Open Label Exploratory Phase IIa Trial to Investigate the Safety and Efficacy of IFX-1 in Treating Patients With Pyoderma Gangrenosum (OPTIMA)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- InflaRx GmbH
- 入组人数
- 19
- 试验地点
- 10
- 主要终点
- Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs)
研究概览
简要总结
The purpose of this study is to determine whether vilobelimab (development name: IFX-1) is safe and effective in the treatment of pyoderma gangrenosum.
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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:
- •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
排除标准
- •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
研究组 & 干预措施
vilobelimab 800 mg 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 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.
干预措施: vilobelimab (Drug)
vilobelimab 1600 mg 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.
干预措施: vilobelimab (Drug)
vilobelimab 2400 mg 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.
干预措施: vilobelimab (Drug)
结局指标
主要结局
Treatment-emergent Adverse Events (TEAEs), Related TEAEs, Serious TEAEs, and Adverse Events of Special Interest (AESIs)
时间窗: From treatment start until end of study (including observational visits), an average of 249 days
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.
次要结局
- Number of Patients With Physician's Global Assessment (PGA) Score ≤3 (Investigator Assessment)(From treatment start until V16 (Day 189))
- Time to Complete Closure of Pyoderma Gangrenosum Target Ulcer (Investigator Assessment) [Days](From treatment start until end of study (including observational visits), an average of 249 days)
- Percentage Change in Wound Healing (Wound Area) by Photographic Assessment(From treatment start until V16 (Day 189))
- Percentage Change in Wound Healing (Wound Volume) by Photographic Assessment(From treatment start until V16 (Day 189))
- Rate of Change Per Day in Area of Target Ulcer by Photographic Assessment From V1 to V16(From treatment start until V16 (Day 189))
- Number of Patients With ≥ 50% Decrease in Area of Target Ulcer by Photographic Assessment at V16(From treatment start until V16 (Day 189))
- Number of Patients With 100% Decrease in Area of Target Ulcer at V16(From treatment start until V16 (Day 189))
- Degree of Erythema of the Target Ulcer at V4, V6, V10 and V16(From treatment start until V16 (Day 189))
- Border Elevation of the Target Ulcer at Visits V4, V6, V10 and V16(From treatment start until V16 (Day 189))
- Percentage Change in Pain by Numeric Rating Scale (NRS) at Visits V4, V6, V10 and V16(From treatment start until V16 (Day 189))
- Percentage Change in Life Quality by DLQI at Visits V4, V6, V10, and V16(From treatment start until V16 (Day 189))