A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- argenx
- 入组人数
- 24
- 试验地点
- 23
- 主要终点
- Efgartigimod serum concentrations in the DBTP
研究概览
简要总结
The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP).
The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (OLTP2). After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks.
More information can be found here: https://clinicaltrials.argenx.com/advancejunior
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
- 12 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Is aged 12 to less than 18 years when completing the informed consent process
- •Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete
- •Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab.
- •Has documented prior response, defined as 1 platelet count of ≥50 × 10^9/L to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin
- •Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy
- •Has documented mean platelet count of less than 30 x10^9/L
排除标准
- •Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP
- •Nonimmune thrombocytopenia
- •ITP-associated critical or severe bleeding
- •History of hereditary thrombocytopenia
研究组 & 干预措施
Placebo IV
Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)
干预措施: Efgartigimod IV (Biological)
Placebo IV
Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)
干预措施: Placebo IV (Other)
Efgartigimod IV
Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)
干预措施: Efgartigimod IV (Biological)
结局指标
主要结局
Efgartigimod serum concentrations in the DBTP
时间窗: Up to 24 weeks
Total IgG levels in the DBTP
时间窗: Up to 24 weeks
次要结局
- Percent change from baseline in total IgG levels in serum over time during the DBTP(Up to 24 weeks)
- Efgartigimod serum concentrations over time during the DBTP(Up to 24 weeks)
- Incidence of AEs, SAEs and AEs leading to IMP discontinuation(Up to 136 weeks)
- Sustained platelet count response between study weeks 19 and 24 of the DBTP and in OLTP1 for participants receiving placebo in the DBTP(Up to 48 weeks)
- Extent of disease control during the DBTP and during the first 24 weeks of OLTP1 for those participants receiving placebo in the DBTP(Up to 48 weeks)
- Changes from baseline for platelet counts over time(Up to 76 weeks)
- Incidence of bleeding, assessed by the Modified Buchanan and Adix Bleeding Score for pediatric ITP(Up to 76 weeks)
- Incidence of ADA and Nab against efgartigimod in serum(Up to 76 weeks)
- Change from baseline in EQ-5D-5L(Up to 76 weeks)
- Change from baseline in KIT Child Self-Report and KIT Parent Impact Report(Up to 76 weeks)
- Change from baseline in peds FACIT-F(Up to 76 weeks)