Clinical trial NCT03971643: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Pyoderma Gangrenosum
Lead sponsor: InflaRx GmbH
Sponsor class: INDUSTRY
Collaborators: name: Innovaderm Research Inc.
First posted: 2019-06-03
Enrollment: 19
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Intervention model description: IV infusions of vilobelimab diluted in sodium chloride
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs)
timeFrame: From treatment start until end of study (including observational visits), an average of 249 days
Secondary outcomes:
measure: Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment)
timeFrame: From treatment start until V16 (Day 189)
measure: Time to Complete Closure of Pyoderma Gangrenosum Target Ulcer (Investigator Assessment) [Days]
timeFrame: From treatment start until end of study (including observational visits), an average of 249 days
measure: Percentage Change in Wound Healing (Wound Area) by Photographic Assessment
timeFrame: From treatment start until V16 (Day 189)
measure: Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment
timeFrame: From treatment start until V16 (Day 189)
measure: Rate of Change Per Day in Area of Target Ulcer by Photographic Assessment From V1 to V16
timeFrame: From treatment start until V16 (Day 189)
measure: Number of Patients With ≥ 50% Decrease in Area of Target Ulcer by Photographic Assessment at V16
timeFrame: From treatment start until V16 (Day 189)
measure: Number of Patients With 100% Decrease in Area of Target Ulcer at V16
timeFrame: From treatment start until V16 (Day 189)
measure: Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16
timeFrame: From treatment start until V16 (Day 189)
measure: Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16
timeFrame: From treatment start until V16 (Day 189)
measure: Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16
timeFrame: From treatment start until V16 (Day 189)
measure: Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16
timeFrame: From treatment start until V16 (Day 189)
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: InflaRx Site #07
state: California
city: Miami
country: United States
facility: InflaRx Site #08
state: Florida
city: Tampa
country: United States
facility: InflaRx Site #03
state: Florida
city: Saint Louis
country: United States
facility: InflaRx Site #10
state: Missouri
city: Columbus
country: United States
facility: InflaRx Site #05
state: Ohio
city: Hershey
country: United States
facility: InflaRx Site #12
state: Pennsylvania
city: Pittsburgh
country: United States
facility: InflaRx Site #09
state: Pennsylvania
city: Richmond Hill
country: Canada
facility: InflaRx Site #01
state: Ontario
city: Rzeszów
country: Poland
facility: InflaRx Site #21
city: Wrocław
country: Poland
facility: InflaRx Site #20
Study officials:
affiliation: InflaRx GmbH
name: Prof. Niels C. Riedemann, M.D., Ph.D.
role: STUDY_DIRECTOR
References: