Vilobelimab for Pyoderma Gangrenosum

No longer recruiting at 88 trial locations

Overseen By Eva Wagner, Dr.

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: InflaRx GmbH

Must not be taking: Antibiotics, Vaccines, Corticosteroids, others

Disqualifiers: Large ulcers, Transplanted skin, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 13

Duration: 26 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test the effectiveness and safety of a new treatment called vilobelimab (also known as Gohibic or IFX-1) for people with ulcerative pyoderma gangrenosum, a painful skin condition that causes open sores. Participants will receive either vilobelimab or a placebo (a substance with no active medicine) to compare results. Those with at least one ulcer that is 5 cm² or larger, surrounded by healthy skin, and who haven't had certain treatments recently may be eligible to join. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to potentially bringing a new treatment to market.

Will I have to stop taking my current medications?

The trial requires that you stop any systemic medical treatment or biological/immunomodulatory therapy for pyoderma gangrenosum (PG) at least 4 weeks before starting the trial. However, if you are on a stable dose of a biologic or immunomodulatory therapy for another condition, you may continue it.

Is there any evidence suggesting that vilobelimab is likely to be safe for humans?

Research has shown that vilobelimab underwent safety testing in earlier studies. In one study, most side effects were mild to moderate, occurring in 15 out of 19 patients, or about 79%. This indicates that while some patients experienced side effects, they were generally not serious.

Vilobelimab has also shown promise in treating conditions like pyoderma gangrenosum and hidradenitis suppurativa, suggesting it might be safe for use in similar skin conditions. As this trial is in a later phase, evidence already indicates that vilobelimab is generally well-tolerated in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for pyoderma gangrenosum?

Researchers are excited about vilobelimab for pyoderma gangrenosum because it targets a specific part of the immune system called complement factor C5a, which plays a role in inflammation. Most traditional treatments for this condition, like corticosteroids and immunosuppressants, focus broadly on reducing immune activity, which can lead to a range of side effects. Vilobelimab, on the other hand, offers a more targeted approach, potentially reducing inflammation with fewer side effects. This specificity not only promises better outcomes but also enhances patient safety and comfort.

What evidence suggests that vilobelimab might be an effective treatment for pyoderma gangrenosum?

Research shows that vilobelimab, which participants in this trial may receive, may help treat pyoderma gangrenosum, a rare skin condition. Previous studies have found that vilobelimab works well and is safe for this condition. For example, one study showed that patients experienced symptom relief, indicating that vilobelimab can effectively reduce symptoms. Additionally, safety data from these studies suggest that the treatment is generally well-tolerated, with most side effects being mild to moderate. Overall, these findings support the potential effectiveness of vilobelimab for people with pyoderma gangrenosum. ^{1 2 3 4 6}

Who Is on the Research Team?

Alex GO Loayza, Prof, MD

Principal Investigator

university

Are You a Good Fit for This Trial?

Adults diagnosed with ulcerative pyoderma gangrenosum (PG) can join this trial. They must have a PG symptom score of 10 or more and at least one qualifying PG ulcer larger than 5 cm2. People are excluded if they've had certain treatments for PG, infections needing systemic treatment, vaccinations within two weeks before the trial starts, or previous exposure to vilobelimab.

Inclusion Criteria

The doctor has confirmed that you have ulcerative pyoderma gangrenosum and your PARACELSUS score is 10 points or more. If your score is less than 10, the doctor needs to explain why they still think you have ulcerative pyoderma gangrenosum.

I have a skin ulcer larger than 5 cm2 that can be measured in two ways.

Exclusion Criteria

I have not had any systemic treatment for PG in the last 4 weeks.

I haven't taken antibiotics or anti-infectives for an infection in the last 2 weeks.

I have been taking more than 10 mg/day of prednisone or its equivalent for the past 4 weeks.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive vilobelimab or placebo intravenously every 2 weeks for 26 weeks

26 weeks

13 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Vilobelimab

Trial Overview The trial is testing the effectiveness and safety of a drug called vilobelimab against a placebo in treating ulcerative pyoderma gangrenosum. Participants will be randomly assigned to receive either vilobelimab or a placebo without knowing which one they're getting.

How Is the Trial Designed?

Group I: vilobelimab Experimental Treatment 1 Intervention

Patients will be treated with vilobelimab IV, Q2W for 26 weeks

Group II: Placebo Placebo Group 1 Intervention

Patients will receive placebo IV in the same schedule as patients in Arm 1

Vilobelimab is already approved in United States, European Union for the following indications:

Approved in United States as Gohibic for:

• COVID-19 in hospitalized adults requiring mechanical ventilation or extracorporeal membrane oxygenation

Approved in European Union as Gohibic for:

• SARS-CoV-2-induced acute respiratory distress syndrome (ARDS) in adults receiving systemic corticosteroids

Who Is Running the Clinical Trial?

