NCT04687072已完成3 期
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and the Safety of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- argenx
- 入组人数
- 207
- 试验地点
- 200
- 主要终点
- Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24
研究概览
简要总结
This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.
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 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Placebo PH20 SC
Placebo Comparator
Patients receiving placebo PH20 SC treatment
干预措施: Placebo PH20 SC (Other)
Efgartigimod PH20 SC
Experimental
Patients receiving efgartigimod PH20 SC treatment
干预措施: Efgartigimod PH20 SC (Biological)
结局指标
主要结局
Percentage of Participants With Chronic Immune Thrombocytopenia (ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24
时间窗: Up to 6 weeks (between Weeks 19 and 24)
A participant was considered a responder for this endpoint (i.e., had a sustained platelet count response) if the participant had platelet counts of ≥50 × 10\^9/L for ≥4 of the 6 analysis visits between Weeks 19 and 24.
次要结局
- Extent of Disease Control Over the 24-Week Treatment Period in the Chronic ITP Population(Up to 24 weeks)
- Percentage of Participants in the Overall Population (Chronic and Persistent ITP) With a Sustained Platelet Count Response Between Weeks 19 and 24(Up to 6 weeks (between Weeks 19 and 24))
- Percentage of Participants in the Overall Population With Sustained Platelet Count Response Between Weeks 17 and 24(Up to 8 weeks (between Weeks 17 and 24))
- Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time During the 24-week Treatment Period(Up to 24 weeks)
- Extent of Disease Control Until Week 12 in the Overall Population(Up to 12 weeks)
- Percentage of Participants in the Overall Population Achieving Overall Platelet Count Response at Any Time Until Week 12(Up to 12 weeks)
- Mean Change From Baseline in Platelet Count at Each Visit in the Overall Population(Baseline and Weeks 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20, 21, 22, 23, 24, Safety and Efficacy Follow-up Visit 1 (SEFU1) (up to Week 29), and SEFU2 (up to Week 33))
- Time to Platelet Count Response in the Overall Population(Up to 24 weeks)
- Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population(Up to 24 weeks)
- Extent of Disease Control Over the 24-Week Treatment Period in the Overall Population for Participants With Baseline Platelet Count of <15×10^9/L(Up to 24 weeks)
- Number of the World Health Organization (WHO)-Classified Bleeding Events (Grade ≥1) in the Overall Population(Up to 24 weeks)
- Percentage of Participants With a Platelet Count International Working Group (IWG) Response(Up to 24 weeks)
- Rate of Receipt of Rescue Therapy (Rescue Per Participant Per Month) in the Overall Population(Up to 24 weeks)
- Percentage of Participants for Whom Dose and/or Frequency of Concurrent ITP Therapies Have Increased at Week 12 or Later in the Overall Population(Up to 13 weeks (between Weeks 12 and 24))
- Change From Baseline in Functional Assessment of Chronic Illness Therapy Fatigue Scale (FACIT-Fatigue) at Week 24 in the Overall Population(Baseline and Week 24)
- Change From Baseline in Functional Assessment of Cancer Therapy Questionnaire-Th6 (Fact-Th6) at Week 24 in the Overall Population(Baseline and Weeks 4, 8, 12, 16, 20, and 24)
- Change From Baseline in Short Form-36 (SF-36) at Week 24 in the Overall Population(Baseline and Week 24)
- Incidence and Prevalence of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population(Up to 35 weeks)
- Titers of Antibodies to Efgartigimod and/or rHuPH20 in the Overall Population(Weeks 3, 7, 11, 15, 19, 23, 24, SEFU1 (up to Week 29), and SEFU2 (up to Week 33))
- Incidence and Prevalence of Neutralizing Antibodies (NAb) to Efgartigimod and/or rHuPH20 in the Overall Population(Up to 35 weeks)
- Serum Efgartigimod Trough Concentration (Ctrough) in the Overall Population(Predose on Weeks 1, 2, 3, 17, 19, 21, 23, and 24)
- Percentage Change From Baseline in Total IgG in the Overall Population(Baseline and Weeks 1, 2, 3, 17, 19, 21, 23, and 24)
- Number of Participants With Antiplatelet Antibodies in the Overall Population(Weeks 7, 15, 23, and 24)
研究者
研究点 (200)
Loading locations...
相似试验
已完成
3 期
Study to Investigate CSL112 in Subjects With Acute Coronary SyndromeAcute Coronary SyndromeNCT03473223CSL Behring18,226
已完成
3 期
A Study to Evaluate The Efficacy, Safety and Immunogenicity of Inactivated SARS-CoV-2 Vaccines (Vero Cell) in Healthy Population Aged 18 Years Old and AboveCOVID-19NCT04510207China National Biotec Group Company Limited44,101
已完成
3 期
Evaluation of the Efficacy, Durability, Safety, and Tolerability of RP-G28 in Patients With Lactose IntoleranceLactose IntoleranceNCT03597516Ritter Pharmaceuticals, Inc.557
已完成
3 期
A Study to Evaluate the Efficacy and Safety of Ensifentrine for 24 Weeks in Patients With Moderate to Severe Chronic Obstructive Pulmonary DiseaseChronic Obstructive Pulmonary DiseaseNCT05743075Nuance Pharma (shanghai) Co., Ltd526
已完成
3 期
A Phase Ⅲ Study to Evaluate Tildrakizumab in the Treatment of Chinese Subjects With Moderate to Severe Plaquetype PsoriasisPlaque PsoriasisNCT05108766Shenzhen Kangzhe Pharmaceutical Co., Ltd.220