A Randomized, Double-blind, Placebo Controlled, Parallel Group Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of QGE031 in the Treatment of Patients With Bullous Pemphigoid With Disease Refractory to Oral Steroid Treatment
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 20
- 试验地点
- 1
- 主要终点
- Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12
研究概览
简要总结
To evaluate the safety and efficacy of QGE031 versus placebo in patients with bullous pemphigoid. Efficacy will be assessed as a reduction of disease activity. How QGE031 is broken down by the body and the impact it has on different blood and tissue markers will also be explored.
详细描述
This study was planned to be divided into 2 distinct parts. Part 1 was a multicenter, randomized, placebo-controlled study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment. Patients were treated with QGE031 or placebo in a 2:1 ratio.
Part 2 of this study was planned to be a multi-center, open label, dose range finding study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment.
This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( >50% better then placebo)
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 20 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients diagnosed with bullous pemphigoid
- •Stable dose of prednisone at or above 10mg per day but no greater than 1 mg/kg/day
- •Weigh between 40-120kg
- •total IgE level up to 5000 IU/mL
排除标准
- •Use of rituximab within 1 year
- •Other protocol-defined inclusion/exclusion criteria may apply
研究组 & 干预措施
QGE031
QGE031 240 mg Q2W s.c.
干预措施: QGE031 (Drug)
Placebo
Placebo to Match Q2W s.c.
干预措施: Placebo (Drug)
Open Label QGE031
Open Label QGE031 Q2W s.c.
干预措施: QGE031 (Drug)
结局指标
主要结局
Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12
时间窗: 12 weeks
Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 12 weeks based on the CGA-C in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating 'at least marked improvement from baseline' at 12 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement)
次要结局
- Number of Patients Investigator Global Assessment Score Over 12 Weeks(Baseline (week 0), week 6 and week 12)
- Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks(6 weeks)