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Trial record 6 of 3386 for:    adult stem cell transplants | Interventional Studies

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

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ClinicalTrials.gov Identifier: NCT02851407
Recruitment Status : Recruiting
First Posted : August 1, 2016
Last Update Posted : April 27, 2018
Sponsor:
Information provided by (Responsible Party):
Jazz Pharmaceuticals

Brief Summary:
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.

Condition or disease Intervention/treatment Phase
Veno-occlusive Disease Drug: Defibrotide Other: Best Supportive Care Phase 3

Study Type : Interventional
Estimated Enrollment : 400 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Prevention
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
Study Start Date : September 2016
Estimated Primary Completion Date : January 2021
Estimated Study Completion Date : February 2021

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Defibrotide
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
Drug: Defibrotide
Best Supportive Care
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
Other: Best Supportive Care



Primary Outcome Measures :
  1. Veno-Occlusive Disease (VOD)-Free Survival at Day 30 Post-Hemotopoietic Stem Cell Transplant (HSCT) [ Time Frame: 30 Days Post-Transplant ]
    Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD


Secondary Outcome Measures :
  1. Veno-Occlusive Disease (VOD)-Free Survival at Day 100 Post-Hemotopoietic Stem Cell Transplant (HSCT) [ Time Frame: 100 Days Post-Transplant ]
    Comparison of efficacy of defibrotide vs Best Supportive Care (BSC) for the prevention of VOD



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Ages Eligible for Study:   1 Month and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 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 (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 14 weeks after the last dose of defibrotide.
  4. 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.

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 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02851407


Contacts
Contact: Clinical Trial Information Desk HarmonyTrialInfo@jazzpharma.com

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Sponsors and Collaborators
Jazz Pharmaceuticals
Investigators
Study Director: Jazz Pharmaceuticals Jazz Pharmaceuticals

Responsible Party: Jazz Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02851407     History of Changes
Other Study ID Numbers: 15-007
First Posted: August 1, 2016    Key Record Dates
Last Update Posted: April 27, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Keywords provided by Jazz Pharmaceuticals:
stem cell transplant
hematopoietic stem cell transplant (HSCT)
veno-occlusive disease (VOD)
sinosoidial obstruction syndrome (SOS)

Additional relevant MeSH terms:
Hepatic Veno-Occlusive Disease
Liver Diseases
Digestive System Diseases
Vascular Diseases
Cardiovascular Diseases
Defibrotide
Fibrinolytic Agents
Fibrin Modulating Agents
Molecular Mechanisms of Pharmacological Action
Platelet Aggregation Inhibitors