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

Completed · Phase 3

Conditions studied: Veno-occlusive Disease

In brief

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.

Key facts

Study ID

NCT02851407

Run by

Jazz Pharmaceuticals

People needed

372

Starts

2016-09-01

Expected to finish

2020-10-20

Last updated by the study team

2022-03-02

Who can join

Age: 0 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• Phoenix Children's Hospital — Phoenix, Arizona, United States
• University of Arizona Cancer Center — Tucson, Arizona, United States
• Children's Hospital Los Angeles — Los Angeles, California, United States
• Stanford University — Palo Alto, California, United States
• Rady Childrens Hospital San Diego — San Diego, California, United States
• University of California San Francisco — San Francisco, California, United States
• Colorado Children's Hospital — Aurora, Colorado, United States
• Alfred I Dupont Hospital For Children — Wilmington, Delaware, United States
• Nicklaus Childrens Hospital — Miami, Florida, United States
• Johns Hopkins All Children's Hospital — St. Petersburg, Florida, United States
• Children's Healthcare of Atlanta — Atlanta, Georgia, United States
• Ann and Robert H Lurie Childrens Hospital of Chicago — Chicago, Illinois, United States
• Tufts Floating Hospital for Children — Boston, Massachusetts, United States
• Dana Farber Cancer Institute — Boston, Massachusetts, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• Children's Mercy Hospital — Kansas City, Missouri, United States
• Columbia University Medical Center — New York, New York, United States
• Weill Cornell Medical College — New York, New York, United States
• Memorial Sloan Kettering Cancer Center — New York, New York, United States
• Children's Hospital at Montefiore — The Bronx, New York, United States
• Duke University Medical Center — Durham, North Carolina, United States
• University Hospital Case Medical Center — Cleveland, Ohio, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Nationwide Children's Hospital — Columbus, Ohio, United States
• Children's Hospital of Alabama — Birmingham, Alabama, United States

Full record on ClinicalTrials.gov

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