EUCTR2016-002004-10-DE进行中（未招募）1 期
A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Undergoing Hematopoietic Stem Cell Transplant
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 400
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. Patient must be above the age of 1 month as of Study Day 1.
- •2. Patient must be scheduled to undergo allogeneic (adults or pediatric
- •patients) or autologous HSCT (pediatric patients only) and be at high
- •risk or very high risk of developing VOD.
- •a. High-risk patients must meet both of the following criteria (i and ii):
- •i. Patient must be scheduled to receive myeloablative conditioning,
- •defined as either of the following:
- •a. At least 2 alkylating agents (e.g., cyclophosphamide, busulfan,
- •melphalan); the investigator must document in the medical chart that
- •the conditioning regimen is considered to be myeloablative or b. TBI
- •(single dose of =5 Gy, or =8 Gy fractionated dose) and at least 1
- •alkylating agent, and ii. Patient must meet at least 1 of the following criteria:
- •a. Has at least 1 hepatic-related risk factor, as defined by the European
- •Society for Blood and Marrow Transplantation (EBMT) position
- •statement, during screening as follows:
- •Transaminase level >2.5 times the upper limit of normal (ULN) during
- •screening or within 14 days prior to screening on a non-screening test if
- •the test was performed as part of patient's routine standard of care
- •Serum total bilirubin level >1.5 times the ULN during screening or
- •within 14 days prior to screening on a non-screening test if the test was
- •performed as part of patient's routine standard of care
- •Prior history of cirrhosis (with biopsy evidence)
- •Prior history of hepatic fibrosis (by histology or other diagnostic
- •scoring system per institutional guidelines)
- •Prior history of viral hepatitis within 1 year before the start of study
- •treatment as indicated by a positive test for any of the following:
- •– hepatitis A virus (HAV) immunoglobulin M (IgM) (anti-HAV IgM)
- •– hepatitis B virus (HBV) core immunoglobulin G (IgG) or IgM (anti-HBc
- •IgG or anti-HBc IgM)
- •– HBV surface antigen (HBsAg)
- •– HBV DNA by polymerase chain reaction (PCR) or nucleic acid
- •amplification testing (NAAT)
- •– hepatitis C virus (HCV) antibody (anti-HCV) and HCV RNA by PCR or
- •Any prior hepatic irradiation, including abdominal irradiation covering
- •the hepatic area
- •Documented diagnosis and confirmed evidence of iron overload in
- •medical notes (repeated serum ferritin >2000 ng/mL and/or liver iron
- •content =5.0 mg/gdw as estimated by magnetic resonance imaging T2*)
- •within 3 months prior to screening.
- •b. Has advanced-stage neuroblastoma requiring myeloablative
- •conditioning.
- •b. Very high-risk patients must meet 1 of the following criteria:
- •i. Osteopetrosis and patient must be scheduled to receive myeloablative
- •conditioning defined as either of the following:
- •a. At least 2 alkylating agents (e.g., cyclophosphamide, busulfan,
- •melphalan); the investigator must document in the medical chart that
- •the conditioning regimen is considered to be myeloablative or
- •b. TBI (single dose of =5 Gy, or =8 Gy fractionated dose) and at least 1
- •alkylating agent
- •ii. Primary HLH, Griscelli II Chediak-Higashi syndrome, HermanskyPudiak
- 另有 10 项未显示
排除标准
- •1. Patient has hemodynamic instability within 24 hours before the start
- •of study treatment.
- •2. Patient has acute bleeding that is clinically significant within 24 hours
- •before the start of study treatment, defined as either of the following (a
- •a. hemorrhage requiring >15 cc/kg of packed red blood cells (e.g.,
- •pediatric patient weighing 20 kg and requiring 300 cc packed red blood
- •cells/24 hours, or an adult weighing >70 kg and requiring 3 units of
- •packed red blood cells/24 hours) to replace blood loss, or
- •b. bleeding from a site which, in the investigator's opinion, constitutes a
- •potential life-threatening source (e.g., pulmonary hemorrhage or central
- •nervous system bleeding), irrespective of amount of blood loss
- •3. Patient used any medication that increases the risk of bleeding within
- •24 hours before the start of study treatment, including, but not limited
- •to, systemic heparin, low molecular weight heparin, heparin analogs,
- •alteplase (tPA), streptokinase, urokinase,
- •antithrombin III (ATIII), and oral anticoagulants including warfarin, and
- •other agents that increase the risk of bleeding. Patients may receive
- •heparin or other anticoagulants for routine central venous line
- •management and intermittent dialysis or ultrafiltration. Fibrinolytic
- •instillation for central venous line occlusion is also permitted. Note:
- •Heparin use will be allowed in both treatment arms (up to a maximum of
- •100 U/kg/day).
- •4. Patient is using or plans to use an investigational agent for the
- •prevention or treatment of VOD.
- •5. Patient, in the opinion of the investigator, may not be able to comply
- •with the safety monitoring requirements of the study.
- •6. Patient or parent/legal guardian or representative has a psychiatric
- •illness that would prevent the patient or parent/legal guardian or
- •representative from giving informed consent and/or assent.
- •7. Patient has a serious active disease or co-morbid medical condition, as
- •judged by the investigator, which would interfere with the conduct of
- •this study.
- •8. Patient is pregnant or lactating and does not agree to stop
- •breastfeeding.
- •9. Patient has a known history of hypersensitivity to defibrotide or any of
- •the excipients.
- •10. Patient or parent/legal guardian or representative lacks the full
- •mental capacity to understand and sign a written informed consent.
- •11. Patient is receiving or plans to receive other investigational therapy
- •during study.
研究者
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