EUCTR2019-003829-18-GR进行中（未招募）1 期
A Phase 3 Study of Danicopan (ALXN2040) as Add-on Therapy to a C5 Inhibitor (Eculizumab or Ravulizumab) in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Clinically Evident Extravascular Hemolysis (EVH)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 84
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
- •Diagnosis of PNH
- •Clinically evident extravascular hemolysis (EVH) defined by:
- •- Anemia (Hgb =9.5 gram/deciliter) with absolute reticulocyte count =120 x 10^9/liter.
- •- At least 1 packed red blood cell or whole blood transfusion within 6 months prior to the start of the study.
- •Receiving a C5 inhibitor for at least 6 months prior to Day.
- •Platelet count =30,000/microliters (µL)
- •Absolute neutrophil counts =750/µL.
- •Documentation of/or willingness to receive vaccinations and prophylactic antibiotics as required.
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 82
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 2
排除标准
- •History of a major organ transplant or hematopoietic stem cell transplantation (HSCT).
- •Participants with known aplastic anemia or other bone marrow failure that requires HSCT or other therapies including anti-thymocyte globulin and/or immunosuppressants, unless the dosage regimen of immunosuppressant has been stable for at least 12 weeks before Day 1 and patient is expected to remain on stable doses through Week 24.
- •Known or suspected complement deficiency.
- •Laboratory abnormalities at screening, including:
- •- Alanine aminotransferase >2 x ULN (> 3 x ULN in the case of patients with documented liver iron overload defined by serum ferritin values = 500 ng/mL). The inclusion of patients with documented iron overload and ALT>2 X ULN will be done in a case by case basis, with prior discussion with the Medical Monitor.
- •- Direct bilirubin >2 x ULN - patients who, in the opinion of investigator, have direct bilirubin > 2 x ULN due to EVH and/or patients with documented Gilbert’s Syndrome. If Gilbert's syndrome is
- •suspected, the patient will be tested for this condition at screening.
- •Estimated glomerular filtration rate <30 milliliters/minute/1.73 meter squared and/or are on dialysis.
- •Evidence of hepatitis B (positive hepatitis surface antigen [HBsAg] or positive core antibody [anti-HBc] with negative surface antibody [anti-HBs]) or hepatitis C viral infection (HCV antibody positive), except for patients with documented successful treatment and documented sustained virologic response (SVR) at Screening
- •Evidence of human immunodeficiency virus (HIV antibody positive) infection at Screening
研究者
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