InflaRx GmbH

Lead Sponsor

Trials

16

Recruited

2,300+

Founded

2007

Headquarters

Jena, Germany

Known For

Inflammation research

Top Products

Gohibic (vilobelimab)

Published Research Related to This Trial

In active vitiligo patients, there is a significant increase in pro-inflammatory dendritic cells (CD11b+CD11c+) and pro-inflammatory cytokine IL-17A, which may contribute to skin depigmentation.

Conversely, anti-inflammatory dendritic cells (CD11b+) are reduced in active vitiligo, suggesting an imbalance in immune response that could exacerbate the condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dendritic cells sub-sets are associated with inflammatory cytokine production in progressive vitiligo disease. Srivastava, N., Bishnoi, A., Parsad, D., et al.[2022]

Treatment with a PD-L1 fusion protein significantly reversed depigmentation in Pmel-1 vitiligo mice, suggesting its potential as a therapeutic target for vitiligo.

The mechanism of action involves an increase in regulatory T cells (Tregs) in the skin, spleen, and circulation, indicating that PD-L1 therapy may inhibit the immune response responsible for depigmentation.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

PD-L1 reverses depigmentation in Pmel-1 vitiligo mice by increasing the abundance of Tregs in the skin. Miao, X., Xu, R., Fan, B., et al.[2019]

In a review of 148 cases of difficult-to-treat pyoderma gangrenosum (PG), it was found that the condition is more prevalent in females and those with inflammatory bowel disease, but most patients healed within a typical timeframe despite needing three or more treatments.

The study suggests that initiating biologic therapies earlier may be beneficial for PG patients, highlighting the need for standardized terminology in the field to improve treatment comparisons and outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Recalcitrant Pyoderma Gangrenosum: Clinical Burden and Unmet Needs. Becker, SL., Velasco, R., Ortega-Loayza, AG.[2023]

The pilot registry, which included 52 patients with pyoderma gangrenosum (PG), found that systemic immunomodulators used at the baseline visit were significantly associated with improved healing outcomes (P = 0.048).

Most patients were treated with first-line systemic immunosuppressants, highlighting the need for standardized wound care regimens and data collection to better evaluate treatment effectiveness in PG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum study pilot registry: The first step to a better understanding. Orfaly, VE., Reese, AM., Friedman, M., et al.[2022]

A 38-year-old male with inflammatory vitiligo with raised borders (IVRB) showed significant improvement after receiving two courses of oral prednisone and whole-body narrow-band ultraviolet B (NB-UVB) therapy.

The treatment not only halted the spread of the disease but also led to substantial repigmentation, suggesting that early and aggressive intervention can be effective for this rare subtype of vitiligo.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Repigmentation of extensive inflammatory vitiligo with raised borders using early and aggressive treatment. Gunasekera, N., Murphy, GF., Sheth, VM.[2015]

Vedolizumab is an effective treatment for moderate to severe Crohn's disease and ulcerative colitis, as demonstrated in phase III GEMINI studies, which included induction and maintenance therapy.

A case report highlights that vedolizumab may also help manage severe pyoderma gangrenosum in a Crohn's disease patient, although more clinical data is needed to confirm its efficacy for extraintestinal manifestations.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Vedolizumab (Entyvio®) for the Treatment of Pyoderma Gangrenosum in a Crohn's Disease Patient. Groudan, K., Gupta, K., Singhania, R.[2021]

In a 26-week study involving 22 Japanese patients with pyoderma gangrenosum, adalimumab led to a significant reduction in ulcer size, with 54.5% of patients achieving complete skin re-epithelialization by week 26.

Adalimumab was generally well tolerated, although 18 patients experienced adverse events, primarily infections, indicating that while effective, monitoring for side effects is important.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An update on adalimumab for pyoderma gangrenosum. Yamamoto, T.[2021]

A young boy with refractory idiopathic childhood pyoderma gangrenosum, who had been suffering from the condition since age 3, showed poor responses to various cytotoxic and steroid treatments.

After starting infliximab, the boy experienced a dramatic improvement and achieved full recovery, which was maintained over a 2-year follow-up period.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Infliximab in treatment of idiopathic refractory childhood pyoderma gangrenosum (PG). Salehzadeh, F., Mohammadikebar, Y., Enteshary, A., et al.[2020]

Infliximab, a monoclonal antibody that targets TNF-alpha, was successfully used to treat a 57-year-old female patient with refractory pyoderma gangrenosum linked to inactive Crohn's disease.

This case suggests that Infliximab can be a safe and effective treatment option for pyoderma gangrenosum associated with Crohn's disease, even when the bowel disease is not currently active, although multiple infusions may be necessary.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Infliximab for treatment of pyoderma gangrenosum associated with clinically inactive Crohn's disease. A case report. Kouklakis, G., Moschos, J., Leontiadis, GI., et al.[2015]

A patient with treatment-resistant pyoderma gangrenosum, associated with inflammatory bowel disease, did not respond to multiple immunosuppressive therapies but showed significant improvement after switching to adalimumab, a fully humanized anti-TNF-alpha monoclonal antibody.

