跳至主要内容
MedPath Trial logoMedPath TrialMedPath Trial Home
临床试验/NCT02851407
NCT02851407已完成3 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

Jazz Pharmaceuticals114 个研究点 分布在 5 个国家目标入组 372 人开始时间: 2016年9月1日最近更新:
Share to TwitterShare to FacebookShare to LinkedInShare to Reddit
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
372
试验地点
114
主要终点
Veno-occlusive Disease (VOD)-Free Survival by Day +30 Post-Hematopoietic Stem Cell Transplant (HSCT) Per the Independent Endpoint Adjudication Committee (EPAC)
研究概览研究设计入排标准研究组 & 干预措施结局指标研究者研究点记录与标识符相似试验

研究概览

简要总结

This study is to compare the efficacy and safety of defibrotide prophylaxis in addition to best supportive care versus best supportive care alone in the prevention of hepatic veno- occlusive disease (VOD) in adult and pediatric patients undergoing hematopoietic stem cell transplant who are at high risk or very high risk of developing VOD.

研究设计

研究类型
Interventional
分配方式
Randomized
干预模型
Parallel
主要目的
Prevention
盲法
None

入排标准

年龄范围
1 Month 至 —（Child, Adult, Older Adult）
性别
All
接受健康志愿者
否

入选标准

  • •Patient must be above the age of 1 month as of the start date of study treatment.
  • •Patient must be scheduled to undergo allogeneic hematopoietic stem cell transplant (HSCT) (adults or pediatric patients) or autologous HSCT (pediatric patients only) and be at high risk or very high risk of developing veno-occlusive disease (VOD).
  • •Female patients (and female partners of male patients) of childbearing potential who are sexually active must agree to use a highly effective method of contraception with their partners during exposure to defibrotide and for 1 week after the last dose of defibrotide.
  • •Adult patients must be able to understand and sign a written informed consent. For minor patients, the parent/legal guardian or representative must be able to understand and sign a written informed consent. Assent, when appropriate, will be obtained according to institutional guidelines.

排除标准

  • •Patient has hemodynamic instability within 24 hours before the start of study treatment.
  • •Patient has acute bleeding that is clinically significant within 24 hours before the start of study treatment.
  • •Patient used any medication that increases the risk of bleeding within 24 hours before the start of study treatment.
  • •Patient is using or plans to use an investigational agent for the prevention or treatment of VOD.
  • •Patient, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.
  • •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.
  • •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.
  • •Patient is pregnant or lactating and does not agree to stop breastfeeding.
  • •Patient has a known history of hypersensitivity to defibrotide or any of the excipients.
  • •Patient or parent/legal guardian or representative lacks the full mental capacity to understand and sign a written informed consent.
  • •Patient is receiving or plans to receive other investigational therapy during study.

研究组 & 干预措施

Defibrotide

Experimental

Defibrotide is administered intravenously at a dose of 25 mg/kg/day in addition to best supportive care on the day before the first day of the conditioning regimen and will continue (for those patients without a VOD diagnosis) for a recommended minimum of 21 days and end no later than Day +30 post HSCT

干预措施: Defibrotide (Drug)

Best Supportive Care

Other

Best supportive care alone (without the addition of defibrotide) according to institutional guidelines and patient need, is administered on the first day of conditioning and will continue until Day +30 post HSCT or hospital discharge, whichever is sooner, or diagnosis of VOD, if applicable

干预措施: Best Supportive Care (Other)

结局指标

主要结局

Veno-occlusive Disease (VOD)-Free Survival by Day +30 Post-Hematopoietic Stem Cell Transplant (HSCT) Per the Independent Endpoint Adjudication Committee (EPAC)

时间窗: Day +30 Post-HSCT

VOD-free survival is a composite of survival status and VOD occurrence as determined by modified Seattle criteria adjudicated by a blinded independent EPAC. An event is defined as a VOD diagnosis (as assessed by the EPAC) or death, whichever, is earlier, up to and including Day +30 post-HSCT. The values reported below are participants who did not experience VOD or death by Day +30 post-HSCT.

