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

a study on Veno-occlusive Disease

Summary

for people ages 1 month and up (full criteria)
at San Francisco, California and other locations
study started
estimated completion

Description

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.

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

Keywords

Veno-occlusive Diseasestem cell transplanthematopoietic stem cell transplant (HSCT)veno-occlusive disease (VOD)sinosoidial obstruction syndrome (SOS)Hepatic Veno-Occlusive DiseaseDefibrotideBest Supportive Care

Eligibility

You can join if…

Open to people ages 1 month and up

  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 1 week 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.

You CAN'T join if...

  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.

Locations

  • University of California San Francisco accepting new patients
    San FranciscoCalifornia94158United States
  • Stanford University accepting new patients
    Palo AltoCalifornia94304United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Jazz Pharmaceuticals
ID
NCT02851407
Phase
Phase 3
Study Type
Interventional
Last Updated