A Phase 1B/2, Multicenter, Open-Label, Safety, and Dose-Finding Study of SYNT001 in Subjects With Pemphigus (Vulgaris or Foliaceus)
试验速览
- 阶段
- 1 期
- 状态
- 终止
- 入组人数
- 8
- 试验地点
- 1
- 主要终点
- Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
研究概览
简要总结
This was a multicenter, open-label safety study to determine the dose regimen of SYNT001 (ALXN1830) administered intravenously in participants with pemphigus (vulgaris or foliaceus).
详细描述
This study planned to evaluate 2 cohorts: up to 8 participants to receive 5 weekly intravenous (IV) doses of ALXN1830 at 10 milligram/kilogram (mg/kg) (Cohort 1) and up to 12 participants to receive 3 x 30 mg/kg weekly doses of ALXN1830 IV (loading) followed by 5 x 10 mg/kg doses of ALXN1830 IV every other week or 10 weekly doses of ALXN1830 IV (maintenance) (Cohort 2).
This study was terminated after the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy were characterized in participants with pemphigus at a single dose level (10 mg/kg) in Cohort 1, before any participants were enrolled in Cohort 2.
The study consisted of 3 periods: Screening, Treatment, and Follow-Up.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 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
- •Participants must have meet the following criteria to be included:
- •Were willing and able to read, understand and sign an informed consent form
- •Documented diagnosis of pemphigus vulgaris or foliaceus
- •Were required to use medically acceptable contraception
排除标准
- •Participants meeting any of the following criteria were excluded:
- •Were unable or unwilling to comply with the protocol
- •Active non-hematologic malignancy or history of non-hematologic malignancy in the 3 years prior to screening (exclusive of non-melanoma skin cancer and cervical cancer in situ)
- •Positive for human immunodeficiency virus (HIV) or hepatitis C antibody
- •Positive for hepatitis B surface antigen
- •IV immunoglobulin treatment within 30 days of screening
- •Any exposure to an investigational drug or device within the 30 days prior to screening
- •Plasmapheresis or immunoadsorption within 30 days of screening
- •Participant had any current medical condition that, in the opinion of the Investigator, may have compromised their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results
研究组 & 干预措施
Cohort 1: ALXN1830
Participants received 5 doses of ALXN1830 10 mg/kg administered weekly.
干预措施: ALXN1830 (Drug)
Cohort 2: ALXN1830
Participants were to receive 3 doses of ALXN1830 30 mg/kg administered weekly (loading) followed by 5 doses of ALXN1830 10 mg/kg administered every other week or 10 weekly doses of ALXN1830 IV (maintenance).
干预措施: ALXN1830 (Drug)
结局指标
主要结局
Count Of Participants Reporting Treatment-emergent Adverse Events (TEAEs)
时间窗: Day 1 (after first dose) through Day 112
A TEAE was defined as any adverse event (AE) that starts on or after the first dose of study drug or occurs prior to the first dose and worsens in severity on or after the first dose of study drug, during the Treatment Period and Follow-up Period. A TEAE was considered "serious" (Grade 3) if, in the view of either the investigator or sponsor, it resulted in any of the following outcomes: death, life-threatening adverse drug event, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect, or an event that may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously listed outcomes. A summary of serious and all other non-serious adverse events, regardless of causality, is located in the Reported Adverse Events module.
次要结局
- Maximum Percent Reduction Of Mean Total Immunoglobulin G (IgG) Levels From Baseline(Baseline through Day 112)
- Maximum Percent Reduction In Mean Pemphigus Disease Area Index (PDAI) Total Activity Score From Baseline(Baseline through Day 112)
- Maximum Percent Reduction Of Mean Circulating Immune Complexes (CIC) Levels From Baseline(Baseline through Day 112)
- Maximum Percent Reduction Of Mean Anti-Desmoglein (Dsg) 1 And 3 Antibodies From Baseline(Baseline through Day 112)