This case suggests that adalimumab may be an effective treatment option for pyoderma gangrenosum, particularly in patients who do not respond to other TNF-alpha inhibitors like infliximab.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Adalimumab therapy for recalcitrant pyoderma gangrenosum. Fonder, MA., Cummins, DL., Ehst, BD., et al.[2020]

IFX-1, a complement C5a inhibitor, shows promise in treating pyoderma gangrenosum (PG) by reducing neutrophil activation and inflammation, which are key factors in the disease's pathogenesis.

Early clinical investigations suggest that IFX-1 may be effective for PG and other neutrophilic conditions, but further studies are needed to determine the best dosing and treatment strategies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pyoderma gangrenosum: proposed pathogenesis and current use of biologics with an emphasis on complement C5a inhibitor IFX-1. Lu, JD., Milakovic, M., Ortega-Loayza, AG., et al.[2022]

New diagnostic criteria for pyoderma gangrenosum have been proposed to help speed up diagnosis and treatment, which is crucial to reduce complications and improve patient outcomes.

For treatment, local immunosuppressive therapies are recommended for mild cases, while severe cases may require systemic glucocorticoids, ciclosporin, or TNF-alpha inhibitors, with other biologics also being explored.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Pyoderma gangrenosum]. Burian, EA., Karlsmark, T., Fogh, K., et al.[2021]

Pyoderma gangrenosum (PG) is primarily treated with systemic agents, including corticosteroids, cyclosporine A, and biologics like infliximab and adalimumab, with the best evidence supporting the use of cyclosporine, prednisolone, and infliximab, particularly in patients with ulcerative colitis or Crohn's disease.

Future treatments for PG may improve due to ongoing clinical studies that aim to enhance understanding of the disease and develop targeted therapies that offer higher efficacy and fewer side effects compared to traditional immunosuppressants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment options for pyoderma gangrenosum. Quist, SR., Kraas, L.[2019]

Plasmacytoid dendritic cells (pDC) are involved in the immune response in progressive vitiligo, producing interferon (IFN)-alpha and contributing to the local immune environment.

The presence of MxA, a protein linked to IFN-α, in perilesional skin suggests that early immune responses may drive the progression of vitiligo, highlighting a new innate immune pathway in the disease's development.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Type I interferon signature in the initiation of the immune response in vitiligo. Bertolotti, A., Boniface, K., Vergier, B., et al.[2014]

In a study of 20 patients with active non-segmental vitiligo (NSV) undergoing 36 sessions of narrowband ultraviolet B (NB-UVB) therapy, significant changes were observed in immune markers: levels of IL-17 and IL-22 decreased while FoxP3 levels increased after treatment.

The study suggests that NB-UVB therapy may improve vitiligo by restoring the balance between Th17 cells (which promote inflammation) and regulatory T cells (Tregs, which help control immune responses), indicating a potential mechanism of action for this treatment.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

T helper 17 and Tregs: a novel proposed mechanism for NB-UVB in vitiligo. Hegazy, RA., Fawzy, MM., Gawdat, HI., et al.[2023]

Citations

1. inflarx.de inflarx.de/Home/Investors/Press-Releases/Press-Release~2025-05-InflaRx-Announces-Outcome-of-Interim-Analysis-for-Vilobelimab-Phase-3-Trial-in-Pyoderma-Gangrenosum~.html

Press Release The results showed that vilobelimab treatment improved survival with a relative reduction in 28-day all-cause mortality of 23.9% compared to ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03971643

Exploratory Study of IFX-1 in Patients With Pyoderma ... The purpose of this study is to determine whether vilobelimab (development name: IFX-1) is safe and effective in the treatment of pyoderma gangrenosum.

3. dermatologytimes.com dermatologytimes.com/view/inflarx-presents-efficacy-and-safety-data-on-vilobelimab-at-aad-2025

InflaRX Presents Efficacy and Safety Data on Vilobelimab ... Vilobelimab shows promise in treating pyoderma gangrenosum and hidradenitis suppurativa, with significant efficacy and safety data presented at ...

4. clinicaltrials.gov clinicaltrials.gov/study/NCT05964413

Study Details | NCT05964413 | Phase III Trial to Investigate ... A randomized, double-blind, placebo-controlled, multicenter, adaptive phase III trial to investigate efficacy and safety of vilobelimab in the treatment of ...

5. jaad.org jaad.org/article/S0190-9622(25)01127-2/fulltext

63560 Vilobelimab Safety in Pyoderma Gangrenosum ... Results: Efficacy was reported elsewhere. Adverse events (AE) were mostly mild to moderate and occurred in 15 of 19 patients (78.9%): six (100.0%) in Group ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03971643

Exploratory Study of IFX-1 in Patients With Pyoderma ... The purpose of this study is to determine whether vilobelimab (development name: IFX-1) is safe and effective in the treatment of pyoderma gangrenosum.