobelimab: IV infusions of vilobelimab diluted in sodium chloride.
All patients received vilobelimab 800 mg three times during the first week (Days 1, 4, and 8).
Starting at Day 15:
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.
Arm Title
vilobelimab 2400 mg Q2W
Arm Type
Experimental
Arm Description
Dose finding with a total of 15 doses of vilobelimab.
Vilobelimab: IV infusions of vilobelimab diluted in sodium chloride.
All patients received vilobelimab 800 mg three times during the first week (Days 1, 4, and 8).
Starting at Day 15:
Group 3 (N=7) received vilobelimab 2400 mg every Q2W.
Intervention Type
Drug
Intervention Name(s)
vilobelimab
Other Intervention Name(s)
IFX-1, CaCP29
Intervention Description
IV infusions of vilobelimab diluted in sodium chloride.
Primary Outcome Measure Information:
Title
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs)
Description
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.
Time Frame
From treatment start until end of study (including observational visits), an average of 249 days
Secondary Outcome Measure Information:
Title
Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment)
Description
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
Time Frame
From treatment start until V16 (Day 189)
Title
Time to Complete Closure of Pyoderma Gangrenosum Target Ulcer (Investigator Assessment) [Days]
Description
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
Time Frame
From treatment start until end of study (including observational visits), an average of 249 days
Title
Percentage Change in Wound Healing (Wound Area) by Photographic Assessment
Description
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.
Time Frame
From treatment start until V16 (Day 189)
Title
Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment
Description
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.
Time Frame
From treatment start until V16 (Day 189)
Title
Rate of Change Per Day in Area of Target Ulcer by Photographic Assessment From V1 to V16
Description
Efficacy endpoint: Rate of change per day in area of target ulcer [mm²/day] between Visits V1 and V16 (photographic assessment)
Time Frame
From treatment start until V16 (Day 189)
Title
Number of Patients With ≥ 50% Decrease in Area of Target Ulcer by Photographic Assessment at V16
Description
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
Time Frame
From treatment start until V16 (Day 189)
Title
Number of Patients With 100% Decrease in Area of Target Ulcer at V16
Description
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)
Time Frame
From treatment start until V16 (Day 189)
Title
Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16
Description
Efficacy endpoint: Degree of erythema of the target ulcer at Visit V4, V6, V10 and V16 (EOT) (investigator assessment)
Time Frame
From treatment start until V16 (Day 189)
Title
Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16
Description
Efficacy endpoint: Border elevation of the target ulcer at visits V4, V6, V10 and V16 (EOT) (investigator assessment)
Time Frame
From treatment start until V16 (Day 189)
Title
Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16
Description
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).
Time Frame
From treatment start until V16 (Day 189)
Title
Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16
Description
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).
Time Frame
From treatment start until V16 (Day 189)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Diagnosis of an ulcerative form of pyoderma gangrenosum confirmed by the investigator
In addition, the subject must fulfill at least 3 of the following 6 criteria at screening:
History of
Pathergy (ulcer occurring at the sites of trauma)
Personal history of inflammatory bowel disease or inflammatory arthritis
History of papule, pustule or vesicle that rapidly ulcerated
Clinical examination (or photographic evidence) of
Peripheral erythema, undermining border, and tenderness at site of ulceration
Multiple ulcerations (at least 1 occurring on the lower leg)
Cribriform or "wrinkled paper" scar(s) at sites of healed ulcers
Subject has a minimum of 1 evaluable ulcer (≥2 cm2) on the lower extremity at screening
Exclusion Criteria:
Pyoderma gangrenosum target ulcer for more than 3 years before screening
Surgical wound debridement within the previous 2 weeks before screening
Use of intravenous antibacterials, antivirals, anti-fungals, or anti-parasitic agents within 30 days before screening
Any drug treatment for pyoderma gangrenosum including corticosteroids (>10 mg), intralesional steroids, cyclosporine A, biologicals and immunosuppressives (with the exception of antibiotics for wound superinfection) used within a time of 5 half-lives of the drug before screening
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Prof. Niels C. Riedemann, M.D., Ph.D.
Organizational Affiliation
InflaRx GmbH
Official's Role
Study Director
Facility Information:
Facility Name
InflaRx Site #07
City
Sacramento
State/Province
California
ZIP/Postal Code
95816
Country
United States
Facility Name
InflaRx Site #08
City
Miami
State/Province
Florida
ZIP/Postal Code
33125
Country
United States
Facility Name
InflaRx Site #03
City
Tampa
State/Province
Florida
ZIP/Postal Code
33613
Country
United States
Facility Name
InflaRx Site #10
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
InflaRx Site #05
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
InflaRx Site #12
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
InflaRx Site #09
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
InflaRx Site #01
City
Richmond Hill
State/Province
Ontario
Country
Canada
Facility Name
InflaRx Site #21
City
Rzeszów
ZIP/Postal Code
35-055
Country
Poland
Facility Name
InflaRx Site #20
City
Wrocław
ZIP/Postal Code
50-566
Country
Poland
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Exploratory Study of IFX-1 in Patients With Pyoderma Gangrenosum
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