EUCTR2016-002499-29-DE进行中（未招募）1 期
A Phase 3, Open-Label, Multicenter Study of ALXN1210 in Children and Adolescents with Atypical Hemolytic Uremic Syndrome (aHUS)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 31
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
- •Cohort 1 inclusion criteria:
- •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
- •Cohort 2 inclusion criteria:
- •1. Patients between 12 and <18 years of age who have been treated with eculizumab for aHUS for at least 90 days prior to Screening
- •2. Patients with a documented diagnosis of aHUS
- •3. Patients with clinical evidence of response to eculizumab indicated by stable TMA parameters at Screening
- •4. Documented meningococcal vaccination not more than 3 years prior to dosing, and vaccination against Streptococcus pneumoniae and
- •Haemophilus influenzae
- •5. 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: 23
- •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
排除标准
- •1. ADAMTS13 deficiency (Activity < 5%)
- •2. Shiga toxin-related hemolytic uremic syndrome (STEC-HUS)
- •3. Positive direct Coombs test
- •4. Females who plan to become pregnant during the study or are currently pregnant 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 known genetic defects of cobalamin C metabolism
- •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)
- •10. For Cohort 2 patients, prior use of complement inhibitors other than eculizumab.
- •11. For Cohort 2 patients, any known abnormal TMA parameters within 90 days prior to Screening
研究者
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