EUCTR2020-003884-25-DE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
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 450
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 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
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 40
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 408
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •Medical Conditions
- •1) More than 15 vaso-occlusive crises within the past 12 months prior to screening
- •2) Female who is breast feeding or pregnant
- •3) Hepatic dysfunction characterized by:
- •- Alanine aminotransferase (ALT) > 4.0 × upper limit of normal (ULN)
- •- 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 anti-coagulation 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.
- •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) Use of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study
- •6) Receipt of prior cellular-based therapy (e.g., hematopoietic cell transplant, gene modification therapy)
研究者
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