NCT00628498已完成3 期
Defibrotide for Patients With Hepatic Veno-occlusive Disease: A Treatment IND Study
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 1,206
- 试验地点
- 125
- 主要终点
- Survival by Day+100 Post Stem Cell Transplant or Chemotherapy
研究概览
简要总结
Single arm, open-label study to provide Defibrotide to patients diagnosed with VOD. Defibrotide is no longer available though the Emergency Use IND mechanism (also known as compassionate use, or single patient named use). This protocol is the only mechanism by which Defibrotide can be made available to patients in the U.S.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Entry criteria include the following:
- •Clinical diagnosis of VOD, made by Baltimore Criteria, Modified Seattle Criteria, or biopsy proven:
- •1.1 Baltimore Criteria- Bilirubin ≥2 mg/dL and at least 2 of the following clinical findings:
- •Ascites (radiographic or physical exam)
- •Weight gain of ≥5% compared to the day of conditioning-- if this value is not available, the weight on the date of admission to the SCT unit may be used)
- •Hepatomegaly; increased over baseline.
- •1.2 Modified Seattle Criteria: At least two of the following
- •Bilirubin ≥2 mg/dL
- •Ascites (radiographic or physical exam) and/or weight gain ≥5% above baseline weight (defined as weight on the first day of conditioning- if this value is not available, the weight on the date of admission to the SCT unit may be used)
- •hepatomegaly increased over baseline
- •1.3 Patients that do not meet the Baltimore Criteria or Modified Seattle Criteria and have biopsy proven VOD are eligible.
- •Patient must also provide written informed consent.
排除标准
- •Use of any medication which increases the risk of hemorrhage is disallowed. Use of heparin or other anticoagulants is disallowed within 12 hours unless being used for routine central venous line management, fibrinolytic instillation for central venous line occlusion, intermittent dialysis or ultrafiltration of CVVH.
- •Clinically significant uncontrolled acute bleeding, defined as hemorrhage requiring > 15 cc/kg of packed red blood cells (e.g., a pediatric patient weighing 20 kg and requiring > 300cc of packed red blood cells/24 hours, or an adult patient weighing 70 kg and requiring >3 units of packed red blood cells/24 hours) to replace blood loss, OR bleeding from a site which in the Investigator's opinion constitutes a potential life-threatening source (e.g. pulmonary hemorrhage or CNS bleeding), irrespective of amount of blood loss, at any point from the date of SCT through the date of severe VOD diagnosis.
- •Hemodynamic instability as defined by a requirement for multiple pressors, or inability to maintain mean arterial pressure (for children: to maintain mean arterial pressure within 1 standard deviation of age-adjusted levels) with single pressor support.
- •Woman who are pregnant.
研究组 & 干预措施
Defibrotide
Experimental
Defibrotide 25 mg/kg day given in 4 divided doses approximately every 6 hours
干预措施: Defibrotide (Drug)
结局指标
主要结局
Survival by Day+100 Post Stem Cell Transplant or Chemotherapy
时间窗: Day +100 from HSCT or 100 days from start of chemotherapy
次要结局
未报告次要终点
研究者
研究点 (125)
Loading locations...
相似试验
No Longer Available
不适用
Compassionate Use of Defibrotide for Patients With Veno-occlusive Disease of the LiverHepatic Veno-occlusive DiseaseNCT00143546St. Jude Children's Research Hospital
No Longer Available
不适用
Defibrotide Compassionate Use for Patients With Life Threatening Veno-Occlusive Disease of the LiverLiver DiseasesNCT00263978Emory University
终止
2 期
A Safety and Efficacy Study of Defibrotide in the Prevention of Chimeric Antigen Receptor-T-cell-associated NeurotoxicityNeurotoxicity SyndromesDLBCLNCT03954106Jazz Pharmaceuticals25
已完成
4 期
Defibrotide in the Human Endotoxemia Model --- an Exploratory Trial Investigating the Effects and the Mechanisms of DefibrotideHealthy VolunteersEndotoxemiaNCT02876601Bernd Jilma20
已完成
不适用
European VOD RegistryHepatic Veno-Occlusive DiseaseNCT03032016Jazz Pharmaceuticals176