EUCTR2020-000761-16-IT进行中（未招募）1 期
A Phase 3, Open-label, Single Arm, Multicenter Study of Ravulizumab in Addition to Best Supportive Care in Pediatric Participants (from 1 month to < 18 years of age) with Thrombotic Microangiopathy (TMA) after Hematopoietic Stem Cell Transplantation (HSCT) - Ravulizumab in Pediatric Participants with TMA after HSCT
ALEXION PHARMACEUTICALS INCORPORATED0 个研究点目标入组 40 人开始时间: 2021年5月24日最近更新:
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 40
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
- •1. 1 month of age up to < 18 years of age at the time of signing the informed consent
- •2. Participants who received HSCT within the past 6 months at the time of Screening
- •3. A TMA diagnosis, based on all of the following criteria occurring simultaneously:
- •De novo thrombocytopenia or platelet transfusion refractoriness, where:
- •De novo anemia or increase in transfusion requirements
- •Either one of the following markers of hemolysis:
- •-Lactate dehydrogenase > 1.5 × ULN or,
- •- Presence of schistocytes = 2 high power field (HPF)
- •Proteinuria on spot urinalysis
- •Presence of hypertension
- •4. Participants must have HSCT-TMA that persists for at least 72 hours after initial management of
- •any triggering agent/condition
- •5.Body weight = 5 kg at Screening
- •6. Male or female Contraceptive use by men or women should be consistent with local regulations
- •regarding the methods of contraception for those participating in clinical studies
- •7. Vaccinated against meningococcal infections if clinically feasible, according to institutional
- •guidelines for immune reconstitution after HSCT. Participants must be re-vaccinated against
- •Haemophilus influenzae type b (Hib) and Streptococcus pneumoniae if clinically feasible,
- •according to institutional guidelines for immune reconstitution after HSCT. All participants should
- •be administered coverage with prophylactic antibiotics according to institutional posttransplant
- •infection prophylaxis guidances including coverage against N. meningiditis for at least 2 weeks
- •after meningococcal vaccination. Participants who cannot receive meningococcal vaccine should
- •receive antibiotic prophylaxis coverage against N. meningiditis the entire Treatment Period and for
- •8 months following the final dose of ravulizumab.
- •8. Participants or their legally authorized representative must be capable of giving signed informed
- •consent which includes compliance with the requirements and restrictions listed in the informed
- •consent or assent form and in this protocol
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range:
- •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. Known familial or acquired 'a disintegrin and metalloproteinase with a thrombospondin type 1
- •motif, member 13' (ADAMTS13) deficiency (activity < 5%)
- •2. Known Shiga toxin-related hemolytic uremic syndrome (ST-HUS)
- •3. Positive direct Coombs test
- •4. Diagnosis or suspicion of disseminated intravascular coagulation (DIC)
- •5. Known bone marrow/graft failure
- •6. Diagnosis of veno-occlusive disease (VOD)
- •7. Human immunodeficiency virus (HIV) infection (evidenced by HIV-1 or HIV-2 antibody titer,
- •8. Unresolved meningococcal disease
- •9. Presence or suspicion of sepsis (treated or untreated) within 7 days prior to Screening
- •10. Pregnancy or breastfeeding
- •11. Hypersensitivity to murine proteins or to 1 of the excipients of ravulizumab
- •12. Previously or currently treated with a complement inhibitor
研究者
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