Clinical Trials /

Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients

NCT02851407

Description:

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.

Recruiting Status:

Completed

Phase:

Phase 3

Trial Eligibility

Document

Title

  • Brief Title: Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients
  • Official Title: 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

Clinical Trial IDs

  • ORG STUDY ID: 15-007
  • NCT ID: NCT02851407

Conditions

  • Veno-occlusive Disease

Interventions

DrugSynonymsArms
DefibrotideDefibrotide

Purpose

This study is to compare the efficacy and safety of defibrotide versus best supportive care 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.

Trial Arms

NameTypeDescriptionInterventions
DefibrotideExperimentalDefibrotide 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
  • Defibrotide
Best Supportive CareOtherBest supportive care alone (without the addition of defibrotide) according to institutional guidelines 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

    Eligibility Criteria

            Inclusion Criteria:
    
              1. Patient must be above the age of 1 month as of the start date of study treatment.
    
              2. Patient must be scheduled to undergo allogeneic or autologous 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).
    
              3. Female patients of childbearing potential who are sexually active must agree to use a
                 medically acceptable method of contraception throughout the entire study period and
                 for 4 weeks after the last dose of study drug; male patients with female partners of
                 childbearing potential must agree to use a medically acceptable method of
                 contraception for 6 months after the last dose of study drug.
    
              4. Patient and/or the 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.
    
            Exclusion Criteria:
    
              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.
    
              3. Patient used any medication that increases the risk of bleeding within 24 hours
                 before the start of study treatment.
    
              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 has a psychiatric illness that would prevent the patient or 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.
          
    Maximum Eligible Age:N/A
    Minimum Eligible Age:1 Month
    Eligible Gender:All
    Healthy Volunteers:No

    Primary Outcome Measures

    Measure:Veno-Occlusive Disease (VOD)-Free Survival at Day 30 Post-Hemotopoietic Stem Cell Transplant (HSCT)
    Time Frame:30 Days Post-Transplant
    Safety Issue:
    Description:Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD

    Secondary Outcome Measures

    Measure:Veno-Occlusive Disease (VOD)-Free Survival at Day 100 Post-Hemotopoietic Stem Cell Transplant (HSCT)
    Time Frame:100 Days Post-Transplant
    Safety Issue:
    Description:Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD

    Details

    Phase:Phase 3
    Primary Purpose:Interventional
    Overall Status:Recruiting
    Lead Sponsor:Jazz Pharmaceuticals

    Trial Keywords

    • stem cell transplant
    • hematopoietic stem cell transplant (HSCT)
    • veno-occlusive disease (VOD)
    • sinosoidial obstruction syndrome (SOS)

    Last Updated

    March 17, 2017