Study Comparing Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients
NCT ID: NCT02851407
Last Updated: 2022-03-02
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
372 participants
INTERVENTIONAL
2016-09-01
2020-10-20
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
PREVENTION
NONE
Study Groups
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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
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
Best Supportive Care
Interventions
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Defibrotide
Best Supportive Care
Eligibility Criteria
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Inclusion Criteria
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.
Exclusion Criteria
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.
1 Month
ALL
No
Sponsors
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Jazz Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Jazz Pharmaceuticals
Role: STUDY_DIRECTOR
Jazz Pharmaceuticals
Locations
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Children's Hospital of Alabama
Birmingham, Alabama, United States
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
Doernbecher Children's Hospital
Portland, Oregon, United States
Penn State Milton S Hershey Medical Center
Hershey, Pennsylvania, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States
Medical University of South Carolina - PPDS
Charleston, South Carolina, United States
Vanderbilt Ingram Cancer Center
Nashville, Tennessee, United States
Children's Medical Center Dallas
Dallas, Texas, United States
Cook Childrens Hospital
Fort Worth, Texas, United States
MD Anderson Cancer Center
Houston, Texas, United States
Texas Childrens Hospital
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, United States
Royal Adelaide Hospital
Adelaide, South Australia, Australia
Royal Children's Hospital Melbourne
Melbourne, Victoria, Australia
Cliniques Universitaires Saint-Luc
Brussels, , Belgium
UZ Gent
Ghent, , Belgium
UZ Leuven Gasthuisberg
Leuven, , Belgium
UZ Leuven
Leuven, , Belgium
Centre Hospitalier Universitaire du Sart Tilman
Liège, , Belgium
Alberta Children's Hospital
Calgary, Alberta, Canada
Sainte Justine Hospital
Montreal, Quebec, Canada
Hopital Jean Minjoz
Besançon, , France
Institut Paoli Calmettes
Marseille, , France
Hôpital Saint Antoine
Paris, , France
CHU de Poitiers
Poitiers, , France
Institut Universitaire du Cancer de Toulouse - Oncopôle
Toulouse, , France
Klinikum Frankfurt Oder GmbH
Frankfurt (Oder), Brandenburg, Germany
Universitätsklinikum der RWTH Aachen
Aachen, North Rhine-Westphalia, Germany
Universitätsklinikum Münster
Münster, North Rhine-Westphalia, Germany
Universitätsklinikum Carl Gustav Carus an der TU Dresden
Dresden, Saxony, Germany
Universitätsklinikum Leipzig
Leipzig, Saxony, Germany
Universitätsklinikum Hamburg Eppendorf
Hamburg, , Germany
Klinikum der Universitat Regensburg
Regensburg, , Germany
Rambam Health Care Campus
Haifa, , Israel
Rambam Health Corporation
Haifa, , Israel
Hadassah Ein Kerem Hospital
Jerusalem, , Israel
Schneider Children Medical Center of Israel
Petah Tikva, , Israel
Chaim Sheba Medical Center
Ramat Gan, , Israel
Tel Aviv Sourasky Medical Center
Tel Aviv, , Israel
11. Azienda Ospedaliero Universitaria Ospedali Riuniti di Ancona-Umberto I G M Lancisi G Salesi
Ancona, , Italy
Azienda Ospedaliero - Universitaria
Catania, , Italy
Azienda Ospedaliera Universitaria Careggi
Florence, , Italy
AORMN Marche Nord
Pesaro, , Italy
Ospedale Pediatrico Bambino Gesù
Roma, , Italy
Fondazione Policlinico Universitario A Gemelli
Roma, , Italy
Anjo Kosei Hospital
