A Phase III, Randomized, Double-Blind, Placebo-Controlled Study of QL0911 in Pediatric Patients With Primary Immune Thrombocytopenia.
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 入组人数
- 60
- 主要终点
- Percentage of Participants With a Durable Platelet Response
研究概览
简要总结
The purpose of this study is to evaluate efficacy and of safety QL0911 in the treatment of thrombocytopenia in pediatric patients with previously treated chronic 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
- 1 Year 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Age ≥ 1 years old and < 18 years old.
- •Diagnosed primary ITP for at least 12 months;
- •Had received at least one first-line ITP treatment with no response or recurrence after treatment;
- •Had a platelet count <30×10^9/L within 48 hours before the first dose;
- •Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2;
- •Patients of child-bearing potential must agree to use effective contraception during the study and for 3 months following the last dose of study treatment.
排除标准
- •Had a history of bone marrow stem cell abnormalities or myelodysplastic syndrome other than ITP-specific changes.
- •Underwent splenectomy within 12 weeks before the first dose;
- •Had received ITP treatments (including rescue treatment) within 2 weeks before the first dose;
- •Had received romiplostim (Nplate®) or eltrombopag (Revolade®), rhTPO or other agents that stimulate TPO receptors (also known as c-Mpl)within 4 weeks before the first dose;
- •Had received antibody-based therapies within 14 weeks before the first dose.
- •Patients with concurrent or past malignant disease.
- •Serum creatinine or total bilirubin >1.5*ULN) alanine transaminase (ALT) or aspartate transaminase (AST) >3* ULN.
- •Had received antibody-based therapies within 14 weeks before the first dose.
- •Had prothrombin time (PT) or prothrombin time-international normalized ratio (PT-INR) or activated partial thromboplastin time (APTT) exceeded 20% of the reference range of normal values.
研究组 & 干预措施
QL0911
Participants received once weekly subcutaneous QL0911 at a starting dose of 1 µg/kg; weekly dose increases continued in increments of 1 µg/kg/week to a maximum dose of 10 µg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L.
干预措施: QL0911 (Drug)
placebo
Participants received weekly subcutaneous placebo.
干预措施: Placebo (Drug)
结局指标
主要结局
Percentage of Participants With a Durable Platelet Response
时间窗: Week 18 to week 25
A participant with durable platelet response was defined as achieving at least 6 weekly platelet counts of ≥ 50 x 10\^9/L from week 18 to week 25. If a platelet count from a participant was not available (missing) in a certain week, that week was imputed as non-response for that participant. Platelet counts were not deemed as a positive response for 4 weeks after the administration of rescue medication.
次要结局
- Percentage of Participants With an Overall Platelet Response(24 weeks)
- The proportion of subjects with a weekly platelet count ≥ 30 × 10^9/Land at least twice the baseline platelet count without bleeding during the double-blind period of 24 weeks.(24 weeks)
- Number of Weeks With Platelet Response(24 weeks)
- Percentage of Participants Who Received Rescue Medication During the Treatment Period.(24 weeks)
- Number of Participants With Treatment-emergent Adverse Events (TEAEs)(Up to Week 38)
- Number of Participants With Antidrug Antibodies (ADAs) , Anti endogenous TPO antibody and Neutralizing Antibodies (Nab)；(Up to Week 38)