NCT04224688终止3 期
A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 30
- 试验地点
- 29
- 主要终点
- Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks
研究概览
简要总结
The purpose of this study is to demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment and assess safety and tolerability of rozanolixizumab in adult study participants with primary immune thrombocytopenia (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)
盲法说明
Investigators are blinded to the treatment code, they will see the platelet values.
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
- •Study participant must be ≥18 years of age at the time of the Screening Visit
- •Study participant has a diagnosis of persistent (>3 months duration) or chronic (>12 months duration) primary immune thrombocytopenia (ITP) at the Screening Visit
- •Study participant has a documented intolerance or insufficient response to two or more appropriate standard of care ITP treatments prior to Screening
- •Study participants must have prior history of a response to a previous ITP therapy.
- •If taking allowed drugs, study participant must be on stable doses during defined time periods prior to Baseline (Day 1)
- •Study participant has a documented history of low platelet count (<30×10^9/L) prior to Screening
- •Study participant has a platelet count measurement at Screening and at Baseline (Day 1) with an average of the two <30×10^9/L and no single count may be >35×10^9/L (using local laboratories)
- •Study participant has a current or history of a peripheral blood smear consistent with ITP
- •Study participants may be male or female:
- •A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study treatment and refrain from donating sperm during this period
- •A female participant is eligible to participate if she is not pregnant as confirmed by a negative serum pregnancy test and not planning to get pregnant during the participation in the study, not breastfeeding, and at least one of the following conditions applies:
- •Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the dose of study treatment
排除标准
- •Participant has a history of arterial or venous thromboembolism (eg, stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires current anticoagulant treatment
- •Study participant has clinically significant bleeding that warrants immediate platelet adjustment (eg, menorrhagia with significant drop in hemoglobin)
- •Study participant has a known hypersensitivity to any components of the study medication or any other anti-neonatal Fc receptor (FcRn) medications
- •Study participant has evidence of a secondary cause of immune thrombocytopenia (clear association with other medical conditions eg, of untreated H. pylori infection, leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease or is drug induced), participant has a multiple immune cytopenia (eg, Evan's syndrome) etc.
- •Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
- •Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
- •Study participant has a history of a major organ transplant or hematopoietic stem cell/marrow transplant
- •Study participant has experienced intracranial bleed in the last 6 months prior to the Screening Visit
- •Study participant has a history of coagulopathy disorders other than ITP
- •Study participant with current or medical history of immunoglobulin A (IgA) deficiency, or a measurement of IgA <50 mg/dL at the Screening Visit
- •Study participant has undergone a splenectomy in the 2 years prior to the Baseline Visit
研究组 & 干预措施
Rozanolixizumab
Experimental
Study participants randomized to this arm will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs.
干预措施: Rozanolixizumab (Drug)
Placebo
Placebo Comparator
Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.
干预措施: Placebo (Other)
结局指标
主要结局
Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks
时间窗: During the last 12 weeks (Week 13 to Week 25)
Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10\^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported.
次要结局
- Time to First Rescue Therapy(From Baseline to first rescue therapy (up to Week 25))
- Time to First Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/L: Time From Starting Treatment to Achievement of First Response of ≥50×10^9/L(Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25))
- Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period(Week 1 up to Week 25)
- Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8(Baseline to Day 8)
- Percentage of Participants With Response Defined as Platelet Count ≥30*10^9/L and at Least Doubling of Baseline, at Least 2 Separate Occasions at Two Adjacent Nominal Visits at Least 7 Days Apart, and Absence of Bleeding(From Baseline during Treatment Period (up to Week 25))
- Change From Baseline to Week 25 in Primary Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score(From Baseline during Treatment Period (up to Week 25))
- Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)(From Baseline to end of Safety Follow-Up Period (up to Week 31))
- Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (ie, Study Discontinuation)(From Baseline to end of Safety Follow-Up Period (up to Week 31))
研究者
研究点 (29)
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