Anjo, , Japan
Hamanomachi Hospital
Fukuoka, , Japan
Fukushima Medical University Hospital
Fukushima, , Japan
Kobe University Hospital
Hyōgo, , Japan
Kanagawa Children's Medical Center
Kanagawa, , Japan
National Hospital Organization Kumamoto Medical Center
Kumamoto, , Japan
Japanese Red Cross Nagoya Daiichi Hospital
Nagoya, , Japan
Hyogo College of Medicine
Nishinomiya, , Japan
Osaka International Cancer Institute
Osaka, , Japan
Osaka City University Hospital
Osaka, , Japan
Hokkaido University Hospital
Sapporo, , Japan
Toranomon Hospital
Tokyo, , Japan
Tokyo Metropolitan Cancer and Infectious Diseases Center Komagome Hospital
Tokyo, , Japan
Medical Hospital Tokyo Medical and Dental University
Tokyo, , Japan
Auckland City Hospital
Auckland, , New Zealand
Seoul National University Hospital
Seoul, , South Korea
Severance Hospital at Yonsei University Health System
Seoul, , South Korea
Asan Medical Center
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
The Catholic University of Korea Seoul St. Mary's Hospital
Seoul, , South Korea
Hospital Universitario Germans Trias i Pujol
Badalona, Barcelona, Spain
Hospital Universitario Vall d Hebron
Barcelona, , Spain
Hospital Universitario Reina Sofia
Córdoba, , Spain
Hospital Sant Joan de Deu - PIN
Esplugues de Llobregat, , Spain
Hospital Infantil Universitario Niño Jesus
Madrid, , Spain
Hospital Universitario Ramon y Cajal
Madrid, , Spain
Hospital Universitario La Paz
Madrid, , Spain
Hospital Regional Universitario de Malaga Hospital General
Málaga, , Spain
Hospital General Universitario Morales Meseguer
Murcia, , Spain
Hospital Universitario de Salamanca
Salamanca, , Spain
Hospital Universitario Virgen del Rocio
Seville, , Spain
Akdeniz University Medical Faculty Department of Pediatrics
Antalya, Konyaalti, Turkey (Türkiye)
Erciyes University Medical Faculty
Kayseri, Talas, Turkey (Türkiye)
Medicana International Ankara Hospital
Ankara, , Turkey (Türkiye)
Medical Park Antalya Hospital
Antalya, , Turkey (Türkiye)
Ege Universitesi Tip Fakultesi
Bornova, , Turkey (Türkiye)
Acibadem Universitesi Tip Fakultesi Atakent Hastanesi
Istanbul, , Turkey (Türkiye)
Acibadem Adana Hospital
Seyhan, , Turkey (Türkiye)
Birmingham Children's Hospital
Birmingham, , United Kingdom
Beatson West of Scotland Cancer Centre
Glasgow, , United Kingdom
Royal Hospital for Children
Glasgow, , United Kingdom
St. James University Hospital
Leeds, , United Kingdom
Kings College Hospital
London, , United Kingdom
Great Ormond Street Hospital
London, , United Kingdom
Countries
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References
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Corbacioglu S, Grupp SA, Richardson PG, Duarte R, Pagliuca A, Ruutu T, Mahadeo K, Carreras E. Prevention of veno-occlusive disease/sinusoidal obstruction syndrome: a never-ending story and no easy answer. Bone Marrow Transplant. 2023 Aug;58(8):839-841. doi: 10.1038/s41409-023-02007-2. Epub 2023 May 25. No abstract available.
Grupp SA, Corbacioglu S, Kang HJ, Teshima T, Khaw SL, Locatelli F, Maertens J, Stelljes M, Stepensky P, Lopez P, Amber V, Pagliuca A, Richardson PG, Mohty M. Defibrotide plus best standard of care compared with best standard of care alone for the prevention of sinusoidal obstruction syndrome (HARMONY): a randomised, multicentre, phase 3 trial. Lancet Haematol. 2023 May;10(5):e333-e345. doi: 10.1016/S2352-3026(23)00011-X. Epub 2023 Mar 28.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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15-007
Identifier Type: -
Identifier Source: org_study_id
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