A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 38
- 试验地点
- 30
- 主要终点
- Incidence and severity of serious adverse events (SAEs).
研究概览
简要总结
The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia.
详细描述
This is a randomized, double-blind, placebo-controlled Phase II study in which approximately 36 patients will be randomized in a 1:1:1 ratio to receive either ARGX-113 Dose A, or ARGX-113 Dose B body weight or placebo in 4 infusions administered 1-week apart in addition to Standard-of-Care (SoC) treatment. Patients aged 18 to 85 years (inclusive) with confirmed primary immune thrombocytopenia (ITP) who have a platelet count ˂ 30 × 109/L and who are receiving oral corticosteroids and/or permitted oral immunosuppressants and/or Thrombopoietin receptor (TPO-R) agonist as SoC which must be maintained on a stable dose and frequency for at least 4 weeks prior to Screening.
The study will include a 2-week Screening, a 3-week Treatment period, and an 21-week follow-up (FU) period. The study is followed by an open label period where patients will be given the option to be treated with ARGX-113 Dose A in cycles of 4 weekly infusions with a minimum of 4 weeks apart. Patients may receive rescue therapy during the study at the discretion the investigator when deemed medically necessary.
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 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients aged ≥ 18 to ≤ 85 years.
- •Must receive SoC treatment for ITP that has been stable in dose and frequency for at least 4 weeks prior to Screening. SoC may include oral corticosteroids and/or permitted oral immunosuppressants and/or TPO-R agonist.
- •Confirmed diagnosis of ITP with blood platelet counts < 30 × 109/L and who have not experienced major bleeding in the last 4 weeks prior to Screening.
排除标准
- •Use of anticoagulants, or any drug with antiplatelet effect within 3 weeks prior to Screening.
- •Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
- •Use of Intravenous immunoglobulin G (IVIg) or anti-D immunoglobulin treatment within 4 weeks prior to screening.
- •Use of recombinant thrombopoietin at any time.
- •Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
- •Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine, danazol, mycophenolate mofetil, mycophenolate sodium which must have been stable for at least 4 weeks prior to Screening.
- •Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
- •Received vaccinations within 4 weeks prior to Screening or planned during the study.
- •At Screening, have clinically significant laboratory abnormalities
- •History of any thrombotic or embolic event within 12 months prior to Screening.
- •Known auto-immune disease other than ITP.
研究组 & 干预措施
ARGX-113 Dose A + SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
干预措施: ARGX-113 (Drug)
ARGX-113 Dose B +SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
干预措施: ARGX-113 (Drug)
Placebo + SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
干预措施: Placebo (Other)
结局指标
主要结局
Incidence and severity of serious adverse events (SAEs).
时间窗: After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
次要结局
- Frequency and proportion of patients with initial response(Over the study period (up to 13 weeks).)