A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of the Oral Factor D (FD) Inhibitor ACH-0145228 in Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients as Monotherapy and with an Approved C5 Inhibitor as Background Therapy

Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc.0 个研究点目标入组 26 人开始时间: 2020年6月16日最近更新: 2020年10月13日

适应症

试验速览

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•Male or female greater than or equal to 18 years of age, female subjects of child-bearing potential
•For newly identified PNH patients must meet one of the following conditions:
•1. PNH patients not currently receiving an approved C5 inhibitor must have:
•- PNH Type III erythrocyte and/or granulocyte clone size =10% with adequate reticulocytosis (absolute reticulocyte count =100×10?/L).
•- Anemia (Hgb <10.5 g/dL).
•- LDH =1.5× upper limit of normal (ULN).
•- Platelet count =30,000/µL without the need for platelet transfusions.
•- Absolute neutrophil count (ANC) =750/ µL.
•2. PNH patients with a sub-optimal response to background therapy with an approved C5 inhibitor must have:
•- Stable background regimen of an approved C5 inhibitor (at least 24 weeks for eculizumab, or at least 2 doses for ravulizumab), without change in dose or interval for at least the past 8 weeks.
•- Anemia (Hgb <10 g/dL).
•- Adequate reticulocytosis (absolute reticulocyte count =100×109/L).
•Platelet count =30,000/µL without the need for platelet transfusions.
•- Absolute neutrophil count (ANC) =750/ µL.
•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 25
•F.1.3 Elderly (>=65 years) yes
•F.1.3.1 Number of subjects for this age range 1

•- Estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2 and/or are on dialysis.
•- History of a major organ transplant (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant ([HSCT] unless HSCT engraftment has failed).
•- History of dosing with an investigational agent other than danicopan within 30 days or 5 half-lives of the investigational agent prior to ACH 0145228 administration, whichever is greater.
•- New patients in the monotherapy group with a history of dosing with eculizumab at any dose or interval within the past 75 days before study medication administration or 300 days for ravulizumab.
•- Known or suspected complement deficiency.
•- Contraindication to one or more of the required vaccinations that may be used in the study.
•- History of seizure disorder unless seizure free without the use of antiepileptic medications for the past 5 years prior to screening.

发起方

Achillion Pharmaceuticals, Inc., a wholly owned subsidiary of Alexion Pharmaceuticals Inc.