An Exploratory Clinical Study of Golidocitinib in Adult Patients With Primary Immune Thrombocytopenia (ITP)
试验速览
- 阶段
- 1 期
- 状态
- 尚未招募
- 入组人数
- 138
- 试验地点
- 1
- 主要终点
- Part A: Incidence and severity of Adverse Events (AEs)
研究概览
简要总结
This is a multicenter clinical study to evaluate the safety and efficacy of golidocitinib in patients with primary immune thrombocytopenia (ITP). The study consists of two parts: Part A dose escalation and Part B dose expansion. Part A is designed to obtain the safety profile of golidocitinib in patients with ITP and the recommended dose for the randomized cohort in Part B. Part B is a randomized, double-blind, placebo-controlled study, and the primary objective of this part is to evaluate the preliminary efficacy of golidocitinib in patients with ITP.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
盲法说明
Part A-Open-label, Part B-Double-blind
入排标准
- 年龄范围
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male and female participants between 18 and 80 years old.
- •Primary ITP for >3 months.
- •An average of two platelet counts (⩾ 7 days apart) of < 30 × 10⁹/L.
- •Have relapsed or treatment-resistant to previous ITP therapies, including corticosteroids and at least one other ITP therapy.
- •Have history of response to previous treatments.
- •Adequate hematologic, hepatic, and renal fuction.
- •Participants willing to comply with contraceptive restrictions.
排除标准
- •Diagnosed with secondary immune thrombocytopenia, or there is evidence that the patient has a secondary cause of immune thrombocytopenia.
- •Previous or current therapy and comedications meet exclusion criteria.
- •Patients with major caridiovascular disease, active infetion, maligancy or uncontrolled systemic disease.
- •Women who are breast feeding.
- •History of hypersensitivity to sudy drug with a similar chemical structure or class.
- •Previously received JAK inhibitors.
研究组 & 干预措施
Golidocitinib Part B
Golidocitinib at recommended phase 2 doses, administered orally, once daily.
干预措施: Golidocitinib (Drug)
Placebo Part B
Placebo at recommended phase 2 doses, administered orally, once daily.
干预措施: Placebo (Drug)
Golidocitinib Part A
Golidocitinib dose escalation, administered orally, once daily.
干预措施: Golidocitinib (Drug)
结局指标
主要结局
Part A: Incidence and severity of Adverse Events (AEs)
时间窗: 1 year
The severity was graded based on the National Cancer Institute's Common Terminology Criteria for Adverse Events.
Part B: Durable response rate
时间窗: up to 24 weeks
The proportion of patients with a platelet count ≥ 50 × 10⁹/L on at least four of six scheduled visits between weeks 14 and 24
次要结局
- Part A: Durable response rate(up to 24 weeks)
- Part B: Incidence and severity of Adverse Events (AEs)(1 year)
- Part A and Part B: Overall response rate(up to 24 weeks)
- Part A and Part B: Complete response rate(up to 24 weeks)
- Part A and Part B: Early response rate(up to 1 week)
- Part A and Part B: Initial response rate(up to 4 weeks)
- Part A and Part B: Sustained response rate(up to 24 weeks)
- Part A and Part B: Time to response(up to 24 weeks)
- Part A and Part B: Cumulative response time(up to 24 weeks)
- Part A and Part B: The proportion of patients requiring rescue therapy(up to 24 weeks)
- Part A and Part B: The proportion of patients experencing bleeding (≥ Grade 2)(up to 24 weeks)
- Part A and Part B: Changes of ITP-BAT bleeding score(up to 24 weeks)
研究者
Xiao Hui Zhang
Associate Director
Peking University People's Hospital