A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)

Alexion Pharmaceuticals Incorporated0 个研究点目标入组 16 人开始时间: 2018年3月15日最近更新: 2019年12月10日

研究概览

暂无简介。

•1. Patients from birth up to < 18 years of age and weighing = 5 kg at the time of consent.
•2. Evidence of thrombotic microangiopathy (TMA), including low platelet count, hemolysis (breaking of red blood cells inside of blood vessels), and decreased kidney function.
•3. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and Haemophilus influenzae
•4. Female patients of childbearing potential must use highly effective contraception starting at screening and continuing until at least 8 months after the last dose of ALXN1210
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 16
•F.1.2 Adults (18-64 years) no
•F.1.2.1 Number of subjects for this age range
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•1. ADAMTS13 deficiency (Activity < 5%)
•2. Shiga toxin-related hemolytic uremic syndrome (STEC-HUS)
•3. Positive direct Coombs test
•4. Pregnancy or breastfeeding
•5. Identified drug exposure-related hemolytic uremic syndrome (HUS)
•6. Bone marrow transplant (BMT)/hematopoietic stem cell transplant (HSCT) within last 6 months prior to start of Screening
•7. HUS related to vitamin B12 deficiency
•8. Systemic sclerosis (scleroderma), systemic lupus erythematosus (SLE), or antiphospholipid antibody positivity or syndrome
•9. Chronic dialysis (defined as dialysis on a regular basis as renal replacement therapy for ESKD)

发起方

Alexion Pharmaceuticals Incorporated