A Randomized, Double-Blind, Placebo-Controlled Phase II/III Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of HMPL-523 in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 发起方
- Hutchmed
- 入组人数
- 111
- 试验地点
- 39
- 主要终点
- Phase II: overall Hb response rate
研究概览
简要总结
Phase II Study: To evaluate the safety and preliminary efficacy of HMPL-523 in adult patients with wAIHA.
Phase III Study(Part A): Confirmation of Efficacy safety and of HMPL-523 in adult patients with wAIHA.
Phase III Study (Part B): To further evaluate the long-term safety and tolerability of HMPL-523 in adult patients with wAIHA.
详细描述
Phase II Study: An eight-week randomized, placebo-controlled, double-blind phase followed by at least a 16-week open-label HMPL-523 treatment to evaluate the safety and preliminary efficacy of HMPL-523. The primary endpoint was the proportion of patients with overall Hb response by week 24.
Phase III study (Part A):A 24-week randomized, placebo-controlled double-blind phase to evaluate efficacy and safety of HMPL-523. The primary endpoint was the proportion of patients who achieve a durable response during weeks 5 to 24.
Phase III Study (Part B): An open-label Phase evaluating long-term safety and efficacy of HMPL-523 treatment. Eligible patients include those with lack of efficacy during the 20-week Phase III Part A treatment, completion of the Phase II study, or completion of 24-week Phase III Part A treatment with investigators assessment of potential benefit from open-label treatment with HMPL-523.
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
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Voluntarily signed the informed consent form (ICF);
- •Males or females aged 18 to 75 years;
- •Patients diagnosed with primary wAIHA or secondary wAIHA whose underlying diseases are stable;
- •Organs in good function.
排除标准
- •Patients with other types of AIHA other than wAIHA;
- •Patients with secondary wAIHA with unstable underlying disease;
- •Patients with drug-induced secondary wAIHA;
- •Patients with infections requiring systemic treatment;
- •Patients previously treated with Syk inhibitors (e.g., fostamatinib);
- •Patients with known allergy to the active ingredients or excipients of the study drug;
- •Patients with serious psychological or mental disorder;
- •Alcoholic or drug abuser;
- •Female patients who are pregnant and lactating.
研究组 & 干预措施
HMPL-523
Phase II: Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 8 weeks and at least 16 weeks open-label treatment.
Phase III:
Part A (Randomized, Double-Blind Phase): Eligible subjects will receive 300 mg HMPL-523 treatment once daily for 24 weeks.
Part B (Open-label Phase): Eligible subjects will roll-over into the open-label phase and receive treatment with HMPL-523 at the same dose administered after 24-week randomized controlled trial phase (Part A) or in Phase II. Treatment will continue until 24 weeks after the last subject is enrolled in Part B.
干预措施: HMPL-523(300mg PO QD) (Drug)
Placebo
Phase II: Eligible subjects will receive 300 mg HMPL-523 matched placebo treatment once daily for 8 weeks.
Phase III:
Part A (Randomized, Double-Blind Phase): Eligible subjects will receive 300 mg HMPL-523 matched placebo treatment once daily for 24 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Phase II: overall Hb response rate
时间窗: 24Weeks
Phase II: Overall Hb response rate: the proportion of patients with overall Hb response by Week 24
次要结局
- Phase III: Durable Hb response rate(24Weeks)