次要结局

  • Veno-Occlusive Disease (VOD)-Free Survival by Day +100 Post-Hematopoietic Stem Cell Transplant (HSCT) Per the Independent Endpoint Adjudication Committee (EPAC)(Day +100 Post-HSCT)
  • Percentage of Participants With Veno-Occlusive Disease (VOD) by Day +30 Post-Hematopoietic Stem Cell Transplant (HSCT)(Day +30 Post-HSCT)
  • Veno-Occlusive Disease (VOD)-Free Survival Rate by Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT)(Day +180 Post-HSCT)
  • Non-Relapse Mortality (NRM) for Defibrotide (DP) and Best Supportive Care (BSC) by Days +100 and +180 Post-Hematopoietic Stem Cell Transplant (HSCT)(Days +100 and +180 Post-HSCT)
  • Percentage of Participants With Veno-Occlusive Disease (VOD)-Associated Multi-Organ Dysfunction (MOD) by Days +30 and Days +100 Post-Hematopoietic Stem Cell Transplant (HSCT) in Patients Who Developed VOD(Days +30 and +100 Post-HSCT)
  • Percentage of Participants Who Had Resolution of Veno-Occlusive Disease (VOD) by Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT)(Day +180 Post-HSCT)
  • Time to Resolution of Veno-Occlusive Disease (VOD) by Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT)(Day +180 Post-HSCT)
  • Percentage of Participants With Veno-Occlusive Disease (VOD) After Day +30 Post-Hematopoietic Stem Cell Transplant (HSCT) up to Days +100 and +180 Post-HSCT(Days +100 and +180 Post-HSCT)
  • Change in 5-Level EuroQol-5D (EQ-5D-5L) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Adult Participants Age ≥ 16 Years: Mobility(Day +180 Post-HSCT)
  • Change in 5-Level EuroQol-5D (EQ-5D-5L) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Adult Participants Age ≥ 16 Years: Self-Care(Day +180 Post-HSCT)
  • Change in 5-Level EuroQol-5D (EQ-5D-5L) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Adult Participants Age ≥ 16 Years: Activity(Day +180 Post-HSCT)
  • Change in 5-Level EuroQol-5D (EQ-5D-5L) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Adult Participants Age ≥ 16 Years: Pain(Day +180 Post-HSCT)
  • Change in 5-Level EuroQol-5D (EQ-5D-5L) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Adult Participants Age ≥ 16 Years: Anxiety(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 4 and ≤ 7 Years: Mobility(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 4 and ≤ 7 Years: Self-Care(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 4 and ≤ 7 Years: Activity(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 4 and ≤ 7 Years: Pain(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 4 and ≤ 7 Years: Anxiety(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 8 and ≤ 15 Years: Mobility(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 8 and ≤ 15 Years: Self-Care(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 8 and ≤ 15 Years: Activity(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 8 and ≤ 15 Years: Pain(Day +180 Post-HSCT)
  • Change in EuroQol-5D for Youth (EQ-5D-Y) Dimensions From Baseline to Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT) for Pediatric Participants Age ≥ 8 and ≤ 15 Years: Anxiety(Day +180 Post-HSCT)
  • Maximum Plasma Concentration (Cmax) of Defibrotide Prophylaxis During the Prophylaxis Phase(Day +1 and +7 Post-HSCT)
  • Area Under the Defibrotide Concentration-Time Curve (AUClast) of Defibrotide Prophylaxis During the Prophylaxis Phase(Day +1 and +7 Post-HSCT)
  • Mean Clearance of Defibrotide Prophylaxis During the Prophylaxis Phase(Day +1 and +7 Post-HSCT)
  • Volume of Distribution of Defibrotide Prophylaxis During the Prophylaxis Phase(Day +1 and +7 Post-HSCT)
  • Maximum Plasma Concentration (Cmax) of Defibrotide Prophylaxis During the Rescue Phase(Day +14 Post-VOD Treatment)
  • Area Under the Defibrotide Concentration-Time Curve (AUClast) of Defibrotide Prophylaxis During the Rescue Phase(Day +14 Post-VOD Treatment)
  • Volume of Distribution of Defibrotide Prophylaxis During the Rescue Phase(Day +14 Post-VOD Treatment)
  • Percentage of Participants With Grades 2, 3, and 4 Acute Graft-Versus-Host-Disease (GvHD) by Days +30, +100, and +180 Post-Hematopoietic Stem Cell Transplant (HSCT) in the Prophylaxis Phase(Days +30, +100, and +180 Post-HSCT)
  • Percentage of Participants With Grades 2, 3, and 4 Acute Graft-Versus-Host-Disease (GvHD) by Days +30, +100, and +180 Post-Hematopoietic Stem Cell Transplant (HSCT) in the Rescue Phase(Days +30, +100, and +180 Post-HSCT)
  • Percentage of Participants With Chronic Graft-Versus-Host-Disease (GvHD) by Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT)(Day +180 Post-HSCT)
  • Number of Participants With Graft Failure During the Prophylaxis Phase and Rescue Phase(Day +180 Post-HSCT)
  • Number of Participants With Neutrophil Engraftment by Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT)(Day +180 Post-HSCT)
  • Number of Participants With Platelet Engraftment by Day +180 Post-Hematopoietic Stem Cell Transplant (HSCT)(Day +180 Post-HSCT)

研究者

申办方类型
Industry
责任方
Sponsor

研究点 (114)

Loading locations...

相似试验

已完成
2 期
An Open-Label Study of Defibrotide for the Prevention of Acute Graft-versus-Host-Disease (AGvHD)Acute-graft-versus-host DiseaseGraft-versus-host Disease
NCT03339297Jazz Pharmaceuticals152
已完成
2 期
Pediatric Trial Investigating the Incidence & Outcome of Veno-Occlusive Disease With the Prophylactic Use of DefibrotideHepatic Veno-Occlusive Disease
NCT00272948European Society for Blood and Marrow Transplantation360
已完成
3 期
Assess the Efficacy and Safety of Personalized Prophylaxis Human-cl rhFVIII in Patients With Severe Haemophilia ASevere Haemophilia A
NCT02256917Octapharma58
招募中
2 期
Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell DiseaseSickle Cell Disease
NCT02675959New York Medical College40
Unknown
不适用
Prophylactic Entecavir for HBV Reactivation in Past HBV Infected Patients With Hematopoietic Stem Cell TransplantationHepatitis B ReactivationHematopoietic Stem Cell TransplantationAntiviral DrugHBV
NCT04942886Seoul St. Mary's Hospital226