A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ALXN2050 (ACH-0145228) in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy - Study of the Oral Factor D (FD) Inhibitor ALXN2050 in PNH Patients as Monotherapy

Alexion Pharmaceuticals Inc.0 个研究点目标入组 26 人开始时间: 2021年7月14日最近更新: 2021年11月8日

适应症

试验速览

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•All patients must meet all of the following conditions:
•1. Diagnosis of PNH.
•2. Male or female, = 18 years of age (or minimum adult age in accordance with local legal requirements).
•Eligibility Criteria Specific for Group 1:
•1. PNH Patients who have no history of treatment with any complement inhibitor at any dose.
•2. PNH Type III erythrocyte or granulocyte clone size =10%
•3. Absolute reticulocyte count =100×10^9/liter [L].
•4. Anemia (Hgb <10.5 grams/deciliter [g/dL]).
•5. LDH =1.5× upper limit of normal.
•6. Platelet count =30,000/microliter (µL) without the need for platelet transfusions.
•7. Absolute neutrophil count (ANC) =750/ µL.
•Eligibility Criteria Specific for Group 2:
•Patients on stable eculizumab switching to ALXN2050 (Group 2) must meet the following criteria:
•1. Stable background regimen of at least 24 weeks for eculizumab without change in dose or interval for at least the past 8 weeks
•2. Anemia (Hgb <10 g/dL)
•3. Absolute reticulocyte count =100×10^9/L
•4. Platelet count =30,000/µL without the need for platelet transfusions
•5. Absolute neurophil count (ANC) =750/ µL
•Eligibility Criteria Specific for Group 3:
•1. Patient received danicopan during Study ACH-471-103.
•Are the trial subjects under 18? no
•Number of subjects for this age range: 0
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 25
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

•1. History of a major organ transplant or hematopoietic stem cell/marrow transplant .
•2. Known aplastic anemia or other bone marrow failure that requires HSCT, or if these patients are on immunosuppressive agents such as (but not limited to) cyclosporine, tacrolimus, mycophenolate, or others for less than 24 weeks prior to enrollment.
•3. Known underlying bleeding disorders (eg, coagulation factor deficiencies, idiopathic thrombocytopenic purpura, Von Willebrand disease) or any other conditions leading to anemia not primarily associated with PNH.
•4. Estimated glomerular filtration rate <30 milliliters/minute/1.73 meters squared and/or are on dialysis.

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