NCT03764618已完成3 期
A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 90
- 试验地点
- 102
- 主要终点
- Durable Hemoglobin Response
研究概览
简要总结
The primary objective of this study is to assess the efficacy of fostamatinib in subjects with warm antibody autoimmune hemolytic anemia (wAIHA).
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 100 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Subject must have a diagnosis of primary or secondary warm Antibody Autoimmune Hemolytic Anemia (wAIHA) as documented by a positive direct antiglobulin test (DAT) specific for anti-IgG or anti-IgA.
- •Have failed or not tolerated at least one prior wAIHA treatment regimen, including steroids, rituximab, azathioprine, cyclophosphamide, cyclosporine, MMF, danazol, vincristine, ESA or splenectomy (folate, iron or other supplements do not fulfill this criterion).
- •Have haptoglobin <LLN or total bilirubin >ULN or lactate dehydrogenase (LDH) >ULN.
- •At screening, subject's hemoglobin level must be ≤9 g/dL OR if hemoglobin value >9 g/dL and <10 g/dL, subject must be on an allowed wAIHA treatment AND the subject must have documented symptoms related to anemia (e.g., weakness, dizziness, fatigue, shortness of breath, chest pain).
- •Karnofsky performance status (KPS) ≥
- •Subject's concurrent treatment for wAIHA may consist of no more than two of any of the following agents: azathioprine, steroids, ESAs, mycophenolate mofetil, dapsone or danazol at a stable dose
排除标准
- •Subject with other types of Antibody Autoimmune Hemolytic Anemia (AIHA) (e.g., cold antibody AIHA, cold agglutinin syndrome, mixed type AIHA, or paroxysmal cold hemoglobinuria).
- •Subject has AIHA secondary to autoimmune disease, including systemic lupus erythematosus (SLE), or lymphoid malignancy if the underlying disease is not stable or is not well-controlled on current therapy, per investigator medical judgement.
- •Subject has uncontrolled or poorly controlled hypertension, defined as systolic blood pressure ≥135 mmHg or diastolic blood pressure ≥85 mmHg, whether or not the subject is receiving anti-hypertensive treatment.
- •Subject has one or more of the following laboratory abnormalities at screening: neutrophil count of <1,000/μL or platelet count of <30,000/μL, unless due to Evans syndrome; transaminase levels (i.e., alanine aminotransferase [ALT] or aspartate aminotransferase [AST]) >1.5 x ULN.
- •Has documented active hepatitis B or hepatitis C infection or HIV infection.
研究组 & 干预措施
Fostamatinib
Experimental
Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to fostamatinib 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator.
干预措施: Fostamatinib disodium (Drug)
Placebo
Placebo Comparator
Initial dose is 100 mg by mouth (PO) twice a day (bid). At week 4 dose will be increased to placebo 150 mg PO bid if subjects have adequately tolerated the study drug in the opinion of the Investigator.
干预措施: Placebo (Drug)
结局指标
主要结局
Durable Hemoglobin Response
时间窗: 24 Weeks
Proportion of subjects achieving a hemoglobin level ≥ 10 g/dL with an increase from Baseline in hemoglobin level of ≥ 2 g/dL on 3 consecutive available visits during the 24-week treatment period.
次要结局
- A Hemoglobin Response by Week 24(24 weeks)
- Change From Baseline in Hemoglobin Level of 2 g/dL or Greater(24 weeks)
- Change in Hemoglobin From Baseline to End of Treatment(24 weeks)
- Use of Rescue Antibody Autoimmune Hemolytic Anemia (AIHA) Regimens Use After Week 4(24 weeks)
- Change in Functional Assessment of Chronic Illness Therapy - Fatigue Scale (FACIT-F)(24 weeks)
研究者
研究点 (102)
Loading locations...
相似试验
已完成
3 期
A Efficacy and Safety Study of Fostamatinib in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)Immune Thrombocytopenic PurpuraNCT02076412Rigel Pharmaceuticals74
已完成
3 期
A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)Immune Thrombocytopenic PurpuraNCT02076399Rigel Pharmaceuticals76
已完成
2 期
Safety and Efficacy Study of Fostamatinib to Treat Immunoglobin A (IgA) NephropathyIGA NephropathyNCT02112838Rigel Pharmaceuticals76
已完成
2 期
A Safety and Efficacy Study of R935788 in the Treatment of Warm Antibody Autoimmune Hemolytic Anemia (AIHA)Warm Antibody Autoimmune Hemolytic AnemiaNCT02612558Rigel Pharmaceuticals26
已完成
3 期
Evaluation of Effectiveness of Two Dosing Regimens of Fostamatinib Compared to Placebo in Patients With Rheumatoid Arthritis (RA) Who Are Taking Methotrexate But Not Responding.Rheumatoid ArthritisNCT01197521AstraZeneca923