CTIS2024-511535-97-00进行中（未招募）1 期
An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral Etavopivat, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease - 4202-HEM-301
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 420
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 0 至 65+（—）
- 性别
- All
入选标准
- •1) Provision of consent, 2) Patient has a confirmed diagnosis of sickle cell disease, 3) 2-15 episodes of documented vaso-occlusive crises in the past 12 months, 4) Hemoglobin = 5.5 and = 10.5 g/dL (= 55 and = 105 g/L) during screening, 5) Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment, 6) Female patients of childbearing potential must use acceptable methods of contraception; male patients are willing to use acceptable methods of contraception, 7)Patients on crizanlizumab or L-glutamine oral powder (Endari®) treatment at the time of consent may be eligible if they: • Have been on a stable dose for = 12 months at the time of consent (i.e., no changes to the dose except for changes to weight or for safety reasons) • Have been = 80% compliant with the planned regimen during the 12 months prior to the time of consent • Meet the VOC eligibility requirement in Inclusion Criterion 4.
排除标准
- •Medical Conditions 1) More than 15 vaso-occlusive crises within the past 12 months prior to screening, Prior/Concomitant Therapy 1) Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion), 2) Receiving or use of concomitant medications that are strong inducers of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study, 3) Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study, 4) Use of an experimental selectin antagonist (e.g., monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study, 5) Uso de eritropoyetina u otro tratamiento con factor de crecimiento hematopoyético dentro de los 28 días posteriores al inicio del tratamiento del estudio o la necesidad anticipada de dichos agentes durante el estudio., 6) Receipt of prior cellular-based therapy (e.g., hematopoietic cell transplant, gene modification therapy), 2) Female who is breast feeding or pregnant, 3) Hepatic dysfunction characterized by: - Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN) OR - Direct bilirubin > 3.0 × ULN, 4) Known HIV positive, 5) Active hepatitis B or hepatitis C infection, 6) Severe renal dysfunction or on chronic dialysis, 7) History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to the following: - Unstable angina pectoris or myocardial infarction or elective coronary intervention - Congestive heart failure requiring hospitalization - Uncontrolled clinically significant arrhythmias - Symptomatic pulmonary hypertension, 8) History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage, 9) History of deep venous thrombosis requiring systemic anticoagulation therapy for = 6 weeks, occurring within 6 months prior to Day 1 of study treatment. Note: patients on = 6 months of chronic or prophylactic anti-coagulation therapy are allowed on study.
研究者
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