NCT04039477撤回2 期
A Phase 2 Randomized, Dose-blind, Multicenter Study to Evaluate the Safety and Efficacy of KZR-616 in the Treatment of Patients With Autoimmune Hemolytic Anemia (AIHA) and Immune Thrombocytopenia (ITP)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 试验地点
- 2
- 主要终点
- Mean change from Baseline to Week 13 in hematologic parameters of interest in evaluable patients (Hgb for AIHA; Platelets [PLT] for ITP)
研究概览
简要总结
This is a Phase 2 randomized, dose-blind, multicenter study designed to evaluate the safety, tolerability, efficacy, Pharmacokinetics (PK), and Pharmacodynamics (PD) of treatment with KZR-616 in patients with active Autoimmune Hemolytic Anemia or Immune Thrombocytopenia.
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
- •Adult patients must be at least 18 years of age at the time of signing informed consent at Screening
- •Body Mass Index (BMI) equal to or greater than 18 kg/m2
- •Have a documented diagnosis of primary or secondary AIHA, ITP, or primary Evans syndrome
- •AIHA or ITP disease activity as follows::
- •ITP: Per central or local laboratory assessments on 2 separate occasions ≥7 days apart during Screening, a mean Platelet (PLT) ≤30×109/L with no individual PLT >35×109/L; or for those patients receiving a constant dose of permitted treatments for ITP: a mean PLT <50×109/L, with no count >55×109/L
- •AIHA: Hgb ≤10 g/dL and presence of any 2 of the following:
- •i. Haptoglobin <lower limit of normal (LLN) ii. Corrected reticulocyte count >upper limit of normal (ULN) iii. LDH >ULN iv. Indirect bilirubin >ULN.
- •Documented inadequate response on intolerance to ≥1 standard treatment approach for AIHA or ≥2 standard treatment approaches for ITP
排除标准
- •Systemic Lupus Erythematosus (SLE) with confirmed anti-phospholipid antibody syndrome, the presence of positive lupus anti-coagulant test, moderate-high titer anti-cardiolipin IgG or IgM or moderate-high titer anti-beta2-globuilin IgG or IgM or severe central nervous system involvement
- •History of clinically significant coagulopathy, hereditary thrombocytopenia, anemia, or family history of thrombocytopenia
- •History of primary immunodeficiency
- •Use of nonpermitted medications within the specified washout periods prior to screening
- •Recent serious or ongoing infection, or risk for serious infection
- •Any of the following laboratory values at Screening:
- •Estimated glomerular filtration rate (eGFR) <45 ml/min
- •Absolute neutrophil count (ANC) <1.5×109/L (1500/mm3)
- •Serum aspartate transaminase (AST), serum alanine transaminase (ALT) or serum alkaline phosphatase >2.5×ULN
- •Thyroid stimulating hormone if outside of the central laboratory normal range and considered clinically significant
- •International normalized ratio (INR) or activated partial thromboplastin time (aPTT) >1.5×ULN
- •Immunoglobulin G (IgG) <500 mg/dL
- •For ITP patients only: total bilirubin >1.5×ULN (3×ULN for patients with documented Gilbert's syndrome).
- •Presence of New York Heart Association Class III or IV heart failure, or uncontrolled blood pressure, or prolonged QT interval
- •Major surgery within 12 weeks before Screening or planned during the study period
- •History of any thrombotic or embolic event within 12 months prior to Screening
- •Clinical evidence of significant unstable or uncontrolled diseases
- •Any active or suspected malignancy or history of documented malignancy within the last 5 years before Screening, except appropriately excised and cured cervical carcinoma in situ or basal or squamous cell carcinoma of the skin, or non-muscle invasive bladder cancer
研究组 & 干预措施
Arm A - KZR-616 30mg
Experimental
KZR-616 30mg Subcutaneous (SC) injection weekly for 13 weeks
干预措施: KZR-616 (Drug)
Arm B - KZR-616 45mg
Experimental
KZR-616 30 mg SC injection weekly for 1 dose then 45mg weekly for 12 weeks.
干预措施: KZR-616 (Drug)
结局指标
主要结局
Mean change from Baseline to Week 13 in hematologic parameters of interest in evaluable patients (Hgb for AIHA; Platelets [PLT] for ITP)
时间窗: 13 weeks
次要结局
- Mean change from Baseline to Week 13 in hematologic parameters of interest (Hgb for AIHA; PLT for ITP) in the intent to treat (ITT) population(13 weeks)
- Proportion of patients over time with a response(Through study completion, up to 25 weeks)
- Mean change from Baseline over time in hematologic parameters of interest (Hgb for AIHA; PLT for ITP)(Through study completion, up to 25 weeks)
- Proportion of patients over time with loss of response(Through study completion, up to 25 weeks)
- Mean change from Baseline over time in Hct(Through study completion, up to 25 weeks)
- For AIHA: Proportion of patients with an Hgb >12 g/dL or 2 g/dL higher than Baseline at W13(13 weeks)
- For AIHA: Number of blood transfusions and units of blood administered over time(Through study completion, up to 25 weeks)
- Safety and tolerability of KZR-616 in patients with AIHA or ITP as assessed by monitoring incidence and severity of adverse events (AEs)(Through study completion, up to 25 weeks)
- Time to peak plasma concentration (Tmax) following KZR-616 injection(Day 29)
- Proportion of patients with a response at Week 13(13 weeks)
- Time to response(Through study completion, up to 25 weeks)
- Proportion of patients over time with sustained response(Through study completion, up to 25 weeks)
- Mean change from Baseline over time in Lactate Dehydrogenase (LDH)(Through study completion, up to 25 weeks)
- Change from Baseline over time in Patient Global Assessment scores(Baseline and every 4 weeks for 25 weeks)
- For ITP: Number of platelet transfusions and units of platelets administered over time(Through study completion, up to 25 weeks)
- Peak Plasma Concentration (Cmax) following KZR-616 injection(Day 29)
- Area under the plasma concentration versus time curve (AUC) following KZR-616 injection(Day 29)
- Half-life (T1/2) following KZR-616 injection(Day 29)
研究者
研究点 (2)
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